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Clinical Trial Results:
A 52 week, multi-center, randomized, double-blind placebo-controlled study to assess the clinical efficacy and safety of ligelizumab (QGE031) in decreasing the sensitivity to peanuts in patients with peanut allergy

Summary
EudraCT number	2020-005339-56
Trial protocol	FR ES DE IT DK NL
Global end of trial date	27 Nov 2023

Results information
Results version number	v1(current)
This version publication date	07 Jun 2024
First version publication date	07 Jun 2024
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

CQGE031G12301

ISRCTN number

US NCT number

WHO universal trial number (UTN)

Sponsor organisation name

Novartis Pharma AG

Sponsor organisation address

Novartis Campus, Basel, Switzerland,

Public contact

Clinical Disclosure Office, Novartis Pharma AG, 41 613241111, Novartis.email@Novartis.com

Scientific contact

Clinical Disclosure Office, Novartis Pharma AG, 41 613241111, Novartis.email@Novartis.com

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

Yes

EMA paediatric investigation plan number(s)

EMEA-001811-PIP03-20

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?

Yes

Analysis stage

Final

Date of interim/final analysis

27 Nov 2023

Is this the analysis of the primary completion data?

Global end of trial reached?

Yes

Global end of trial date

27 Nov 2023

Was the trial ended prematurely?

Yes

Main objective of the trial

The primary objective was to evaluate the efficacy of ligelizumab 240 mg and 120 mg (subcutaneous injection once every 4 weeks (SC q4w)) compared to placebo, as measured by the proportion of participants who can tolerate a single dose of >= 600 mg (1044 mg cumulative tolerated dose) of peanut protein without dose-limiting symptoms during the double-blind placebo-controlled food challenge (DBPCFC) at Week 12

Protection of trial subjects

The study was in compliance with the ethical principles derived from the Declaration of Helsinki and the International Conference on Harmonization (ICH) Good Clinical Practice (GCP) guidelines. All the local regulatory requirements pertinent to safety of trial subjects were also followed during the conduct of the trial.

Background therapy

Evidence for comparator

Actual start date of recruitment

07 Dec 2021

Long term follow-up planned

Independent data monitoring committee (IDMC) involvement?

Yes

Number of subjects enrolled per country

Country: Number of subjects enrolled

Australia: 9

Country: Number of subjects enrolled

Canada: 35

Country: Number of subjects enrolled

Denmark: 1

Country: Number of subjects enrolled

France: 13

Country: Number of subjects enrolled

Germany: 22

Country: Number of subjects enrolled

Italy: 2

Country: Number of subjects enrolled

Japan: 7

Country: Number of subjects enrolled

Netherlands: 4

Country: Number of subjects enrolled

Spain: 9

Country: Number of subjects enrolled

United States: 109

Worldwide total number of subjects

211

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)

119

Adults (18-64 years)

From 65 to 84 years

85 years and over

Subject disposition

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Recruitment details

Screening details

This study was conducted in 56 centers in 10 countries worldwide

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, Carer

Are arms mutually exclusive

Yes

ligelizumab 240 mg

Arm description

ligelizumab 240 mg subcutaneous injection for 52 weeks

Arm type

Experimental

Investigational medicinal product name

ligelizumab

Investigational medicinal product code

Other name

QGE031

Pharmaceutical forms

Solution for injection in pre-filled syringe

Routes of administration

Subcutaneous use

Dosage and administration details

2 injections of 1.0 mL ligelizumab from Day 1 through Week 52

ligelizumab 120 mg

Arm description

ligelizumab 120 mg subcutaneous injection for 52 weeks

Arm type

Experimental

Investigational medicinal product name

Placebo

Investigational medicinal product code

Other name

Pharmaceutical forms

Solution for injection in pre-filled syringe

Routes of administration

Subcutaneous use

Dosage and administration details

1 injection of 1.0 mL placebo from Day 1 through Week 52

Investigational medicinal product name

ligelizumab

Investigational medicinal product code

Other name

QGE031

Pharmaceutical forms

Solution for injection in pre-filled syringe

Routes of administration

Subcutaneous use

Dosage and administration details

1 injection of 1.0 mL ligelizumab from Day 1 through Week 52

Placebo 8 weeks and ligelizumab 240 mg

Arm description

Placebo subcutaneous injection for first 8 weeks and ligelizumab 240 mg subcutaneous injection for 44 weeks

Arm type

Experimental

Investigational medicinal product name

Placebo

Investigational medicinal product code

Other name

Pharmaceutical forms

Solution for injection in pre-filled syringe

Routes of administration

Subcutaneous use

Dosage and administration details

2 injections of 1.0 mL placebo at Day 1 and Week 4

Investigational medicinal product name

ligelizumab

Investigational medicinal product code

Other name

QGE031

Pharmaceutical forms

Solution for injection in pre-filled syringe

Routes of administration

Subcutaneous use

Dosage and administration details

2 injections of 1.0 mL ligelizumab from Week 8 through Week 52

Placebo 8 weeks and ligelizumab 120 mg

Arm description

Placebo subcutaneous injection for first 8 weeks and ligelizumab 120 mg subcutaneous injection for 44 weeks

Arm type

Experimental

Investigational medicinal product name

ligelizumab

Investigational medicinal product code

Other name

QGE031

Pharmaceutical forms

Solution for injection in pre-filled syringe

Routes of administration

Subcutaneous use

Dosage and administration details

1 injection of 1.0 mL ligelizumab from Week 8 through Week 52

Investigational medicinal product name

Placebo

Investigational medicinal product code

Other name

Pharmaceutical forms

Solution for injection in pre-filled syringe

Routes of administration

Subcutaneous use

Dosage and administration details

1 injection of 1.0 mL placebo from Week 8 through Week 52

Investigational medicinal product name

Placebo

Investigational medicinal product code

Other name

Pharmaceutical forms

Solution for injection in pre-filled syringe

Routes of administration

Subcutaneous use

Dosage and administration details

2 injections of 1.0 mL placebo at Day 1 and Week 4

Placebo 16 weeks and ligelizumab 240 mg

Arm description

Placebo subcutaneous injection for first 16 weeks and ligelizumab 240 mg subcutaneous injection for 36 weeks

Arm type

Experimental

Investigational medicinal product name

Placebo

Investigational medicinal product code

Other name

Pharmaceutical forms

Solution for injection in pre-filled syringe

Routes of administration

Subcutaneous use

Dosage and administration details

2 injections of 1.0 mL placebo at Day 1 and Week 12

Investigational medicinal product name

ligelizumab

Investigational medicinal product code

Other name

QGE031

Pharmaceutical forms

Solution for injection in pre-filled syringe

Routes of administration

Subcutaneous use

Dosage and administration details

2 injections of 1.0 mL ligelizumab from Week 16 through Week 52

Placebo 16 weeks and ligelizumab 120 mg

Arm description

Placebo subcutaneous injection for first 16 weeks and ligelizumab 120 mg subcutaneous injection for 36 weeks

Arm type

Experimental

Investigational medicinal product name

ligelizumab

Investigational medicinal product code

Other name

QGE031

Pharmaceutical forms

Solution for injection in pre-filled syringe

Routes of administration

Subcutaneous use

Dosage and administration details

1 injection of 1.0 mL ligelizumab from Week 16 through Week 52

Investigational medicinal product name

Placebo

Investigational medicinal product code

Other name

Pharmaceutical forms

Solution for injection in pre-filled syringe

Routes of administration

Subcutaneous use

Dosage and administration details

1 injection of 1.0 mL placebo from Week 16 through Week 52

Investigational medicinal product name

Placebo

Investigational medicinal product code

Other name

Pharmaceutical forms

Solution for injection in pre-filled syringe

Routes of administration

Subcutaneous use

Dosage and administration details

2 injections of 1.0 mL placebo at Day 1 and Week 12

Number of subjects in period 1	ligelizumab 240 mg	ligelizumab 120 mg	Placebo 8 weeks and ligelizumab 240 mg	Placebo 8 weeks and ligelizumab 120 mg	Placebo 16 weeks and ligelizumab 240 mg	Placebo 16 weeks and ligelizumab 120 mg
Started	47	51	46	44	11	12
Randomized Analysis Set (RAS)	47	51	46	44	11	12
Full Analysis Set (FAS)	47	51	46	44	11	12
Safety Set (SAF)	47	51	46	44	11	12
Completed	44	40	41	37	8	11
Not completed	3	11	5	7	3	1
Physician decision	1	-	-	1	-	-
Subject decision	2	10	3	3	3	-
Adverse event, non-fatal	-	-	1	-	-	-
Lost to follow-up	-	-	1	1	-	-
Guardian decision	-	1	-	2	-	1

Baseline characteristics

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Reporting group title

ligelizumab 240 mg

Reporting group description

ligelizumab 240 mg subcutaneous injection for 52 weeks

Reporting group title

ligelizumab 120 mg

Reporting group description

ligelizumab 120 mg subcutaneous injection for 52 weeks

Reporting group title

Placebo 8 weeks and ligelizumab 240 mg

Reporting group description

Placebo subcutaneous injection for first 8 weeks and ligelizumab 240 mg subcutaneous injection for 44 weeks

Reporting group title

Placebo 8 weeks and ligelizumab 120 mg

Reporting group description

Placebo subcutaneous injection for first 8 weeks and ligelizumab 120 mg subcutaneous injection for 44 weeks

Reporting group title

Placebo 16 weeks and ligelizumab 240 mg

Reporting group description

Placebo subcutaneous injection for first 16 weeks and ligelizumab 240 mg subcutaneous injection for 36 weeks

Reporting group title

Placebo 16 weeks and ligelizumab 120 mg

Reporting group description

Placebo subcutaneous injection for first 16 weeks and ligelizumab 120 mg subcutaneous injection for 36 weeks

Reporting group values	ligelizumab 240 mg	ligelizumab 120 mg	Placebo 8 weeks and ligelizumab 240 mg	Placebo 8 weeks and ligelizumab 120 mg	Placebo 16 weeks and ligelizumab 240 mg	Placebo 16 weeks and ligelizumab 120 mg	Total
Number of subjects	47	51	46	44	11	12	211
Age Categorical
Units: Participants
12 - 17 years	27	29	25	24	6	8	119
18 - 55 years	20	22	21	20	5	4	92
Age Continuous
Units: Years
arithmetic mean (standard deviation)	19.5 ( 7.50 )	18.8 ( 6.27 )	18.7 ( 6.95 )	20.0 ( 7.75 )	17.4 ( 4.57 )	16.1 ( 3.58 )	-
Sex: Female, Male
Units: Participants
Female	25	25	19	20	3	6	98
Male	22	26	27	24	8	6	113
Race (NIH/OMB)
Units: Subjects
American Indian or Alaska Native	0	0	0	0	0	0	0
Asian	8	10	2	1	2	2	25
Native Hawaiian or Other Pacific Islander	0	0	0	0	0	0	0
Black or African American	6	3	1	2	0	0	12
White	31	36	42	39	7	10	165
More than one race	1	1	0	2	1	0	5
Unknown or Not Reported	1	1	1	0	1	0	4
Number of participants with at least one food allergy
Units: Subjects
1 food allergy	12	15	13	11	5	3	59
2 food allergies	10	5	8	4	1	1	29
3 food allergies	5	5	2	5	3	1	21
4 food allergies	8	7	6	4	0	1	26
5 food allergies	0	4	4	3	1	2	14
> 5 food allergies	12	15	13	17	1	4	62
MTD (maximum tolerated dose) of peanut protein (mg) - n (%)
Units: Subjects
< 1 mg	2	5	2	2	1	0	12
1 mg	4	4	6	9	1	2	26
3 mg	14	14	11	11	1	1	52
10 mg	9	14	7	11	4	5	50
30 mg	18	14	20	11	4	4	71
Poly-sensitized to food
Poly-sensitization is defined as specific Immunoglobulin E (sIgE) >=0.35kUA/L for a food allergen in the panel other than peanut.
Units: Subjects
Poly-sensitized to food = Yes	29	27	22	27	8	8	121
Poly-sensitized to food = No	18	24	24	17	3	4	90
Poly-allergic to food
Poly-allergic: Participants experiencing more than one food allergies in medical history.
Units: Subjects
Poly-allergic to food = Yes	35	36	33	33	6	9	152
Poly-allergic to food = No	12	15	13	11	5	3	59
History of anaphylactic reaction to food
Units: Subjects
History of anaphylactic reaction to food = Yes	32	36	29	32	9	8	146
History of anaphylactic reaction to food = No	14	15	16	12	2	4	63
History of anaphylactic reaction to food = Unknown	1	0	1	0	0	0	2
Time to diagnosis of peanut allergy
Units: Years
arithmetic mean (standard deviation)	16.287 ( 7.9393 )	16.381 ( 6.9248 )	15.556 ( 7.5799 )	17.748 ( 7.8969 )	15.598 ( 5.3525 )	14.971 ( 3.5291 )	-
Peanut specific Immunoglobulin E (IgE)
Units: IU/mL
arithmetic mean (standard deviation)	109.021 ( 147.9877 )	80.644 ( 117.6312 )	131.948 ( 189.7757 )	116.198 ( 128.9151 )	51.606 ( 48.6951 )	262.136 ( 543.7764 )	-
Total Immunoglobulin E (IgE)
Units: IU/mL
arithmetic mean (standard deviation)	524.35 ( 444.009 )	482.25 ( 478.047 )	455.38 ( 397.960 )	436.31 ( 335.113 )	325.15 ( 208.482 )	635.42 ( 535.099 )	-
Peanut Skin Prick Test (SPT) (undiluted): Size of mean wheal diameter
Size of Mean wheal diameter (mm) = [(Mean of peanut wheal longest diameter + Mean of peanut wheal orthogonal diameter)/2]- average of the non-missing negative control diameters
Units: mm
arithmetic mean (standard deviation)	13.83 ( 5.720 )	14.61 ( 6.007 )	13.23 ( 6.414 )	16.82 ( 8.262 )	14.45 ( 4.845 )	12.17 ( 3.664 )	-
Peanut Skin Prick Test (SPT) (average across dilutions): Size of mean wheal diameter
Size of Mean wheal diameter (mm) = [(Mean of peanut wheal longest diameter + Mean of peanut wheal orthogonal diameter)/2]- average of the non-missing negative control diameters. Range of dilutions (1:10 to 1:100,000).
Units: mm
arithmetic mean (standard deviation)	7.232 ( 2.8339 )	7.273 ( 2.7253 )	7.508 ( 2.7624 )	7.907 ( 2.8866 )	6.575 ( 2.3809 )	7.743 ( 2.3547 )	-

Subject analysis set title

Placebo

Subject analysis set type

Sub-group analysis

Subject analysis set description

Placebo subcutaneous injection for first 16 weeks and ligelizumab 120 mg OR 240 mg subcutaneous injection for 36 weeks

Subject analysis set title

Placebo

Subject analysis set type

Sub-group analysis

Subject analysis set description

Placebo subcutaneous injection for first 16 weeks and ligelizumab 120 mg OR 240 mg subcutaneous injection for 36 weeks

Subject analysis set title

Placebo

Subject analysis set type

Sub-group analysis

Subject analysis set description

Placebo subcutaneous injection for first 16 weeks and ligelizumab 120 mg OR 240 mg subcutaneous injection for 36 weeks

Subject analysis set title

Placebo

Subject analysis set type

Sub-group analysis

Subject analysis set description

Placebo subcutaneous injection for first 16 weeks and ligelizumab 120 mg OR 240 mg subcutaneous injection for 36 weeks

Subject analysis set title

Placebo

Subject analysis set type

Sub-group analysis

Subject analysis set description

Placebo subcutaneous injection for first 16 weeks and ligelizumab 120 mg OR 240 mg subcutaneous injection for 36 weeks

Subject analysis set title

Placebo

Subject analysis set type

Sub-group analysis

Subject analysis set description

Placebo subcutaneous injection for first 16 weeks and ligelizumab 120 mg OR 240 mg subcutaneous injection for 36 weeks

Subject analysis set title

Placebo

Subject analysis set type

Sub-group analysis

Subject analysis set description

Placebo subcutaneous injection for first 16 weeks and ligelizumab 120 mg OR 240 mg subcutaneous injection for 36 weeks

Subject analysis sets values	Placebo	Placebo	Placebo	Placebo	Placebo	Placebo	Placebo
Number of subjects	23	20	19	16	9	8	5
Age Categorical
Units: Participants
12 - 17 years
18 - 55 years
Age Continuous
Units: Years
arithmetic mean (standard deviation)	( )	( )	( )	( )	( )	( )	( )
Sex: Female, Male
Units: Participants
Female
Male
Race (NIH/OMB)
Units: Subjects
American Indian or Alaska Native
Asian
Native Hawaiian or Other Pacific Islander
Black or African American
White
More than one race
Unknown or Not Reported
Number of participants with at least one food allergy
Units: Subjects
1 food allergy
2 food allergies
3 food allergies
4 food allergies
5 food allergies
> 5 food allergies
MTD (maximum tolerated dose) of peanut protein (mg) - n (%)
Units: Subjects
< 1 mg
1 mg
3 mg
10 mg
30 mg
Poly-sensitized to food
Poly-sensitization is defined as specific Immunoglobulin E (sIgE) >=0.35kUA/L for a food allergen in the panel other than peanut.
Units: Subjects
Poly-sensitized to food = Yes
Poly-sensitized to food = No
Poly-allergic to food
Poly-allergic: Participants experiencing more than one food allergies in medical history.
Units: Subjects
Poly-allergic to food = Yes
Poly-allergic to food = No
History of anaphylactic reaction to food
Units: Subjects
History of anaphylactic reaction to food = Yes
History of anaphylactic reaction to food = No
History of anaphylactic reaction to food = Unknown
Time to diagnosis of peanut allergy
Units: Years
arithmetic mean (standard deviation)	1 ( )	( )	( )	-2.38 ( 3.788 )	( )	( )	( )
Peanut specific Immunoglobulin E (IgE)
Units: IU/mL
arithmetic mean (standard deviation)	1 ( )	( )	( )	-2.38 ( 3.788 )	( )	( )	( )
Total Immunoglobulin E (IgE)
Units: IU/mL
arithmetic mean (standard deviation)	1 ( )	( )	( )	-2.38 ( 3.788 )	( )	( )	( )
Peanut Skin Prick Test (SPT) (undiluted): Size of mean wheal diameter
Size of Mean wheal diameter (mm) = [(Mean of peanut wheal longest diameter + Mean of peanut wheal orthogonal diameter)/2]- average of the non-missing negative control diameters
Units: mm
arithmetic mean (standard deviation)	1 ( )	( )	( )	-2.38 ( 3.788 )	( )	( )	( )
Peanut Skin Prick Test (SPT) (average across dilutions): Size of mean wheal diameter
Size of Mean wheal diameter (mm) = [(Mean of peanut wheal longest diameter + Mean of peanut wheal orthogonal diameter)/2]- average of the non-missing negative control diameters. Range of dilutions (1:10 to 1:100,000).
Units: mm
arithmetic mean (standard deviation)	1 ( )	( )	( )	-2.38 ( 3.788 )	( )	( )	( )

End points

Top of page

Reporting group title

ligelizumab 240 mg

Reporting group description

ligelizumab 240 mg subcutaneous injection for 52 weeks

Reporting group title

ligelizumab 120 mg

Reporting group description

ligelizumab 120 mg subcutaneous injection for 52 weeks

Reporting group title

Placebo 8 weeks and ligelizumab 240 mg

Reporting group description

Placebo subcutaneous injection for first 8 weeks and ligelizumab 240 mg subcutaneous injection for 44 weeks

Reporting group title

Placebo 8 weeks and ligelizumab 120 mg

Reporting group description

Placebo subcutaneous injection for first 8 weeks and ligelizumab 120 mg subcutaneous injection for 44 weeks

Reporting group title

Placebo 16 weeks and ligelizumab 240 mg

Reporting group description

Placebo subcutaneous injection for first 16 weeks and ligelizumab 240 mg subcutaneous injection for 36 weeks

Reporting group title

Placebo 16 weeks and ligelizumab 120 mg

Reporting group description

Placebo subcutaneous injection for first 16 weeks and ligelizumab 120 mg subcutaneous injection for 36 weeks

Subject analysis set title

Placebo

Subject analysis set type

Sub-group analysis

Subject analysis set description

Placebo subcutaneous injection for first 16 weeks and ligelizumab 120 mg OR 240 mg subcutaneous injection for 36 weeks

Subject analysis set title

Placebo

Subject analysis set type

Sub-group analysis

Subject analysis set description

Placebo subcutaneous injection for first 16 weeks and ligelizumab 120 mg OR 240 mg subcutaneous injection for 36 weeks

Subject analysis set title

Placebo

Subject analysis set type

Sub-group analysis

Subject analysis set description

Placebo subcutaneous injection for first 16 weeks and ligelizumab 120 mg OR 240 mg subcutaneous injection for 36 weeks

Subject analysis set title

Placebo

Subject analysis set type

Sub-group analysis

Subject analysis set description

Placebo subcutaneous injection for first 16 weeks and ligelizumab 120 mg OR 240 mg subcutaneous injection for 36 weeks

Subject analysis set title

Placebo

Subject analysis set type

Sub-group analysis

Subject analysis set description

Placebo subcutaneous injection for first 16 weeks and ligelizumab 120 mg OR 240 mg subcutaneous injection for 36 weeks

Subject analysis set title

Placebo

Subject analysis set type

Sub-group analysis

Subject analysis set description

Placebo subcutaneous injection for first 16 weeks and ligelizumab 120 mg OR 240 mg subcutaneous injection for 36 weeks

Subject analysis set title

Placebo

Subject analysis set type

Sub-group analysis

Subject analysis set description

Placebo subcutaneous injection for first 16 weeks and ligelizumab 120 mg OR 240 mg subcutaneous injection for 36 weeks

Primary: Percentage of participants who tolerated a single dose of >= 600 mg (1044 mg cumulative tolerated dose) of peanut protein without dose-limiting symptoms at Week 12

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End point title

Percentage of participants who tolerated a single dose of >= 600 mg (1044 mg cumulative tolerated dose) of peanut protein without dose-limiting symptoms at Week 12 ^[1]

End point description

Responder rate was defined as the percentage of participants tolerating a single dose of >= 600 mg (1044 mg cumulative tolerated dose) of peanut protein without dose-limiting symptoms during the double blind placebo controlled food challenge (DBPCFC) conducted at Week 12. The cumulative tolerated dose is the sum of the tolerated doses, not including the reactive dose. Dose-limiting symptoms indicate a true allergic reaction occurring during administration of a single dose of peanut protein at the DBPCFC that should preclude the administration of any further doses in the view of the investigator. Symptoms that require administration of any rescue medication were considered dose-limiting symptoms. Participants with treatment discontinuation or missing more than 1 doses of study drug prior to Week 12 due to reasons other than operational complications caused by public health emergency were considered non-responders.

End point type

Primary

End point timeframe

Week 12

Notes
[1] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
Justification: This outcome measure applies to ligelizumab 120 mg arm, ligelizumab 240 mg arm, and placebo 16-week to ligelizumab 120/240 mg arm (labelled as placebo for efficacy estimands).

End point values	ligelizumab 240 mg	ligelizumab 120 mg	Placebo
Number of subjects analysed	47	51	23
Units: Participants	21	8	1

Single dose of >= 600 mg without DLT at Week 12

Comparison groups

ligelizumab 120 mg v Placebo

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

P-value

= 0.073

Method

Regression, Logistic

Parameter type

Odds ratio (OR)

Point estimate

5.1

Confidence interval

level

95%

sides

2-sided

lower limit

0.8

upper limit

100.98

Single dose of >= 600 mg without DLT at Week 12

Comparison groups

ligelizumab 240 mg v Placebo

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

P-value

= 0.002

Method

Regression, Logistic

Parameter type

Odds ratio (OR)

Point estimate

25.83

Confidence interval

level

95%

sides

2-sided

lower limit

4.34

upper limit

506.78

Secondary: Percentage of participants who tolerated a single dose of >= 1000 mg (2044 mg cumulative tolerated dose) of peanut protein without dose-limiting symptoms at Week 12

Top of page

End point title

Percentage of participants who tolerated a single dose of >= 1000 mg (2044 mg cumulative tolerated dose) of peanut protein without dose-limiting symptoms at Week 12 ^[2]

End point description

Responder rate was defined as the percentage of participants tolerating a single dose of >= 1000 mg (2044 mg cumulative tolerated dose) of peanut protein without dose-limiting symptoms during the double blind placebo controlled food challenge (DBPCFC) conducted at Week 12. The cumulative tolerated dose is the sum of the tolerated doses, not including the reactive dose. Dose-limiting symptoms indicate a true allergic reaction occurring during administration of a single dose of peanut protein at the DBPCFC that should preclude the administration of any further doses in the view of the investigator. Symptoms that require administration of any rescue medication were considered dose-limiting symptoms. Participants with treatment discontinuation or missing more than 1 doses of study drug prior to Week 12 due to reasons other than operational complications caused by public health emergency were considered non-responders.

End point type

Secondary

End point timeframe

Week 12

Notes
[2] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
Justification: This outcome measure applies to ligelizumab 120 mg arm, ligelizumab 240 mg arm, and placebo 16-week to ligelizumab 120/240 mg arm (labelled as placebo for efficacy estimands).

End point values	ligelizumab 240 mg	ligelizumab 120 mg	Placebo
Number of subjects analysed	47	51	23
Units: Participants	14	6	1

Single dose of >= 1000 mg without DLT at Week 12

Comparison groups

ligelizumab 120 mg v Placebo

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

P-value

= 0.164

Method

Regression, Logistic

Parameter type

Odds ratio (OR)

Point estimate

3.02

Confidence interval

level

95%

sides

2-sided

lower limit

0.45

upper limit

59.93

Single dose of >= 1000 mg without DLT at Week 12

Comparison groups

ligelizumab 240 mg v Placebo

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

P-value

= 0.018

Method

Regression, Logistic

Parameter type

Odds ratio (OR)

Point estimate

9.86

Confidence interval

level

95%

sides

2-sided

lower limit

1.69

upper limit

188.85

Secondary: Percentage of maximum severity of symptoms occurring at any challenge dose of peanut protein up to and including 1000 mg at Week 12

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End point title

Percentage of maximum severity of symptoms occurring at any challenge dose of peanut protein up to and including 1000 mg at Week 12 ^[3]

End point description

Symptom severity occurring at any challenge dose of peanut protein up to and including 1000 mg during the DBPCFC conducted at Week 12 was categorized as 4 levels: None, Mild, Moderate, Severe. The CoFAR grading system was used to categorize the symptom severity as mild, moderate and severe. Symptom severity for participants who completed DBPCFC without any symptom were categorized as none.

End point type

Secondary

End point timeframe

Week 12

Notes
[3] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
Justification: This outcome measure applies to ligelizumab 120 mg arm, ligelizumab 240 mg arm, and placebo 16-week to ligelizumab 120/240 mg arm (labelled as placebo for efficacy estimands).

End point values	ligelizumab 240 mg	ligelizumab 120 mg	Placebo
Number of subjects analysed	47	51	23
Units: Participants
None	5	2	0
Mild	15	10	2
Moderate	24	37	13
Severe	3	2	8

Max. severity of symptoms at any dose at Week 12

Comparison groups

ligelizumab 120 mg v Placebo

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

P-value

= 0.001

Method

proportional odds model

Parameter type

Odds ratio (OR)

Point estimate

5.05

Confidence interval

level

95%

sides

2-sided

lower limit

1.8

upper limit

14.36

Max. severity of symptoms at any dose at Week 12

Comparison groups

ligelizumab 240 mg v Placebo

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

P-value

< 0.001

Method

proportional odds model

Parameter type

Odds ratio (OR)

Point estimate

10.59

Confidence interval

level

95%

sides

2-sided

lower limit

3.63

upper limit

31.56

Secondary: Percentage of participants who tolerated a single dose of 3000 mg (5044 mg cumulative tolerated dose) of peanut protein without dose-limiting symptoms at Week 12

Top of page

End point title

Percentage of participants who tolerated a single dose of 3000 mg (5044 mg cumulative tolerated dose) of peanut protein without dose-limiting symptoms at Week 12 ^[4]

End point description

Responder rate was defined as the percentage of participants tolerating a single dose of 3000 mg (5044 mg cumulative tolerated dose) of peanut protein without dose-limiting symptoms during the double blind placebo controlled food challenge (DBPCFC) conducted at Week 12. The cumulative tolerated dose is the sum of the tolerated doses, not including the reactive dose. Dose-limiting symptoms indicate a true allergic reaction occurring during administration of a single dose of peanut protein at the DBPCFC that should preclude the administration of any further doses in the view of the investigator. Symptoms that require administration of any rescue medication were considered dose-limiting symptoms. Participants with treatment discontinuation or missing more than 1 doses of study drug prior to Week 12 due to reasons other than operational complications caused by public health emergency were considered non-responders.

End point type

Secondary

End point timeframe

Week 12

Notes
[4] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
Justification: This outcome measure applies to ligelizumab 120 mg arm, ligelizumab 240 mg arm, and placebo 16-week to ligelizumab 120/240 mg arm (labelled as placebo for efficacy estimands).

End point values	ligelizumab 240 mg	ligelizumab 120 mg	Placebo
Number of subjects analysed	47	51	23
Units: Participants	7	5	1

Single dose of >= 3000 mg without DLT at Week 12

Comparison groups

ligelizumab 120 mg v Placebo

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

P-value

= 0.247

Method

Regression, Logistic

Parameter type

Odds ratio (OR)

Point estimate

2.21

Confidence interval

level

95%

sides

2-sided

lower limit

0.3

upper limit

45.56

Single dose of >= 3000 mg without DLT at Week 12

Comparison groups

ligelizumab 240 mg v Placebo

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

P-value

= 0.138

Method

Regression, Logistic

Parameter type

Odds ratio (OR)

Point estimate

3.47

Confidence interval

level

95%

sides

2-sided

lower limit

0.51

upper limit

70.08

Secondary: Percentage of participants who tolerated a single dose of >= 1000 mg (2044 mg cumulative tolerated dose) of peanut protein without dose-limiting symptoms at Week 12 after 4 weeks of Treatment

Top of page

End point title

Percentage of participants who tolerated a single dose of >= 1000 mg (2044 mg cumulative tolerated dose) of peanut protein without dose-limiting symptoms at Week 12 after 4 weeks of Treatment ^[5]

End point description

Responder rate was defined as the percentage of participants tolerating a single dose of >= 1000 mg (2044 mg cumulative tolerated dose) of peanut protein without dose-limiting symptoms during the double blind placebo controlled food challenge (DBPCFC) conducted at Week 12 after 4 weeks of treatment (8 weeks of placebo + 4 weeks of ligelizumab treatment versus 12 weeks of placebo). The cumulative tolerated dose is the sum of the tolerated doses, not including the reactive dose. Participants with treatment discontinuation or missing more than 1 doses of study drug prior to Week 12 due to reasons other than operational complications caused by public health emergency were considered non-responders.

End point type

Secondary

End point timeframe

Week 12

Notes
[5] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
Justification: This outcome measure applies to Placebo 8 weeks and ligelizumab 240 mg, Placebo 8 weeks and ligelizumab 120 mg and placebo 16-week to ligelizumab 120/240 mg arm (labelled as placebo for efficacy estimands).

End point values	Placebo 8 weeks and ligelizumab 240 mg	Placebo 8 weeks and ligelizumab 120 mg	Placebo
Number of subjects analysed	46	44	23
Units: Participants	8	1	1

>= 1000 mg without DLT at Week 12 after 4 wks Tx

Comparison groups

Placebo 8 weeks and ligelizumab 240 mg v Placebo

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

P-value

= 0.082

Method

Regression, Logistic

Parameter type

Odds ratio (OR)

Point estimate

4.73

Confidence interval

level

95%

sides

2-sided

lower limit

0.74

upper limit

93.16

>= 1000 mg without DLT at Week 12 after 4 wks Tx

Comparison groups

Placebo 8 weeks and ligelizumab 120 mg v Placebo

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

P-value

= 0.632

Method

Regression, Logistic

Parameter type

Odds ratio (OR)

Point estimate

0.61

Confidence interval

level

95%

sides

2-sided

lower limit

0.02

upper limit

16.43

Secondary: Percentage of participants who tolerated the specified peanut protein dose without dose-limiting symptoms at Week 52

Top of page

End point title

Percentage of participants who tolerated the specified peanut protein dose without dose-limiting symptoms at Week 52 ^[6]

End point description

Responder rate was defined as the percentage of participants tolerating the specified peanut protein doses (>= 600 mg (1044 mg cumulative tolerated dose), >= 1000 mg (2044 mg cumulative tolerated dose) or 3000 mg (5044 mg cumulative tolerated dose)) of peanut protein without dose-limiting symptoms during the double blind placebo controlled food challenge (DBPCFC) conducted at Week 52. The cumulative tolerated dose is the sum of the tolerated doses, not including the reactive dose. Dose-limiting symptoms indicate a true allergic reaction occurring during administration of a single dose of peanut protein at the DBPCFC that should preclude the administration of any further doses in the view of the investigator. Symptoms that require administration of any rescue medication were considered dose-limiting symptoms.

End point type

Secondary

End point timeframe

Week 52

Notes
[6] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
Justification: This outcome measure applies to ligelizumab 120 mg arm, ligelizumab 240 mg arm, Placebo 8 weeks and ligelizumab 240 mg, Placebo 8 weeks and ligelizumab 120 mg and placebo 16-week to ligelizumab 120/240 mg arm (labelled as placebo for efficacy estimands).

End point values	ligelizumab 240 mg	ligelizumab 120 mg	Placebo 8 weeks and ligelizumab 240 mg	Placebo 8 weeks and ligelizumab 120 mg
Number of subjects analysed	3	2	2	4
Units: Participants
MTD >= 600 mg	2	1	0	1
MTD >= 1000 mg	1	1	0	0
MTD = 3000 mg	1	1	0	0

No statistical analyses for this end point

Secondary: Change from baseline in maximum tolerated dose (MTD) of peanut protein without dose-limiting symptoms during the DBPCFC at Week 12 and Week 52

Top of page

End point title

Change from baseline in maximum tolerated dose (MTD) of peanut protein without dose-limiting symptoms during the DBPCFC at Week 12 and Week 52 ^[7]

End point description

Change from baseline in maximum tolerated dose (MTD) of peanut protein without dose-limiting symptoms during the DBPCFC at Week 12 and Week 52. Dose-limiting symptoms indicate a true allergic reaction occurring during administration of a single dose of peanut protein at the DBPCFC that should preclude the administration of any further doses in the view of the investigator.

End point type

Secondary

End point timeframe

Baseline, Week 12, Week 52

Notes
[7] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
Justification: This outcome measure applies to ligelizumab 120 mg arm, ligelizumab 240 mg arm, Placebo 8 weeks and ligelizumab 240 mg, Placebo 8 weeks and ligelizumab 120 mg and placebo 16-week to ligelizumab 120/240 mg arm (labelled as placebo for efficacy estimands).

End point values	ligelizumab 240 mg	ligelizumab 120 mg	Placebo 8 weeks and ligelizumab 240 mg	Placebo 8 weeks and ligelizumab 120 mg	Placebo
Number of subjects analysed	45	48	46	40	20
Units: mg
geometric mean (geometric coefficient of variation)
Week 12	274.05 ( 501.051 )	104.31 ( 794.248 )	148.98 ( 754.171 )	44.29 ( 768.803 )	46.62 ( 555.207 )
Week 52	800.41 ( 178.687 )	164.23 ( 459916.375 )	89.55 ( 408.957 )	146.48 ( 113.009 )	999 ( 999 )

No statistical analyses for this end point

Secondary: Change from baseline in peanut-specific immunoglobulin E (IgE) at Week 12 and Week 52

Top of page

End point title

Change from baseline in peanut-specific immunoglobulin E (IgE) at Week 12 and Week 52 ^[8]

End point description

Change from baseline of serum levels of peanut-specific immunoglobulin E (IgE)

End point type

Secondary

End point timeframe

Baseline, Week 12, Week 52

Notes
[8] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
Justification: This outcome measure applies to ligelizumab 120 mg arm, ligelizumab 240 mg arm, Placebo 8 weeks and ligelizumab 240 mg, Placebo 8 weeks and ligelizumab 120 mg and placebo 16-week to ligelizumab 120/240 mg arm (labelled as placebo for efficacy estimands).

End point values	ligelizumab 240 mg	ligelizumab 120 mg	Placebo 8 weeks and ligelizumab 240 mg	Placebo 8 weeks and ligelizumab 120 mg	Placebo
Number of subjects analysed	44	46	45	39	20
Units: kilounits per liter (kU/L)
arithmetic mean (standard deviation)
Week 12	258.28 ( 282.477 )	295.60 ( 321.451 )	302.22 ( 323.846 )	400.03 ( 611.676 )	-35.52 ( 179.040 )
Week 52	362.36 ( 436.857 )	297.94 ( 285.827 )	338.63 ( 435.532 )	361.44 ( 587.852 )	174.75 ( 433.889 )

No statistical analyses for this end point

Secondary: Change from baseline in peanut-specific immunoglobulin G4 (IgG4) at Week 12 and Week 52

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End point title

Change from baseline in peanut-specific immunoglobulin G4 (IgG4) at Week 12 and Week 52 ^[9]

End point description

Change from baseline of serum levels of peanut-specific immunoglobulin G4 (IgG4)

End point type

Secondary

End point timeframe

Baseline, Week 12, Week 52

Notes
[9] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
Justification: This outcome measure applies to ligelizumab 120 mg arm, ligelizumab 240 mg arm, Placebo 8 weeks and ligelizumab 240 mg, Placebo 8 weeks and ligelizumab 120 mg and placebo 16-week to ligelizumab 120/240 mg arm (labelled as placebo for efficacy estimands).

End point values	ligelizumab 240 mg	ligelizumab 120 mg	Placebo 8 weeks and ligelizumab 240 mg	Placebo 8 weeks and ligelizumab 120 mg	Placebo
Number of subjects analysed	46	46	45	40	19
Units: mg/L
arithmetic mean (standard deviation)
Week 12	-0.09 ( 0.65 )	0.35 ( 3.178 )	0.12 ( 0.903 )	0.11 ( 0.789 )	0.03 ( 0.469 )
Week 52	-0.01 ( 0.608 )	0.73 ( 3.103 )	0.21 ( 0.909 )	0.01 ( 0.768 )	-0.09 ( 1.126 )

No statistical analyses for this end point

Secondary: Change from baseline in total and domain scores in the Food Allergy Quality of Life Questionnaire (FAQLQ) Teenager Form (FAQLQ-TF)

Top of page

End point title

Change from baseline in total and domain scores in the Food Allergy Quality of Life Questionnaire (FAQLQ) Teenager Form (FAQLQ-TF) ^[10]

End point description

The Food Allergy Quality of Life Questionnaire (FAQLQ) Teenager Form (FAQLQ-TF) completed by adolescents aged 13-17 is a self-reported instrument intended to assess the effect of food allergy on the participant’s health-related quality of life (HRQoL). Each question is scored on a 7-point scale (coded as 1-7 in analysis, with a higher level indicating greater impairment in HRQoL). The total score and the domain scores are the arithmetic average of all completed items: Emotional impact (EI) (item no. 5, 12, 19-23), Allergen avoidance and dietary restrictions (AADR) (item no. 1-4, 6-10, 16), Risk of accidental exposure (RAE) (item no. 11, 13-15, 17, 18). If more than one item in any domain is missing, a domain score should not be calculated for that case. A total score could still be calculated if 20% or fewer of the items are missing.

End point type

Secondary

End point timeframe

Baseline, Week 12 Part 1 (10 Days Before Day 1 Oral Food Challenge (OFC)), Week 12 Part 2 (3 Days After Day 2 Oral Food Challenge (OFC))

Notes
[10] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
Justification: This outcome measure applies to ligelizumab 120 mg arm, ligelizumab 240 mg arm, Placebo 8 weeks and ligelizumab 240 mg, Placebo 8 weeks and ligelizumab 120 mg and placebo 16-week to ligelizumab 120/240 mg arm (labelled as placebo for efficacy estimands).

End point values	ligelizumab 240 mg	ligelizumab 120 mg	Placebo 8 weeks and ligelizumab 240 mg	Placebo 8 weeks and ligelizumab 120 mg	Placebo
Number of subjects analysed	17	20	15	16	9
Units: Unit on a scale
arithmetic mean (standard deviation)
Week 12 Part 1 - Total Score	-0.09 ( 0.833 )	-0.01 ( 0.779 )	-0.30 ( 0.647 )	-0.07 ( 0.972 )	0.27 ( 0.835 )
Week 12 Part 2 - Total Score	-0.49 ( 0.839 )	-0.13 ( 0.869 )	-0.08 ( 0.879 )	0.04 ( 1.255 )	0.09 ( 0.954 )
Week 12 Part 1 - EI	-0.03 ( 0.896 )	-0.12 ( 0.846 )	-0.40 ( 0.745 )	-0.11 ( 1.062 )	0.27 ( 1.034 )
Week 12 Part 2 - EI	-0.51 ( 1.057 )	-0.19 ( 0.875 )	-0.23 ( 0.737 )	-0.05 ( 1.348 )	-0.04 ( 1.486 )
Week 12 Part 1 - AADR	-0.15 ( 0.873 )	0.02 ( 0.977 )	-0.35 ( 0.988 )	-0.11 ( 1.130 )	0.11 ( 0.599 )
Week 12 Part 2 - AADR	-0.61 ( 0.946 )	-0.14 ( 1.004 )	-0.08 ( 1.291 )	0.12 ( 1.235 )	0.25 ( 0.628 )
Week 12 Part 1 - RAE	-0.07 ( 1.085 )	0.08 ( 0.868 )	-0.10 ( 0.796 )	0.02 ( 1.016 )	0.54 ( 1.204 )
Week 12 Part 2 - RAE	-0.28 ( 0.718 )	-0.04 ( 1.028 )	0.08 ( 0.733 )	0.03 ( 1.366 )	-0.04 ( 1.253 )

Week 12 Part 1 - Total Score

Comparison groups

ligelizumab 240 mg v Placebo

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

P-value

= 0.173

Method

ANCOVA

Parameter type

LS Mean difference

Point estimate

-0.33

Confidence interval

level

95%

sides

2-sided

lower limit

-1.03

upper limit

0.36

Week 12 Part 1 - Total Score

Comparison groups

ligelizumab 120 mg v Placebo

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

P-value

= 0.202

Method

ANCOVA

Parameter type

LS Mean difference

Point estimate

-0.29

Confidence interval

level

95%

sides

2-sided

lower limit

-0.96

upper limit

0.39

Week 12 Part 2 - Total Score

Comparison groups

ligelizumab 240 mg v Placebo

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

P-value

= 0.123

Method

ANCOVA

Parameter type

LS Mean difference

Point estimate

-0.43

Confidence interval

level

95%

sides

2-sided

lower limit

-1.16

upper limit

0.3

Week 12 Part 2 - Total Score

Comparison groups

ligelizumab 120 mg v Placebo

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

P-value

= 0.258

Method

ANCOVA

Parameter type

LS Mean difference

Point estimate

-0.23

Confidence interval

level

95%

sides

2-sided

lower limit

-0.94

upper limit

0.47

Week 12 Part 2 - Total Score

Comparison groups

Placebo 8 weeks and ligelizumab 240 mg v Placebo

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

P-value

= 0.324

Method

ANCOVA

Parameter type

LS Mean difference

Point estimate

-0.17

Confidence interval

level

95%

sides

2-sided

lower limit

-0.92

upper limit

0.58

Week 12 Part 2 - Total Score

Comparison groups

Placebo 8 weeks and ligelizumab 120 mg v Placebo

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

P-value

= 0.473

Method

ANCOVA

Parameter type

LS Mean difference

Point estimate

-0.03

Confidence interval

level

95%

sides

2-sided

lower limit

-0.78

upper limit

0.73

Week 12 Part 1 - Total Score

Comparison groups

Placebo 8 weeks and ligelizumab 240 mg v Placebo

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

P-value

= 0.151

Method

ANCOVA

Parameter type

LS Mean difference

Point estimate

-0.37

Confidence interval

level

95%

sides

2-sided

lower limit

-1.09

upper limit

0.34

Week 12 Part 1 - Total Score

Comparison groups

Placebo 8 weeks and ligelizumab 120 mg v Placebo

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

P-value

= 0.264

Method

ANCOVA

Parameter type

LS Mean difference

Point estimate

-0.23

Confidence interval

level

95%

sides

2-sided

lower limit

-0.94

upper limit

0.49

Secondary: Change from Baseline in Skin Prick Test (SPT) mean wheal diameters at Week 16/Week 56

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End point title

Change from Baseline in Skin Prick Test (SPT) mean wheal diameters at Week 16/Week 56 ^[11]

End point description

An allergen specific SPT is a commonly used diagnostic tool. In this study a titration SPT using peanut allergen provided additional information on the impact of Immunoglobulin E (IgE) suppression on skin mast cells. The size of the wheal and flare (the longest diameter and the midpoint orthogonal diameter) at each site was evaluated. Considering the study termination, SPT originally scheduled at Week 56 was performed 4 weeks after Week 12 assessment in some patients.

End point type

Secondary

End point timeframe

Baseline, Week 16/Week 56

Notes
[11] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
Justification: This outcome measure applies to ligelizumab 120 mg arm, ligelizumab 240 mg arm, Placebo 8 weeks and ligelizumab 240 mg, Placebo 8 weeks and ligelizumab 120 mg and placebo 16-week to ligelizumab 120/240 mg arm (labelled as placebo for efficacy estimands).

End point values	ligelizumab 240 mg	ligelizumab 120 mg	Placebo 8 weeks and ligelizumab 240 mg	Placebo 8 weeks and ligelizumab 120 mg	Placebo
Number of subjects analysed	36	40	37	36	16
Units: undiluted peanut protein (mm)
arithmetic mean (standard deviation)	-10.85 ( 6.423 )	-8.40 ( 5.805 )	-5.28 ( 5.237 )	-7.25 ( 7.354 )	-2.38 ( 3.788 )

Skin Prick Test (SPT) at Week 16/Week 56

Comparison groups

ligelizumab 240 mg v Placebo

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

P-value

< 0.001

Method

ANCOVA

Parameter type

LS Mean difference

Point estimate

-3.77

Confidence interval

level

95%

sides

2-sided

lower limit

-5.15

upper limit

-2.4

Skin Prick Test (SPT) at Week 16/Week 56

Comparison groups

Placebo 8 weeks and ligelizumab 120 mg v Placebo

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

P-value

= 0.13

Method

ANCOVA

Parameter type

LS Mean difference

Point estimate

-1.68

Confidence interval

level

95%

sides

2-sided

lower limit

-4.62

upper limit

1.25

Skin Prick Test (SPT) at Week 16/Week 56

Comparison groups

Placebo 8 weeks and ligelizumab 240 mg v Placebo

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

P-value

= 0.015

Method

ANCOVA

Parameter type

LS Mean difference

Point estimate

-3.2

Confidence interval

level

95%

sides

2-sided

lower limit

-6.08

upper limit

-0.32

Skin Prick Test (SPT) at Week 16/Week 56

Comparison groups

ligelizumab 120 mg v Placebo

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

P-value

< 0.001

Method

ANCOVA

Parameter type

LS Mean difference

Point estimate

-4.93

Confidence interval

level

95%

sides

2-sided

lower limit

-7.77

upper limit

-2.09

Secondary: Change from baseline in total and domain scores in the Food Allergy Quality of Life Questionnaire (FAQLQ) Adult Form (FAQLQ-AF)

Top of page

End point title

Change from baseline in total and domain scores in the Food Allergy Quality of Life Questionnaire (FAQLQ) Adult Form (FAQLQ-AF) ^[12]

End point description

The Food Allergy Quality of Life Questionnaire (FAQLQ) Adult Form (FAQLQ-AF) completed by adults aged 18-55 is a self-reported instrument intended to assess the effect of food allergy on the participant’s health-related quality of life (HRQoL). Each question is scored on a 7-point scale (coded as 1-7 in analysis, with a higher level indicating greater impairment in HRQoL). The total score and domain scores are the arithmetic average of all completed items: Emotional impact (EI) (item no. 5, 24-29), Allergen avoidance and dietary restrictions (AADR) (item no. 1-4, 6, 8-12, 20), Risk of Accidental Exposure (RAE) (item no. 7, 13-18, 21), Food allergy related health (FAH) (item no. 19, 22, 23). If more than one item in any domain is missing, a domain score should not be calculated for that case. A total score could still be calculated if 20% or fewer of the items are missing.

End point type

Secondary

End point timeframe

Baseline, Week 12 Part 1 (10 Days Before Day 1 Oral Food Challenge (OFC)), Week 12 Part 2 (3 Days After Day 2 Oral Food Challenge (OFC))

Notes
[12] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
Justification: This outcome measure applies to ligelizumab 120 mg arm, ligelizumab 240 mg arm, Placebo 8 weeks and ligelizumab 240 mg, Placebo 8 weeks and ligelizumab 120 mg and placebo 16-week to ligelizumab 120/240 mg arm (labelled as placebo for efficacy estimands).

End point values	ligelizumab 240 mg	ligelizumab 120 mg	Placebo 8 weeks and ligelizumab 240 mg	Placebo 8 weeks and ligelizumab 120 mg	Placebo
Number of subjects analysed	16	17	19	17	9
Units: Unit on a scale
arithmetic mean (standard deviation)
Week 12 Part 1 - Total Score	-0.01 ( 0.560 )	-0.40 ( 0.439 )	-0.20 ( 0.601 )	0.00 ( 0.656 )	0.00 ( 0.330 )
Week 12 Part 2 - Total Score	-0.16 ( 0.742 )	-0.55 ( 0.671 )	-0.60 ( 0.783 )	-0.22 ( 0.727 )	-0.14 ( 0.542 )
Week 12 Part 1 - EI	0.07 ( 0.747 )	-0.31 ( 0.838 )	-0.35 ( 0.601 )	-0.04 ( 0.857 )	-0.06 ( 0.358 )
Week 12 Part 2 - EI	-0.11 ( 0.980 )	-0.47 ( 1.006 )	-0.90 ( 1.027 )	-0.26 ( 0.828 )	0.00 ( 0.553 )
Week 12 Part 1 - AADR	-0.07 ( 0.609 )	-0.41 ( 0.526 )	-0.31 ( 0.805 )	-0.12 ( 0.731 )	-0.05 ( 0.508 )
Week 12 Part 2 - AADR	-0.10 ( 0.788 )	-0.74 ( 0.585 )	-0.70 ( 1.106 )	-0.20 ( 0.749 )	-0.15 ( 0.532 )
Week 12 Part 1 - RAE	0.06 ( 0.647 )	-0.40 ( 0.427 )	0.02 ( 0.834 )	0.18 ( 0.794 )	0.10 ( 0.471 )
Week 12 Part 2 - RAE	-0.15 ( 0.750 )	-0.31 ( 0.917 )	-0.34 ( 0.468 )	-0.26 ( 0.821 )	-0.33 ( 0.549 )
Week 12 Part 1 - FAH	-0.17 ( 1.082 )	-0.53 ( 0.717 )	-0.05 ( 0.788 )	0.06 ( 1.056 )	0.04 ( 1.073 )
Week 12 Part 2 - FAH	-0.47 ( 0.864 )	-0.73 ( 0.854 )	-0.22 ( 0.770 )	-0.06 ( 1.153 )	0.07 ( 1.140 )

Week 12 Part 1 - Total Score

Comparison groups

ligelizumab 240 mg v Placebo

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

P-value

= 0.597

Method

ANCOVA

Parameter type

LS Mean difference

Point estimate

0.06

Confidence interval

level

95%

sides

2-sided

lower limit

-0.44

upper limit

0.57

Week 12 Part 1 - Total Score

Comparison groups

ligelizumab 120 mg v Placebo

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

P-value

= 0.064

Method

ANCOVA

Parameter type

LS Mean difference

Point estimate

-0.39

Confidence interval

level

95%

sides

2-sided

lower limit

-0.89

upper limit

0.11

Week 12 Part 1 - Total Score

Comparison groups

Placebo 8 weeks and ligelizumab 240 mg v Placebo

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

P-value

= 0.191

Method

ANCOVA

Parameter type

LS Mean difference

Point estimate

-0.22

Confidence interval

level

95%

sides

2-sided

lower limit

-0.72

upper limit

0.28

Week 12 Part 2 - Total Score

Comparison groups

Placebo 8 weeks and ligelizumab 120 mg v Placebo

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

P-value

= 0.368

Method

ANCOVA

Parameter type

LS Mean difference

Point estimate

-0.1

Confidence interval

level

95%

sides

2-sided

lower limit

-0.68

upper limit

0.48

Week 12 Part 2 - Total Score

Comparison groups

ligelizumab 240 mg v Placebo

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

P-value

= 0.537

Method

ANCOVA

Parameter type

LS Mean difference

Point estimate

0.03

Confidence interval

level

95%

sides

2-sided

lower limit

-0.57

upper limit

0.62

Week 12 Part 2 - Total Score

Comparison groups

ligelizumab 120 mg v Placebo

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

P-value

= 0.141

Method

ANCOVA

Parameter type

LS Mean difference

Point estimate

-0.32

Confidence interval

level

95%

sides

2-sided

lower limit

-0.91

upper limit

0.27

Week 12 Part 2 - Total Score

Comparison groups

Placebo 8 weeks and ligelizumab 240 mg v Placebo

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

P-value

= 0.063

Method

ANCOVA

Parameter type

LS Meand difference

Point estimate

-0.45

Confidence interval

level

95%

sides

2-sided

lower limit

-1.04

upper limit

0.13

Week 12 Part 1 - Total Score

Comparison groups

Placebo 8 weeks and ligelizumab 120 mg v Placebo

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

P-value

= 0.472

Method

ANCOVA

Parameter type

LS Mean difference

Point estimate

-0.02

Confidence interval

level

95%

sides

2-sided

lower limit

-0.51

upper limit

0.48

Secondary: Change from baseline in total scores in the Food Allergy Independent Measure (FAIM) – Teenager Form (FAIM-TF)

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End point title

Change from baseline in total scores in the Food Allergy Independent Measure (FAIM) – Teenager Form (FAIM-TF) ^[13]

End point description

The Food Allergy Independent Measure (FAIM) – Teenager Form (FAIM-TF) completed by adolescents aged 13-17 reflects the participant’s perceived food allergy severity and food allergy-related risk. It consists of six questions (the first four of them assess participant's food allergy expectation outcomes, and the other two reflect aspects of the perceived severity of food allergy). Each question is scored on a 7-point scale (coded as 1-7 in analysis, with a greater score indicating a higher level of perceived risk or chance of adverse events occurring). The total score is the arithmetic average of all completed items. If less than 80% of the items within the score are complete, it was not calculated.

End point type

Secondary

End point timeframe

Baseline, Week 12 Part 1 (10 Days Before Day 1 Oral Food Challenge (OFC)), Week 12 Part 2 (3 Days After Day 2 Oral Food Challenge (OFC))

Notes
[13] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
Justification: This outcome measure applies to ligelizumab 120 mg arm, ligelizumab 240 mg arm, Placebo 8 weeks and ligelizumab 240 mg, Placebo 8 weeks and ligelizumab 120 mg and placebo 16-week to ligelizumab 120/240 mg arm (labelled as placebo for efficacy estimands).

End point values	ligelizumab 240 mg	ligelizumab 120 mg	Placebo 8 weeks and ligelizumab 240 mg	Placebo 8 weeks and ligelizumab 120 mg	Placebo
Number of subjects analysed	17	19	15	14	9
Units: Unit on a scale
arithmetic mean (standard deviation)
Week 12 Part 1 - Total Score	-0.03 ( 0.751 )	-0.11 ( 0.760 )	-0.04 ( 0.529 )	0.31 ( 0.989 )	0.19 ( 0.930 )
Week 12 Part 2 - Total Score	-0.31 ( 0.952 )	0.31 ( 0.784 )	-0.07 ( 0.524 )	0.39 ( 1.133 )	0.25 ( 1.306 )

Week 12 Part 1 - Total Score

Comparison groups

ligelizumab 240 mg v Placebo

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

P-value

= 0.225

Method

ANCOVA

Parameter type

LS Mean difference

Point estimate

-0.24

Confidence interval

level

95%

sides

2-sided

lower limit

-0.87

upper limit

0.39

Week 12 Part 1 - Total Score

Comparison groups

Placebo 8 weeks and ligelizumab 240 mg v Placebo

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

P-value

= 0.179

Method

ANCOVA

Parameter type

LS Mean difference

Point estimate

-0.3

Confidence interval

level

95%

sides

2-sided

lower limit

-0.95

upper limit

0.34

Week 12 Part 2 - Total Score

Comparison groups

Placebo 8 weeks and ligelizumab 120 mg v Placebo

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

P-value

= 0.514

Method

ANCOVA

Parameter type

LS Mean difference

Point estimate

0.01

Confidence interval

level

95%

sides

2-sided

lower limit

-0.7

upper limit

0.72

Week 12 Part 2 - Total Score

Comparison groups

ligelizumab 240 mg v Placebo

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

P-value

= 0.06

Method

ANCOVA

Parameter type

LS Mean difference

Point estimate

-0.52

Confidence interval

level

95%

sides

2-sided

lower limit

-1.18

upper limit

0.14

Week 12 Part 2 - Total Score

Comparison groups

ligelizumab 120 mg v Placebo

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

P-value

= 0.404

Method

ANCOVA

Parameter type

LS Mean difference

Point estimate

-0.08

Confidence interval

level

95%

sides

2-sided

lower limit

-0.72

upper limit

0.56

Week 12 Part 2 - Total Score

Comparison groups

Placebo 8 weeks and ligelizumab 240 mg v Placebo

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

P-value

= 0.135

Method

ANCOVA

Parameter type

LS Mean difference

Point estimate

-0.38

Confidence interval

level

95%

sides

2-sided

lower limit

-1.06

upper limit

0.3

Week 12 Part 1 - Total Score

Comparison groups

ligelizumab 120 mg v Placebo

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

P-value

= 0.153

Method

ANCOVA

Parameter type

LS Mean difference

Point estimate

-0.32

Confidence interval

level

95%

sides

2-sided

lower limit

-0.93

upper limit

0.3

Week 12 Part 1 - Total Score

Comparison groups

Placebo 8 weeks and ligelizumab 120 mg v Placebo

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

P-value

= 0.631

Method

ANCOVA

Parameter type

LS Mean difference

Point estimate

0.11

Confidence interval

level

95%

sides

2-sided

lower limit

-0.55

upper limit

0.77

Secondary: Change from baseline in total scores in the Food Allergy Independent Measure (FAIM) – Adult Form (FAIM-AF)

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End point title

Change from baseline in total scores in the Food Allergy Independent Measure (FAIM) – Adult Form (FAIM-AF) ^[14]

End point description

The Food Allergy Independent Measure (FAIM) – Adult Form (FAIM-AF) completed by adults aged 18-55 reflects the participant’s perceived food allergy severity and food allergy-related risk. It consists of six questions (the first four of them assess participant's food allergy expectation outcomes, and the other two reflect aspects of the perceived severity of food allergy). Each question is scored on a 7-point scale (coded as 1-7 in analysis, with a greater score indicating a higher level of perceived risk or chance of adverse events occurring). The total score is the arithmetic average of all completed items. If less than 80% of the items within the score are complete, it was calculated.

End point type

Secondary

End point timeframe

Baseline, Week 12 Part 1 (10 Days Before Day 1 Oral Food Challenge (OFC)), Week 12 Part 2 (3 Days After Day 2 Oral Food Challenge (OFC))

Notes
[14] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
Justification: This outcome measure applies to ligelizumab 120 mg arm, ligelizumab 240 mg arm, Placebo 8 weeks and ligelizumab 240 mg, Placebo 8 weeks and ligelizumab 120 mg and placebo 16-week to ligelizumab 120/240 mg arm (labelled as placebo for efficacy estimands).

End point values	ligelizumab 240 mg	ligelizumab 120 mg	Placebo 8 weeks and ligelizumab 240 mg	Placebo 8 weeks and ligelizumab 120 mg	Placebo
Number of subjects analysed	16	17	16	17	8
Units: Unit on a scale
arithmetic mean (standard deviation)
Week 12 Part 1 - Total Score	0.17 ( 0.574 )	-0.23 ( 0.496 )	-0.09 ( 0.694 )	0.00 ( 0.489 )	0.17 ( 0.309 )
Week 12 Part 2 - Total Score	-0.23 ( 0.681 )	-0.36 ( 0.562 )	-0.27 ( 0.492 )	-0.24 ( 0.479 )	0.07 ( 0.703 )

Week 12 Part 1 - Total Score

Comparison groups

ligelizumab 240 mg v Placebo

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

P-value

= 0.574

Method

ANCOVA

Parameter type

LS Mean difference

Point estimate

0.04

Confidence interval

level

95%

sides

2-sided

lower limit

-0.42

upper limit

0.51

Week 12 Part 1 - Total Score

Comparison groups

Placebo 8 weeks and ligelizumab 240 mg v Placebo

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

P-value

= 0.069

Method

ANCOVA

Parameter type

LS Mean difference

Point estimate

-0.36

Confidence interval

level

95%

sides

2-sided

lower limit

-0.83

upper limit

0.12

Week 12 Part 1 - Total Score

Comparison groups

ligelizumab 120 mg v Placebo

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

P-value

= 0.056

Method

ANCOVA

Parameter type

LS Mean difference

Point estimate

-0.37

Confidence interval

level

95%

sides

2-sided

lower limit

-0.83

upper limit

0.09

Week 12 Part 2 - Total Score

Comparison groups

Placebo 8 weeks and ligelizumab 240 mg v Placebo

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

P-value

= 0.048

Method

ANCOVA

Parameter type

LS Mean difference

Point estimate

-0.49

Confidence interval

level

95%

sides

2-sided

lower limit

-1.06

upper limit

0.09

Week 12 Part 2 - Total Score

Comparison groups

ligelizumab 120 mg v Placebo

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

P-value

= 0.071

Method

ANCOVA

Parameter type

LS Mean difference

Point estimate

-0.42

Confidence interval

level

95%

sides

2-sided

lower limit

-0.98

upper limit

0.14

Week 12 Part 2 - Total Score

Comparison groups

ligelizumab 240 mg v Placebo

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

P-value

= 0.248

Method

ANCOVA

Parameter type

LS Mean difference

Point estimate

-0.2

Confidence interval

level

95%

sides

2-sided

lower limit

-0.76

upper limit

0.37

Week 12 Part 2 - Total Score

Comparison groups

Placebo 8 weeks and ligelizumab 120 mg v Placebo

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

P-value

= 0.142

Method

ANCOVA

Parameter type

LS Mean difference

Point estimate

-0.3

Confidence interval

level

95%

sides

2-sided

lower limit

-0.85

upper limit

0.25

Week 12 Part 1 - Total Score

Comparison groups

Placebo 8 weeks and ligelizumab 120 mg v Placebo

Number of subjects included in analysis

Analysis specification

Pre-specified

Analysis type

P-value

= 0.205

Method

ANCOVA

Parameter type

LS Mean difference

Point estimate

-0.19

Confidence interval

level

95%

sides

2-sided

lower limit

-0.65

upper limit

0.27

Secondary: Change from baseline in the SF-36v2 Physical Component Score (PCS) and Mental Component Score (MCS)

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End point title

Change from baseline in the SF-36v2 Physical Component Score (PCS) and Mental Component Score (MCS) ^[15]

End point description

The SF-36v2 Health Survey is a 36-item instrument that measures generic health-related quality of life. It is designed for use in surveys of general and specific populations, health policy evaluations and clinical practice and research. It contains 8 scales and 2 component summary indices evaluating physical, social and emotional functioning in addition to general health perceptions and mental health. Responses to items allow for direct calculation of scale scores, while the physical component summary (PCS) and mental component summary (MCS) scores are computed from weighted scale scores. For all scales and summary measures, higher scores indicate better health outcomes (PCS and MCS scores range 0 to 100).

End point type

Secondary

End point timeframe

Baseline, Week 12 Part 2 (3 Days After Day 2 Oral Food Challenge (OFC))

Notes
[15] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
Justification: This outcome measure applies to ligelizumab 120 mg arm, ligelizumab 240 mg arm, Placebo 8 weeks and ligelizumab 240 mg, Placebo 8 weeks and ligelizumab 120 mg and placebo 16-week to ligelizumab 120/240 mg arm (labelled as placebo for efficacy estimands).

End point values	ligelizumab 240 mg	ligelizumab 120 mg	Placebo 8 weeks and ligelizumab 240 mg	Placebo 8 weeks and ligelizumab 120 mg	Placebo
Number of subjects analysed	10	11	10	12	5
Units: Unit on a scale
arithmetic mean (standard deviation)
Physical Component Score	0.51 ( 8.022 )	0.38 ( 2.912 )	2.13 ( 3.909 )	-2.19 ( 5.090 )	3.15 ( 5.342 )
Mental Component Score	-0.15 ( 9.950 )	-0.12 ( 8.175 )	-1.78 ( 8.458 )	-3.26 ( 8.256 )	-9.43 ( 14.662 )

No statistical analyses for this end point

Adverse events

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Timeframe for reporting adverse events

Adverse events were collected from first dose of study treatment to 30 days after last dose of study medication, up to approximately 22 months.

Adverse event reporting additional description

Any sign or symptom that occurred during the conduct of the trial and safety follow-up.

Assessment type

Systematic

Dictionary name

MedDRA

Dictionary version

26.1

Reporting group title

Placebo controlled period (Up to Week 8) QGE031 240 mg SCq4w

Reporting group description

Placebo controlled period (Up to Week 8) QGE031 240 mg SCq4w

Reporting group title

Placebo controlled period (Up to Week 8) QGE031 120 mg SCq4w

Reporting group description

Placebo controlled period (Up to Week 8) QGE031 120 mg SCq4w

Reporting group title

Placebo controlled period (Up to Week 8) Placebo

Reporting group description

Placebo controlled period (Up to Week 8) Placebo

Reporting group title

Entire study period (Up to Week 68) QGE031 120 mg SCq4w

Reporting group description

Entire study period (Up to Week 68) QGE031 120 mg SCq4w

Reporting group title

Placebo controlled period (Up to Week 16) QGE031 120 mg SCq4w

Reporting group description

Placebo controlled period (Up to Week 16) QGE031 120 mg SCq4w

Reporting group title

Placebo controlled period (Up to Week 16) Placebo

Reporting group description

Placebo controlled period (Up to Week 16) Placebo

Reporting group title

Entire study period (Up to Week 68) QGE031 240 mg SCq4w

Reporting group description

Entire study period (Up to Week 68) QGE031 240 mg SCq4w

Reporting group title

Placebo controlled period (Up to Week 16) QGE031 240 mg SCq4w

Reporting group description

Placebo controlled period (Up to Week 16) QGE031 240 mg SCq4w

Serious adverse events	Placebo controlled period (Up to Week 8) QGE031 240 mg SCq4w	Placebo controlled period (Up to Week 8) QGE031 120 mg SCq4w	Placebo controlled period (Up to Week 8) Placebo	Entire study period (Up to Week 68) QGE031 120 mg SCq4w	Placebo controlled period (Up to Week 16) QGE031 120 mg SCq4w	Placebo controlled period (Up to Week 16) Placebo	Entire study period (Up to Week 68) QGE031 240 mg SCq4w	Placebo controlled period (Up to Week 16) QGE031 240 mg SCq4w
Total subjects affected by serious adverse events
subjects affected / exposed	0 / 47 (0.00%)	0 / 51 (0.00%)	0 / 113 (0.00%)	1 / 101 (0.99%)	0 / 51 (0.00%)	0 / 23 (0.00%)	0 / 99 (0.00%)	0 / 47 (0.00%)
number of deaths (all causes)	0	0	0	0	0	0	0	0
number of deaths resulting from adverse events	0	0	0	0	0	0	0	0
Injury, poisoning and procedural complications
Post procedural complication
subjects affected / exposed	0 / 47 (0.00%)	0 / 51 (0.00%)	0 / 113 (0.00%)	1 / 101 (0.99%)	0 / 51 (0.00%)	0 / 23 (0.00%)	0 / 99 (0.00%)	0 / 47 (0.00%)
occurrences causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 1	0 / 0	0 / 0	0 / 0	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0

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

Non-serious adverse events	Placebo controlled period (Up to Week 8) QGE031 240 mg SCq4w	Placebo controlled period (Up to Week 8) QGE031 120 mg SCq4w	Placebo controlled period (Up to Week 8) Placebo	Entire study period (Up to Week 68) QGE031 120 mg SCq4w	Placebo controlled period (Up to Week 16) QGE031 120 mg SCq4w	Placebo controlled period (Up to Week 16) Placebo	Entire study period (Up to Week 68) QGE031 240 mg SCq4w	Placebo controlled period (Up to Week 16) QGE031 240 mg SCq4w
Total subjects affected by non serious adverse events
subjects affected / exposed	21 / 47 (44.68%)	17 / 51 (33.33%)	27 / 113 (23.89%)	53 / 101 (52.48%)	20 / 51 (39.22%)	9 / 23 (39.13%)	57 / 99 (57.58%)	26 / 47 (55.32%)
Nervous system disorders
Dizziness
subjects affected / exposed	2 / 47 (4.26%)	0 / 51 (0.00%)	1 / 113 (0.88%)	2 / 101 (1.98%)	0 / 51 (0.00%)	0 / 23 (0.00%)	3 / 99 (3.03%)	3 / 47 (6.38%)
occurrences all number	2	0	1	2	0	0	3	3
Headache
subjects affected / exposed	2 / 47 (4.26%)	3 / 51 (5.88%)	5 / 113 (4.42%)	12 / 101 (11.88%)	4 / 51 (7.84%)	1 / 23 (4.35%)	10 / 99 (10.10%)	4 / 47 (8.51%)
occurrences all number	2	3	7	22	6	1	14	4
General disorders and administration site conditions
Injection site induration
subjects affected / exposed	3 / 47 (6.38%)	0 / 51 (0.00%)	2 / 113 (1.77%)	2 / 101 (1.98%)	1 / 51 (1.96%)	0 / 23 (0.00%)	9 / 99 (9.09%)	3 / 47 (6.38%)
occurrences all number	4	0	2	2	1	0	15	4
Injection site erythema
subjects affected / exposed	5 / 47 (10.64%)	7 / 51 (13.73%)	0 / 113 (0.00%)	13 / 101 (12.87%)	8 / 51 (15.69%)	0 / 23 (0.00%)	15 / 99 (15.15%)	6 / 47 (12.77%)
occurrences all number	6	8	0	28	13	0	27	8
Injection site swelling
subjects affected / exposed	2 / 47 (4.26%)	4 / 51 (7.84%)	0 / 113 (0.00%)	4 / 101 (3.96%)	4 / 51 (7.84%)	0 / 23 (0.00%)	6 / 99 (6.06%)	2 / 47 (4.26%)
occurrences all number	3	4	0	7	5	0	8	3
Pyrexia
subjects affected / exposed	1 / 47 (2.13%)	0 / 51 (0.00%)	0 / 113 (0.00%)	1 / 101 (0.99%)	0 / 51 (0.00%)	0 / 23 (0.00%)	5 / 99 (5.05%)	1 / 47 (2.13%)
occurrences all number	1	0	0	1	0	0	5	1
Injection site pruritus
subjects affected / exposed	3 / 47 (6.38%)	0 / 51 (0.00%)	0 / 113 (0.00%)	1 / 101 (0.99%)	0 / 51 (0.00%)	0 / 23 (0.00%)	4 / 99 (4.04%)	3 / 47 (6.38%)
occurrences all number	3	0	0	2	0	0	4	3
Injection site pain
subjects affected / exposed	2 / 47 (4.26%)	3 / 51 (5.88%)	4 / 113 (3.54%)	4 / 101 (3.96%)	3 / 51 (5.88%)	0 / 23 (0.00%)	6 / 99 (6.06%)	3 / 47 (6.38%)
occurrences all number	2	4	4	10	4	0	12	3
Injection site oedema
subjects affected / exposed	3 / 47 (6.38%)	1 / 51 (1.96%)	0 / 113 (0.00%)	3 / 101 (2.97%)	2 / 51 (3.92%)	0 / 23 (0.00%)	7 / 99 (7.07%)	4 / 47 (8.51%)
occurrences all number	6	1	0	7	2	0	14	9
Gastrointestinal disorders
Vomiting
subjects affected / exposed	0 / 47 (0.00%)	0 / 51 (0.00%)	3 / 113 (2.65%)	1 / 101 (0.99%)	0 / 51 (0.00%)	2 / 23 (8.70%)	1 / 99 (1.01%)	0 / 47 (0.00%)
occurrences all number	0	0	3	1	0	3	1	0
Respiratory, thoracic and mediastinal disorders
Nasal congestion
subjects affected / exposed	1 / 47 (2.13%)	1 / 51 (1.96%)	1 / 113 (0.88%)	4 / 101 (3.96%)	1 / 51 (1.96%)	0 / 23 (0.00%)	5 / 99 (5.05%)	2 / 47 (4.26%)
occurrences all number	1	1	1	4	1	0	6	2
Cough
subjects affected / exposed	1 / 47 (2.13%)	0 / 51 (0.00%)	3 / 113 (2.65%)	2 / 101 (1.98%)	0 / 51 (0.00%)	2 / 23 (8.70%)	7 / 99 (7.07%)	4 / 47 (8.51%)
occurrences all number	1	0	3	3	0	2	7	4
Asthma
subjects affected / exposed	2 / 47 (4.26%)	0 / 51 (0.00%)	1 / 113 (0.88%)	3 / 101 (2.97%)	0 / 51 (0.00%)	0 / 23 (0.00%)	7 / 99 (7.07%)	2 / 47 (4.26%)
occurrences all number	2	0	1	4	0	0	11	4
Skin and subcutaneous tissue disorders
Urticaria
subjects affected / exposed	5 / 47 (10.64%)	1 / 51 (1.96%)	1 / 113 (0.88%)	6 / 101 (5.94%)	1 / 51 (1.96%)	0 / 23 (0.00%)	11 / 99 (11.11%)	7 / 47 (14.89%)
occurrences all number	8	1	1	8	1	0	14	10
Infections and infestations
COVID-19
subjects affected / exposed	1 / 47 (2.13%)	2 / 51 (3.92%)	2 / 113 (1.77%)	14 / 101 (13.86%)	5 / 51 (9.80%)	0 / 23 (0.00%)	7 / 99 (7.07%)	5 / 47 (10.64%)
occurrences all number	1	2	2	14	5	0	7	5
Influenza
subjects affected / exposed	1 / 47 (2.13%)	1 / 51 (1.96%)	0 / 113 (0.00%)	6 / 101 (5.94%)	3 / 51 (5.88%)	0 / 23 (0.00%)	5 / 99 (5.05%)	2 / 47 (4.26%)
occurrences all number	1	1	0	6	3	0	5	2
Viral upper respiratory tract infection
subjects affected / exposed	0 / 47 (0.00%)	0 / 51 (0.00%)	2 / 113 (1.77%)	1 / 101 (0.99%)	0 / 51 (0.00%)	2 / 23 (8.70%)	2 / 99 (2.02%)	0 / 47 (0.00%)
occurrences all number	0	0	2	1	0	2	2	0
Upper respiratory tract infection
subjects affected / exposed	1 / 47 (2.13%)	3 / 51 (5.88%)	7 / 113 (6.19%)	9 / 101 (8.91%)	4 / 51 (7.84%)	2 / 23 (8.70%)	8 / 99 (8.08%)	3 / 47 (6.38%)
occurrences all number	1	4	8	15	8	3	12	6
Nasopharyngitis
subjects affected / exposed	4 / 47 (8.51%)	2 / 51 (3.92%)	5 / 113 (4.42%)	14 / 101 (13.86%)	3 / 51 (5.88%)	3 / 23 (13.04%)	19 / 99 (19.19%)	8 / 47 (17.02%)
occurrences all number	4	2	6	17	3	4	23	10

More information

Top of page

Were there any global substantial amendments to the protocol? Yes

12 Apr 2022

The main purpose of Protocol Amendment 1 was to: • Change the data review mode for the DMC from semi-blinded to unblinded as per request of the DMC from 12-Oct-2021; • Adjust inclusion criterion number 4: The amended cutoff for positive peanut-specific IgE (peanut sIgE) was set at ≥ 0.35 kUA/L at Screening Visit 1 to avoid excluding participants with otherwise strong evidence supporting the diagnosis of peanut allergy (based on medical history and SPT); • Clarify and harmonize usage of prohibited medication and medication allowed under certain conditions; • Add serum tryptase as biomarker; • Reduce the number of stool samples required to determine participant eligibility at screening from three to one in asymptomatic participants; • Define two entry timepoints for participants who wish to enter the Extension study: at Week 68 (end of follow-up) for the first one third of participants or Week 52 (end of treatment) for the remaining participants. It also included other clarifications, minor updates, and corrections of typographical errors across the document.

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.

Due to EudraCT system limitations, which EMA is aware of, data using 999 as data points in this record are not an accurate representation of the clinical trial results. Please use https://www.novctrd.com/#/ for complete trial results.

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Clinical trials

Clinical Trial Results: A 52 week, multi-center, randomized, double-blind placebo-controlled study to assess the clinical efficacy and safety of ligelizumab (QGE031) in decreasing the sensitivity to peanuts in patients with peanut allergy

Trial information

Trial information

Subject disposition

Subject disposition

Baseline characteristics

Baseline characteristics

End points

End points

Primary: Percentage of participants who tolerated a single dose of >= 600 mg (1044 mg cumulative tolerated dose) of peanut protein without dose-limiting symptoms at Week 12

Primary: Percentage of participants who tolerated a single dose of >= 600 mg (1044 mg cumulative tolerated dose) of peanut protein without dose-limiting symptoms at Week 12

Secondary: Percentage of participants who tolerated a single dose of >= 1000 mg (2044 mg cumulative tolerated dose) of peanut protein without dose-limiting symptoms at Week 12

Secondary: Percentage of participants who tolerated a single dose of >= 1000 mg (2044 mg cumulative tolerated dose) of peanut protein without dose-limiting symptoms at Week 12

Secondary: Percentage of maximum severity of symptoms occurring at any challenge dose of peanut protein up to and including 1000 mg at Week 12

Secondary: Percentage of maximum severity of symptoms occurring at any challenge dose of peanut protein up to and including 1000 mg at Week 12

Secondary: Percentage of participants who tolerated a single dose of 3000 mg (5044 mg cumulative tolerated dose) of peanut protein without dose-limiting symptoms at Week 12

Secondary: Percentage of participants who tolerated a single dose of 3000 mg (5044 mg cumulative tolerated dose) of peanut protein without dose-limiting symptoms at Week 12

Secondary: Percentage of participants who tolerated a single dose of >= 1000 mg (2044 mg cumulative tolerated dose) of peanut protein without dose-limiting symptoms at Week 12 after 4 weeks of Treatment

Secondary: Percentage of participants who tolerated a single dose of >= 1000 mg (2044 mg cumulative tolerated dose) of peanut protein without dose-limiting symptoms at Week 12 after 4 weeks of Treatment

Secondary: Percentage of participants who tolerated the specified peanut protein dose without dose-limiting symptoms at Week 52

Secondary: Percentage of participants who tolerated the specified peanut protein dose without dose-limiting symptoms at Week 52

Secondary: Change from baseline in maximum tolerated dose (MTD) of peanut protein without dose-limiting symptoms during the DBPCFC at Week 12 and Week 52

Secondary: Change from baseline in maximum tolerated dose (MTD) of peanut protein without dose-limiting symptoms during the DBPCFC at Week 12 and Week 52

Secondary: Change from baseline in peanut-specific immunoglobulin E (IgE) at Week 12 and Week 52

Secondary: Change from baseline in peanut-specific immunoglobulin E (IgE) at Week 12 and Week 52

Secondary: Change from baseline in peanut-specific immunoglobulin G4 (IgG4) at Week 12 and Week 52

Secondary: Change from baseline in peanut-specific immunoglobulin G4 (IgG4) at Week 12 and Week 52

Secondary: Change from baseline in total and domain scores in the Food Allergy Quality of Life Questionnaire (FAQLQ) Teenager Form (FAQLQ-TF)

Secondary: Change from baseline in total and domain scores in the Food Allergy Quality of Life Questionnaire (FAQLQ) Teenager Form (FAQLQ-TF)

Secondary: Change from Baseline in Skin Prick Test (SPT) mean wheal diameters at Week 16/Week 56

Secondary: Change from Baseline in Skin Prick Test (SPT) mean wheal diameters at Week 16/Week 56

Secondary: Change from baseline in total and domain scores in the Food Allergy Quality of Life Questionnaire (FAQLQ) Adult Form (FAQLQ-AF)

Secondary: Change from baseline in total and domain scores in the Food Allergy Quality of Life Questionnaire (FAQLQ) Adult Form (FAQLQ-AF)

Secondary: Change from baseline in total scores in the Food Allergy Independent Measure (FAIM) – Teenager Form (FAIM-TF)

Secondary: Change from baseline in total scores in the Food Allergy Independent Measure (FAIM) – Teenager Form (FAIM-TF)

Secondary: Change from baseline in total scores in the Food Allergy Independent Measure (FAIM) – Adult Form (FAIM-AF)

Secondary: Change from baseline in total scores in the Food Allergy Independent Measure (FAIM) – Adult Form (FAIM-AF)

Secondary: Change from baseline in the SF-36v2 Physical Component Score (PCS) and Mental Component Score (MCS)

Secondary: Change from baseline in the SF-36v2 Physical Component Score (PCS) and Mental Component Score (MCS)

Adverse events

Adverse events

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Substantial protocol amendments (globally)

Interruptions (globally)

Limitations and caveats

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Clinical Trial Results:
A 52 week, multi-center, randomized, double-blind placebo-controlled study to assess the clinical efficacy and safety of ligelizumab (QGE031) in decreasing the sensitivity to peanuts in patients with peanut allergy