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Clinical Trial Results:
A Phase 2, Randomized, Placebo-Controlled Study to Evaluate Safety, Tolerability, and Efficacy of TAK-079 in Patients With Generalized Myasthenia Gravis

Summary
EudraCT number	2019-003383-47
Trial protocol	IT
Global end of trial date	12 Jul 2022

Results information
Results version number	v1(current)
This version publication date	19 Apr 2023
First version publication date	19 Apr 2023
Other versions

Trial Information
Subject Disposition
Baseline Characteristics
End Points
Adverse Events
More Information

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Trial information

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Sponsor protocol code

TAK-079-1005

ISRCTN number

US NCT number

NCT04159805

WHO universal trial number (UTN)

U1111-1234-4442

Sponsor organisation name

Takeda

Sponsor organisation address

95 Hayden Avenue, Lexington, United States, MA 02421

Public contact

Study Director, Takeda, +1 877-825-3327, TrialDisclosures@takeda.com

Scientific contact

Study Director, Takeda, +1 877-825-3327, TrialDisclosures@takeda.com

Is trial part of an agreed paediatric investigation plan (PIP)

Does article 45 of REGULATION (EC) No 1901/2006 apply to this trial?

Does article 46 of REGULATION (EC) No 1901/2006 apply to this trial?

Analysis stage

Final

Date of interim/final analysis

12 Jul 2022

Is this the analysis of the primary completion data?

Global end of trial reached?

Yes

Global end of trial date

12 Jul 2022

Was the trial ended prematurely?

Main objective of the trial

The main objective of this trial is to evaluate the safety and tolerability of TAK-079 in participants with generalized myasthenia gravis (MG) who are receiving stable background therapy for MG.

Protection of trial subjects

All study participants were required to read and sign an Informed Consent Form.

Background therapy

Evidence for comparator

Actual start date of recruitment

14 Jan 2020

Long term follow-up planned

Independent data monitoring committee (IDMC) involvement?

Number of subjects enrolled per country

Country: Number of subjects enrolled

Canada: 2

Country: Number of subjects enrolled

Poland: 5

Country: Number of subjects enrolled

Serbia: 17

Country: Number of subjects enrolled

Spain: 4

Country: Number of subjects enrolled

United States: 8

Worldwide total number of subjects

EEA total number of subjects

Number of subjects enrolled per age group

In utero

Preterm newborn - gestational age < 37 wk

Newborns (0-27 days)

Infants and toddlers (28 days-23 months)

Children (2-11 years)

Adolescents (12-17 years)

Adults (18-64 years)

From 65 to 84 years

85 years and over

Subject disposition

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Recruitment details

Participants took part in the study at 15 investigative sites in the United States, Spain, Poland, Serbia, and Canada from 14 January 2020 to 12 July 2022.

Screening details

Participants with a diagnosis of Myasthenia Gravis (MG) were enrolled in 1:1:1 ratio to receive TAK-079 matching placebo, TAK-079 300 mg or TAK-079 600 mg in combination with standard background therapy.

Period 1 title

Overall Study (overall period)

Is this the baseline period?

Yes

Allocation method

Randomised - controlled

Blinding used

Double blind

Roles blinded

Subject, Investigator, Data analyst, Carer

Are arms mutually exclusive

Yes

TAK-079 Placebo-matching

Arm description

TAK-079 placebo-matching injection, subcutaneously (SC), once weekly in combination with standard background therapy for 8 weeks.

Arm type

Placebo

Investigational medicinal product name

TAK-079 Placebo

Investigational medicinal product code

Other name

Pharmaceutical forms

Solution for injection

Routes of administration

Subcutaneous use

Dosage and administration details

TAK-079 placebo-matching SC injection

TAK-079 300 mg

Arm description

TAK-079 300 mg injection, SC, once weekly in combination with standard background therapy for 8 weeks.

Arm type

Experimental

Investigational medicinal product name

TAK-079

Investigational medicinal product code

Other name

Mezagitamab

Pharmaceutical forms

Solution for injection

Routes of administration

Subcutaneous use

Dosage and administration details

TAK-079 SC injection

TAK-079 600 mg

Arm description

TAK-079 600 mg injection, SC, once weekly in combination with standard background therapy for 8 weeks.

Arm type

Experimental

Investigational medicinal product name

TAK-079

Investigational medicinal product code

Other name

Mezagitamab

Pharmaceutical forms

Solution for injection

Routes of administration

Subcutaneous use

Dosage and administration details

TAK-079 SC injection

Number of subjects in period 1	TAK-079 Placebo-matching	TAK-079 300 mg	TAK-079 600 mg
Started	12	12	12
Completed	12	10	10
Not completed	0	2	2
Consent withdrawn by subject	-	2	2

Baseline characteristics

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Reporting group title

TAK-079 Placebo-matching

Reporting group description

TAK-079 placebo-matching injection, subcutaneously (SC), once weekly in combination with standard background therapy for 8 weeks.

Reporting group title

TAK-079 300 mg

Reporting group description

TAK-079 300 mg injection, SC, once weekly in combination with standard background therapy for 8 weeks.

Reporting group title

TAK-079 600 mg

Reporting group description

TAK-079 600 mg injection, SC, once weekly in combination with standard background therapy for 8 weeks.

Reporting group values	TAK-079 Placebo-matching	TAK-079 300 mg	TAK-079 600 mg	Total
Number of subjects	12	12	12
Age Categorical
Units: Subjects
Age continuous
Units: years
arithmetic mean (standard deviation)	46.5 ± 18.03	45.3 ± 12.47	56.3 ± 14.42	-
Gender categorical
Units: Subjects
Female	9	6	7	22
Male	3	6	5	14
Ethnicity (NIH/OMB)
Units: Subjects
Hispanic or Latino	2	0	1	3
Not Hispanic or Latino	9	12	11	32
Unknown or Not Reported	1	0	0	1
Race (NIH/OMB)
Units: Subjects
American Indian or Alaska Native	0	0	0	0
Asian	0	0	1	1
Native Hawaiian or Other Pacific Islander	0	0	0	0
Black or African American	1	0	0	1
White	10	12	11	33
More than one race	0	0	0	0
Unknown or Not Reported	1	0	0	1
Region of Enrollment
Units: Subjects
Canada Canada	0	1	1	2
Poland Poland	1	2	2	5
Serbia Serbia	5	7	5	17
Spain Spain	3	0	1	4
United States United States	3	2	3	8
Weight
Units: kg
arithmetic mean (standard deviation)	85.11 ± 25.607	86.28 ± 21.132	88.38 ± 25.672	-
Height
Units: cm
arithmetic mean (standard deviation)	164.67 ± 9.435	175.39 ± 8.911	171.38 ± 10.910	-
Quantitative Myasthenia Gravis (QMG) Scale Score at Baseline
Physician-reported scale to assess MG disease severity by quantifying several body functions by physical exam. Each question is graded on a 4-point scale from 0=normal to 3=severe with a total score of 0 to 39; the higher score indicates greater disease burden.
Units: score on a scale
arithmetic mean (standard deviation)	11.4 ± 5.21	12.9 ± 6.47	12.8 ± 4.26	-
Myasthenia Gravis Composite (MGC) Scale Score at Baseline
An assessment scale of MG disease activity based on a combination of participant- and physician-reported items. Each question is graded on 4 levels of impact from normal to severe with a total score of 0 to 50; the higher score indicates worse MG disease activity.
Units: score on a scale
arithmetic mean (standard deviation)	14.7 ± 5.80	16.8 ± 6.58	15.3 ± 6.00	-
Revised 15-item Myasthenia Gravis Quality of Life Scale (MG-QoL15r) Scale Score at Baseline
A participant-reported score that assesses the participant's perception of impairment and disability and the degree to which the patient tolerates disease manifestations. Each question is graded on a 3-point scale from 0=normal to 2=severe with a total score of 0 to 30; the higher score indicates worse MG disease activity.
Units: score on a scale
arithmetic mean (standard deviation)	11.5 ± 4.15	17.1 ± 6.76	13.9 ± 4.72	-
Body Mass Index (BMI)
BMI=[weight(kg) / height(m)^2].
Units: kg/m^2
arithmetic mean (standard deviation)	31.33 ± 8.867	27.74 ± 4.852	30.22 ± 9.315	-
Myasthenia Gravis Activities of Daily Living (MG-ADL) Scale Score at Baseline
Participant-reported scale to assess MG symptoms to evaluate capacity to perform activities of daily living. Each question is graded on a 4-point scale from 0=normal to 3=severe with a total score of 0 to 24; the higher score indicates greater functional impairment and disability.
Units: score on a scale
arithmetic mean (standard deviation)	7.9 ± 1.78	9.3 ± 2.49	8.4 ± 2.23	-

End points

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Reporting group title

TAK-079 Placebo-matching

Reporting group description

TAK-079 placebo-matching injection, subcutaneously (SC), once weekly in combination with standard background therapy for 8 weeks.

Reporting group title

TAK-079 300 mg

Reporting group description

TAK-079 300 mg injection, SC, once weekly in combination with standard background therapy for 8 weeks.

Reporting group title

TAK-079 600 mg

Reporting group description

TAK-079 600 mg injection, SC, once weekly in combination with standard background therapy for 8 weeks.

Primary: Percentage of Participants With Treatment Emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), Grade 3 or Higher TEAEs, and Adverse Event (AE) Leading to TAK-079 Discontinuation

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End point title

Percentage of Participants With Treatment Emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), Grade 3 or Higher TEAEs, and Adverse Event (AE) Leading to TAK-079 Discontinuation ^[1]

End point description

AE=any untoward medical occurrence in clinical investigation participant administered drug;does not necessarily have to have causal relationship with this treatment.TEAE=AE with onset that occurs after receiving study drug.SAE is AE resulting in any of following outcomes or deemed significant for any other reason:death;initial or prolonged inpatient hospitalization;life-threatening experience(immediate risk of dying); persistent or significant disability/incapacity;congenital anomaly.Severity of TEAEs was graded using National cancer institute-Common Terminology Criteria for Adverse Events(NCI-CTCAE) version 4.03 definitions of Grade 1 through Grade 5 where Grade 1=mild symptoms,Grade 2=moderate symptoms,Grade 3=Severe or medically significant but not immediately life-threatening,Grade 4=life-threatening consequences and Grade 5=death related to AEs.Percentages are rounded off to whole number at single decimal. Safety Analysis Set=participants receiving at least 1 dose of study drug.

End point type

Primary

End point timeframe

From signing the informed consent form up to end of long-term follow-up (up to Week 32)

Notes
[1] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
Justification: Only descriptive statistical analysis was performed for this endpoint.

End point values	TAK-079 Placebo-matching	TAK-079 300 mg	TAK-079 600 mg
Number of subjects analysed	12	12	12
Units: percentage of participants
number (not applicable)
TEAE	66.7	75.0	91.7
SAE	8.3	8.3	8.3
Grade 3 or Higher TEAEs	16.7	8.3	8.3
AE Leading to TAK-079 Discontinuation	0.0	0.0	0.0

No statistical analyses for this end point

Secondary: Change From Baseline in Myasthenia Gravis Activities of Daily Living (MG-ADL) Scale Score

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End point title

Change From Baseline in Myasthenia Gravis Activities of Daily Living (MG-ADL) Scale Score

End point description

Participant-reported scale to assess MG symptoms to evaluate capacity to perform activities of daily living. Each question is graded on a 4-point scale from 0=normal to 3=severe with a total score of 0 to 24; the higher score indicates greater functional impairment and disability. Negative change indicates improvement. Mixed-effects model for repeated measures (MMRM) was used for the analysis. Full Analysis Set included all randomised participants who had baseline and at least 1 post-baseline efficacy assessment. n=Number analysed is the number of participants available for analysis at the specified timepoint. 9999=Data not reported for 0 participants.

End point type

Secondary

End point timeframe

Baseline up to Week 32

End point values	TAK-079 Placebo-matching	TAK-079 300 mg	TAK-079 600 mg
Number of subjects analysed	12	12	12
Units: score on a scale
arithmetic mean (standard deviation)
Change From Baseline at Week 4(n=12,12,12)	-2.8 ± 2.67	-2.5 ± 2.47	-1.8 ± 1.66
Change From Baseline at Week 6(n=12,11,11)	-4.0 ± 2.73	-4.2 ± 2.64	-2.3 ± 2.15
Change From Baseline at Week 8(n=12,11,12)	-3.8 ± 2.80	-4.3 ± 3.10	-2.0 ± 3.16
Change From Baseline at Week 10(n=11,11,11)	-4.0 ± 3.52	-5.0 ± 2.41	-2.0 ± 2.79
Change From Baseline at Week 12(n=11,11,12)	-4.2 ± 3.19	-4.2 ± 2.56	-2.5 ± 2.68
Change From Baseline at Week 14(n=11,10,10)	-4.0 ± 4.38	-4.8 ± 2.20	-2.0 ± 3.30
Change From Baseline at Week 16(n=10,10,10)	-4.1 ± 3.21	-4.3 ± 2.79	-3.1 ± 3.48
Change From Baseline at Week 20(n=0,8,11)	9999 ± 9999	-3.9 ± 3.60	-2.9 ± 3.11
Change From Baseline at Week 24(n=0,9,9)	9999 ± 9999	-4.1 ± 3.62	-3.0 ± 3.57
Change From Baseline at Week 28(n=0,10,9)	9999 ± 9999	-3.9 ± 3.96	-3.1 ± 3.37
Change From Baseline at Week 32(n=0,9,8)	9999 ± 9999	-2.3 ± 4.61	-2.8 ± 3.99

Statistical Analysis 2

Statistical analysis description

Change From Baseline at Week 4

Comparison groups

TAK-079 Placebo-matching v TAK-079 600 mg

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.299 ^[2]

Method

MMRM

Parameter type

Difference in LS Mean

Point estimate

1.01

Confidence interval

level

95%

sides

2-sided

lower limit

-0.94

upper limit

2.97

Notes
[2] - From MMRM analysis over all post-baseline visits, with the change from baseline as the outcome, treatment group, visit, and treatment-by-visit interaction as factors, and adjusted by baseline value and baseline-by-visit interaction.

Statistical Analysis 1

Statistical analysis description

Change From Baseline at Week 4

Comparison groups

TAK-079 300 mg v TAK-079 Placebo-matching

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.772 ^[3]

Method

MMRM

Parameter type

Difference in Least Square (LS) Mean

Point estimate

0.29

Confidence interval

level

95%

sides

2-sided

lower limit

-1.72

upper limit

2.3

Notes
[3] - From MMRM analysis over all post-baseline visits, with the change from baseline as the outcome, treatment group, visit, and treatment-by-visit interaction as factors, and adjusted by baseline value and baseline-by-visit interaction.

Statistical Analysis 5

Statistical analysis description

Change From Baseline at Week 8

Comparison groups

TAK-079 Placebo-matching v TAK-079 300 mg

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.887 ^[4]

Method

MMRM

Parameter type

Difference in LS Mean

Point estimate

-0.18

Confidence interval

level

95%

sides

2-sided

lower limit

-2.82

upper limit

2.45

Notes
[4] - From MMRM analysis over all post-baseline visits, with the change from baseline as the outcome, treatment group, visit, and treatment-by-visit interaction as factors, and adjusted by baseline value and baseline-by-visit interaction.

Statistical Analysis 6

Statistical analysis description

Change From Baseline at Week 8

Comparison groups

TAK-079 Placebo-matching v TAK-079 600 mg

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.162 ^[5]

Method

MMRM

Parameter type

Difference in LS Mean

Point estimate

1.8

Confidence interval

level

95%

sides

2-sided

lower limit

-0.76

upper limit

4.36

Notes
[5] - From MMRM analysis over all post-baseline visits, with the change from baseline as the outcome, treatment group, visit, and treatment-by-visit interaction as factors, and adjusted by baseline value and baseline-by-visit interaction.

Statistical Analysis 4

Statistical analysis description

Change From Baseline at Week 6

Comparison groups

TAK-079 Placebo-matching v TAK-079 600 mg

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.091 ^[6]

Method

MMRM

Parameter type

Difference in LS Mean

Point estimate

1.86

Confidence interval

level

95%

sides

2-sided

lower limit

-0.32

upper limit

4.04

Notes
[6] - From MMRM analysis over all post-baseline visits, with the change from baseline as the outcome, treatment group, visit, and treatment-by-visit interaction as factors, and adjusted by baseline value and baseline-by-visit interaction.

Statistical Analysis 3

Statistical analysis description

Change From Baseline at Week 6

Comparison groups

TAK-079 Placebo-matching v TAK-079 300 mg

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.924 ^[7]

Method

MMRM

Parameter type

Difference in LS Mean

Point estimate

-0.11

Confidence interval

level

95%

sides

2-sided

lower limit

-2.34

upper limit

2.13

Notes
[7] - From MMRM analysis over all post-baseline visits, with the change from baseline as the outcome, treatment group, visit, and treatment-by-visit interaction as factors, and adjusted by baseline value and baseline-by-visit interaction.

Statistical Analysis 7

Statistical analysis description

Change From Baseline at Week 10

Comparison groups

TAK-079 Placebo-matching v TAK-079 300 mg

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.784 ^[8]

Method

MMRM

Parameter type

Difference in LS Mean

Point estimate

-0.36

Confidence interval

level

95%

sides

2-sided

lower limit

-3.01

upper limit

2.29

Notes
[8] - From MMRM analysis over all post-baseline visits, with the change from baseline as the outcome, treatment group, visit, and treatment-by-visit interaction as factors, and adjusted by baseline value and baseline-by-visit interaction.

Statistical Analysis 9

Statistical analysis description

Change From Baseline at Week 12

Comparison groups

TAK-079 Placebo-matching v TAK-079 300 mg

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.724 ^[9]

Method

MMRM

Parameter type

Difference in LS Mean

Point estimate

0.42

Confidence interval

level

95%

sides

2-sided

lower limit

-1.98

upper limit

2.82

Notes
[9] - From MMRM analysis over all post-baseline visits, with the change from baseline as the outcome, treatment group, visit, and treatment-by-visit interaction as factors, and adjusted by baseline value and baseline-by-visit interaction.

Statistical Analysis 8

Statistical analysis description

Change From Baseline at Week 10

Comparison groups

TAK-079 Placebo-matching v TAK-079 600 mg

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.166 ^[10]

Method

MMRM

Parameter type

Difference in LS Mean

Point estimate

1.79

Confidence interval

level

95%

sides

2-sided

lower limit

-0.79

upper limit

4.37

Notes
[10] - From MMRM analysis over all post-baseline visits, with the change from baseline as the outcome, treatment group, visit, and treatment-by-visit interaction as factors, and adjusted by baseline value and baseline-by-visit interaction.

Statistical Analysis 10

Statistical analysis description

Change From Baseline at Week 12

Comparison groups

TAK-079 Placebo-matching v TAK-079 600 mg

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.124 ^[11]

Method

MMRM

Parameter type

Difference in LS Mean

Point estimate

1.81

Confidence interval

level

95%

sides

2-sided

lower limit

-0.52

upper limit

4.14

Notes
[11] - From MMRM analysis over all post-baseline visits, with the change from baseline as the outcome, treatment group, visit, and treatment-by-visit interaction as factors, and adjusted by baseline value and baseline-by-visit interaction.

Statistical Analysis 11

Statistical analysis description

Change From Baseline at Week 14

Comparison groups

TAK-079 Placebo-matching v TAK-079 300 mg

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.856 ^[12]

Method

MMRM

Parameter type

Difference in LS Mean

Point estimate

0.29

Confidence interval

level

95%

sides

2-sided

lower limit

-2.91

upper limit

3.48

Notes
[12] - From MMRM analysis over all post-baseline visits, with the change from baseline as the outcome, treatment group, visit, and treatment-by-visit interaction as factors, and adjusted by baseline value and baseline-by-visit interaction.

Statistical Analysis 13

Statistical analysis description

Change From Baseline at Week 16

Comparison groups

TAK-079 Placebo-matching v TAK-079 300 mg

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.944 ^[13]

Method

MMRM

Parameter type

Difference in LS Mean

Point estimate

0.11

Confidence interval

level

95%

sides

2-sided

lower limit

-3.14

upper limit

3.37

Notes
[13] - From MMRM analysis over all post-baseline visits, with the change from baseline as the outcome, treatment group, visit, and treatment-by-visit interaction as factors, and adjusted by baseline value and baseline-by-visit interaction.

Statistical Analysis 12

Statistical analysis description

Change From Baseline at Week 14

Comparison groups

TAK-079 Placebo-matching v TAK-079 600 mg

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.292 ^[14]

Method

MMRM

Parameter type

Difference in LS Mean

Point estimate

1.64

Confidence interval

level

95%

sides

2-sided

lower limit

-1.48

upper limit

4.76

Notes
[14] - From MMRM analysis over all post-baseline visits, with the change from baseline as the outcome, treatment group, visit, and treatment-by-visit interaction as factors, and adjusted by baseline value and baseline-by-visit interaction.

Statistical Analysis 14

Statistical analysis description

Change From Baseline at Week 16

Comparison groups

TAK-079 Placebo-matching v TAK-079 600 mg

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.589 ^[15]

Method

MMRM

Parameter type

Difference in LS Mean

Point estimate

0.85

Confidence interval

level

95%

sides

2-sided

lower limit

-2.33

upper limit

4.02

Notes
[15] - From MMRM analysis over all post-baseline visits, with the change from baseline as the outcome, treatment group, visit, and treatment-by-visit interaction as factors, and adjusted by baseline value and baseline-by-visit interaction.

Secondary: Change From Baseline in Quantitative Myasthenia Gravis (QMG) Scale Score

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End point title

Change From Baseline in Quantitative Myasthenia Gravis (QMG) Scale Score

End point description

Physician-reported scale to assess MG disease severity by quantifying several body functions by physical exam. Each question is graded on a 4-point scale from 0=normal to 3=severe with a total score of 0 to 39; the higher score indicates greater disease burden. Negative change indicates improvement. MMRM was used for the analysis. Full Analysis Set included all randomised participants who had baseline and at least 1 post-baseline efficacy assessment. n=Number analysed is the number of participants available for analysis at the specified timepoint. 9999=Dta was not collected for 0 participants.

End point type

Secondary

End point timeframe

Baseline up to Week 32

End point values	TAK-079 Placebo-matching	TAK-079 300 mg	TAK-079 600 mg
Number of subjects analysed	12	12	12
Units: score on a scale
arithmetic mean (standard deviation)
Change From Baseline at Week 4(n=12,12,12)	-1.0 ± 2.34	-2.0 ± 2.70	-0.2 ± 2.89
Change From Baseline at Week 6(n=11,12,12)	-0.9 ± 1.76	-2.5 ± 3.24	-1.3 ± 3.10
Change From Baseline at Week 8(n=12,11,11)	-1.0 ± 2.30	-3.4 ± 3.35	-0.9 ± 3.27
Change From Baseline at Week 10(n=11,11,11)	-1.4 ± 2.87	-3.2 ± 3.09	-0.7 ± 4.03
Change From Baseline at Week 12(n=11,11,12)	-2.8 ± 2.68	-3.9 ± 2.55	-0.8 ± 3.51
Change From Baseline at Week 14(n=11,10,10)	-1.9 ± 3.11	-3.5 ± 2.51	-0.6 ± 4.65
Change From Baseline at Week 16(n=10,10,10)	-1.2 ± 3.22	-3.3 ± 3.43	-0.3 ± 4.81
Change From Baseline at Week 20(n=0,8,11)	9999 ± 9999	-1.9 ± 3.56	-0.5 ± 4.27
Change From Baseline at Week 24(n=0,9,9)	9999 ± 9999	-2.2 ± 4.47	-0.6 ± 3.78
Change From Baseline at Week 28(n=0,10,9)	9999 ± 9999	-1.7 ± 3.80	-0.8 ± 4.52
Change From Baseline at Week 32(n=0,9,8)	9999 ± 9999	-0.7 ± 4.27	-0.1 ± 4.79

Statistical Analysis 3

Statistical analysis description

Change From Baseline at Week 6

Comparison groups

TAK-079 Placebo-matching v TAK-079 300 mg

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.463 ^[16]

Method

MMRM

Parameter type

Difference in LS Mean

Point estimate

-0.94

Confidence interval

level

95%

sides

2-sided

lower limit

-3.53

upper limit

1.65

Notes
[16] - From MMRM analysis over all post-baseline visits, with the change from baseline as the outcome, treatment group, visit, and treatment-by-visit interaction as factors, and adjusted by baseline value and baseline-by-visit interaction.

Statistical Analysis 2

Statistical analysis description

Change From Baseline at Week 4

Comparison groups

TAK-079 Placebo-matching v TAK-079 600 mg

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.418 ^[17]

Method

MMRM

Parameter type

Difference in LS Mean

Point estimate

0.9

Confidence interval

level

95%

sides

2-sided

lower limit

-1.34

upper limit

3.14

Notes
[17] - From MMRM analysis over all post-baseline visits, with the change from baseline as the outcome, treatment group, visit, and treatment-by-visit interaction as factors, and adjusted by baseline value and baseline-by-visit interaction.

Statistical Analysis 1

Statistical analysis description

Change From Baseline at Week 4

Comparison groups

TAK-079 Placebo-matching v TAK-079 300 mg

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.406 ^[18]

Method

MMRM

Parameter type

Difference in LS Mean

Point estimate

-0.93

Confidence interval

level

95%

sides

2-sided

lower limit

-3.17

upper limit

1.31

Notes
[18] - From MMRM analysis over all post-baseline visits, with the change from baseline as the outcome, treatment group, visit, and treatment-by-visit interaction as factors, and adjusted by baseline value and baseline-by-visit interaction.

Statistical Analysis 6

Statistical analysis description

Change From Baseline at Week 8

Comparison groups

TAK-079 Placebo-matching v TAK-079 600 mg

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.687 ^[19]

Method

MMRM

Parameter type

Difference in LS Mean

Point estimate

0.52

Confidence interval

level

95%

sides

2-sided

lower limit

-2.1

upper limit

3.13

Notes
[19] - From MMRM analysis over all post-baseline visits, with the change from baseline as the outcome, treatment group, visit, and treatment-by-visit interaction as factors, and adjusted by baseline value and baseline-by-visit interaction.

Statistical Analysis 5

Statistical analysis description

Change From Baseline at Week 8

Comparison groups

TAK-079 Placebo-matching v TAK-079 300 mg

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.17 ^[20]

Method

MMRM

Parameter type

Difference in LS Mean

Point estimate

-1.8

Confidence interval

level

95%

sides

2-sided

lower limit

-4.41

upper limit

0.82

Notes
[20] - From MMRM analysis over all post-baseline visits, with the change from baseline as the outcome, treatment group, visit, and treatment-by-visit interaction as factors, and adjusted by baseline value and baseline-by-visit interaction.

Statistical Analysis 4

Statistical analysis description

Change From Baseline at Week 6

Comparison groups

TAK-079 Placebo-matching v TAK-079 600 mg

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.936 ^[21]

Method

MMRM

Parameter type

Difference in LS Mean

Point estimate

-0.1

Confidence interval

level

95%

sides

2-sided

lower limit

-2.7

upper limit

2.49

Notes
[21] - From MMRM analysis over all post-baseline visits, with the change from baseline as the outcome, treatment group, visit, and treatment-by-visit interaction as factors, and adjusted by baseline value and baseline-by-visit interaction.

Statistical Analysis 7

Statistical analysis description

Change From Baseline at Week 10

Comparison groups

TAK-079 Placebo-matching v TAK-079 300 mg

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.374 ^[22]

Method

MMRM

Parameter type

Difference in LS Mean

Point estimate

-1.24

Confidence interval

level

95%

sides

2-sided

lower limit

-4.05

upper limit

1.57

Notes
[22] - From MMRM analysis over all post-baseline visits, with the change from baseline as the outcome, treatment group, visit, and treatment-by-visit interaction as factors, and adjusted by baseline value and baseline-by-visit interaction.

Statistical Analysis 10

Statistical analysis description

Change From Baseline at Week 12

Comparison groups

TAK-079 Placebo-matching v TAK-079 600 mg

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.106 ^[23]

Method

MMRM

Parameter type

Difference in LS Mean

Point estimate

2.12

Confidence interval

level

95%

sides

2-sided

lower limit

-0.48

upper limit

4.71

Notes
[23] - From MMRM analysis over all post-baseline visits, with the change from baseline as the outcome, treatment group, visit, and treatment-by-visit interaction as factors, and adjusted by baseline value and baseline-by-visit interaction.

Statistical Analysis 9

Statistical analysis description

Change From Baseline at Week 12

Comparison groups

TAK-079 Placebo-matching v TAK-079 300 mg

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.69 ^[24]

Method

MMRM

Parameter type

Difference in LS Mean

Point estimate

-0.51

Confidence interval

level

95%

sides

2-sided

lower limit

-3.12

upper limit

2.09

Notes
[24] - From MMRM analysis over all post-baseline visits, with the change from baseline as the outcome, treatment group, visit, and treatment-by-visit interaction as factors, and adjusted by baseline value and baseline-by-visit interaction.

Statistical Analysis 8

Statistical analysis description

Change From Baseline at Week 10

Comparison groups

TAK-079 Placebo-matching v TAK-079 600 mg

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.478 ^[25]

Method

MMRM

Parameter type

Difference in LS Mean

Point estimate

0.99

Confidence interval

level

95%

sides

2-sided

lower limit

-1.82

upper limit

3.8

Notes
[25] - From MMRM analysis over all post-baseline visits, with the change from baseline as the outcome, treatment group, visit, and treatment-by-visit interaction as factors, and adjusted by baseline value and baseline-by-visit interaction.

Statistical Analysis 13

Statistical analysis description

Change From Baseline at Week 16

Comparison groups

TAK-079 Placebo-matching v TAK-079 300 mg

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.431 ^[26]

Method

MMRM

Parameter type

Difference in LS Mean

Point estimate

-1.37

Confidence interval

level

95%

sides

2-sided

lower limit

-4.91

upper limit

2.17

Notes
[26] - From MMRM analysis over all post-baseline visits, with the change from baseline as the outcome, treatment group, visit, and treatment-by-visit interaction as factors, and adjusted by baseline value and baseline-by-visit interaction.

Statistical Analysis 12

Statistical analysis description

Change From Baseline at Week 14

Comparison groups

TAK-079 Placebo-matching v TAK-079 600 mg

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.144 ^[27]

Method

MMRM

Parameter type

Difference in LS Mean

Point estimate

2.19

Confidence interval

level

95%

sides

2-sided

lower limit

-0.81

upper limit

5.18

Notes
[27] - From MMRM analysis over all post-baseline visits, with the change from baseline as the outcome, treatment group, visit, and treatment-by-visit interaction as factors, and adjusted by baseline value and baseline-by-visit interaction.

Statistical Analysis 11

Statistical analysis description

Change From Baseline at Week 14

Comparison groups

TAK-079 Placebo-matching v TAK-079 300 mg

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.547 ^[28]

Method

MMRM

Parameter type

Difference in LS Mean

Point estimate

-0.89

Confidence interval

level

95%

sides

2-sided

lower limit

-3.89

upper limit

2.12

Notes
[28] - From MMRM analysis over all post-baseline visits, with the change from baseline as the outcome, treatment group, visit, and treatment-by-visit interaction as factors, and adjusted by baseline value and baseline-by-visit interaction.

Statistical Analysis 14

Statistical analysis description

Change From Baseline at Week 16

Comparison groups

TAK-079 Placebo-matching v TAK-079 600 mg

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.488

Method

MMRM

Parameter type

Difference in LS Mean

Point estimate

1.2

Confidence interval

level

95%

sides

2-sided

lower limit

-2.34

upper limit

4.74

Secondary: Change From Baseline in Myasthenia Gravis Composite (MGC) Scale Score

Top of page

End point title

Change From Baseline in Myasthenia Gravis Composite (MGC) Scale Score

End point description

An assessment scale of MG disease activity based on a combination of participant- and physician-reported items. Each question is graded on 4 levels of impact from normal to severe with a total score of 0 to 50; the higher score indicates worse MG disease activity. Negative change indicates improvement. MMRM was used for the analysis. Full Analysis Set included all randomised participants who had baseline and at least 1 post-baseline efficacy assessment. n=Number analysed is the number of participants available for analysis at the specified timepoint. 9999=Data was not reported for 0 participants.

End point type

Secondary

End point timeframe

Baseline up to Week 32

End point values	TAK-079 Placebo-matching	TAK-079 300 mg	TAK-079 600 mg
Number of subjects analysed	12	12	12
Units: score on a scale
arithmetic mean (standard deviation)
Change From Baseline at Week 4(n=12,12,12)	-4.2 ± 3.69	-4.9 ± 3.82	-1.1 ± 5.92
Change From Baseline at Week 6(n=12,11,11)	-7.3 ± 4.29	-6.1 ± 5.24	-4.9 ± 4.74
Change From Baseline at Week 8(n=11,11,12)	-5.5 ± 4.03	-7.4 ± 4.63	-2.4 ± 6.10
Change From Baseline at Week 10(n=11,11,11)	-7.8 ± 5.86	-7.6 ± 5.07	-2.6 ± 6.55
Change From Baseline at Week 12(n=11,11,12)	-7.3 ± 5.00	-8.5 ± 3.11	-5.1 ± 5.11
Change From Baseline at Week 14(n=11,10,10)	-6.1 ± 6.52	-9.7 ± 3.83	-0.9 ± 8.25
Change From Baseline at Week 16(n=10,10,10)	-6.6 ± 4.95	-9.2 ± 5.18	-2.9 ± 6.45
Change From Baseline at Week 20(n=0,8,11)	9999 ± 9999	-7.3 ± 5.20	-2.6 ± 7.21
Change From Baseline at Week 24(n=0,9,9)	9999 ± 9999	-7.1 ± 5.78	-3.3 ± 7.30
Change From Baseline at Week 28(n=0,10,9)	9999 ± 9999	-5.6 ± 6.40	-4.1 ± 7.59
Change From Baseline at Week 32(n=0,9,8)	9999 ± 9999	-3.4 ± 8.13	-2.5 ± 8.07

Statistical Analysis 1

Statistical analysis description

Change From Baseline at Week 4

Comparison groups

TAK-079 Placebo-matching v TAK-079 300 mg

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.855 ^[29]

Method

MMRM

Parameter type

Difference in LS Mean

Point estimate

-0.34

Confidence interval

level

95%

sides

2-sided

lower limit

-4.14

upper limit

3.45

Notes
[29] - From MMRM analysis over all post-baseline visits, with the change from baseline as the outcome, treatment group, visit, and treatment-by-visit interaction as factors, and adjusted by baseline value and baseline-by-visit interaction.

Statistical Analysis 6

Statistical analysis description

Change From Baseline at Week 8

Comparison groups

TAK-079 Placebo-matching v TAK-079 600 mg

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.13 ^[30]

Method

MMRM

Parameter type

Difference in LS Mean

Point estimate

3.26

Confidence interval

level

95%

sides

2-sided

lower limit

-1.02

upper limit

7.54

Notes
[30] - From MMRM analysis over all post-baseline visits, with the change from baseline as the outcome, treatment group, visit, and treatment-by-visit interaction as factors, and adjusted by baseline value and baseline-by-visit interaction.

Statistical Analysis 5

Statistical analysis description

Change From Baseline at Week 8

Comparison groups

TAK-079 Placebo-matching v TAK-079 300 mg

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.662 ^[31]

Method

MMRM

Parameter type

Difference in LS Mean

Point estimate

-0.94

Confidence interval

level

95%

sides

2-sided

lower limit

-5.29

upper limit

3.41

Notes
[31] - From MMRM analysis over all post-baseline visits, with the change from baseline as the outcome, treatment group, visit, and treatment-by-visit interaction as factors, and adjusted by baseline value and baseline-by-visit interaction.

Statistical Analysis 4

Statistical analysis description

Change From Baseline at Week 6

Comparison groups

TAK-079 Placebo-matching v TAK-079 600 mg

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.154 ^[32]

Method

MMRM

Parameter type

Difference in LS Mean

Point estimate

3.13

Confidence interval

level

95%

sides

2-sided

lower limit

-1.25

upper limit

7.5

Notes
[32] - From MMRM analysis over all post-baseline visits, with the change from baseline as the outcome, treatment group, visit, and treatment-by-visit interaction as factors, and adjusted by baseline value and baseline-by-visit interaction.

Statistical Analysis 3

Statistical analysis description

Change From Baseline at Week 6

Comparison groups

TAK-079 Placebo-matching v TAK-079 300 mg

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.41 ^[33]

Method

MMRM

Parameter type

Difference in LS Mean

Point estimate

1.8

Confidence interval

level

95%

sides

2-sided

lower limit

-2.61

upper limit

6.2

Notes
[33] - From MMRM analysis over all post-baseline visits, with the change from baseline as the outcome, treatment group, visit, and treatment-by-visit interaction as factors, and adjusted by baseline value and baseline-by-visit interaction.

Statistical Analysis 2

Statistical analysis description

Change From Baseline at Week 4

Comparison groups

TAK-079 Placebo-matching v TAK-079 600 mg

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.093 ^[34]

Method

MMRM

Parameter type

Difference in LS Mean

Point estimate

3.19

Confidence interval

level

95%

sides

2-sided

lower limit

-0.56

upper limit

6.95

Notes
[34] - From MMRM analysis over all post-baseline visits, with the change from baseline as the outcome, treatment group, visit, and treatment-by-visit interaction as factors, and adjusted by baseline value and baseline-by-visit interaction.

Statistical Analysis 13

Statistical analysis description

Change From Baseline at Week 16

Comparison groups

TAK-079 Placebo-matching v TAK-079 300 mg

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.677 ^[35]

Method

MMRM

Parameter type

Difference in LS Mean

Point estimate

-1.01

Confidence interval

level

95%

sides

2-sided

lower limit

-5.98

upper limit

3.95

Notes
[35] - From MMRM analysis over all post-baseline visits, with the change from baseline as the outcome, treatment group, visit, and treatment-by-visit interaction as factors, and adjusted by baseline value and baseline-by-visit interaction.

Statistical Analysis 12

Statistical analysis description

Change From Baseline at Week 14

Comparison groups

TAK-079 Placebo-matching v TAK-079 600 mg

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.08 ^[36]

Method

MMRM

Parameter type

Difference in LS Mean

Point estimate

5.2

Confidence interval

level

95%

sides

2-sided

lower limit

-0.67

upper limit

11.06

Notes
[36] - From MMRM analysis over all post-baseline visits, with the change from baseline as the outcome, treatment group, visit, and treatment-by-visit interaction as factors, and adjusted by baseline value and baseline-by-visit interaction.

Statistical Analysis 11

Statistical analysis description

Change From Baseline at Week 14

Comparison groups

TAK-079 Placebo-matching v TAK-079 300 mg

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.764 ^[37]

Method

MMRM

Parameter type

Difference in LS Mean

Point estimate

-0.88

Confidence interval

level

95%

sides

2-sided

lower limit

-6.8

upper limit

5.05

Notes
[37] - From MMRM analysis over all post-baseline visits, with the change from baseline as the outcome, treatment group, visit, and treatment-by-visit interaction as factors, and adjusted by baseline value and baseline-by-visit interaction.

Statistical Analysis 10

Statistical analysis description

Change From Baseline at Week 12

Comparison groups

TAK-079 Placebo-matching v TAK-079 600 mg

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.158 ^[38]

Method

MMRM

Parameter type

Difference in LS Mean

Point estimate

2.65

Confidence interval

level

95%

sides

2-sided

lower limit

-1.09

upper limit

6.4

Notes
[38] - From MMRM analysis over all post-baseline visits, with the change from baseline as the outcome, treatment group, visit, and treatment-by-visit interaction as factors, and adjusted by baseline value and baseline-by-visit interaction.

Statistical Analysis 9

Statistical analysis description

Change From Baseline at Week 12

Comparison groups

TAK-079 Placebo-matching v TAK-079 300 mg

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.909 ^[39]

Method

MMRM

Parameter type

Difference in LS Mean

Point estimate

-0.22

Confidence interval

level

95%

sides

2-sided

lower limit

-4.03

upper limit

3.6

Notes
[39] - From MMRM analysis over all post-baseline visits, with the change from baseline as the outcome, treatment group, visit, and treatment-by-visit interaction as factors, and adjusted by baseline value and baseline-by-visit interaction.

Ststistical Analysis 8

Statistical analysis description

Change From Baseline at Week 10

Comparison groups

TAK-079 Placebo-matching v TAK-079 600 mg

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.039 ^[40]

Method

MMRM

Parameter type

Difference in LS Mean

Point estimate

4.93

Confidence interval

level

95%

sides

2-sided

lower limit

0.26

upper limit

9.6

Notes
[40] - From MMRM analysis over all post-baseline visits, with the change from baseline as the outcome, treatment group, visit, and treatment-by-visit interaction as factors, and adjusted by baseline value and baseline-by-visit interaction.

Statistical Analysis 14

Statistical analysis description

Change From Baseline at Week 16

Comparison groups

TAK-079 Placebo-matching v TAK-079 600 mg

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.055 ^[41]

Method

MMRM

Parameter type

Difference in LS Mean

Point estimate

4.81

Confidence interval

level

95%

sides

2-sided

lower limit

-0.1

upper limit

9.73

Notes
[41] - From MMRM analysis over all post-baseline visits, with the change from baseline as the outcome, treatment group, visit, and treatment-by-visit interaction as factors, and adjusted by baseline value and baseline-by-visit interaction.

Statistical Analysis 7

Statistical analysis description

Change From Baseline at Week 10

Comparison groups

TAK-079 Placebo-matching v TAK-079 300 mg

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.586 ^[42]

Method

MMRM

Parameter type

Difference in LS Mean

Point estimate

1.27

Confidence interval

level

95%

sides

2-sided

lower limit

-3.44

upper limit

5.98

Notes
[42] - From MMRM analysis over all post-baseline visits, with the change from baseline as the outcome, treatment group, visit, and treatment-by-visit interaction as factors, and adjusted by baseline value and baseline-by-visit interaction.

Secondary: Change From Baseline in Revised 15-item Myasthenia Gravis Quality of Life Scale (MG-QoL15r) Scale Score

Top of page

End point title

Change From Baseline in Revised 15-item Myasthenia Gravis Quality of Life Scale (MG-QoL15r) Scale Score

End point description

A participant-reported score that assesses the participant's perception of impairment and disability and the degree to which the participant tolerates disease manifestations. Each question is graded on a 3-point scale from 0=normal to 2=severe with a total score of 0 to 30; the higher score indicates worse MG disease activity. Negative change indicates improvement. MMRM was used for the analysis. Full Analysis Set included all randomised participants who had baseline and at least 1 post-baseline efficacy assessment. n=Number analysed is the number of participants available for analysis at the specified timepoint.

End point type

Secondary

End point timeframe

Baseline up to Week 32

End point values	TAK-079 Placebo-matching	TAK-079 300 mg	TAK-079 600 mg
Number of subjects analysed	12	12	12
Units: score on a scale
arithmetic mean (standard deviation)
Change From Baseline at Week 4(n=12,12,12)	-3.1 ± 3.85	-3.9 ± 4.46	-3.2 ± 4.43
Change From Baseline at Week 6(n=12,11,11)	-4.3 ± 4.11	-5.8 ± 6.00	-3.4 ± 3.53
Change From Baseline at Week 8(n=12,11,12)	-3.2 ± 4.00	-5.6 ± 6.53	-3.3 ± 3.74
Change From Baseline at Week 10(n=11,11,11)	-2.5 ± 6.12	-6.3 ± 6.07	-3.9 ± 3.70
Change From Baseline at Week 12(n=11,11,12)	-2.5 ± 5.32	-6.1 ± 7.25	-4.1 ± 4.40
Change From Baseline at Week 14(n=11,10,10)	-2.0 ± 7.06	-6.4 ± 6.98	-0.9 ± 5.49
Change From Baseline at Week 16(n=10,10,10)	-3.8 ± 4.44	-5.8 ± 6.83	-2.3 ± 6.43
Change From Baseline at Week 20(n=0,8,11)	9999 ± 9999	-3.3 ± 7.01	-4.7 ± 5.48
Change From Baseline at Week 24(n=0,8,9)	9999 ± 9999	-7.1 ± 7.95	-3.8 ± 5.31
Change From Baseline at Week 28(n=0,10,8)	9999 ± 9999	-4.8 ± 10.36	-4.0 ± 6.16
Change From Baseline at Week 32(n=0,9,8)	9999 ± 9999	-6.2 ± 7.31	-2.4 ± 4.78

Statistical Analysis 1

Statistical analysis description

Change From Baseline at Week 4

Comparison groups

TAK-079 Placebo-matching v TAK-079 300 mg

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.78 ^[43]

Method

MMRM

Parameter type

Difference in LS Mean

Point estimate

0.52

Confidence interval

level

95%

sides

2-sided

lower limit

-3.23

upper limit

4.27

Notes
[43] - From a MMRM analysis over all post-baseline visits, with the change from baseline as the outcome, treatment group, visit, and treatment-by-visit interaction as factors, and adjusted by baseline value and baseline-by-visit interaction.

Statistical Analysis 3

Statistical analysis description

Change From Baseline at Week 6

Comparison groups

TAK-079 Placebo-matching v TAK-079 300 mg

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.984 ^[44]

Method

MMRM

Parameter type

Difference in LS Mean

Point estimate

-0.04

Confidence interval

level

95%

sides

2-sided

lower limit

-4.22

upper limit

4.14

Notes
[44] - From a MMRM analysis over all post-baseline visits, with the change from baseline as the outcome, treatment group, visit, and treatment-by-visit interaction as factors, and adjusted by baseline value and baseline-by-visit interaction.

Statistical Analysis 4

Statistical analysis description

Change From Baseline at Week 6

Comparison groups

TAK-079 Placebo-matching v TAK-079 600 mg

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.213 ^[45]

Method

MMRM

Parameter type

Difference in LS Mean

Point estimate

2.44

Confidence interval

level

95%

sides

2-sided

lower limit

-1.47

upper limit

6.35

Notes
[45] - From a MMRM analysis over all post-baseline visits, with the change from baseline as the outcome, treatment group, visit, and treatment-by-visit interaction as factors, and adjusted by baseline value and baseline-by-visit interaction.

Statistical Analysis 5

Statistical analysis description

Change From Baseline at Week 8

Comparison groups

TAK-079 Placebo-matching v TAK-079 300 mg

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.371 ^[46]

Method

MMRM

Parameter type

Difference in LS Mean

Point estimate

-1.97

Confidence interval

level

95%

sides

2-sided

lower limit

-6.4

upper limit

2.45

Notes
[46] - From a MMRM analysis over all post-baseline visits, with the change from baseline as the outcome, treatment group, visit, and treatment-by-visit interaction as factors, and adjusted by baseline value and baseline-by-visit interaction.

Statistical Analysis 6

Statistical analysis description

Change From Baseline at Week 8

Comparison groups

TAK-079 Placebo-matching v TAK-079 600 mg

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.956 ^[47]

Method

MMRM

Parameter type

Difference in LS Mean

Point estimate

0.11

Confidence interval

level

95%

sides

2-sided

lower limit

-4.01

upper limit

4.23

Notes
[47] - From a MMRM analysis over all post-baseline visits, with the change from baseline as the outcome, treatment group, visit, and treatment-by-visit interaction as factors, and adjusted by baseline value and baseline-by-visit interaction.

Statistical Analysis 2

Statistical analysis description

Change From Baseline at Week 4

Comparison groups

TAK-079 Placebo-matching v TAK-079 600 mg

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.771 ^[48]

Method

MMRM

Parameter type

Difference in LS Mean

Point estimate

0.5

Confidence interval

level

95%

sides

2-sided

lower limit

-2.99

upper limit

3.99

Notes
[48] - From a MMRM analysis over all post-baseline visits, with the change from baseline as the outcome, treatment group, visit, and treatment-by-visit interaction as factors, and adjusted by baseline value and baseline-by-visit interaction.

Statistical Analysis 7

Statistical analysis description

Change From Baseline at Week 10

Comparison groups

TAK-079 Placebo-matching v TAK-079 300 mg

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.641 ^[49]

Method

MMRM

Parameter type

Difference in LS Mean

Point estimate

-1.06

Confidence interval

level

95%

sides

2-sided

lower limit

-5.65

upper limit

3.53

Notes
[49] - From a MMRM analysis over all post-baseline visits, with the change from baseline as the outcome, treatment group, visit, and treatment-by-visit interaction as factors, and adjusted by baseline value and baseline-by-visit interaction.

Statistical Analysis 11

Statistical analysis description

Change From Baseline at Week 14

Comparison groups

TAK-079 Placebo-matching v TAK-079 300 mg

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.699 ^[50]

Method

MMRM

Parameter type

Difference in LS Mean

Point estimate

-1.14

Confidence interval

level

95%

sides

2-sided

lower limit

-7.07

upper limit

4.8

Notes
[50] - From a MMRM analysis over all post-baseline visits, with the change from baseline as the outcome, treatment group, visit, and treatment-by-visit interaction as factors, and adjusted by baseline value and baseline-by-visit interaction.

Statistical Analysis 10

Statistical analysis description

Change From Baseline at Week 12

Comparison groups

TAK-079 Placebo-matching v TAK-079 600 mg

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.769 ^[51]

Method

MMRM

Parameter type

Difference in LS Mean

Point estimate

-0.68

Confidence interval

level

95%

sides

2-sided

lower limit

-5.37

upper limit

Notes
[51] - From a MMRM analysis over all post-baseline visits, with the change from baseline as the outcome, treatment group, visit, and treatment-by-visit interaction as factors, and adjusted by baseline value and baseline-by-visit interaction.

Statistical Analysis 8

Statistical analysis description

Change From Baseline at Week 10

Comparison groups

TAK-079 Placebo-matching v TAK-079 600 mg

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.876 ^[52]

Method

MMRM

Parameter type

Difference in LS Mean

Point estimate

-0.33

Confidence interval

level

95%

sides

2-sided

lower limit

-4.61

upper limit

3.95

Notes
[52] - From a MMRM analysis over all post-baseline visits, with the change from baseline as the outcome, treatment group, visit, and treatment-by-visit interaction as factors, and adjusted by baseline value and baseline-by-visit interaction.

Statistical Analysis 9

Statistical analysis description

Change From Baseline at Week 12

Comparison groups

TAK-079 Placebo-matching v TAK-079 300 mg

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.458 ^[53]

Method

MMRM

Parameter type

Difference in LS Mean

Point estimate

-1.86

Confidence interval

level

95%

sides

2-sided

lower limit

-6.91

upper limit

3.18

Notes
[53] - From a MMRM analysis over all post-baseline visits, with the change from baseline as the outcome, treatment group, visit, and treatment-by-visit interaction as factors, and adjusted by baseline value and baseline-by-visit interaction.

Statistical Analysis 13

Statistical analysis description

Change From Baseline at Week 16

Comparison groups

TAK-079 Placebo-matching v TAK-079 300 mg

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.944 ^[54]

Method

MMRM

Parameter type

Difference in LS Mean

Point estimate

0.2

Confidence interval

level

95%

sides

2-sided

lower limit

-5.55

upper limit

5.95

Notes
[54] - From a MMRM analysis over all post-baseline visits, with the change from baseline as the outcome, treatment group, visit, and treatment-by-visit interaction as factors, and adjusted by baseline value and baseline-by-visit interaction.

Statistical Analysis 12

Statistical analysis description

Change From Baseline at Week 14

Comparison groups

TAK-079 Placebo-matching v TAK-079 600 mg

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.687 ^[55]

Method

MMRM

Parameter type

Difference in LS Mean

Point estimate

1.1

Confidence interval

level

95%

sides

2-sided

lower limit

-4.41

upper limit

6.62

Notes
[55] - From a MMRM analysis over all post-baseline visits, with the change from baseline as the outcome, treatment group, visit, and treatment-by-visit interaction as factors, and adjusted by baseline value and baseline-by-visit interaction.

Statistical Analysis 14

Statistical analysis description

Change From Baseline at Week 16

Comparison groups

TAK-079 Placebo-matching v TAK-079 600 mg

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.351 ^[56]

Method

MMRM

Parameter type

Difference in LS Mean

Point estimate

2.48

Confidence interval

level

95%

sides

2-sided

lower limit

-2.87

upper limit

7.83

Notes
[56] - From a MMRM analysis over all post-baseline visits, with the change from baseline as the outcome, treatment group, visit, and treatment-by-visit interaction as factors, and adjusted by baseline value and baseline-by-visit interaction.

Secondary: Change From Baseline in Anti-acetylcholine Receptor (AChR) Antibody Levels

Top of page

End point title

Change From Baseline in Anti-acetylcholine Receptor (AChR) Antibody Levels

End point description

Clinical laboratory evaluations of anti-AChR antibodies were tested to monitor disease activity. MMRM was used for the analysis. Full Analysis Set included all randomised participants who had baseline and at least 1 post-baseline efficacy assessment. Subjects analysed is the number of participants available for analyses. n=Number analysed is the number of participants available for analysis at the specified timepoint. 9999=Data not reported for 0 participants.

End point type

Secondary

End point timeframe

Baseline up to Week 32

End point values	TAK-079 Placebo-matching	TAK-079 300 mg	TAK-079 600 mg
Number of subjects analysed	11	10	12
Units: nmol/L
arithmetic mean (standard deviation)
Change From Baseline at Week 2(n=11,10,12)	-6.300 ± 25.3514	-19.452 ± 50.5551	-3.450 ± 5.7767
Change From Baseline at Week 3(n=11,10,12)	-3.989 ± 22.9732	-14.821 ± 44.4776	-2.509 ± 17.5221
Change From Baseline at Week 4(n=11,10,12)	-0.976 ± 11.7759	-24.253 ± 51.2627	-13.680 ± 87.1669
Change From Baseline at Week 5(n=11,10,12)	-8.199 ± 13.9571	-32.251 ± 58.3701	-9.831 ± 26.2188
Change From Baseline at Week 6(n=11,9,11)	-19.152 ± 49.2221	-27.518 ± 52.6672	-2.148 ± 23.2543
Change From Baseline at Week 7(n=11,9,11)	-11.235 ± 26.2159	-33.261 ± 56.3495	-9.882 ± 16.2972
Change From Baseline at Week 8(n=10,9,12)	-7.731 ± 27.8994	-39.386 ± 72.3110	-5.668 ± 10.4493
Change From Baseline at Week 10(n=10,9,11)	-37.473 ± 120.9186	-37.083 ± 60.8092	-6.654 ± 7.8236
Change From Baseline at Week 12(n=10,9,12)	-0.832 ± 31.0318	-38.430 ± 64.4594	-7.827 ± 11.2120
Change From Baseline at Week 14(n=9,8,10)	-12.729 ± 49.8823	-40.771 ± 74.3624	-8.058 ± 15.8407
Change From Baseline at Week 16(n=7,8,10)	-3.501 ± 9.8729	-49.843 ± 77.6316	-4.909 ± 8.8478
Change From Baseline at Week 20(n=0,7,11)	9999 ± 9999	-52.217 ± 83.1537	-4.378 ± 7.7297
Change From Baseline at Week 24(n=0,7,9)	9999 ± 9999	-43.079 ± 77.4438	-4.802 ± 10.5639
Change From Baseline at Week 28(n=0,8,9)	9999 ± 9999	-73.786 ± 111.0044	-6.044 ± 11.3755
Change From Baseline at Week 32(n=0,7,8)	9999 ± 9999	-51.296 ± 67.3437	-5.485 ± 11.8380

Statistical Analysis 2

Statistical analysis description

Change From Baseline at Week 2

Comparison groups

TAK-079 Placebo-matching v TAK-079 600 mg

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.328 ^[57]

Method

MMRM

Parameter type

Difference in LS Mean

Point estimate

-15.77

Confidence interval

level

95%

sides

2-sided

lower limit

-47.5

upper limit

15.96

Notes
[57] - From MMRM analysis over all post-baseline visits, with the change from baseline as the outcome, treatment group, visit, and treatment-by-visit interaction as factors, and adjusted by baseline value and baseline-by-visit interaction.

Statistical Analysis 1

Statistical analysis description

Change From Baseline at Week 2

Comparison groups

TAK-079 Placebo-matching v TAK-079 300 mg

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.09 ^[58]

Method

MMRM

Parameter type

Difference in LS Mean

Point estimate

-27.9

Confidence interval

level

95%

sides

2-sided

lower limit

-60.18

upper limit

4.38

Notes
[58] - From MMRM analysis over all post-baseline visits, with the change from baseline as the outcome, treatment group, visit, and treatment-by-visit interaction as factors, and adjusted by baseline value and baseline-by-visit interaction.

Statistical Analysis 3

Statistical analysis description

Change From Baseline at Week 3

Comparison groups

TAK-079 Placebo-matching v TAK-079 300 mg

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.441 ^[59]

Method

MMRM

Parameter type

Difference in LS Mean

Point estimate

-12.65

Confidence interval

level

95%

sides

2-sided

lower limit

-44.94

upper limit

19.63

Notes
[59] - From MMRM analysis over all post-baseline visits, with the change from baseline as the outcome, treatment group, visit, and treatment-by-visit interaction as factors, and adjusted by baseline value and baseline-by-visit interaction.

Statistical Analysis 4

Statistical analysis description

Change From Baseline at Week 3

Comparison groups

TAK-079 Placebo-matching v TAK-079 600 mg

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.931 ^[60]

Method

MMRM

Parameter type

Difference in LS Mean

Point estimate

1.39

Confidence interval

level

95%

sides

2-sided

lower limit

-30.33

upper limit

33.12

Notes
[60] - From MMRM analysis over all post-baseline visits, with the change from baseline as the outcome, treatment group, visit, and treatment-by-visit interaction as factors, and adjusted by baseline value and baseline-by-visit interaction.

Statistical Analysis 5

Statistical analysis description

Change From Baseline at Week 4

Comparison groups

TAK-079 Placebo-matching v TAK-079 300 mg

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.087 ^[61]

Method

MMRM

Parameter type

Difference in LS Mean

Point estimate

-28.18

Confidence interval

level

95%

sides

2-sided

lower limit

-60.46

upper limit

4.11

Notes
[61] - From MMRM analysis over all post-baseline visits, with the change from baseline as the outcome, treatment group, visit, and treatment-by-visit interaction as factors, and adjusted by baseline value and baseline-by-visit interaction.

Statistical Analysis 6

Statistical analysis description

Change From Baseline at Week 4

Comparison groups

TAK-079 Placebo-matching v TAK-079 600 mg

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.783 ^[62]

Method

MMRM

Parameter type

Difference in LS Mean

Point estimate

4.44

Confidence interval

level

95%

sides

2-sided

lower limit

-27.29

upper limit

36.16

Notes
[62] - From MMRM analysis over all post-baseline visits, with the change from baseline as the outcome, treatment group, visit, and treatment-by-visit interaction as factors, and adjusted by baseline value and baseline-by-visit interaction.

Statistical Analysis 7

Statistical analysis description

Change From Baseline at Week 5

Comparison groups

TAK-079 Placebo-matching v TAK-079 300 mg

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.077 ^[63]

Method

MMRM

Parameter type

Difference in LS Mean

Point estimate

-29.08

Confidence interval

level

95%

sides

2-sided

lower limit

-61.36

upper limit

3.21

Notes
[63] - From MMRM analysis over all post-baseline visits, with the change from baseline as the outcome, treatment group, visit, and treatment-by-visit interaction as factors, and adjusted by baseline value and baseline-by-visit interaction.

Statistical Analysis 10

Statistical analysis description

Change From Baseline at Week 6

Comparison groups

TAK-079 Placebo-matching v TAK-079 600 mg

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 1 ^[64]

Method

MMRM

Parameter type

Difference in LS Mean

Point estimate

Confidence interval

level

95%

sides

2-sided

lower limit

-32.18

upper limit

32.19

Notes
[64] - From MMRM analysis over all post-baseline visits, with the change from baseline as the outcome, treatment group, visit, and treatment-by-visit interaction as factors, and adjusted by baseline value and baseline-by-visit interaction.

Statistical Analysis 9

Statistical analysis description

Change From Baseline at Week 6

Comparison groups

TAK-079 Placebo-matching v TAK-079 300 mg

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.4 ^[65]

Method

MMRM

Parameter type

Difference in LS Mean

Point estimate

-14.14

Confidence interval

level

95%

sides

2-sided

lower limit

-47.19

upper limit

18.91

Notes
[65] - From MMRM analysis over all post-baseline visits, with the change from baseline as the outcome, treatment group, visit, and treatment-by-visit interaction as factors, and adjusted by baseline value and baseline-by-visit interaction.

Statistical Analysis 8

Statistical analysis description

Change From Baseline at Week 5

Comparison groups

TAK-079 Placebo-matching v TAK-079 600 mg

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.33 ^[66]

Method

MMRM

Parameter type

Difference in LS Mean

Point estimate

-15.72

Confidence interval

level

95%

sides

2-sided

lower limit

-47.45

upper limit

Notes
[66] - From MMRM analysis over all post-baseline visits, with the change from baseline as the outcome, treatment group, visit, and treatment-by-visit interaction as factors, and adjusted by baseline value and baseline-by-visit interaction.

Statistical Analysis 11

Statistical analysis description

Change From Baseline at Week 7

Comparison groups

TAK-079 Placebo-matching v TAK-079 300 mg

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.117 ^[67]

Method

MMRM

Parameter type

Difference in LS Mean

Point estimate

-26.47

Confidence interval

level

95%

sides

2-sided

lower limit

-59.64

upper limit

6.69

Notes
[67] - From MMRM analysis over all post-baseline visits, with the change from baseline as the outcome, treatment group, visit, and treatment-by-visit interaction as factors, and adjusted by baseline value and baseline-by-visit interaction.

Statistical Analysis 15

Statistical analysis description

Change From Baseline at Week 10

Comparison groups

TAK-079 Placebo-matching v TAK-079 300 mg

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.815 ^[68]

Method

MMRM

Parameter type

Difference in LS Mean

Point estimate

-3.97

Confidence interval

level

95%

sides

2-sided

lower limit

-37.34

upper limit

29.4

Notes
[68] - From MMRM analysis over all post-baseline visits, with the change from baseline as the outcome, treatment group, visit, and treatment-by-visit interaction as factors, and adjusted by baseline value and baseline-by-visit interaction.

Statistical Analysis 14

Statistical analysis description

Change From Baseline at Week 8

Comparison groups

TAK-079 Placebo-matching v TAK-079 600 mg

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.215 ^[69]

Method

MMRM

Parameter type

Difference in LS Mean

Point estimate

-20.48

Confidence interval

level

95%

sides

2-sided

lower limit

-52.92

upper limit

11.95

Notes
[69] - From MMRM analysis over all post-baseline visits, with the change from baseline as the outcome, treatment group, visit, and treatment-by-visit interaction as factors, and adjusted by baseline value and baseline-by-visit interaction.

Statistical Analysis 13

Statistical analysis description

Change From Baseline at Week 8

Comparison groups

TAK-079 Placebo-matching v TAK-079 300 mg

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.005 ^[70]

Method

MMRM

Parameter type

Difference in LS Mean

Point estimate

-47.91

Confidence interval

level

95%

sides

2-sided

lower limit

-81.54

upper limit

-14.29

Notes
[70] - From MMRM analysis over all post-baseline visits, with the change from baseline as the outcome, treatment group, visit, and treatment-by-visit interaction as factors, and adjusted by baseline value and baseline-by-visit interaction.

Statistical Analysis 12

Statistical analysis description

Change From Baseline at Week 7

Comparison groups

TAK-079 Placebo-matching v TAK-079 600 mg

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.436 ^[71]

Method

MMRM

Parameter type

Difference in LS Mean

Point estimate

-12.75

Confidence interval

level

95%

sides

2-sided

lower limit

-44.94

upper limit

19.44

Notes
[71] - From MMRM analysis over all post-baseline visits, with the change from baseline as the outcome, treatment group, visit, and treatment-by-visit interaction as factors, and adjusted by baseline value and baseline-by-visit interaction.

Statistical Analysis 16

Statistical analysis description

Change From Baseline at Week 10

Comparison groups

TAK-079 Placebo-matching v TAK-079 600 mg

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.794 ^[72]

Method

MMRM

Parameter type

Difference in LS Mean

Point estimate

-4.31

Confidence interval

level

95%

sides

2-sided

lower limit

-36.84

upper limit

28.22

Notes
[72] - From MMRM analysis over all post-baseline visits, with the change from baseline as the outcome, treatment group, visit, and treatment-by-visit interaction as factors, and adjusted by baseline value and baseline-by-visit interaction.

Statistical Analysis 12

Statistical analysis description

Change From Baseline at Week 12

Comparison groups

TAK-079 Placebo-matching v TAK-079 300 mg

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.033 ^[73]

Method

MMRM

Parameter type

Difference in LS Mean

Point estimate

-36.78

Confidence interval

level

95%

sides

2-sided

lower limit

-70.48

upper limit

-3.08

Notes
[73] - From MMRM analysis over all post-baseline visits, with the change from baseline as the outcome, treatment group, visit, and treatment-by-visit interaction as factors, and adjusted by baseline value and baseline-by-visit interaction.

Statistical Analysis 21

Statistical analysis description

Week 16

Comparison groups

TAK-079 Placebo-matching v TAK-079 300 mg

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.001 ^[74]

Method

MMRM

Parameter type

Difference in LS Mean

Point estimate

-62.98

Confidence interval

level

95%

sides

2-sided

lower limit

-100.64

upper limit

-25.31

Notes
[74] - From MMRM analysis over all post-baseline visits, with the change from baseline as the outcome, treatment group, visit, and treatment-by-visit interaction as factors, and adjusted by baseline value and baseline-by-visit interaction.

Statistical Analysis 20

Statistical analysis description

Week 14

Comparison groups

TAK-079 Placebo-matching v TAK-079 600 mg

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.036 ^[75]

Method

MMRM

Parameter type

Difference in LS Mean

Point estimate

-36.94

Confidence interval

level

95%

sides

2-sided

lower limit

-71.39

upper limit

-2.48

Notes
[75] - From MMRM analysis over all post-baseline visits, with the change from baseline as the outcome, treatment group, visit, and treatment-by-visit interaction as factors, and adjusted by baseline value and baseline-by-visit interaction.

Statistical Analysis 19

Statistical analysis description

Week 14

Comparison groups

TAK-079 Placebo-matching v TAK-079 300 mg

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.002 ^[76]

Method

MMRM

Parameter type

Difference in LS Mean

Point estimate

-56.21

Confidence interval

level

95%

sides

2-sided

lower limit

-91.69

upper limit

-20.73

Notes
[76] - From MMRM analysis over all post-baseline visits, with the change from baseline as the outcome, treatment group, visit, and treatment-by-visit interaction as factors, and adjusted by baseline value and baseline-by-visit interaction.

Statistical Analysis 18

Statistical analysis description

Week 12

Comparison groups

TAK-079 Placebo-matching v TAK-079 600 mg

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.513 ^[77]

Method

MMRM

Parameter type

Difference in LS Mean

Point estimate

-10.79

Confidence interval

level

95%

sides

2-sided

lower limit

-43.26

upper limit

21.67

Notes
[77] - From MMRM analysis over all post-baseline visits, with the change from baseline as the outcome, treatment group, visit, and treatment-by-visit interaction as factors, and adjusted by baseline value and baseline-by-visit interaction.

Statistical Analysis 22

Statistical analysis description

Week 16

Comparison groups

TAK-079 Placebo-matching v TAK-079 600 mg

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

superiority

P-value

= 0.13 ^[78]

Method

MMRM

Parameter type

Difference in LS Mean

Point estimate

-28.81

Confidence interval

level

95%

sides

2-sided

lower limit

-66.12

upper limit

8.51

Notes
[78] - From MMRM analysis over all post-baseline visits, with the change from baseline as the outcome, treatment group, visit, and treatment-by-visit interaction as factors, and adjusted by baseline value and baseline-by-visit interaction.

Secondary: Change From Baseline in Anti- Muscle-specific Tyrosine Kinase (MuSK) Titer Levels

Top of page

End point title

Change From Baseline in Anti- Muscle-specific Tyrosine Kinase (MuSK) Titer Levels

End point description

Clinical laboratory evaluations of anti-MuSK antibodies were tested to monitor disease activity. Data is reported for participants who were positive for anti-MuSK antibodies at baseline. Full Analysis Set included all randomised participants who had baseline and at least 1 post-baseline efficacy assessment. Subjects analysed is the number of participants available for analyses. n=Number analysed is the number of participants available for analysis at the specified timepoint. 99999=SD was not estimable for 1 participant. 9999=Data was not reported for 0 participants.

End point type

Secondary

End point timeframe

Baseline up to Week 32

End point values	TAK-079 Placebo-matching	TAK-079 300 mg	TAK-079 600 mg
Number of subjects analysed	1	2	0 ^[79]
Units: titer
arithmetic mean (standard deviation)
Change From Baseline at Week 2(n=1,2,0)	0.0 ± 99999	-160.0 ± 226.27	±
Change From Baseline at Week 3(n=1,2,0)	0.0 ± 99999	-200.0 ± 169.71	±
Change From Baseline at Week 4(n=1,2,0)	0.0 ± 99999	-240.0 ± 339.41	±
Change From Baseline at Week 5(n=1,2,0)	0.0 ± 99999	-240.0 ± 339.41	±
Change From Baseline at Week 6(n=1,2,0)	0.0 ± 99999	-240.0 ± 339.41	±
Change From Baseline at Week 7(n=1,2,0)	0.0 ± 99999	-240.0 ± 339.41	±
Change From Baseline at Week 8(n=1,2,0)	0.0 ± 99999	-280.0 ± 395.98	±
Change From Baseline at Week 10(n=1,2,0)	0.0 ± 99999	-280.0 ± 395.98	±
Change From Baseline at Week 12(n=1,2,0)	0.0 ± 99999	-300.0 ± 424.26	±
Change From Baseline at Week 14(n=1,2,0)	0.0 ± 99999	-300.0 ± 424.26	±
Change From Baseline at Week 16(n=1,2,0)	0.0 ± 99999	-300.0 ± 424.26	±
Change From Baseline at Week 20(n=0,1,0)	9999 ± 9999	0.0 ± 99999	±
Change From Baseline at Week 24(n=0,2,0)	9999 ± 9999	-310.0 ± 438.41	±
Change From Baseline at Week 28(n=0,2,0)	9999 ± 9999	-310.0 ± 438.41	±
Change From Baseline at Week 32(n=0,2,0)	9999 ± 9999	-310.0 ± 438.41	±

Notes
[79] - Data was not analyzed of this arm group.

No statistical analyses for this end point

Secondary: Percentage of Participants With 2-point Reduction in MG-ADL Total Score

Top of page

End point title

Percentage of Participants With 2-point Reduction in MG-ADL Total Score

End point description

The percentage of responders with at least a 2-point reduction in MG-ADL total score from baseline is reported. MG-ADL is a participant-reported scale to assess MG symptoms to evaluate capacity to perform activities of daily living. Each question is graded on a 4-point scale from 0=normal to 3=severe with a total score of 0 to 24; the higher score indicates greater functional impairment and disability. Percentages are rounded off to whole number at the nearest decimal. Full Analysis Set included all randomised participants who had baseline and at least 1 post-baseline efficacy assessment.

End point type

Secondary

End point timeframe

At Weeks 4, 6, 8, 10, 12, 14, 16, 20, 24, 28 and 32

End point values	TAK-079 Placebo-matching	TAK-079 300 mg	TAK-079 600 mg
Number of subjects analysed	12	12	12
Units: percentage of participants
number (not applicable)
Week 4	50.00	75.00	58.33
Week 6	91.67	75.00	58.33
Week 8	83.33	66.67	66.67
Week 10	83.33	83.33	50.00
Week 12	75.00	75.00	50.00
Week 14	66.67	75.00	50.00
Week 16	66.67	66.67	33.33
Week 20	0.0	66.67	58.33
Week 24	0.0	58.33	58.33
Week 28	0.0	58.33	58.33
Week 32	0.0	41.67	41.67

No statistical analyses for this end point

Secondary: Percentage of Participants With 3-point Reduction in QMG Total Score

Top of page

End point title

Percentage of Participants With 3-point Reduction in QMG Total Score

End point description

The percentage of responders with at least a 3-point reduction in QMG total score from baseline is reported. QMG is a physician-reported scale to assess MG disease severity by quantifying several body functions by physical exam. Each question is graded on a 4-point scale from 0=normal to 3=severe with a total score of 0 to 39; the higher score indicates greater disease burden. Percentages are rounded off to whole number at the nearest decimal. Full Analysis Set included all randomised participants who had baseline and at least 1 post-baseline efficacy assessment.

End point type

Secondary

End point timeframe

At Weeks 4, 6, 8, 10, 12, 14, 16, 20, 24, 28 and 32

End point values	TAK-079 Placebo-matching	TAK-079 300 mg	TAK-079 600 mg
Number of subjects analysed	12	12	12
Units: percentage of participants
number (not applicable)
Week 4	25.00	50.00	16.67
Week 6	16.67	41.67	33.33
Week 8	33.33	66.67	33.33
Week 10	33.33	66.67	33.33
Week 12	41.67	75.00	25.00
Week 14	33.33	66.67	25.00
Week 16	33.33	58.33	33.33
Week 20	0.0	41.67	33.33
Week 24	0.0	41.67	33.33
Week 28	0.0	41.67	25.00
Week 32	0.0	16.67	16.67

No statistical analyses for this end point

Secondary: Percentage of Participants With 3-point Reduction in MGC Total Score

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End point title

Percentage of Participants With 3-point Reduction in MGC Total Score

End point description

The percentage of responders with at least a 3-point reduction in MGC total score from baseline is reported. MGC is an assessment scale of MG disease activity based on a combination of participant- and physician-reported items. Each question is graded on 4 levels of impact from normal to severe with a total score of 0 to 50; the higher score indicates worse MG disease activity. Percentages are rounded off to whole number at the nearest decimal. Full Analysis Set included all randomised participants who had baseline and at least 1 post-baseline efficacy assessment.

End point type

Secondary

End point timeframe

At Weeks 4, 6, 8, 10, 12, 14, 16, 20, 24, 28 and 32

End point values	TAK-079 Placebo-matching	TAK-079 300 mg	TAK-079 600 mg
Number of subjects analysed	12	12	12
Units: percentage of participants
number (not applicable)
Week 4	66.67	75.00	41.67
Week 6	83.33	66.67	58.33
Week 8	83.33	83.33	58.33
Week 10	83.33	75.00	50.00
Week 12	75.00	91.67	58.33
Week 14	66.67	83.33	50.00
Week 16	66.67	75.00	41.67
Week 20	0.0	75.00	58.33
Week 24	0.0	66.67	50.00
Week 28	0.0	58.33	50.00
Week 32	0.0	41.67	25.00

No statistical analyses for this end point

Adverse events

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Timeframe for reporting adverse events

From signing the informed consent form up to end of long-term follow-up (up to Week 32)

Adverse event reporting additional description

At each visit the investigator had to document any occurrence of adverse events and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to study treatment. Safety Analysis Set included participants who received at least 1 dose of study drug.

Assessment type

Systematic

Dictionary name

MedDRA

Dictionary version

25.0

Reporting group title

TAK-079 Placebo-matching

Reporting group description

TAK-079 placebo-matching injection, SC, once weekly in combination with standard background therapy for 8 weeks.

Reporting group title

TAK-079 600 mg

Reporting group description

TAK-079 600 mg injection, SC, once weekly in combination with standard background therapy for 8 weeks.

Reporting group title

TAK-079 300 mg

Reporting group description

TAK-079 300 mg injection, SC, once weekly in combination with standard background therapy for 8 weeks.

Serious adverse events	TAK-079 Placebo-matching	TAK-079 600 mg	TAK-079 300 mg
Total subjects affected by serious adverse events
subjects affected / exposed	1 / 12 (8.33%)	1 / 12 (8.33%)	1 / 12 (8.33%)
number of deaths (all causes)	0	0	0
number of deaths resulting from adverse events	0	0	0
Nervous system disorders
Myasthenia gravis
subjects affected / exposed	0 / 12 (0.00%)	1 / 12 (8.33%)	0 / 12 (0.00%)
occurrences causally related to treatment / all	0 / 0	0 / 1	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0
Gastrointestinal disorders
Enteritis
subjects affected / exposed	1 / 12 (8.33%)	0 / 12 (0.00%)	0 / 12 (0.00%)
occurrences causally related to treatment / all	0 / 1	0 / 0	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0
Psychiatric disorders
Suicidal ideation
subjects affected / exposed	0 / 12 (0.00%)	0 / 12 (0.00%)	1 / 12 (8.33%)
occurrences causally related to treatment / all	0 / 0	0 / 0	0 / 1
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0
Infections and infestations
Gastroenteritis
subjects affected / exposed	1 / 12 (8.33%)	0 / 12 (0.00%)	0 / 12 (0.00%)
occurrences causally related to treatment / all	0 / 1	0 / 0	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0

Frequency threshold for reporting non-serious adverse events: 5%

Non-serious adverse events	TAK-079 Placebo-matching	TAK-079 600 mg	TAK-079 300 mg
Total subjects affected by non serious adverse events
subjects affected / exposed	8 / 12 (66.67%)	11 / 12 (91.67%)	9 / 12 (75.00%)
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Basal cell carcinoma
subjects affected / exposed	0 / 12 (0.00%)	1 / 12 (8.33%)	0 / 12 (0.00%)
occurrences all number	0	1	0
Vascular disorders
Haematoma
subjects affected / exposed	1 / 12 (8.33%)	0 / 12 (0.00%)	0 / 12 (0.00%)
occurrences all number	1	0	0
Hypertension
subjects affected / exposed	1 / 12 (8.33%)	0 / 12 (0.00%)	0 / 12 (0.00%)
occurrences all number	1	0	0
General disorders and administration site conditions
Asthenia
subjects affected / exposed	1 / 12 (8.33%)	1 / 12 (8.33%)	0 / 12 (0.00%)
occurrences all number	3	1	0
Feeling cold
subjects affected / exposed	0 / 12 (0.00%)	1 / 12 (8.33%)	0 / 12 (0.00%)
occurrences all number	0	1	0
Fatigue
subjects affected / exposed	0 / 12 (0.00%)	2 / 12 (16.67%)	1 / 12 (8.33%)
occurrences all number	0	3	1
Early satiety
subjects affected / exposed	1 / 12 (8.33%)	0 / 12 (0.00%)	0 / 12 (0.00%)
occurrences all number	1	0	0
Chills
subjects affected / exposed	0 / 12 (0.00%)	3 / 12 (25.00%)	0 / 12 (0.00%)
occurrences all number	0	3	0
Chest discomfort
subjects affected / exposed	0 / 12 (0.00%)	1 / 12 (8.33%)	0 / 12 (0.00%)
occurrences all number	0	1	0
Feeling hot
subjects affected / exposed	0 / 12 (0.00%)	1 / 12 (8.33%)	0 / 12 (0.00%)
occurrences all number	0	1	0
Influenza like illness
subjects affected / exposed	0 / 12 (0.00%)	0 / 12 (0.00%)	1 / 12 (8.33%)
occurrences all number	0	0	1
Injection site bruising
subjects affected / exposed	0 / 12 (0.00%)	1 / 12 (8.33%)	0 / 12 (0.00%)
occurrences all number	0	1	0
Injection site haematoma
subjects affected / exposed	1 / 12 (8.33%)	0 / 12 (0.00%)	0 / 12 (0.00%)
occurrences all number	1	0	0
Injection site hypertrophy
subjects affected / exposed	0 / 12 (0.00%)	1 / 12 (8.33%)	0 / 12 (0.00%)
occurrences all number	0	1	0
Pyrexia
subjects affected / exposed	1 / 12 (8.33%)	3 / 12 (25.00%)	2 / 12 (16.67%)
occurrences all number	1	3	3
Malaise
subjects affected / exposed	0 / 12 (0.00%)	0 / 12 (0.00%)	1 / 12 (8.33%)
occurrences all number	0	0	1
Injection site pain
subjects affected / exposed	0 / 12 (0.00%)	0 / 12 (0.00%)	1 / 12 (8.33%)
occurrences all number	0	0	5
Immune system disorders
Immunisation reaction
subjects affected / exposed	0 / 12 (0.00%)	1 / 12 (8.33%)	0 / 12 (0.00%)
occurrences all number	0	1	0
Respiratory, thoracic and mediastinal disorders
Dyspnoea exertional
subjects affected / exposed	0 / 12 (0.00%)	1 / 12 (8.33%)	1 / 12 (8.33%)
occurrences all number	0	1	1
Oropharyngeal pain
subjects affected / exposed	1 / 12 (8.33%)	0 / 12 (0.00%)	0 / 12 (0.00%)
occurrences all number	1	0	0
Psychiatric disorders
Depression
subjects affected / exposed	0 / 12 (0.00%)	0 / 12 (0.00%)	1 / 12 (8.33%)
occurrences all number	0	0	1
Investigations
Alanine aminotransferase increased
subjects affected / exposed	0 / 12 (0.00%)	1 / 12 (8.33%)	0 / 12 (0.00%)
occurrences all number	0	1	0
Aspartate aminotransferase increased
subjects affected / exposed	0 / 12 (0.00%)	1 / 12 (8.33%)	0 / 12 (0.00%)
occurrences all number	0	1	0
Blood glucose increased
subjects affected / exposed	1 / 12 (8.33%)	0 / 12 (0.00%)	0 / 12 (0.00%)
occurrences all number	1	0	0
Blood immunoglobulin A decreased
subjects affected / exposed	0 / 12 (0.00%)	1 / 12 (8.33%)	0 / 12 (0.00%)
occurrences all number	0	1	0
Blood lactate dehydrogenase increased
subjects affected / exposed	0 / 12 (0.00%)	1 / 12 (8.33%)	0 / 12 (0.00%)
occurrences all number	0	1	0
Lymphocyte count decreased
subjects affected / exposed	0 / 12 (0.00%)	0 / 12 (0.00%)	1 / 12 (8.33%)
occurrences all number	0	0	1
SARS-CoV-2 test positive
subjects affected / exposed	0 / 12 (0.00%)	1 / 12 (8.33%)	0 / 12 (0.00%)
occurrences all number	0	1	0
Staphylococcus test positive
subjects affected / exposed	1 / 12 (8.33%)	0 / 12 (0.00%)	0 / 12 (0.00%)
occurrences all number	1	0	0
Injury, poisoning and procedural complications
Limb injury
subjects affected / exposed	0 / 12 (0.00%)	1 / 12 (8.33%)	0 / 12 (0.00%)
occurrences all number	0	1	0
Cardiac disorders
Atrioventricular block first degree
subjects affected / exposed	1 / 12 (8.33%)	0 / 12 (0.00%)	1 / 12 (8.33%)
occurrences all number	1	0	1
Nervous system disorders
Headache
subjects affected / exposed	1 / 12 (8.33%)	0 / 12 (0.00%)	0 / 12 (0.00%)
occurrences all number	1	0	0
Dysarthria
subjects affected / exposed	0 / 12 (0.00%)	0 / 12 (0.00%)	1 / 12 (8.33%)
occurrences all number	0	0	1
Hypoaesthesia
subjects affected / exposed	0 / 12 (0.00%)	1 / 12 (8.33%)	0 / 12 (0.00%)
occurrences all number	0	1	0
Myasthenia gravis
subjects affected / exposed	0 / 12 (0.00%)	0 / 12 (0.00%)	1 / 12 (8.33%)
occurrences all number	0	0	1
Paraesthesia
subjects affected / exposed	0 / 12 (0.00%)	0 / 12 (0.00%)	1 / 12 (8.33%)
occurrences all number	0	0	1
Parosmia
subjects affected / exposed	0 / 12 (0.00%)	0 / 12 (0.00%)	1 / 12 (8.33%)
occurrences all number	0	0	1
Tension headache
subjects affected / exposed	1 / 12 (8.33%)	0 / 12 (0.00%)	0 / 12 (0.00%)
occurrences all number	2	0	0
Blood and lymphatic system disorders
Anaemia
subjects affected / exposed	0 / 12 (0.00%)	1 / 12 (8.33%)	0 / 12 (0.00%)
occurrences all number	0	1	0
Thrombocytosis
subjects affected / exposed	1 / 12 (8.33%)	0 / 12 (0.00%)	0 / 12 (0.00%)
occurrences all number	1	0	0
Normochromic normocytic anaemia
subjects affected / exposed	0 / 12 (0.00%)	1 / 12 (8.33%)	0 / 12 (0.00%)
occurrences all number	0	1	0
Lymphopenia
subjects affected / exposed	1 / 12 (8.33%)	0 / 12 (0.00%)	0 / 12 (0.00%)
occurrences all number	1	0	0
Eye disorders
Periorbital oedema
subjects affected / exposed	0 / 12 (0.00%)	0 / 12 (0.00%)	1 / 12 (8.33%)
occurrences all number	0	0	1
Conjunctivitis allergic
subjects affected / exposed	0 / 12 (0.00%)	0 / 12 (0.00%)	1 / 12 (8.33%)
occurrences all number	0	0	1
Diplopia
subjects affected / exposed	0 / 12 (0.00%)	0 / 12 (0.00%)	1 / 12 (8.33%)
occurrences all number	0	0	1
Ocular hypertension
subjects affected / exposed	0 / 12 (0.00%)	1 / 12 (8.33%)	0 / 12 (0.00%)
occurrences all number	0	1	0
Angle closure glaucoma
subjects affected / exposed	0 / 12 (0.00%)	1 / 12 (8.33%)	0 / 12 (0.00%)
occurrences all number	0	1	0
Vitreous detachment
subjects affected / exposed	0 / 12 (0.00%)	1 / 12 (8.33%)	0 / 12 (0.00%)
occurrences all number	0	1	0
Visual impairment
subjects affected / exposed	0 / 12 (0.00%)	0 / 12 (0.00%)	1 / 12 (8.33%)
occurrences all number	0	0	1
Gastrointestinal disorders
Vomiting
subjects affected / exposed	0 / 12 (0.00%)	1 / 12 (8.33%)	0 / 12 (0.00%)
occurrences all number	0	2	0
Odynophagia
subjects affected / exposed	1 / 12 (8.33%)	0 / 12 (0.00%)	0 / 12 (0.00%)
occurrences all number	1	0	0
Nausea
subjects affected / exposed	0 / 12 (0.00%)	1 / 12 (8.33%)	0 / 12 (0.00%)
occurrences all number	0	1	0
Skin and subcutaneous tissue disorders
Pruritus
subjects affected / exposed	0 / 12 (0.00%)	1 / 12 (8.33%)	0 / 12 (0.00%)
occurrences all number	0	1	0
Hyperhidrosis
subjects affected / exposed	0 / 12 (0.00%)	1 / 12 (8.33%)	0 / 12 (0.00%)
occurrences all number	0	1	0
Rash papular
subjects affected / exposed	1 / 12 (8.33%)	1 / 12 (8.33%)	0 / 12 (0.00%)
occurrences all number	1	1	0
Renal and urinary disorders
Urine odour abnormal
subjects affected / exposed	0 / 12 (0.00%)	1 / 12 (8.33%)	0 / 12 (0.00%)
occurrences all number	0	1	0
Musculoskeletal and connective tissue disorders
Arthralgia
subjects affected / exposed	0 / 12 (0.00%)	1 / 12 (8.33%)	0 / 12 (0.00%)
occurrences all number	0	1	0
Muscular weakness
subjects affected / exposed	0 / 12 (0.00%)	0 / 12 (0.00%)	1 / 12 (8.33%)
occurrences all number	0	0	1
Pain in jaw
subjects affected / exposed	0 / 12 (0.00%)	1 / 12 (8.33%)	0 / 12 (0.00%)
occurrences all number	0	1	0
Joint noise
subjects affected / exposed	0 / 12 (0.00%)	1 / 12 (8.33%)	0 / 12 (0.00%)
occurrences all number	0	1	0
Infections and infestations
Abscess limb
subjects affected / exposed	0 / 12 (0.00%)	1 / 12 (8.33%)	0 / 12 (0.00%)
occurrences all number	0	1	0
Abdominal wall abscess
subjects affected / exposed	1 / 12 (8.33%)	0 / 12 (0.00%)	0 / 12 (0.00%)
occurrences all number	1	0	0
Bartholin's abscess
subjects affected / exposed	1 / 12 (8.33%)	0 / 12 (0.00%)	0 / 12 (0.00%)
occurrences all number	1	0	0
Cellulitis
subjects affected / exposed	1 / 12 (8.33%)	0 / 12 (0.00%)	0 / 12 (0.00%)
occurrences all number	1	0	0
Gastroenteritis
subjects affected / exposed	1 / 12 (8.33%)	0 / 12 (0.00%)	0 / 12 (0.00%)
occurrences all number	1	0	0
Nasopharyngitis
subjects affected / exposed	1 / 12 (8.33%)	2 / 12 (16.67%)	0 / 12 (0.00%)
occurrences all number	2	2	0
Upper respiratory tract infection
subjects affected / exposed	0 / 12 (0.00%)	1 / 12 (8.33%)	0 / 12 (0.00%)
occurrences all number	0	1	0
Metabolism and nutrition disorders
Decreased appetite
subjects affected / exposed	1 / 12 (8.33%)	0 / 12 (0.00%)	0 / 12 (0.00%)
occurrences all number	1	0	0

More information

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Were there any global substantial amendments to the protocol? Yes

04 Mar 2020

Following changes were implemented with Protocol Amendment 2: -Updated inclusion criterion to clarify the intent to exclude only pulse steroid therapy. Dosing of corticosteroids with oral therapy every other day to be as acceptable as daily. -Updated exclusion criterion to exclude only those receiving live vaccines and not inactive vaccines. -Updated exclusion criterion to clarify the intent not to exclude a minor, benign, resolved, localized herpes simplex infection not requiring systemic therapy. -Updated exclusion criterion to change “lower limit of normal” to “5 g/L.” As a result of this edit, participants were excluded if the IgG was less than 5 g/L (500 mg/dL) at screening. -Updated exclusion criterion to clarify the intent to exclude participants with active hepatitis C and not those who have been fully cured of the disease. Additionally, more stringent criteria were used to exclude participants with hepatitis B infection. -Updated the SAE reporting procedure to include an acknowledgement of receipt. -Added a global fax number to the SAE Reporting Contact Information. -Descriptors for circulating biomarker samples and quantitative immunoglobulin (Ig) A/IgM/IgG samples were changed from blood to serum. -Updated the version number of the QMG scoring assessment tool, which fixed a typographical error in the definition of severe forced vital capacity (FVC). -Serum samples for hepatitis B, hepatitis C, and human immunodeficiency virus (HIV) were added to Primary Specimen Collection Table. -Added serum pregnancy test at Week 32. -Eligibility criteria regarding immunosuppressive drugs now required a stable dose for at least 3 months before screening. Stable dosing of azathioprine remained at 6 months prior to screening.

28 Sep 2020

Following changes were implemented with Protocol Amendment 3: Added contingency plans for the COVID-19 pandemic by incorporating flexibility for the study participants and investigators, while continuing to maintain participant safety and study integrity as per local site regulations.

01 Feb 2021

Following changes were implemented with Protocol Amendment 4: Clarified the intent of exclusion criterion.

05 May 2021

Following changes were implemented with Protocol Amendment 5: Changed the legal entity name for the sponsor to Takeda Development Center Americas, Inc., 95 Hayden Avenue, Lexington, MA 02421, USA.

Were there any global interruptions to the trial? No

Limitations of the trial such as small numbers of subjects analysed or technical problems leading to unreliable data.

None reported

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Clinical trials

Clinical Trial Results:
A Phase 2, Randomized, Placebo-Controlled Study to Evaluate Safety, Tolerability, and Efficacy of TAK-079 in Patients With Generalized Myasthenia Gravis

Trial information

Trial information

Subject disposition

Subject disposition

Baseline characteristics

Baseline characteristics

End points

End points

Primary: Percentage of Participants With Treatment Emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), Grade 3 or Higher TEAEs, and Adverse Event (AE) Leading to TAK-079 Discontinuation

Primary: Percentage of Participants With Treatment Emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), Grade 3 or Higher TEAEs, and Adverse Event (AE) Leading to TAK-079 Discontinuation

Secondary: Change From Baseline in Myasthenia Gravis Activities of Daily Living (MG-ADL) Scale Score

Secondary: Change From Baseline in Myasthenia Gravis Activities of Daily Living (MG-ADL) Scale Score

Secondary: Change From Baseline in Quantitative Myasthenia Gravis (QMG) Scale Score

Secondary: Change From Baseline in Quantitative Myasthenia Gravis (QMG) Scale Score

Secondary: Change From Baseline in Myasthenia Gravis Composite (MGC) Scale Score

Secondary: Change From Baseline in Myasthenia Gravis Composite (MGC) Scale Score

Secondary: Change From Baseline in Revised 15-item Myasthenia Gravis Quality of Life Scale (MG-QoL15r) Scale Score

Secondary: Change From Baseline in Revised 15-item Myasthenia Gravis Quality of Life Scale (MG-QoL15r) Scale Score

Secondary: Change From Baseline in Anti-acetylcholine Receptor (AChR) Antibody Levels

Secondary: Change From Baseline in Anti-acetylcholine Receptor (AChR) Antibody Levels

Secondary: Change From Baseline in Anti- Muscle-specific Tyrosine Kinase (MuSK) Titer Levels

Secondary: Change From Baseline in Anti- Muscle-specific Tyrosine Kinase (MuSK) Titer Levels

Secondary: Percentage of Participants With 2-point Reduction in MG-ADL Total Score

Secondary: Percentage of Participants With 2-point Reduction in MG-ADL Total Score

Secondary: Percentage of Participants With 3-point Reduction in QMG Total Score

Secondary: Percentage of Participants With 3-point Reduction in QMG Total Score

Secondary: Percentage of Participants With 3-point Reduction in MGC Total Score

Secondary: Percentage of Participants With 3-point Reduction in MGC Total Score

Adverse events

Adverse events

More information

Substantial protocol amendments (globally)

Interruptions (globally)

Limitations and caveats

More information

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Finland
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Clinical trials

Clinical Trial Results: A Phase 2, Randomized, Placebo-Controlled Study to Evaluate Safety, Tolerability, and Efficacy of TAK-079 in Patients With Generalized Myasthenia Gravis

Trial information

Trial information

Subject disposition

Subject disposition

Baseline characteristics

Baseline characteristics

End points

End points

Primary: Percentage of Participants With Treatment Emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), Grade 3 or Higher TEAEs, and Adverse Event (AE) Leading to TAK-079 Discontinuation

Primary: Percentage of Participants With Treatment Emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), Grade 3 or Higher TEAEs, and Adverse Event (AE) Leading to TAK-079 Discontinuation

Secondary: Change From Baseline in Myasthenia Gravis Activities of Daily Living (MG-ADL) Scale Score

Secondary: Change From Baseline in Myasthenia Gravis Activities of Daily Living (MG-ADL) Scale Score

Secondary: Change From Baseline in Quantitative Myasthenia Gravis (QMG) Scale Score

Secondary: Change From Baseline in Quantitative Myasthenia Gravis (QMG) Scale Score

Secondary: Change From Baseline in Myasthenia Gravis Composite (MGC) Scale Score

Secondary: Change From Baseline in Myasthenia Gravis Composite (MGC) Scale Score

Secondary: Change From Baseline in Revised 15-item Myasthenia Gravis Quality of Life Scale (MG-QoL15r) Scale Score

Secondary: Change From Baseline in Revised 15-item Myasthenia Gravis Quality of Life Scale (MG-QoL15r) Scale Score

Secondary: Change From Baseline in Anti-acetylcholine Receptor (AChR) Antibody Levels

Secondary: Change From Baseline in Anti-acetylcholine Receptor (AChR) Antibody Levels

Secondary: Change From Baseline in Anti- Muscle-specific Tyrosine Kinase (MuSK) Titer Levels

Secondary: Change From Baseline in Anti- Muscle-specific Tyrosine Kinase (MuSK) Titer Levels

Secondary: Percentage of Participants With 2-point Reduction in MG-ADL Total Score

Secondary: Percentage of Participants With 2-point Reduction in MG-ADL Total Score

Secondary: Percentage of Participants With 3-point Reduction in QMG Total Score

Secondary: Percentage of Participants With 3-point Reduction in QMG Total Score

Secondary: Percentage of Participants With 3-point Reduction in MGC Total Score

Secondary: Percentage of Participants With 3-point Reduction in MGC Total Score

Adverse events

Adverse events

More information

Substantial protocol amendments (globally)

Interruptions (globally)

Limitations and caveats

More information

Clinical Trial Results:
A Phase 2, Randomized, Placebo-Controlled Study to Evaluate Safety, Tolerability, and Efficacy of TAK-079 in Patients With Generalized Myasthenia Gravis