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Clinical Trial Results:
A Multi-Site, Randomized, Placebo-Controlled, Double-Blind, Multiple Ascending Subcutaneous Dose Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of RO7239361 in Ambulatory Boys With Duchenne Muscular Dystrophy

Summary
EudraCT number	2015-005455-28
Trial protocol	Outside EU/EEA
Global end of trial date	15 Apr 2020

Results information
Results version number	v1(current)
This version publication date	29 Oct 2020
First version publication date	29 Oct 2020
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

CN001-006

ISRCTN number

US NCT number

NCT02515669

WHO universal trial number (UTN)

Sponsor organisation name

F. Hoffmann-La Roche AG

Sponsor organisation address

Grenzacherstrasse 124, Basel, Switzerland, CH-4070

Public contact

F. Hoffmann-La Roche AG, F. Hoffmann-La Roche AG, 41 616878333, global.trial_information@roche.com

Scientific contact

F. Hoffmann-La Roche AG, F. Hoffmann-La Roche AG, 41 616878333, global.trial_information@roche.com

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

Yes

EMA paediatric investigation plan number(s)

EMEA-001793-PIP01-15

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

15 Apr 2020

Is this the analysis of the primary completion data?

Global end of trial reached?

Yes

Global end of trial date

15 Apr 2020

Was the trial ended prematurely?

Yes

Main objective of the trial

The main purpose of this study was to determine the safety and tolerability of RO7239361 in boys with Duchenne Muscular Dystrophy with any genetic mutation.

Protection of trial subjects

All study subjects and parents, guardians, or legally acceptable representatives were required to read and sign an Informed Consent Form.

Background therapy

Evidence for comparator

Actual start date of recruitment

02 Dec 2015

Long term follow-up planned

Yes

Long term follow-up rationale

Safety

Long term follow-up duration

6 Months

Independent data monitoring committee (IDMC) involvement?

Yes

Number of subjects enrolled per country

Country: Number of subjects enrolled

Canada: 4

Country: Number of subjects enrolled

United States: 39

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

Doses for this study are based upon achieving a moderate to high degree of suppression in serum free myostatin at steady-state trough across 3 dose levels. A body weight-tiered, fixed-dose strategy targeting moderate (>50%), high (>85%) and near complete (>95%) suppression of serum free myostatin was used to select the three doses for the study.

Screening details

Starting at Week 5, participants whose weight exceeded or dropped below the dosing weight tier to which they were assigned (by >1kg) were assigned to the new corresponding body weight-based dose within the participants assigned panel.

Period 1 title

24 Week Double-Blind Phase

Is this the baseline period?

Yes

Allocation method

Randomised - controlled

Blinding used

Double blind

Roles blinded

Subject, Investigator, Monitor, Carer, Data analyst, Assessor

Are arms mutually exclusive

Yes

Placebo

Arm description

Placebo subcutaneous injections on specified days

Arm type

Placebo

Investigational medicinal product name

Placebo

Investigational medicinal product code

Other name

Pharmaceutical forms

Solution for injection

Routes of administration

Subcutaneous use

Dosage and administration details

Placebo subcutaneous injections on specified days.

Panel 1 RO7239361

Arm description

RO7239361 subcutaneous injections on specified days. The Panel 1 dose targets achievement of > 50% suppression in levels of free myostatin at trough after 5 weeks of weekly dosing. The Panel 1 body weight tier dose is >15kg at a dose of 4 mg (milligram) /0.8 mL (millilitre).

Arm type

Experimental

Investigational medicinal product name

RO7239361

Investigational medicinal product code

Other name

Pharmaceutical forms

Solution for injection

Routes of administration

Subcutaneous use

Dosage and administration details

Participants in Panel 1 received 4 mg RO7239361 on specified days.

Panel 2 RO7239361

Arm description

RO7239361 subcutaneous injections on specified days. The Panel 2 dose targets achievement of > 85% suppression in levels of free myostatin at trough after 5 weeks of weekly dosing. Panel 2 body weight tier doses are =>15kg to =< 45kg at a dose of 12.5 mg /0.25 mL and =>45kg at a dose of 20 mg /0.4 mL

Arm type

Experimental

Investigational medicinal product name

RO7239361

Investigational medicinal product code

Other name

Pharmaceutical forms

Solution for injection

Routes of administration

Subcutaneous use

Dosage and administration details

Participants in Panel 2 received either 12.5 mg (weight between 15 and 45 kg) or 20 mg (weight >45 kg) RO7239361 on specified days.

Panel 3 and Expansion Panel RO7239361

Arm description

RO7239361 subcutaneous injections on specified days. The Panel 3 and Expansion Panel dose targets achievement of > 95% suppression in levels of free myostatin at trough after 5 weeks of weekly dosing. Panel 3 and Expansion body weight tier doses are =>15kg to =< 45kg at a dose of 35 mg /0.7 mL and =>45kg at a dose of 50 mg /1.0 mL

Arm type

Experimental

Investigational medicinal product name

RO7239361

Investigational medicinal product code

Other name

Pharmaceutical forms

Solution for injection

Routes of administration

Subcutaneous use

Dosage and administration details

Participants in Panel 3 and the Expansion Panel received either 35 mg (weight between 15 and 45 kg) or 50 mg (weight >45 kg) RO7239361 on specified days.

Number of subjects in period 1	Placebo	Panel 1 RO7239361	Panel 2 RO7239361	Panel 3 and Expansion Panel RO7239361
Started	11	7	6	19
Completed	11	7	6	19

Period 2 title

228 Week Open-Label Phase

Is this the baseline period?

Allocation method

Not applicable

Blinding used

Not blinded

Are arms mutually exclusive

Yes

Panel 1 RO7239361

Arm description

Arm type

Experimental

Investigational medicinal product name

RO7239361

Investigational medicinal product code

Other name

Pharmaceutical forms

Solution for injection

Routes of administration

Subcutaneous use

Dosage and administration details

Participants in Panel 1 received 4 mg RO7239361 on specified days.

Panel 2 RO7239361

Arm description

Arm type

Experimental

Investigational medicinal product name

RO7239361

Investigational medicinal product code

Other name

Pharmaceutical forms

Solution for injection

Routes of administration

Subcutaneous use

Dosage and administration details

Participants in Panel 2 received either 12.5 mg (weight between 15 and 45 kg) or 20 mg (weight >45 kg) RO7239361 on specified days.

Panel 3 and Expansion Panel RO7239361

Arm description

Arm type

Experimental

Investigational medicinal product name

RO7239361

Investigational medicinal product code

Other name

Pharmaceutical forms

Solution for injection

Routes of administration

Subcutaneous use

Dosage and administration details

Participants in Panel 3 and the Expansion Panel received either 35 mg (weight between 15 and 45 kg) or 50 mg (weight >45 kg) RO7239361 on specified days.

Number of subjects in period 2	Panel 1 RO7239361	Panel 2 RO7239361	Panel 3 and Expansion Panel RO7239361
Started	9	8	26
Completed	0	0	0
Not completed	9	8	26
Subject Request to Discontinue Treatment	-	2	3
Unspecified	-	1	4
Missing	2	2	2
Administrative Reason by Sponsor	7	2	17
Lack of efficacy	-	1	-

Baseline characteristics

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Reporting group title

Placebo

Reporting group description

Placebo subcutaneous injections on specified days

Reporting group title

Panel 1 RO7239361

Reporting group description

Reporting group title

Panel 2 RO7239361

Reporting group description

Reporting group title

Panel 3 and Expansion Panel RO7239361

Reporting group description

Reporting group values	Placebo	Panel 1 RO7239361	Panel 2 RO7239361	Panel 3 and Expansion Panel RO7239361	Total
Number of subjects	11	7	6	19	43
Age categorical
Units: Subjects
Children (2-11 years)	11	7	6	19	43
Age Continuous
Units: Years
arithmetic mean (standard deviation)	8.8 ± 1.33	8.0 ± 2.24	8.0 ± 1.79	8.1 ± 1.81	-
Sex: Female, Male
Units: Participants
Female	0	0	0	0	0
Male	11	7	6	19	43

End points

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Reporting group title

Placebo

Reporting group description

Placebo subcutaneous injections on specified days

Reporting group title

Panel 1 RO7239361

Reporting group description

Reporting group title

Panel 2 RO7239361

Reporting group description

Reporting group title

Panel 3 and Expansion Panel RO7239361

Reporting group description

Reporting group title

Panel 1 RO7239361

Reporting group description

Reporting group title

Panel 2 RO7239361

Reporting group description

Reporting group title

Panel 3 and Expansion Panel RO7239361

Reporting group description

Subject analysis set title

Panel 2 RO7239361 12.5mg

Subject analysis set type

Sub-group analysis

Subject analysis set description

Subject analysis set title

Panel 2 RO7239361 20mg

Subject analysis set type

Sub-group analysis

Subject analysis set description

Subject analysis set title

Panel 3 RO7239361 35mg

Subject analysis set type

Sub-group analysis

Subject analysis set description

RO7239361 subcutaneous injections on specified days. RO7239361 subcutaneous injections on specified days, The Panel 2 dose targets achievement of > 85% suppression in levels of free myostatin at trough after 5 weeks of weekly dosing. Panel 2 body weight tier doses are =>15kg to =< 45kg at a dose of 12.5 mg /0.25 mL and =>45kg at a dose of 20 mg /0.4 mL

Subject analysis set title

Expansion Panel RO7239361 35mg

Subject analysis set type

Sub-group analysis

Subject analysis set description

Subject analysis set title

Expansion Panel RO7239361 50mg

Subject analysis set type

Sub-group analysis

Subject analysis set description

Subject analysis set title

Panel 3 RO7239361

Subject analysis set type

Sub-group analysis

Subject analysis set description

Subject analysis set title

Expansion Panel RO7239361

Subject analysis set type

Sub-group analysis

Subject analysis set description

Subject analysis set title

Panel 3 RO7239361

Subject analysis set type

Sub-group analysis

Subject analysis set description

Subject analysis set title

Placebo, then RO7239361

Subject analysis set type

Sub-group analysis

Subject analysis set description

Placebo subcutaneous injections on specified days. At the end of the DB period these participants switched to treatment to treatment with RO7239361 at dose levels of either Panel 1, Panel 2 or Panel 3.

Subject analysis set title

Panel 1 RO7239361 Whole Study

Subject analysis set type

Sub-group analysis

Subject analysis set description

Subject analysis set title

Panel 2 RO7239361 Whole Study

Subject analysis set type

Sub-group analysis

Subject analysis set description

Subject analysis set title

Panel 3 and Expansion Panel RO7239361 Whole Study

Subject analysis set type

Sub-group analysis

Subject analysis set description

Primary: Safety Summary for the 24 Week Double-Blind Phase

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End point title

Safety Summary for the 24 Week Double-Blind Phase ^[1]

End point description

Percentage of participants with fatalities, adverse event (AEs) and serious adverse events (SAEs) up to Week 24. Participants in Panel 1 received 4 mg RO7239361; participants in Panel 2 received either 12.5 mg (weight between 15 and 45 kg) or 20 mg (weight >45 kg) RO7239361, and participants in Panel 3 and the Expansion Panel received either 35 mg (weight between 15 and 45 kg) or 50 mg (weight >45 kg) RO7239361.

End point type

Primary

End point timeframe

Baseline to Week 24

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: No statistical analyses were planned for this safety endpoint.

End point values	Placebo	Panel 1 RO7239361	Panel 2 RO7239361	Panel 3 and Expansion Panel RO7239361
Number of subjects analysed	11	7	6	19
Units: percentage of participants
number (not applicable)
Death	0	0	0	0
Adverse Events (AEs)	81.8	71.4	100.0	94
AEs leading to study withdrawal	0	0	0	0
Serious Adverse Events (SAE)	9.1	0	0	5.3
Very Severe AEs	0	0	0	0
Severe AEs	0	0	0	0
Related AEs	27.3	14.3	50.0	36.8

No statistical analyses for this end point

Primary: Safety Summary up to Week 72

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End point title

Safety Summary up to Week 72 ^[2]

End point description

Percentage of participants with fatalities, adverse event (AEs) and serious adverse events (SAEs) up to Week 72. Participants in Panel 1 received 4 mg RO7239361; participants in Panel 2 received either 12.5 mg (weight between 15 and 45 kg) or 20 mg (weight >45 kg) RO7239361, and participants in Panel 3 and the Expansion Panel received either 35 mg (weight between 15 and 45 kg) or 50 mg (weight >45 kg) RO7239361.

End point type

Primary

End point timeframe

Baseline to Week 72

Notes
[2] - 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: No statistical analyses were planned for this safety endpoint.

End point values	Panel 1 RO7239361	Panel 2 RO7239361	Panel 3 and Expansion Panel RO7239361
Number of subjects analysed	9	8	26
Units: percentage of participants
number (not applicable)
Death	0	0	0
Adverse Events (AEs)	100.0	100.0	96.2
AEs leading to study withdrawal	0	0	0
Serious Adverse Events (SAE)	0	25.0	7.7
Very Severe AEs	0	0	0
Severe AEs	0	25.0	7.7
Related AEs	77.8	62.5	50.0

No statistical analyses for this end point

Secondary: Maximum Observed Serum Concentrations (Cmax) of RO7239361 at Steady State for 4 mg QW, 12.5 mg QW and 35 mg QW doses.

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End point title

Maximum Observed Serum Concentrations (Cmax) of RO7239361 at Steady State for 4 mg QW, 12.5 mg QW and 35 mg QW doses. ^[3]

End point description

PK parameter estimates at steady state. Steady state was achieved following approximately 12 weeks QW administration. Panel: 1 = 4mg QW, 2 = 12.5mg QW, 3 = 35mg QW Participants in Panel 1 received 4 mg RO7239361; participants in Panel 2 received either 12.5 mg (weight between 15 and 45 kg) or 20 mg (weight >45 kg) RO7239361, and participants in Panel 3 and the Expansion Panel received either 35 mg (weight between 15 and 45 kg) or 50 mg (weight >45 kg) RO7239361. No participants received the Panel 2 20mg dose.

End point type

Secondary

End point timeframe

Day 1: predose, 3, 6, 72 and 96 hours (h) postdose; Days 8, 15 and 22: predose; Day 29: predose and 96 h postdose; Weeks 12 and 24: predose

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: Results are only reported for the arms receiving the doses of RO7239361 as indicated.

End point values	Panel 1 RO7239361	Panel 2 RO7239361	Panel 3 and Expansion Panel RO7239361
Number of subjects analysed	7	6	19
Units: ng/ml
geometric mean (geometric coefficient of variation)	3217 ± 15.8	8490 ± 21.5	24242 ± 26.3

No statistical analyses for this end point

Secondary: Maximum Observed Serum Concentrations (Cmax) of RO7239361 at Steady State for 50 mg QW dose.

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End point title

Maximum Observed Serum Concentrations (Cmax) of RO7239361 at Steady State for 50 mg QW dose. ^[4]

End point description

PK parameter estimates at steady state following approximately 12 weeks QW administration. Panel 3 = 50 mg QW Participants in Panel 1 received 4 mg RO7239361; participants in Panel 2 received either 12.5 mg (weight between 15 and 45 kg) or 20 mg (weight >45 kg) RO7239361, and participants in Panel 3 and the Expansion Panel received either 35 mg (weight between 15 and 45 kg) or 50 mg (weight >45 kg) RO7239361. 9999=not calculable as data are reported for one subject.

End point type

Secondary

End point timeframe

Day 1: predose, 3, 6, 72 and 96 h postdose; Days 8, 15 and 22: predose; Day 29: predose and 96 h postdose; Weeks 12 and 24: predose

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: Results are only reported for the arm receiving the dose of RO7239361 as indicated.

End point values	Panel 3 and Expansion Panel RO7239361
Number of subjects analysed	19
Units: ng/mL
geometric mean (geometric coefficient of variation)	23297 ± 9999

No statistical analyses for this end point

Secondary: Time of Maximum Observed Serum Concentrations (Tmax) of RO7239361 at Steady State for 4 mg QW, 12.5 mg QW and 35 mg QW doses.

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End point title

Time of Maximum Observed Serum Concentrations (Tmax) of RO7239361 at Steady State for 4 mg QW, 12.5 mg QW and 35 mg QW doses. ^[5]

End point description

PK parameter estimates at steady state. Steady state was achieved following approximately 12 weeks QW administration. Panel: 1 = 4mg QW, 2 = 12.5mg QW, 3 = 35mg QW. Results for the Panel 3 50mg QW dose level are represented in Outcome Measure 6. Participants in Panel 1 received 4 mg RO7239361; participants in Panel 2 received either 12.5 mg (weight between 15 and 45 kg) or 20 mg (weight >45 kg) RO7239361, and participants in Panel 3 and the Expansion Panel received either 35 mg (weight between 15 and 45 kg) or 50 mg (weight >45 kg) RO7239361.

End point type

Secondary

End point timeframe

Day 1: predose, 3, 6, 72 and 96 h postdose; Days 8, 15 and 22: predose; Day 29: predose and 96 h postdose; Weeks 12 and 24: predose

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: Results are only reported for the arms receiving the doses of RO7239361 as indicated.

End point values	Panel 1 RO7239361	Panel 2 RO7239361	Panel 3 and Expansion Panel RO7239361
Number of subjects analysed	7	6	18
Units: hour
median (full range (min-max))	28 (12 to 48)	24 (11 to 47)	30 (11 to 65)

No statistical analyses for this end point

Secondary: Time of Maximum Observed Serum Concentrations (Tmax) of RO7239361 at Steady State for 50 mg QW dose.

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End point title

Time of Maximum Observed Serum Concentrations (Tmax) of RO7239361 at Steady State for 50 mg QW dose. ^[6]

End point description

End point type

Secondary

End point timeframe

Day 1: predose, 3, 6, 72 and 96 h postdose; Days 8, 15 and 22: predose; Day 29: predose and 96 h postdose; Weeks 12 and 24: predose

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: Results are only reported for the arm receiving the dose of RO7239361 as indicated.

End point values	Panel 3 and Expansion Panel RO7239361
Number of subjects analysed	1
Units: hour
median (full range (min-max))	44 (44 to 44)

No statistical analyses for this end point

Secondary: Area Under the Concentration-Time Curve from Time Zero to Time of Next Dosing (AUCtau) of RO7239361 at Steady State for 4 mg QW, 12.5 mg QW and 35 mg QW doses.

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End point title

Area Under the Concentration-Time Curve from Time Zero to Time of Next Dosing (AUCtau) of RO7239361 at Steady State for 4 mg QW, 12.5 mg QW and 35 mg QW doses. ^[7]

End point description

PK parameter estimates at steady state. Steady state was achieved following approximately 12 weeks QW administration. Panel: 1 = 4mg QW, 2 = 12.5mg QW, 3 = 35mg QW Results for the Panel 3 50mg QW dose level are represented in Outcome Measure 8. Participants in Panel 1 received 4 mg RO7239361; participants in Panel 2 received either 12.5 mg (weight between 15 and 45 kg) or 20 mg (weight >45 kg) RO7239361, and participants in Panel 3 and the Expansion Panel received either 35 mg (weight between 15 and 45 kg) or 50 mg (weight >45 kg) RO7239361.

End point type

Secondary

End point timeframe

Day 1: predose, 3, 6, 72 and 96 h postdose; Days 8, 15 and 22: predose; Day 29: predose and 96 h postdose; Weeks 12 and 24: predose

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: Results are only reported for the arms receiving the doses of RO7239361 as indicated.

End point values	Panel 1 RO7239361	Panel 2 RO7239361	Panel 3 and Expansion Panel RO7239361
Number of subjects analysed	7	6	18
Units: ng•day/mL
geometric mean (geometric coefficient of variation)	18676 ± 23	51461 ± 18	150609 ± 24

No statistical analyses for this end point

Secondary: Area Under the Concentration-Time Curve from Time Zero to Time of Next Dosing (AUCtau) of RO7239361 at Steady State for 50 mg QW dose.

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End point title

Area Under the Concentration-Time Curve from Time Zero to Time of Next Dosing (AUCtau) of RO7239361 at Steady State for 50 mg QW dose. ^[8]

End point description

End point type

Secondary

End point timeframe

Day 1: predose, 3, 6, 72 and 96 h postdose; Days 8, 15 and 22: predose; Day 29: predose and 96 h postdose; Weeks 12 and 24: predose

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: Results are only reported for the arm receiving the dose of RO7239361 as indicated.

End point values	Panel 3 and Expansion Panel RO7239361
Number of subjects analysed	1
Units: ng•day/mL
geometric mean (geometric coefficient of variation)	151000 ± 9999

No statistical analyses for this end point

Secondary: RO7239361 Trough Concentrations

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End point title

RO7239361 Trough Concentrations ^[9]

End point description

Trough concentrations of RO7239361 at different dose levels. Panel 1 = 4mg, Panel 2 = 12.5mg and 20mg, Panel 3 = 35mg, Expansion Panels = 35mg and 50mg. 9999=participant did not receive treatment at visit or sample was not taken; 99999=not evaluable as only data from one participant reported here Participants in Panel 1 received 4 mg RO7239361; participants in Panel 2 received either 12.5 mg (weight between 15 and 45 kg) or 20 mg (weight >45 kg) RO7239361, and participants in Panel 3 and the Expansion Panel received either 35 mg (weight between 15 and 45 kg) or 50 mg (weight >45 kg) RO7239361.

End point type

Secondary

End point timeframe

Day 1: predose, 3, 6, 72 and 96 h postdose; Days 8, 15 and 22: predose; Day 29: predose and 96 h postdose; Weeks 12 and 24: predose

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: Results are only reported for arms receiving RO7239361.

End point values	Panel 1 RO7239361	Panel 2 RO7239361 12.5mg	Panel 2 RO7239361 20mg	Panel 3 RO7239361 35mg	Expansion Panel RO7239361 35mg	Expansion Panel RO7239361 50mg
Number of subjects analysed	7	7	1	7	16	1
Units: ng/mL
geometric mean (geometric coefficient of variation)
Day 1 (n=7,6,0,5,12,1)	825.343 ± 23	2115.951 ± 20	9999 ± 9999	6559.364 ± 26	6017.978 ± 23	5020.000 ± 99999
Day 15 (n=7,6,0,6,12,1)	1258.746 ± 35	3697.590 ± 20	9999 ± 9999	9957.352 ± 34	9944.183 ± 24	9980.000 ± 99999
Day 22 (n=5,6,0,5,11,0)	1640.052 ± 15	4220.280 ± 32	9999 ± 9999	12383.747 ± 30	13215.188 ± 24	999 ± 9999
Day 29 (n=7,6,0,6,12,0)	2015.695 ± 32	5107.668 ± 25	9999 ± 9999	14110.005 ± 34	15094.455 ± 19	9999 ± 9999
Day 84 (n=8,6,0,7,16,1)	2179.385 ± 26	6484.502 ± 19	9999 ± 9999	19332.421 ± 20	18789.382 ± 18	27800.000 ± 99999
Day 168 (n=9,7,1,7,14,1)	2438.595 ± 16	6100.315 ± 25	6340.000 ± 99999	19923.182 ± 41	12580.299 ± 41	26200.000 ± 99999

No statistical analyses for this end point

Secondary: Percentage of Participants with Positive Anti-RO7239361 Antibodies (ADA) Assessment, Double-Blind Phase

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End point title

Percentage of Participants with Positive Anti-RO7239361 Antibodies (ADA) Assessment, Double-Blind Phase

End point description

End point type

Secondary

End point timeframe

Day 8 through Week 24, baseline and on-study information represented in table.

End point values	Placebo	Panel 1 RO7239361	Panel 2 RO7239361	Panel 3 and Expansion Panel RO7239361
Number of subjects analysed	11	7	6	19
Units: percentage of participants
number (not applicable)
Baseline (BL): ADA Positive	0	0	0	0
BL: ADA Negative	0	71.4	100.0	89.5
BL: Missing	100.0	28.6	0	10.5
On-treatment: ADA Positive	0	0	0	0
On-treatment: Persistent Positive	0	0	0	0
On-treatment: Only last sample Positive	0	0	0	0
On-treatment: Other Positive	0	0	0	0
On-treatment: ADA Negative	0	100.0	100.0	100.0
On-treatment: ADA Negative, BL Positive, No Boost	0	0	0	0
On-treatment: Missing	100.0	0	0	0

No statistical analyses for this end point

Secondary: Percentage of Participants with Positive Anti-RO7239361 Antibodies (ADA) Assessment up to Week 72

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End point title

Percentage of Participants with Positive Anti-RO7239361 Antibodies (ADA) Assessment up to Week 72

End point description

A positive ADA sample is defined as one in which the presence of detectable ADAs is confirmed to be specific to RO7239361. A participant is considered as ADA positive if, after initiation of treatment, they have an ADA positive relative to baseline sample. Double-blind phase data for placebo participants is not included. Placebo participants in each arm moved on to RO7239361 upon entering the open label phase. Participants in Panel 1 received 4 mg RO7239361; participants in Panel 2 received either 12.5 mg (weight between 15 and 45 kg) or 20 mg (weight >45 kg) RO7239361, and participants in Panel 3 and the Expansion Panel received either 35 mg (weight between 15 and 45 kg) or 50 mg (weight >45 kg) RO7239361.

End point type

Secondary

End point timeframe

Day 8 through Week 72, baseline and on-study information represented in table.

End point values	Panel 1 RO7239361	Panel 2 RO7239361	Panel 3 and Expansion Panel RO7239361
Number of subjects analysed	9	8	26
Units: percentage of participants
number (not applicable)
Baseline (BL): ADA Positive	0	0	0
BL: ADA Negative	77.8	87.5	88.5
BL: Missing	22.2	12.5	11.5
On-treatment: ADA Positive	0	0	3.8
On-treatment: Persistent Positive	0	0	0
On-treatment: Only last sample Positive	0	0	3.8
On-treatment: Other Positive	0	0	0
On-treatment: ADA Negative	100.0	100.0	96.2
On-treatment: ADA Negative, BL Positive, No Boost	0	0	0
On-treatment: Missing	0	0	0

No statistical analyses for this end point

Secondary: Serum Concentration of Free Myostatin in the Double-Blind Phase

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End point title

Serum Concentration of Free Myostatin in the Double-Blind Phase

End point description

Participants in Panel 1 received 4 mg RO7239361; participants in Panel 2 received either 12.5 mg (weight between 15 and 45 kg) or 20 mg (weight >45 kg) RO7239361, and participants in Panel 3 and the Expansion Panel received either 35 mg (weight between 15 and 45 kg) or 50 mg (weight >45 kg) RO7239361. The pharmacodynamics (PD) data set included all available data from subjects for whom PD measurements were available at baseline and at least one other timepoint.

End point type

Secondary

End point timeframe

Baseline through Week 24

End point values	Placebo	Panel 1 RO7239361	Panel 2 RO7239361	Panel 3 and Expansion Panel RO7239361
Number of subjects analysed	11	7	6	19
Units: pg/mL
arithmetic mean (standard deviation)
Baseline (n=11,6,6,16)	1405.1 ± 809.57	815.3 ± 155.14	1391.3 ± 1084.97	1121.8 ± 526.45
Day 4 (n=8,5,4,18)	1356.6 ± 641.27	421.4 ± 70.60	321.3 ± 165.91	58.3 ± 33.20
Day 5 (n=9,7,6,18)	1438.2 ± 809.18	446.6 ± 108.81	357.8 ± 166.27	66.1 ± 39.07
Day 8 (n=10,6,6,19)	1014.2 ± 549.26	522.7 ± 131.51	479.2 ± 319.13	206.8 ± 419.21
Day 15 (n=11,7,6,19)	1266.9 ± 611.27	478.4 ± 111.44	280.8 ± 148.53	54.8 ± 61.85
Day 22 (n=11,7,6,17)	1317.1 ± 728.48	382.4 ± 82.58	284.8 ± 324.16	35.3 ± 51.45
Day 29 (n=11,7,6,17)	1214.0 ± 676.80	372.3 ± 79.97	179.0 ± 137.39	24.8 ± 27.24
Day 33 (n=11,7,6,18)	1141.4 ± 606.99	291.1 ± 77.55	115.7 ± 84.49	24.3 ± 30.64
Week 12 (n=10,6,4,18)	1366.5 ± 631.28	307.8 ± 97.84	110.8 ± 49.80	16.2 ± 11.60
Week 24 (n=11,7,6,18)	1194.0 ± 599.59	297.1 ± 74.62	160.8 ± 122.75	23.4 ± 25.34

No statistical analyses for this end point

Secondary: Percent Inhibition of Free Myostatin in the Double-Blind Phase

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End point title

Percent Inhibition of Free Myostatin in the Double-Blind Phase

End point description

End point type

Secondary

End point timeframe

Baseline through Week 24

End point values	Placebo	Panel 1 RO7239361	Panel 2 RO7239361	Panel 3 and Expansion Panel RO7239361
Number of subjects analysed	11	7	6	19
Units: percentage inhibition
arithmetic mean (standard deviation)
Day 4 (n=8,5,4,18)	0.595 ± 23.6952	49.689 ± 11.7394	73.533 ± 9.8637	95.334 ± 2.4477
Day 5 (n=9,7,6,18)	3.114 ± 20.3395	46.506 ± 9.0917	69.700 ± 10.3411	94.545 ± 2.7608
Day 8 (n=10,6,6,19)	23.917 ± 31.1378	38.411 ± 7.0320	62.524 ± 9.7074	84.797 ± 22.1958
Day 15 (n=11,7,6,19)	5.744 ± 18.6190	40.763 ± 10.0071	77.090 ± 6.2939	96.488 ± 1.9665
Day 22 (n=11,7,6,17)	5.561 ± 11.7425	51.836 ± 9.3488	81.693 ± 5.7392	97.927 ± 1.1259
Day 29 (n=11,7,6,17)	12.599 ± 16.1591	52.926 ± 6.6001	86.894 ± 2.9590	98.202 ± 1.4590
Day 33 (n=11,7,6,18)	15.662 ± 17.3640	61.796 ± 4.5760	91.557 ± 2.6444	97.042 ± 5.4443
Week 12 (n=10,6,4,18)	5.289 ± 15.5082	60.254 ± 9.4283	92.454 ± 1.7243	98.415 ± 1.2882
Week 24 (n=11,7,6,18)	9.910 ± 17.8109	63.790 ± 5.7031	88.392 ± 5.1669	97.921 ± 2.2143

No statistical analyses for this end point

Secondary: Serum Concentration of Drug-Myostatin Complex in the Double-Blind Phase

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End point title

Serum Concentration of Drug-Myostatin Complex in the Double-Blind Phase

End point description

End point type

Secondary

End point timeframe

Baseline through Week 24

End point values	Placebo	Panel 1 RO7239361	Panel 2 RO7239361	Panel 3 and Expansion Panel RO7239361
Number of subjects analysed	11	7	6	19
Units: ng/mL
arithmetic mean (standard deviation)
Day 4 (n=9,3,4,7)	1.178 ± 1.8833	5.070 ± 0.9456	12.606 ± 5.7623	14.696 ± 5.7926
Day 5 (n=8,5,6,6)	2.119 ± 2.9826	5.845 ± 2.5725	14.856 ± 7.6131	17.308 ± 7.5955
Day 8 (n=10,6,6,7)	4.924 ± 12.9975	7.041 ± 2.3470	19.906 ± 9.8664	21.609 ± 11.0880
Day 15 (n=11,7,6,7)	0.613 ± 0.2102	14.067 ± 7.1096	29.229 ± 10.4929	45.289 ± 18.7600
Day 22 (n=10,7,6,6)	1.326 ± 1.3410	11.985 ± 4.8243	36.811 ± 26.8560	52.841 ± 21.5542
Day 29 (n=11,7,6,7)	1.593 ± 3.1007	15.735 ± 4.2010	43.236 ± 34.0300	67.255 ± 27.9376
Day 33 (n=9,7,4,7)	1.157 ± 1.0036	18.542 ± 3.9874	46.279 ± 24.1457	77.142 ± 32.4153
Week 12 (n=10,6,5,14)	7.082 ± 15.2289	28.556 ± 22.7890	79.555 ± 17.2126	88.402 ± 29.8080
Week 24 (n=8,7,5,7)	3.589 ± 7.8108	25.997 ± 6.4384	83.262 ± 64.2281	101.406 ± 45.4197

No statistical analyses for this end point

Secondary: Fold Change from Baseline in Contractile Versus Non-contractile Content for Muscles in the Right Thigh in the Double-Blind Phase

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End point title

Fold Change from Baseline in Contractile Versus Non-contractile Content for Muscles in the Right Thigh in the Double-Blind Phase ^[10]

End point description

Ratio of contractile vs non-contractile content is contractile content / non-contractile content. Fold change from baseline of the ratio is defined as the ratio of fold change from baseline of contractile content vs fold change from baseline of non-contractile content. Right thigh measurements. W12 = Week 12, W24 = Week 24. Participants in Panel 1 received 4 mg RO7239361; participants in Panel 2 received either 12.5 mg (weight between 15 and 45 kg) or 20 mg (weight >45 kg) RO7239361, and participants in Panel 3 and the Expansion Panel received either 35 mg (weight between 15 and 45 kg) or 50 mg (weight >45 kg) RO7239361.

End point type

Secondary

End point timeframe

Baseline through Week 24

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: Results for Panel 3 and the Expansion Panel were reported separately by Subject analysis sets.

End point values	Placebo	Panel 1 RO7239361	Panel 2 RO7239361	Panel 3 RO7239361	Expansion Panel RO7239361
Number of subjects analysed	11	7	6	6	13
Units: Fold Change
arithmetic mean (standard error)
W12: ADDUCTOR LONGUS (n=7,3,5,4,11)	1.04 ± 0.291	0.95 ± 0.088	1.16 ± 0.330	2.27 ± 1.122	2.36 ± 0.875
W12: ADDUCTOR MAGNUS (n=8,5,4,5,11)	0.69 ± 0.104	0.88 ± 0.283	1.12 ± 0.281	0.93 ± 0.255	1.04 ± 0.101
W12: BICEPS FEMORIS LONG (n=7,4,6,4,11)	0.78 ± 0.221	0.94 ± 0.304	1.57 ± 0.556	1.10 ± 0.280	1.26 ± 0.237
W12: BICEPS FEMORIS SHORT (n=7,4,4,5,11)	0.92 ± 0.269	2.77 ± 1.144	2.03 ± 1.564	0.79 ± 0.162	1.31 ± 0.301
W12: GRACILIS (n=6,4,4,4,11)	1.13 ± 0.318	7.57 ± 5.372	0.98 ± 0.176	3.41 ± 2.474	5.42 ± 3.871
W12:RECTUS FEMORIS (n=7,6,6,4,11)	0.74 ± 0.185	0.55 ± 0.134	4.49 ± 2.729	0.99 ± 0.345	2.02 ± 0.764
W12: SARTORIUS (n=6,4,5,4,10)	1.78 ± 0.771	0.69 ± 0.307	1.04 ± 0.443	0.90 ± 0.396	2.00 ± 0.683
W12: SEMIMEMBRANOSUS (n=8,5,6,6,13)	0.82 ± 0.230	0.82 ± 0.284	1.47 ± 0.777	0.86 ± 0.276	1.31 ± 0.194
W12: SEMITENDINOSUS (n=8,4,5,4,11)	0.78 ± 0.262	2.95 ± 1.016	1.31 ± 0.260	1.42 ± 0.317	1.17 ± 0.160
W12: VASTUS INTERMEDIUS (n=7,3,4,4,10)	2.59 ± 1.749	1.11 ± 0.292	0.62 ± 0.258	0.86 ± 0.157	1.64 ± 0.377
W12: VASTUS LATERALIS (n=7,5,6,5,12)	0.66 ± 0.160	0.89 ± 0.272	1.10 ± 0.333	1.78 ± 0.455	1.70 ± 0.334
W12: VASTUS MEDIALIS (n=6,4,6,4,12)	1.08 ± 0.269	1.26 ± 0.112	2.97 ± 1.981	0.85 ± 0.280	5.96 ± 3.376
W24: ADDUCTOR LONGUS (n=9,4,4,5,11)	0.81 ± 0.290	1.14 ± 0.269	1.11 ± 0.471	1.36 ± 0.718	2.41 ± 0.813
W24: ADDUCTOR MAGNUS (n=10,6,4,5,11)	0.66 ± 0.112	0.94 ± 0.296	0.59 ± 0.144	1.61 ± 0.697	1.14 ± 0.146
W24: BICEPS FEMORIS LONG (n=8,5,5,4,12)	1.07 ± 0.511	1.13 ± 0.315	1.00 ± 0.349	1.29 ± 0.238	2.10 ± 1.044
W24: BICEPS FEMORIS SHORT (n=7,5,4,5,12)	0.91 ± 0.195	2.24 ± 1.027	2.15 ± 1.003	1.24 ± 0.411	2.02 ± 0.416
W24: GRACILIS (n=9,3,3,4,9)	1.43 ± 0.686	3.03 ± 2.564	1.37 ± 0.465	1.45 ± 0.748	5.17 ± 3.540
W24:RECTUS FEMORIS (n=8,6,5,4,11)	0.94 ± 0.256	0.84 ± 0.220	7.43 ± 6.216	0.80 ± 0.262	3.01 ± 1.612
W24: SARTORIUS (n=7,5,4,4,10)	1.90 ± 0.872	0.73 ± 0.207	0.78 ± 0.375	3.24 ± 1.850	2.45 ± 0.842
W24: SEMIMEMBRANOSUS (n=10,6,5,4,12)	0.66 ± 0.126	0.90 ± 0.361	0.72 ± 0.157	0.94 ± 0.250	1.39 ± 0.256
W24: SEMITENDINOSUS (n=9,5,4,6,11)	0.56 ± 0.119	1.59 ± 0.520	1.43 ± 0.524	1.04 ± 0.254	1.22 ± 0.221
W24: VASTUS INTERMEDIUS (n=7,4,3,4,10)	0.95 ± 0.412	1.47 ± 0.446	0.63 ± 0.355	0.96 ± 0.095	1.47 ± 0.318
W24: VASTUS LATERALIS (n=10,5,5,4,12)	0.78 ± 0.159	1.88 ± 0.905	1.45 ± 0.596	1.43 ± 0.256	2.03 ± 0.484
W24: VASTUS MEDIALIS (n=8,5,4,4,12)	2.14 ± 0.889	5.16 ± 3.580	6.01 ± 5.478	1.29 ± 0.474	3.50 ± 1.390

No statistical analyses for this end point

Secondary: Change from Baseline in Thigh Muscle Maximal Cross Sectional Area (CSAmax) in the Double-Blind Phase

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End point title

Change from Baseline in Thigh Muscle Maximal Cross Sectional Area (CSAmax) in the Double-Blind Phase ^[11]

End point description

Right thigh measurements. W12 = Week 12, W24 = Week 24. Participants in Panel 1 received 4 mg RO7239361; participants in Panel 2 received either 12.5 mg (weight between 15 and 45 kg) or 20 mg (weight >45 kg) RO7239361, and participants in Panel 3 and the Expansion Panel received either 35 mg (weight between 15 and 45 kg) or 50 mg (weight >45 kg) RO7239361.

End point type

Secondary

End point timeframe

Baseline through Week 24

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: Results for Panel 3 and the Expansion Panel were reported separately by Subject analysis sets.

End point values	Placebo	Panel 1 RO7239361	Panel 2 RO7239361	Expansion Panel RO7239361	Panel 3 RO7239361
Number of subjects analysed	11	7	6	13	6
Units: Percentage Change
arithmetic mean (standard error)
W12: ADDUCTOR LONGUS (n=9,6,6,6,12)	7.29 ± 16.559	8.31 ± 11.767	5.85 ± 11.074	29.98 ± 8.530	14.61 ± 9.755
W12: ADDUCTOR MAGNUS (n=9,6,6,6,12)	31.01 ± 24.708	24.12 ± 11.804	-3.73 ± 29.037	4.01 ± 54.787	53.20 ± 25.328
W12: BICEPS FEMORIS LONG (n=9,6,6,6,12)	34.24 ± 18.328	33.43 ± 35.009	16.72 ± 17.806	23.93 ± 11.289	19.22 ± 9.802
W12: BICEPS FEMORIS SHORT (n=9,6,6,6,12)	14.10 ± 7.724	7.45 ± 11.592	2.69 ± 13.733	9.84 ± 3.882	18.68 ± 7.479
W12: GRACILIS (n=9,6,6,6,12)	20.14 ± 15.489	43.00 ± 28.539	15.28 ± 8.693	23.22 ± 5.440	27.02 ± 5.686
W12:RECTUS FEMORIS (n=9,6,6,6,12)	-7.86 ± 14.391	10.33 ± 11.927	-3.56 ± 14.175	16.01 ± 9.888	34.91 ± 6.037
W12: SARTORIUS (n=9,6,6,6,12)	11.26 ± 6.584	6.59 ± 11.952	-0.85 ± 6.383	18.20 ± 5.519	10.81 ± 11.017
W12: SEMIMEMBRANOSUS (n=9,6,6,6,12)	39.11 ± 15.041	32.57 ± 19.133	16.56 ± 19.998	31.01 ± 10.564	46.70 ± 16.480
W12: SEMITENDINOSUS (n=9,6,6,6,12)	25.03 ± 9.409	-1.24 ± 10.973	33.00 ± 26.730	40.10 ± 10.180	36.69 ± 14.802
W12: VASTUS INTERMEDIUS (n=9,6,6,6,12)	10.47 ± 12.160	23.35 ± 12.704	-52.03 ± 71.421	10.44 ± 7.344	-0.81 ± 19.353
W12: VASTUS LATERALIS (n=9,6,6,6,12)	31.47 ± 39.343	38.32 ± 19.570	14.08 ± 27.902	22.73 ± 15.138	57.86 ± 34.026
W12: VASTUS MEDIALIS (n=9,6,6,6,12)	-17.89 ± 19.358	15.65 ± 8.463	-11.60 ± 15.849	17.89 ± 6.788	21.14 ± 9.746
W24: ADDUCTOR LONGUS (n=11,7,5,6,12)	11.69 ± 15.626	23.41 ± 13.199	12.81 ± 16.393	44.12 ± 13.323	17.15 ± 15.484
W24: ADDUCTOR MAGNUS (n=11,7,5,6,12)	34.17 ± 24.918	21.90 ± 12.345	-6.71 ± 37.311	4.41 ± 52.393	45.05 ± 27.520
W24: BICEPS FEMORIS LONG (n=11,7,5,6,12)	39.21 ± 19.760	42.84 ± 22.086	-5.32 ± 29.140	31.33 ± 13.370	33.70 ± 12.073
W24: BICEPS FEMORIS SHORT (n=11,7,5,6,12)	20.09 ± 9.818	11.83 ± 13.013	-0.44 ± 16.183	18.60 ± 5.394	30.01 ± 13.494
W24: GRACILIS (n=11,7,5,6,12)	8.14 ± 7.148	18.72 ± 16.757	11.21 ± 5.461	40.15 ± 8.382	48.95 ± 17.064
W24:RECTUS FEMORIS (n=11,7,5,6,12)	8.97 ± 10.214	7.68 ± 13.082	-10.01 ± 16.121	38.40 ± 14.293	55.65 ± 27.535
W24: SARTORIUS (n=11,7,5,6,12)	10.75 ± 7.136	0.81 ± 8.143	-14.18 ± 9.481	25.51 ± 6.404	16.63 ± 4.476
W24: SEMIMEMBRANOSUS (n=11,7,5,6,12)	45.83 ± 14.896	30.01 ± 18.631	-11.48 ± 24.468	49.49 ± 12.011	61.03 ± 25.524
W24: SEMITENDINOSUS (n=11,7,5,6,12)	32.00 ± 16.102	36.17 ± 23.285	24.16 ± 23.403	62.55 ± 11.073	73.68 ± 17.594
W24: VASTUS INTERMEDIUS (n=11,7,5,6,12)	14.86 ± 15.072	43.01 ± 11.506	-68.05 ± 86.362	22.55 ± 8.284	41.63 ± 34.926
W24: VASTUS LATERALIS (n=11,7,5,6,12)	12.95 ± 35.465	47.79 ± 26.178	32.64 ± 30.136	46.87 ± 20.919	45.41 ± 43.774
W24: VASTUS MEDIALIS (n=11,7,5,6,12)	-9.09 ± 11.856	9.82 ± 6.965	-13.74 ± 29.664	23.94 ± 11.321	10.46 ± 12.093

No statistical analyses for this end point

Secondary: Percentage of Participants with Positive Anti-RO7239361 Antibodies (ADA) Assessment, Whole Study

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End point title

Percentage of Participants with Positive Anti-RO7239361 Antibodies (ADA) Assessment, Whole Study

End point description

A positive ADA sample is defined as one in which the presence of detectable ADAs is confirmed to be specific to RO7239361. A participant is considered as ADA positive if, after initiation of treatment, they have an ADA positive relative to baseline sample. Double-blind phase data for placebo participants are not included in this Whole Study outcome measure, but are reported in the outcome measure specific to the double-blind period. At the end of the double-blind period participants in the placebo arm switched to one of the RO7239361 arms upon entering the open label phase. Participants in Panel 1 received 4 mg RO7239361; participants in Panel 2 received either 12.5 mg (weight between 15 and 45 kg) or 20 mg (weight >45 kg) RO7239361, and participants in Panel 3 and the Expansion Panel received either 35 mg (weight between 15 and 45 kg) or 50 mg (weight >45 kg) RO7239361.

End point type

Secondary

End point timeframe

Day 8 through Week 228, baseline and on-study information represented in table.

End point values	Panel 1 RO7239361	Panel 2 RO7239361	Panel 3 and Expansion Panel RO7239361
Number of subjects analysed	9	8	26
Units: percentage of participants
number (not applicable)
Baseline (BL): ADA Positive	0	0	0
BL: ADA Negative	100.0	87.5	92.3
BL: Missing	0	12.5	7.7
On-treatment: ADA Positive	0	0	3.8
On-treatment: Persistent Positive	0	0	0
On-treatment: Only last sample Positive	0	0	0
On-treatment: Other Positive	0	0	3.8
On-treatment: ADA Negative	100.0	100.0	96.2
On-treatment: ADA Negative, BL Positive, No Boost	0	0	0
On-treatment: Missing	0	0	0

No statistical analyses for this end point

Secondary: Serum Concentration of Free Myostatin, Whole Study

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End point title

Serum Concentration of Free Myostatin, Whole Study

End point description

Double-blind phase data for placebo participants are not included in this Whole Study outcome measure, but are reported in the outcome measure specific to the double-blind period. At the end of the double-blind period participants in the placebo arm switched to one of the RO7239361 arms upon entering the open label phase. Panel 1 received 4 mg RO7239361; Panel 2 received either 12.5 mg (weight between 15 and 45 kg) or 20 mg (weight >45 kg) RO7239361, and Panel 3 and the Expansion Panel received either 35 mg (weight between 15 and 45 kg) or 50 mg (weight >45 kg) RO7239361. 9999=not calculable as data are reported for one subject. 99999=no data available The pharmacodynamics (PD) data set included all available data from subjects for whom PD measurements were available at baseline and at least one other timepoint. The Whole Study set reported here includes data from RO7239361-treated participants only. 9999=not calculable as data are reported for one subject. 99999=no data available

End point type

Secondary

End point timeframe

Baseline through Week 252

End point values	Panel 1 RO7239361	Panel 2 RO7239361	Panel 3 and Expansion Panel RO7239361
Number of subjects analysed	9	8	25
Units: pg/mL
arithmetic mean (standard deviation)
Baseline (n=8,8,23)	986.8 ± 363.69	1358.4 ± 964.47	1159.3 ± 601.29
Day 4 (n=5,4,18)	421.4 ± 70.60	321.3 ± 165.91	58.3 ± 33.20
Day 5 (n=7,6,18)	446.6 ± 108.81	357.8 ± 166.27	66.1 ± 39.07
Day 8 (n=6,6,19)	522.7 ± 131.51	479.2 ± 319.13	206.8 ± 419.21
Day 15 (n=7,6,19)	478.4 ± 111.44	280.8 ± 148.53	54.8 ± 61.85
Day 22 (n=7,6,17)	382.4 ± 82.58	284.8 ± 324.16	35.3 ± 51.45
Day 29 (n=7,6,17)	372.3 ± 79.97	179.0 ± 137.39	24.8 ± 27.24
Day 33 (n=7,6,18)	291.1 ± 77.55	115.7 ± 84.49	24.3 ± 30.64
Week 12 (n=8,6,25)	394.0 ± 179.78	115.0 ± 62.23	18.1 ± 13.37
Week 24 (n=9,8,24)	357.9 ± 137.56	139.1 ± 111.63	23.0 ± 23.68
Week 25 (n=6,6,17)	331.0 ± 116.78	154.5 ± 115.21	22.8 ± 18.48
Week 36 (n=9,7,16)	340.3 ± 98.24	81.4 ± 36.00	21.1 ± 22.03
Week 48 (n=9,7,16)	401.3 ± 233.76	79.0 ± 37.85	18.6 ± 12.11
Week 60 (n=7,5,9)	284.6 ± 96.92	90.4 ± 39.30	36.3 ± 54.13
Week 72 (n=5,4,3)	326.8 ± 151.29	132.0 ± 146.37	205.7 ± 321.58
Week 84 (n=4,2,15)	534.0 ± 260.31	74.2 ± 13.58	115.6 ± 146.44
Week 108 (n=9,5,16)	320.9 ± 222.10	84.4 ± 61.19	40.2 ± 42.70
Week 132 (n=8,7,21)	283.5 ± 103.73	71.0 ± 40.02	40.2 ± 38.26
Week 144 (n=0,1,0)	99999 ± 99999	95.2 ± 9999	99999 ± 99999
Week 156 (n=7,4,8)	208.7 ± 64.52	48.9 ± 22.42	20.0 ± 0.00
Week 180 (n=2,0,0)	212.5 ± 146.37	99999 ± 99999	99999 ± 99999
Week 252 (n=0,1,0)	99999 ± 99999	20.0 ± 9999	99999 ± 99999

No statistical analyses for this end point

Secondary: Percent Inhibition of Free Myostatin, Whole Study

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End point title

Percent Inhibition of Free Myostatin, Whole Study

End point description

Double-blind phase data for placebo participants are not included in this Whole Study outcome measure, but are reported in the outcome measure specific to the double-blind period. At the end of the double-blind period participants in the placebo arm switched to one of the RO7239361 arms upon entering the open label phase. Participants in Panel 1 received 4 mg RO7239361; participants in Panel 2 received either 12.5 mg (weight between 15 and 45 kg) or 20 mg (weight >45 kg) RO7239361, and participants in Panel 3 and the Expansion Panel received either 35 mg (weight between 15 and 45 kg) or 50 mg (weight >45 kg) RO7239361. The pharmacodynamics (PD) data set included all available data from subjects for whom PD measurements were available at baseline and at least one other timepoint. The Whole Study set reported here includes data from RO7239361-treated participants only. 9999=not calculable as data are reported for one subject. 99999=no data available

End point type

Secondary

End point timeframe

Baseline through Week 252

End point values	Panel 1 RO7239361	Panel 2 RO7239361	Panel 3 and Expansion Panel RO7239361
Number of subjects analysed	8	8	22
Units: percentage inhibition
arithmetic mean (standard deviation)
Day 4 (n=4,4,15)	49.689 ± 11.7394	73.533 ± 9.8637	95.334 ± 2.4477
Day 5 (n=6,6,16)	46.506 ± 9.0917	69.700 ± 10.3411	94.545 ± 2.7608
Day 8 (n=5,6,16)	38.411 ± 7.0320	62.524 ± 9.7074	84.797 ± 22.1958
Day 15 (n=6,6,16)	40.763 ± 10.0071	77.090 ± 6.2939	96.488 ± 1.9665
Day 22 (n=6,6,15)	51.836 ± 9.3488	81.693 ± 5.7392	97.927 ± 1.1259
Day 29 (n=6,6,15)	52.926 ± 6.6001	86.894 ± 2.9590	98.202 ± 1.4590
Day 33 (n=6,6,15)	61.796 ± 4.5760	91.557 ± 2.6444	97.042 ± 5.4443
Week 12 (n=7,6,22)	58.934 ± 8.7345	92.025 ± 2.0548	98.208 ± 1.5605
Week 24 n=8,8,21)	63.199 ± 5.3419	89.664 ± 5.0245	98.009 ± 1.8677
Week 25 (n=5,6,16)	62.446 ± 7.0668	88.689 ± 4.8868	97.974 ± 1.8259
Week 36 (n=8,7,15)	64.103 ± 5.3774	92.057 ± 2.6300	98.470 ± 0.8410
Week 48 (n=8,7,14)	58.161 ± 15.7101	92.237 ± 3.4952	98.036 ± 1.4516
Week 60 (n=6,5,7)	66.280 ± 8.4684	90.974 ± 2.1780	97.177 ± 2.8783
Week 72 (n=4,4,3)	65.956 ± 8.2066	92.536 ± 2.4445	79.193 ± 31.9504
Week 84 (n=4,2,13)	54.990 ± 1.0397	92.328 ± 5.7945	89.744 ± 13.0990
Week 108 (n=8,5,14)	70.498 ± 15.2905	92.412 ± 3.3662	95.884 ± 3.5214
Week 132 (n=8,7,18)	71.129 ± 4.9576	92.935 ± 3.8121	96.713 ± 2.4278
Week 144 (n=0,1,0)	99999 ± 99999	94.732 ± 9999	99999 ± 99999
Week 156 (n=6,4,6)	74.712 ± 5.7764	94.881 ± 1.3726	98.183 ± 0.8086
Week 180 (n=2,0,0)	75.374 ± 9.5968	99999 ± 99999	99999 ± 99999
Week 252 (n=0,1,0)	99999 ± 99999	96.283 ± 9999	99999 ± 99999

No statistical analyses for this end point

Secondary: Serum Concentration of Drug-Myostatin Complex, Whole Study

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End point title

Serum Concentration of Drug-Myostatin Complex, Whole Study

End point description

Participants in the Placebo arm received placebo during the double-blind (DB) period (up to Week 24) and received RO7239361 during the open label (OL) phase. Participants in Panel 1 received 4 mg RO7239361; participants in Panel 2 received either 12.5 mg (weight between 15 and 45 kg) or 20 mg (weight >45 kg) RO7239361, and participants in Panel 3 and the Expansion Panel received either 35 mg (weight between 15 and 45 kg) or 50 mg (weight >45 kg) RO7239361. The pharmacodynamics (PD) data set included all available data from subjects for whom PD measurements were available at baseline and at least one other timepoint. 9999=not calculable as data are reported for one subject. 99999=no data available

End point type

Secondary

End point timeframe

Baseline through Week 252

End point values	Placebo, then RO7239361	Panel 1 RO7239361 Whole Study	Panel 2 RO7239361 Whole Study	Panel 3 and Expansion Panel RO7239361 Whole Study
Number of subjects analysed	11	7	6	18
Units: ng/mL
arithmetic mean (standard deviation)
Day 4 (n=9,3,4,7)	1.178 ± 1.8833	5.070 ± 0.9456	12.606 ± 5.7623	14.696 ± 5.7926
Day 5 (n=8,5,6,6)	2.119 ± 2.9826	5.845 ± 2.5725	14.856 ± 7.6131	17.308 ± 7.5955
Day 8 (n=10,6,6,7)	4.924 ± 12.9975	7.041 ± 2.3470	19.906 ± 9.8664	21.609 ± 11.0880
Day 15 (n=11,7,6,7)	0.613 ± 0.2102	14.067 ± 7.1096	29.229 ± 10.4929	45.289 ± 18.7600
Day 22 (n=10,7,6,6)	1.326 ± 1.3410	11.985 ± 4.8243	36.811 ± 26.8560	52.841 ± 21.5542
Day 29 (n=11,7,6,7)	1.593 ± 3.1007	15.735 ± 4.2010	43.236 ± 34.0300	67.255 ± 27.9376
Day 33 (n=9,7,4,7)	1.157 ± 1.0036	18.542 ± 3.9874	46.279 ± 24.1457	77.142 ± 32.4153
Week 12 (n=10,6,5,14)	7.082 ± 15.2289	28.556 ± 22.7890	79.555 ± 17.2126	88.402 ± 29.8080
Week 24 (end of DB period) (n=8,7,5,7)	3.589 ± 7.8108	25.997 ± 6.4384	83.262 ± 64.2281	101.406 ± 45.4197
Week 25 (Week 1 OL) (n=7,7,5,8)	3.186 ± 6.9734	31.963 ± 8.0240	77.926 ± 56.9987	102.211 ± 40.2284
Week 36 (Week 12 OL) (n=10,6,5,14)	95.788 ± 88.1521	22.584 ± 8.6839	57.164 ± 25.8284	101.234 ± 42.0902
Week 48 (Week 24 OL) (n=9,7,0,18)	90.593 ± 73.7102	27.461 ± 7.2306	99999 ± 99999	103.657 ± 45.8802
Week 60 (Week 36 OL) (n=5,7,5,3)	63.358 ± 37.5591	41.779 ± 34.2407	89.712 ± 38.9214	67.163 ± 8.4904
Week 72 (Week 48 OL) (n=5,5,1,4)	92.086 ± 68.5222	26.740 ± 3.0256	0.550 ± 9999	102.025 ± 31.4717
Week 84 (Week 60 OL) (n=6,2,0,16)	146.950 ± 131.3706	27.110 ± 3.2527	99999 ± 99999	117.719 ± 41.1143
Week 108 (Week 84 OL) (n=10,7,5,18)	122.610 ± 87.1911	36.014 ± 10.9338	66.020 ± 7.2878	114.806 ± 32.7432
Week 132 (Week 108 OL) (n=9,6,5,18)	95.811 ± 48.1440	32.133 ± 8.2848	75.040 ± 14.3015	107.378 ± 37.8814
Week 144 (Week 120 OL) (n=0,1,0,0)	99999 ± 99999	25.600 ± 9999	99999 ± 99999	99999 ± 99999
Week 156 (Week 132 OL) (n=7,7,4,18)	95.529 ± 48.0065	30.186 ± 7.0053	69.325 ± 17.7064	108.100 ± 35.5833
Week 156 (Week 132 OL: Day 1 PFS) (n=2,0,0,0)	64.400 ± 0.5657	99999 ± 99999	99999 ± 99999	99999 ± 99999
Week 156 (Week 132 OL: Week 12 PFS) (n=1,0,0,6)	152.000 ± 9999	99999 ± 99999	99999 ± 99999	125.583 ± 44.4144
Week 168 (Week 144 OL: Day 1 PFS) (n=3,0,3,3)	105.700 ± 54.5772	99999 ± 99999	70.100 ± 25.1213	122.633 ± 91.2542
Week 168 (Week 144 OL: Day 8 PFS) (n=0,0,0,1)	99999 ± 99999	99999 ± 99999	99999 ± 99999	119.000 ± 9999
Week 168 (Week 144 OL: Week 12 PFS) (n=0,0,1,0)	99999 ± 99999	99999 ± 99999	94.900 ± 9999	99999 ± 99999
Week 180 (Week 156 OL) (n=3,7,2,0)	45.933 ± 28.3161	25.589 ± 12.2478	103.300 ± 16.5463	99999 ± 99999
Week 180 (Week 156 OL: Week 12 PFS) (n=0,1,0,0)	99999 ± 99999	62.600 ± 9999	99999 ± 99999	99999 ± 99999
Week 192 (Week 168 OL: Day 1 PFS) (n=0,1,0,0)	99999 ± 99999	18.000 ± 9999	999999 ± 99999	99999 ± 99999
Week 192 (Week 168 OL: Day 45 PFS) (n=0,1,0,0)	99999 ± 999999	80.400 ± 9999	99999 ± 99999	99999 ± 99999
Week 252 (Week 228 OL) (n=10,5,3,17)	96.770 ± 61.6084	52.680 ± 20.8619	87.900 ± 38.5763	106.071 ± 48.6434

No statistical analyses for this end point

Adverse events

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Timeframe for reporting adverse events

Baseline up to Week 252

Adverse event reporting additional description

Upon completion of the double-blind phase, participants who were receiving placebo switched to the equivalent RO7239361 panel dose for the open label phase. All adverse event data is represented according to the phase (double-blind or open-label) during which the participant experienced the event.

Assessment type

Systematic

Dictionary name

MedDRA

Dictionary version

23.0

Reporting group title

Placebo Double-Blind

Reporting group description

Placebo subcutaneous injections on specified days during the double-blind period.

Reporting group title

Panel 1 RO7239361 Double-Blind

Reporting group description

RO7239361 subcutaneous injections on specified days during the double-blind period.

Reporting group title

Panel 2 RO7239361 Double-Blind

Reporting group description

RO7239361 subcutaneous injections on specified days during the double-blind period.

Reporting group title

Panel 3 and Expansion Panel RO7239361 Double-Blind

Reporting group description

RO7239361 subcutaneous injections on specified days during the double-blind period.

Reporting group title

Panel 1 RO7239361 Open-Label

Reporting group description

RO7239361 subcutaneous injections on specified days during the open-label period.

Reporting group title

Panel 2 RO7239361 Open-Label

Reporting group description

RO7239361 subcutaneous injections on specified days during the open-label period.

Reporting group title

Panel 3 and Expansion Panel RO7239361 Open-Label

Reporting group description

RO7239361 subcutaneous injections on specified days during the open-label period.

Serious adverse events	Placebo Double-Blind	Panel 1 RO7239361 Double-Blind	Panel 2 RO7239361 Double-Blind	Panel 3 and Expansion Panel RO7239361 Double-Blind	Panel 1 RO7239361 Open-Label	Panel 2 RO7239361 Open-Label	Panel 3 and Expansion Panel RO7239361 Open-Label
Total subjects affected by serious adverse events
subjects affected / exposed	1 / 11 (9.09%)	0 / 7 (0.00%)	0 / 6 (0.00%)	1 / 19 (5.26%)	1 / 9 (11.11%)	2 / 8 (25.00%)	1 / 26 (3.85%)
number of deaths (all causes)	0	0	0	0	0	0	0
number of deaths resulting from adverse events	0	0	0	0	0	0	0
Injury, poisoning and procedural complications
FEMUR FRACTURE
subjects affected / exposed	0 / 11 (0.00%)	0 / 7 (0.00%)	0 / 6 (0.00%)	0 / 19 (0.00%)	0 / 9 (0.00%)	0 / 8 (0.00%)	1 / 26 (3.85%)
occurrences causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 1
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0
SPINAL COMPRESSION FRACTURE
subjects affected / exposed	0 / 11 (0.00%)	0 / 7 (0.00%)	0 / 6 (0.00%)	1 / 19 (5.26%)	0 / 9 (0.00%)	0 / 8 (0.00%)	0 / 26 (0.00%)
occurrences causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 1	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
SKULL FRACTURE
subjects affected / exposed	1 / 11 (9.09%)	0 / 7 (0.00%)	0 / 6 (0.00%)	0 / 19 (0.00%)	0 / 9 (0.00%)	0 / 8 (0.00%)	0 / 26 (0.00%)
occurrences causally related to treatment / all	0 / 1	0 / 0	0 / 0	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
Psychiatric disorders
CONVERSION DISORDER
subjects affected / exposed	0 / 11 (0.00%)	0 / 7 (0.00%)	0 / 6 (0.00%)	0 / 19 (0.00%)	0 / 9 (0.00%)	1 / 8 (12.50%)	0 / 26 (0.00%)
occurrences causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 1	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0
Musculoskeletal and connective tissue disorders
RHABDOMYOLYSIS
subjects affected / exposed	0 / 11 (0.00%)	0 / 7 (0.00%)	0 / 6 (0.00%)	0 / 19 (0.00%)	0 / 9 (0.00%)	1 / 8 (12.50%)	0 / 26 (0.00%)
occurrences causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 2	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0
Infections and infestations
INFLUENZA
subjects affected / exposed	0 / 11 (0.00%)	0 / 7 (0.00%)	0 / 6 (0.00%)	0 / 19 (0.00%)	0 / 9 (0.00%)	1 / 8 (12.50%)	0 / 26 (0.00%)
occurrences causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 1	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0
APPENDICITIS PERFORATED
subjects affected / exposed	0 / 11 (0.00%)	0 / 7 (0.00%)	0 / 6 (0.00%)	0 / 19 (0.00%)	1 / 9 (11.11%)	0 / 8 (0.00%)	0 / 26 (0.00%)
occurrences causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 0	0 / 1	0 / 0	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0	0 / 0

Frequency threshold for reporting non-serious adverse events: 0%

Non-serious adverse events	Placebo Double-Blind	Panel 1 RO7239361 Double-Blind	Panel 2 RO7239361 Double-Blind	Panel 3 and Expansion Panel RO7239361 Double-Blind	Panel 1 RO7239361 Open-Label	Panel 2 RO7239361 Open-Label	Panel 3 and Expansion Panel RO7239361 Open-Label
Total subjects affected by non serious adverse events
subjects affected / exposed	9 / 11 (81.82%)	5 / 7 (71.43%)	6 / 6 (100.00%)	17 / 19 (89.47%)	9 / 9 (100.00%)	6 / 8 (75.00%)	24 / 26 (92.31%)
Vascular disorders
FLUSHING
subjects affected / exposed	0 / 11 (0.00%)	0 / 7 (0.00%)	1 / 6 (16.67%)	1 / 19 (5.26%)	0 / 9 (0.00%)	0 / 8 (0.00%)	0 / 26 (0.00%)
occurrences all number	0	0	1	1	0	0	0
HAEMATOMA
subjects affected / exposed	0 / 11 (0.00%)	0 / 7 (0.00%)	1 / 6 (16.67%)	1 / 19 (5.26%)	0 / 9 (0.00%)	0 / 8 (0.00%)	0 / 26 (0.00%)
occurrences all number	0	0	1	2	0	0	0
General disorders and administration site conditions
PYREXIA
subjects affected / exposed	3 / 11 (27.27%)	0 / 7 (0.00%)	1 / 6 (16.67%)	5 / 19 (26.32%)	1 / 9 (11.11%)	2 / 8 (25.00%)	4 / 26 (15.38%)
occurrences all number	3	0	1	6	1	2	8
INJECTION SITE BRUISING
subjects affected / exposed	1 / 11 (9.09%)	0 / 7 (0.00%)	2 / 6 (33.33%)	3 / 19 (15.79%)	2 / 9 (22.22%)	0 / 8 (0.00%)	6 / 26 (23.08%)
occurrences all number	1	0	2	3	2	0	13
INJECTION SITE RASH
subjects affected / exposed	1 / 11 (9.09%)	1 / 7 (14.29%)	1 / 6 (16.67%)	0 / 19 (0.00%)	0 / 9 (0.00%)	2 / 8 (25.00%)	0 / 26 (0.00%)
occurrences all number	1	1	9	0	0	3	0
CHILLS
subjects affected / exposed	1 / 11 (9.09%)	0 / 7 (0.00%)	0 / 6 (0.00%)	0 / 19 (0.00%)	0 / 9 (0.00%)	0 / 8 (0.00%)	0 / 26 (0.00%)
occurrences all number	1	0	0	0	0	0	0
INJECTION SITE HAEMORRHAGE
subjects affected / exposed	1 / 11 (9.09%)	0 / 7 (0.00%)	0 / 6 (0.00%)	0 / 19 (0.00%)	0 / 9 (0.00%)	0 / 8 (0.00%)	2 / 26 (7.69%)
occurrences all number	1	0	0	0	0	0	2
INJECTION SITE IRRITATION
subjects affected / exposed	0 / 11 (0.00%)	1 / 7 (14.29%)	0 / 6 (0.00%)	0 / 19 (0.00%)	5 / 9 (55.56%)	0 / 8 (0.00%)	0 / 26 (0.00%)
occurrences all number	0	5	0	0	23	0	0
PERIPHERAL SWELLING
subjects affected / exposed	1 / 11 (9.09%)	0 / 7 (0.00%)	0 / 6 (0.00%)	0 / 19 (0.00%)	0 / 9 (0.00%)	0 / 8 (0.00%)	1 / 26 (3.85%)
occurrences all number	1	0	0	0	0	0	1
INJECTION SITE PAIN
subjects affected / exposed	0 / 11 (0.00%)	0 / 7 (0.00%)	0 / 6 (0.00%)	2 / 19 (10.53%)	0 / 9 (0.00%)	1 / 8 (12.50%)	2 / 26 (7.69%)
occurrences all number	0	0	0	5	0	1	3
FATIGUE
subjects affected / exposed	0 / 11 (0.00%)	0 / 7 (0.00%)	0 / 6 (0.00%)	1 / 19 (5.26%)	0 / 9 (0.00%)	0 / 8 (0.00%)	1 / 26 (3.85%)
occurrences all number	0	0	0	1	0	0	1
INJECTION SITE DISCOMFORT
subjects affected / exposed	0 / 11 (0.00%)	0 / 7 (0.00%)	0 / 6 (0.00%)	1 / 19 (5.26%)	0 / 9 (0.00%)	0 / 8 (0.00%)	0 / 26 (0.00%)
occurrences all number	0	0	0	1	0	0	0
INJECTION SITE ERYTHEMA
subjects affected / exposed	0 / 11 (0.00%)	0 / 7 (0.00%)	0 / 6 (0.00%)	1 / 19 (5.26%)	4 / 9 (44.44%)	0 / 8 (0.00%)	8 / 26 (30.77%)
occurrences all number	0	0	0	2	6	0	13
INJECTION SITE SWELLING
subjects affected / exposed	0 / 11 (0.00%)	0 / 7 (0.00%)	0 / 6 (0.00%)	1 / 19 (5.26%)	0 / 9 (0.00%)	0 / 8 (0.00%)	0 / 26 (0.00%)
occurrences all number	0	0	0	1	0	0	0
INJECTION SITE REACTION
subjects affected / exposed	0 / 11 (0.00%)	0 / 7 (0.00%)	0 / 6 (0.00%)	0 / 19 (0.00%)	2 / 9 (22.22%)	0 / 8 (0.00%)	0 / 26 (0.00%)
occurrences all number	0	0	0	0	2	0	0
INFLUENZA LIKE ILLNESS
subjects affected / exposed	0 / 11 (0.00%)	0 / 7 (0.00%)	0 / 6 (0.00%)	0 / 19 (0.00%)	0 / 9 (0.00%)	0 / 8 (0.00%)	1 / 26 (3.85%)
occurrences all number	0	0	0	0	0	0	1
INJECTION SITE OEDEMA
subjects affected / exposed	0 / 11 (0.00%)	0 / 7 (0.00%)	0 / 6 (0.00%)	0 / 19 (0.00%)	1 / 9 (11.11%)	0 / 8 (0.00%)	0 / 26 (0.00%)
occurrences all number	0	0	0	0	2	0	0
INJECTION SITE PRURITUS
subjects affected / exposed	0 / 11 (0.00%)	0 / 7 (0.00%)	0 / 6 (0.00%)	0 / 19 (0.00%)	0 / 9 (0.00%)	0 / 8 (0.00%)	1 / 26 (3.85%)
occurrences all number	0	0	0	0	0	0	1
Immune system disorders
SEASONAL ALLERGY
subjects affected / exposed	0 / 11 (0.00%)	0 / 7 (0.00%)	0 / 6 (0.00%)	0 / 19 (0.00%)	0 / 9 (0.00%)	1 / 8 (12.50%)	2 / 26 (7.69%)
occurrences all number	0	0	0	0	0	1	4
Reproductive system and breast disorders
TESTICULAR TORSION
subjects affected / exposed	0 / 11 (0.00%)	0 / 7 (0.00%)	0 / 6 (0.00%)	0 / 19 (0.00%)	0 / 9 (0.00%)	0 / 8 (0.00%)	1 / 26 (3.85%)
occurrences all number	0	0	0	0	0	0	1
Respiratory, thoracic and mediastinal disorders
COUGH
subjects affected / exposed	2 / 11 (18.18%)	0 / 7 (0.00%)	0 / 6 (0.00%)	2 / 19 (10.53%)	1 / 9 (11.11%)	4 / 8 (50.00%)	7 / 26 (26.92%)
occurrences all number	2	0	0	2	1	5	11
EPISTAXIS
subjects affected / exposed	1 / 11 (9.09%)	0 / 7 (0.00%)	1 / 6 (16.67%)	1 / 19 (5.26%)	0 / 9 (0.00%)	0 / 8 (0.00%)	2 / 26 (7.69%)
occurrences all number	1	0	1	1	0	0	13
NASAL CONGESTION
subjects affected / exposed	1 / 11 (9.09%)	0 / 7 (0.00%)	1 / 6 (16.67%)	1 / 19 (5.26%)	2 / 9 (22.22%)	0 / 8 (0.00%)	5 / 26 (19.23%)
occurrences all number	1	0	1	1	3	0	5
PRODUCTIVE COUGH
subjects affected / exposed	0 / 11 (0.00%)	0 / 7 (0.00%)	2 / 6 (33.33%)	0 / 19 (0.00%)	0 / 9 (0.00%)	0 / 8 (0.00%)	0 / 26 (0.00%)
occurrences all number	0	0	2	0	0	0	0
ASTHMA
subjects affected / exposed	0 / 11 (0.00%)	0 / 7 (0.00%)	1 / 6 (16.67%)	0 / 19 (0.00%)	0 / 9 (0.00%)	0 / 8 (0.00%)	0 / 26 (0.00%)
occurrences all number	0	0	1	0	0	0	0
HICCUPS
subjects affected / exposed	1 / 11 (9.09%)	0 / 7 (0.00%)	0 / 6 (0.00%)	0 / 19 (0.00%)	0 / 9 (0.00%)	0 / 8 (0.00%)	0 / 26 (0.00%)
occurrences all number	1	0	0	0	0	0	0
NASAL DISCHARGE DISCOLOURATION
subjects affected / exposed	0 / 11 (0.00%)	0 / 7 (0.00%)	1 / 6 (16.67%)	0 / 19 (0.00%)	0 / 9 (0.00%)	0 / 8 (0.00%)	0 / 26 (0.00%)
occurrences all number	0	0	2	0	0	0	0
PARANASAL SINUS HYPERSECRETION
subjects affected / exposed	1 / 11 (9.09%)	0 / 7 (0.00%)	0 / 6 (0.00%)	0 / 19 (0.00%)	0 / 9 (0.00%)	0 / 8 (0.00%)	0 / 26 (0.00%)
occurrences all number	1	0	0	0	0	0	0
OROPHARYNGEAL PAIN
subjects affected / exposed	0 / 11 (0.00%)	0 / 7 (0.00%)	0 / 6 (0.00%)	2 / 19 (10.53%)	0 / 9 (0.00%)	0 / 8 (0.00%)	4 / 26 (15.38%)
occurrences all number	0	0	0	4	0	0	6
RHINORRHOEA
subjects affected / exposed	0 / 11 (0.00%)	0 / 7 (0.00%)	0 / 6 (0.00%)	2 / 19 (10.53%)	0 / 9 (0.00%)	0 / 8 (0.00%)	3 / 26 (11.54%)
occurrences all number	0	0	0	2	0	0	5
BRONCHOSPASM
subjects affected / exposed	0 / 11 (0.00%)	0 / 7 (0.00%)	0 / 6 (0.00%)	0 / 19 (0.00%)	0 / 9 (0.00%)	1 / 8 (12.50%)	0 / 26 (0.00%)
occurrences all number	0	0	0	0	0	1	0
LOWER RESPIRATORY TRACT CONGESTION
subjects affected / exposed	0 / 11 (0.00%)	0 / 7 (0.00%)	0 / 6 (0.00%)	0 / 19 (0.00%)	0 / 9 (0.00%)	0 / 8 (0.00%)	1 / 26 (3.85%)
occurrences all number	0	0	0	0	0	0	1
PLEURAL EFFUSION
subjects affected / exposed	0 / 11 (0.00%)	0 / 7 (0.00%)	0 / 6 (0.00%)	0 / 19 (0.00%)	1 / 9 (11.11%)	0 / 8 (0.00%)	0 / 26 (0.00%)
occurrences all number	0	0	0	0	1	0	0
RHINITIS ALLERGIC
subjects affected / exposed	0 / 11 (0.00%)	0 / 7 (0.00%)	0 / 6 (0.00%)	0 / 19 (0.00%)	0 / 9 (0.00%)	0 / 8 (0.00%)	1 / 26 (3.85%)
occurrences all number	0	0	0	0	0	0	1
SLEEP APNOEA SYNDROME
subjects affected / exposed	0 / 11 (0.00%)	0 / 7 (0.00%)	0 / 6 (0.00%)	0 / 19 (0.00%)	0 / 9 (0.00%)	0 / 8 (0.00%)	1 / 26 (3.85%)
occurrences all number	0	0	0	0	0	0	1
Psychiatric disorders
BRUXISM
subjects affected / exposed	0 / 11 (0.00%)	0 / 7 (0.00%)	0 / 6 (0.00%)	1 / 19 (5.26%)	0 / 9 (0.00%)	0 / 8 (0.00%)	0 / 26 (0.00%)
occurrences all number	0	0	0	1	0	0	0
ANGER
subjects affected / exposed	0 / 11 (0.00%)	0 / 7 (0.00%)	0 / 6 (0.00%)	0 / 19 (0.00%)	0 / 9 (0.00%)	0 / 8 (0.00%)	1 / 26 (3.85%)
occurrences all number	0	0	0	0	0	0	1
ENURESIS
subjects affected / exposed	0 / 11 (0.00%)	0 / 7 (0.00%)	0 / 6 (0.00%)	0 / 19 (0.00%)	0 / 9 (0.00%)	0 / 8 (0.00%)	1 / 26 (3.85%)
occurrences all number	0	0	0	0	0	0	1
Investigations
BLOOD CREATINE PHOSPHOKINASE INCREASED
subjects affected / exposed	1 / 11 (9.09%)	0 / 7 (0.00%)	0 / 6 (0.00%)	0 / 19 (0.00%)	0 / 9 (0.00%)	0 / 8 (0.00%)	0 / 26 (0.00%)
occurrences all number	1	0	0	0	0	0	0
LYMPH NODE PALPABLE
subjects affected / exposed	0 / 11 (0.00%)	0 / 7 (0.00%)	0 / 6 (0.00%)	1 / 19 (5.26%)	0 / 9 (0.00%)	0 / 8 (0.00%)	0 / 26 (0.00%)
occurrences all number	0	0	0	1	0	0	0
Injury, poisoning and procedural complications
ARTHROPOD BITE
subjects affected / exposed	0 / 11 (0.00%)	0 / 7 (0.00%)	1 / 6 (16.67%)	1 / 19 (5.26%)	1 / 9 (11.11%)	0 / 8 (0.00%)	0 / 26 (0.00%)
occurrences all number	0	0	1	1	1	0	0
CONTUSION
subjects affected / exposed	1 / 11 (9.09%)	1 / 7 (14.29%)	0 / 6 (0.00%)	0 / 19 (0.00%)	4 / 9 (44.44%)	0 / 8 (0.00%)	4 / 26 (15.38%)
occurrences all number	1	2	0	0	5	0	4
FALL
subjects affected / exposed	0 / 11 (0.00%)	1 / 7 (14.29%)	0 / 6 (0.00%)	1 / 19 (5.26%)	7 / 9 (77.78%)	0 / 8 (0.00%)	6 / 26 (23.08%)
occurrences all number	0	1	0	1	20	0	8
LIGAMENT SPRAIN
subjects affected / exposed	0 / 11 (0.00%)	1 / 7 (14.29%)	0 / 6 (0.00%)	1 / 19 (5.26%)	2 / 9 (22.22%)	1 / 8 (12.50%)	2 / 26 (7.69%)
occurrences all number	0	1	0	1	2	1	2
SKIN ABRASION
subjects affected / exposed	1 / 11 (9.09%)	0 / 7 (0.00%)	0 / 6 (0.00%)	1 / 19 (5.26%)	1 / 9 (11.11%)	0 / 8 (0.00%)	1 / 26 (3.85%)
occurrences all number	1	0	0	1	1	0	1
EYELID CONTUSION
subjects affected / exposed	1 / 11 (9.09%)	0 / 7 (0.00%)	0 / 6 (0.00%)	0 / 19 (0.00%)	0 / 9 (0.00%)	0 / 8 (0.00%)	0 / 26 (0.00%)
occurrences all number	1	0	0	0	0	0	0
FOOT FRACTURE
subjects affected / exposed	0 / 11 (0.00%)	0 / 7 (0.00%)	0 / 6 (0.00%)	1 / 19 (5.26%)	1 / 9 (11.11%)	0 / 8 (0.00%)	0 / 26 (0.00%)
occurrences all number	0	0	0	1	1	0	0
POST-TRAUMATIC PAIN
subjects affected / exposed	0 / 11 (0.00%)	0 / 7 (0.00%)	0 / 6 (0.00%)	1 / 19 (5.26%)	0 / 9 (0.00%)	0 / 8 (0.00%)	2 / 26 (7.69%)
occurrences all number	0	0	0	1	0	0	2
THERMAL BURN
subjects affected / exposed	0 / 11 (0.00%)	0 / 7 (0.00%)	0 / 6 (0.00%)	0 / 19 (0.00%)	1 / 9 (11.11%)	0 / 8 (0.00%)	1 / 26 (3.85%)
occurrences all number	0	0	0	0	1	0	1
EYE CONTUSION
subjects affected / exposed	0 / 11 (0.00%)	0 / 7 (0.00%)	0 / 6 (0.00%)	0 / 19 (0.00%)	1 / 9 (11.11%)	0 / 8 (0.00%)	0 / 26 (0.00%)
occurrences all number	0	0	0	0	1	0	0
HEAD INJURY
subjects affected / exposed	0 / 11 (0.00%)	0 / 7 (0.00%)	0 / 6 (0.00%)	0 / 19 (0.00%)	1 / 9 (11.11%)	0 / 8 (0.00%)	0 / 26 (0.00%)
occurrences all number	0	0	0	0	1	0	0
UPPER LIMB FRACTURE
subjects affected / exposed	0 / 11 (0.00%)	0 / 7 (0.00%)	0 / 6 (0.00%)	0 / 19 (0.00%)	1 / 9 (11.11%)	0 / 8 (0.00%)	0 / 26 (0.00%)
occurrences all number	0	0	0	0	1	0	0
CORNEAL ABRASION
subjects affected / exposed	0 / 11 (0.00%)	0 / 7 (0.00%)	0 / 6 (0.00%)	0 / 19 (0.00%)	0 / 9 (0.00%)	0 / 8 (0.00%)	1 / 26 (3.85%)
occurrences all number	0	0	0	0	0	0	1
FIBULA FRACTURE
subjects affected / exposed	0 / 11 (0.00%)	0 / 7 (0.00%)	0 / 6 (0.00%)	0 / 19 (0.00%)	2 / 9 (22.22%)	0 / 8 (0.00%)	0 / 26 (0.00%)
occurrences all number	0	0	0	0	2	0	0
HUMERUS FRACTURE
subjects affected / exposed	0 / 11 (0.00%)	0 / 7 (0.00%)	0 / 6 (0.00%)	0 / 19 (0.00%)	0 / 9 (0.00%)	0 / 8 (0.00%)	1 / 26 (3.85%)
occurrences all number	0	0	0	0	0	0	1
JOINT INJURY
subjects affected / exposed	0 / 11 (0.00%)	0 / 7 (0.00%)	0 / 6 (0.00%)	0 / 19 (0.00%)	1 / 9 (11.11%)	0 / 8 (0.00%)	1 / 26 (3.85%)
occurrences all number	0	0	0	0	1	0	1
LIMB INJURY
subjects affected / exposed	0 / 11 (0.00%)	0 / 7 (0.00%)	0 / 6 (0.00%)	0 / 19 (0.00%)	0 / 9 (0.00%)	0 / 8 (0.00%)	2 / 26 (7.69%)
occurrences all number	0	0	0	0	0	0	2
PROCEDURAL PAIN
subjects affected / exposed	0 / 11 (0.00%)	0 / 7 (0.00%)	0 / 6 (0.00%)	0 / 19 (0.00%)	0 / 9 (0.00%)	0 / 8 (0.00%)	1 / 26 (3.85%)
occurrences all number	0	0	0	0	0	0	1
SKIN LACERATION
subjects affected / exposed	0 / 11 (0.00%)	0 / 7 (0.00%)	0 / 6 (0.00%)	0 / 19 (0.00%)	0 / 9 (0.00%)	0 / 8 (0.00%)	1 / 26 (3.85%)
occurrences all number	0	0	0	0	0	0	1
SOFT TISSUE INJURY
subjects affected / exposed	0 / 11 (0.00%)	0 / 7 (0.00%)	0 / 6 (0.00%)	0 / 19 (0.00%)	1 / 9 (11.11%)	0 / 8 (0.00%)	0 / 26 (0.00%)
occurrences all number	0	0	0	0	1	0	0
SPINAL COMPRESSION FRACTURE
subjects affected / exposed	0 / 11 (0.00%)	0 / 7 (0.00%)	0 / 6 (0.00%)	0 / 19 (0.00%)	1 / 9 (11.11%)	0 / 8 (0.00%)	1 / 26 (3.85%)
occurrences all number	0	0	0	0	1	0	1
SUNBURN
subjects affected / exposed	0 / 11 (0.00%)	0 / 7 (0.00%)	0 / 6 (0.00%)	0 / 19 (0.00%)	0 / 9 (0.00%)	0 / 8 (0.00%)	1 / 26 (3.85%)
occurrences all number	0	0	0	0	0	0	1
TIBIA FRACTURE
subjects affected / exposed	0 / 11 (0.00%)	0 / 7 (0.00%)	0 / 6 (0.00%)	0 / 19 (0.00%)	1 / 9 (11.11%)	0 / 8 (0.00%)	0 / 26 (0.00%)
occurrences all number	0	0	0	0	1	0	0
Congenital, familial and genetic disorders
DERMOID CYST
subjects affected / exposed	0 / 11 (0.00%)	0 / 7 (0.00%)	0 / 6 (0.00%)	1 / 19 (5.26%)	0 / 9 (0.00%)	0 / 8 (0.00%)	0 / 26 (0.00%)
occurrences all number	0	0	0	1	0	0	0
Cardiac disorders
SINUS TACHYCARDIA
subjects affected / exposed	0 / 11 (0.00%)	0 / 7 (0.00%)	0 / 6 (0.00%)	1 / 19 (5.26%)	0 / 9 (0.00%)	0 / 8 (0.00%)	0 / 26 (0.00%)
occurrences all number	0	0	0	1	0	0	0
CARDIOMYOPATHY
subjects affected / exposed	0 / 11 (0.00%)	0 / 7 (0.00%)	0 / 6 (0.00%)	0 / 19 (0.00%)	1 / 9 (11.11%)	0 / 8 (0.00%)	0 / 26 (0.00%)
occurrences all number	0	0	0	0	1	0	0
LEFT VENTRICULAR DYSFUNCTION
subjects affected / exposed	0 / 11 (0.00%)	0 / 7 (0.00%)	0 / 6 (0.00%)	0 / 19 (0.00%)	1 / 9 (11.11%)	0 / 8 (0.00%)	0 / 26 (0.00%)
occurrences all number	0	0	0	0	1	0	0
MYOCARDIAL FIBROSIS
subjects affected / exposed	0 / 11 (0.00%)	0 / 7 (0.00%)	0 / 6 (0.00%)	0 / 19 (0.00%)	1 / 9 (11.11%)	0 / 8 (0.00%)	0 / 26 (0.00%)
occurrences all number	0	0	0	0	1	0	0
Nervous system disorders
HEADACHE
subjects affected / exposed	2 / 11 (18.18%)	2 / 7 (28.57%)	1 / 6 (16.67%)	7 / 19 (36.84%)	2 / 9 (22.22%)	0 / 8 (0.00%)	9 / 26 (34.62%)
occurrences all number	2	2	1	19	3	0	27
DIZZINESS
subjects affected / exposed	1 / 11 (9.09%)	0 / 7 (0.00%)	0 / 6 (0.00%)	0 / 19 (0.00%)	0 / 9 (0.00%)	0 / 8 (0.00%)	1 / 26 (3.85%)
occurrences all number	1	0	0	0	0	0	1
MOTOR DYSFUNCTION
subjects affected / exposed	0 / 11 (0.00%)	0 / 7 (0.00%)	0 / 6 (0.00%)	0 / 19 (0.00%)	1 / 9 (11.11%)	0 / 8 (0.00%)	0 / 26 (0.00%)
occurrences all number	0	0	0	0	1	0	0
SINUS HEADACHE
subjects affected / exposed	0 / 11 (0.00%)	0 / 7 (0.00%)	0 / 6 (0.00%)	0 / 19 (0.00%)	1 / 9 (11.11%)	0 / 8 (0.00%)	0 / 26 (0.00%)
occurrences all number	0	0	0	0	1	0	0
Ear and labyrinth disorders
EAR PAIN
subjects affected / exposed	1 / 11 (9.09%)	0 / 7 (0.00%)	0 / 6 (0.00%)	0 / 19 (0.00%)	0 / 9 (0.00%)	0 / 8 (0.00%)	1 / 26 (3.85%)
occurrences all number	1	0	0	0	0	0	1
TYMPANIC MEMBRANE PERFORATION
subjects affected / exposed	0 / 11 (0.00%)	0 / 7 (0.00%)	1 / 6 (16.67%)	0 / 19 (0.00%)	0 / 9 (0.00%)	0 / 8 (0.00%)	0 / 26 (0.00%)
occurrences all number	0	0	1	0	0	0	0
TYMPANIC MEMBRANE HYPERAEMIA
subjects affected / exposed	0 / 11 (0.00%)	0 / 7 (0.00%)	0 / 6 (0.00%)	0 / 19 (0.00%)	0 / 9 (0.00%)	0 / 8 (0.00%)	1 / 26 (3.85%)
occurrences all number	0	0	0	0	0	0	1
Eye disorders
CATARACT
subjects affected / exposed	0 / 11 (0.00%)	0 / 7 (0.00%)	0 / 6 (0.00%)	0 / 19 (0.00%)	0 / 9 (0.00%)	0 / 8 (0.00%)	1 / 26 (3.85%)
occurrences all number	0	0	0	0	0	0	1
DRY EYE
subjects affected / exposed	0 / 11 (0.00%)	0 / 7 (0.00%)	0 / 6 (0.00%)	0 / 19 (0.00%)	0 / 9 (0.00%)	0 / 8 (0.00%)	1 / 26 (3.85%)
occurrences all number	0	0	0	0	0	0	1
VISION BLURRED
subjects affected / exposed	0 / 11 (0.00%)	0 / 7 (0.00%)	0 / 6 (0.00%)	0 / 19 (0.00%)	1 / 9 (11.11%)	0 / 8 (0.00%)	0 / 26 (0.00%)
occurrences all number	0	0	0	0	1	0	0
Gastrointestinal disorders
DIARRHOEA
subjects affected / exposed	2 / 11 (18.18%)	1 / 7 (14.29%)	1 / 6 (16.67%)	2 / 19 (10.53%)	1 / 9 (11.11%)	1 / 8 (12.50%)	9 / 26 (34.62%)
occurrences all number	4	1	1	2	1	3	14
VOMITING
subjects affected / exposed	1 / 11 (9.09%)	1 / 7 (14.29%)	0 / 6 (0.00%)	4 / 19 (21.05%)	4 / 9 (44.44%)	1 / 8 (12.50%)	8 / 26 (30.77%)
occurrences all number	1	1	0	4	11	1	9
ABDOMINAL PAIN UPPER
subjects affected / exposed	1 / 11 (9.09%)	1 / 7 (14.29%)	0 / 6 (0.00%)	2 / 19 (10.53%)	1 / 9 (11.11%)	0 / 8 (0.00%)	3 / 26 (11.54%)
occurrences all number	1	1	0	2	1	0	11
ABDOMINAL PAIN
subjects affected / exposed	1 / 11 (9.09%)	0 / 7 (0.00%)	0 / 6 (0.00%)	1 / 19 (5.26%)	1 / 9 (11.11%)	0 / 8 (0.00%)	1 / 26 (3.85%)
occurrences all number	1	0	0	1	1	0	1
ABDOMINAL DISTENSION
subjects affected / exposed	1 / 11 (9.09%)	0 / 7 (0.00%)	0 / 6 (0.00%)	0 / 19 (0.00%)	0 / 9 (0.00%)	0 / 8 (0.00%)	0 / 26 (0.00%)
occurrences all number	1	0	0	0	0	0	0
BREATH ODOUR
subjects affected / exposed	0 / 11 (0.00%)	0 / 7 (0.00%)	0 / 6 (0.00%)	1 / 19 (5.26%)	0 / 9 (0.00%)	0 / 8 (0.00%)	0 / 26 (0.00%)
occurrences all number	0	0	0	1	0	0	0
CONSTIPATION
subjects affected / exposed	1 / 11 (9.09%)	0 / 7 (0.00%)	0 / 6 (0.00%)	0 / 19 (0.00%)	0 / 9 (0.00%)	0 / 8 (0.00%)	0 / 26 (0.00%)
occurrences all number	1	0	0	0	0	0	0
DYSPEPSIA
subjects affected / exposed	0 / 11 (0.00%)	0 / 7 (0.00%)	1 / 6 (16.67%)	0 / 19 (0.00%)	0 / 9 (0.00%)	0 / 8 (0.00%)	1 / 26 (3.85%)
occurrences all number	0	0	1	0	0	0	1
LIP SWELLING
subjects affected / exposed	0 / 11 (0.00%)	0 / 7 (0.00%)	1 / 6 (16.67%)	0 / 19 (0.00%)	0 / 9 (0.00%)	0 / 8 (0.00%)	0 / 26 (0.00%)
occurrences all number	0	0	1	0	0	0	0
MOUTH ULCERATION
subjects affected / exposed	1 / 11 (9.09%)	0 / 7 (0.00%)	0 / 6 (0.00%)	0 / 19 (0.00%)	0 / 9 (0.00%)	0 / 8 (0.00%)	0 / 26 (0.00%)
occurrences all number	1	0	0	0	0	0	0
NAUSEA
subjects affected / exposed	1 / 11 (9.09%)	0 / 7 (0.00%)	0 / 6 (0.00%)	0 / 19 (0.00%)	1 / 9 (11.11%)	0 / 8 (0.00%)	3 / 26 (11.54%)
occurrences all number	1	0	0	0	1	0	3
GASTRIC ULCER
subjects affected / exposed	0 / 11 (0.00%)	0 / 7 (0.00%)	0 / 6 (0.00%)	0 / 19 (0.00%)	0 / 9 (0.00%)	1 / 8 (12.50%)	0 / 26 (0.00%)
occurrences all number	0	0	0	0	0	1	0
ABDOMINAL PAIN LOWER
subjects affected / exposed	0 / 11 (0.00%)	0 / 7 (0.00%)	0 / 6 (0.00%)	0 / 19 (0.00%)	0 / 9 (0.00%)	0 / 8 (0.00%)	1 / 26 (3.85%)
occurrences all number	0	0	0	0	0	0	1
HAEMATOCHEZIA
subjects affected / exposed	0 / 11 (0.00%)	0 / 7 (0.00%)	0 / 6 (0.00%)	0 / 19 (0.00%)	0 / 9 (0.00%)	0 / 8 (0.00%)	1 / 26 (3.85%)
occurrences all number	0	0	0	0	0	0	3
MALPOSITIONED TEETH
subjects affected / exposed	0 / 11 (0.00%)	0 / 7 (0.00%)	0 / 6 (0.00%)	0 / 19 (0.00%)	0 / 9 (0.00%)	0 / 8 (0.00%)	1 / 26 (3.85%)
occurrences all number	0	0	0	0	0	0	1
MUCOUS STOOLS
subjects affected / exposed	0 / 11 (0.00%)	0 / 7 (0.00%)	0 / 6 (0.00%)	0 / 19 (0.00%)	0 / 9 (0.00%)	0 / 8 (0.00%)	1 / 26 (3.85%)
occurrences all number	0	0	0	0	0	0	1
RECTAL HAEMORRHAGE
subjects affected / exposed	0 / 11 (0.00%)	0 / 7 (0.00%)	0 / 6 (0.00%)	0 / 19 (0.00%)	0 / 9 (0.00%)	0 / 8 (0.00%)	1 / 26 (3.85%)
occurrences all number	0	0	0	0	0	0	1
Skin and subcutaneous tissue disorders
RASH
subjects affected / exposed	0 / 11 (0.00%)	0 / 7 (0.00%)	3 / 6 (50.00%)	1 / 19 (5.26%)	0 / 9 (0.00%)	0 / 8 (0.00%)	2 / 26 (7.69%)
occurrences all number	0	0	4	2	0	0	3
DERMATITIS CONTACT
subjects affected / exposed	1 / 11 (9.09%)	0 / 7 (0.00%)	0 / 6 (0.00%)	1 / 19 (5.26%)	1 / 9 (11.11%)	0 / 8 (0.00%)	0 / 26 (0.00%)
occurrences all number	1	0	0	1	1	0	0
SKIN ULCER
subjects affected / exposed	0 / 11 (0.00%)	0 / 7 (0.00%)	1 / 6 (16.67%)	0 / 19 (0.00%)	0 / 9 (0.00%)	0 / 8 (0.00%)	0 / 26 (0.00%)
occurrences all number	0	0	1	0	0	0	0
DRY SKIN
subjects affected / exposed	0 / 11 (0.00%)	0 / 7 (0.00%)	0 / 6 (0.00%)	2 / 19 (10.53%)	0 / 9 (0.00%)	0 / 8 (0.00%)	0 / 26 (0.00%)
occurrences all number	0	0	0	2	0	0	0
ERYTHEMA
subjects affected / exposed	0 / 11 (0.00%)	0 / 7 (0.00%)	0 / 6 (0.00%)	1 / 19 (5.26%)	0 / 9 (0.00%)	0 / 8 (0.00%)	0 / 26 (0.00%)
occurrences all number	0	0	0	1	0	0	0
INGROWING NAIL
subjects affected / exposed	0 / 11 (0.00%)	0 / 7 (0.00%)	0 / 6 (0.00%)	1 / 19 (5.26%)	0 / 9 (0.00%)	0 / 8 (0.00%)	0 / 26 (0.00%)
occurrences all number	0	0	0	1	0	0	0
ACNE
subjects affected / exposed	0 / 11 (0.00%)	0 / 7 (0.00%)	0 / 6 (0.00%)	0 / 19 (0.00%)	0 / 9 (0.00%)	0 / 8 (0.00%)	1 / 26 (3.85%)
occurrences all number	0	0	0	0	0	0	1
COLD SWEAT
subjects affected / exposed	0 / 11 (0.00%)	0 / 7 (0.00%)	0 / 6 (0.00%)	0 / 19 (0.00%)	0 / 9 (0.00%)	0 / 8 (0.00%)	1 / 26 (3.85%)
occurrences all number	0	0	0	0	0	0	1
EPHELIDES
subjects affected / exposed	0 / 11 (0.00%)	0 / 7 (0.00%)	0 / 6 (0.00%)	0 / 19 (0.00%)	0 / 9 (0.00%)	0 / 8 (0.00%)	1 / 26 (3.85%)
occurrences all number	0	0	0	0	0	0	1
LIVEDO RETICULARIS
subjects affected / exposed	0 / 11 (0.00%)	0 / 7 (0.00%)	0 / 6 (0.00%)	0 / 19 (0.00%)	1 / 9 (11.11%)	0 / 8 (0.00%)	0 / 26 (0.00%)
occurrences all number	0	0	0	0	1	0	0
PRURITUS
subjects affected / exposed	0 / 11 (0.00%)	0 / 7 (0.00%)	0 / 6 (0.00%)	0 / 19 (0.00%)	0 / 9 (0.00%)	0 / 8 (0.00%)	1 / 26 (3.85%)
occurrences all number	0	0	0	0	0	0	1
Renal and urinary disorders
POLYURIA
subjects affected / exposed	0 / 11 (0.00%)	0 / 7 (0.00%)	0 / 6 (0.00%)	1 / 19 (5.26%)	0 / 9 (0.00%)	0 / 8 (0.00%)	0 / 26 (0.00%)
occurrences all number	0	0	0	1	0	0	0
NEPHROLITHIASIS
subjects affected / exposed	0 / 11 (0.00%)	0 / 7 (0.00%)	0 / 6 (0.00%)	0 / 19 (0.00%)	1 / 9 (11.11%)	0 / 8 (0.00%)	0 / 26 (0.00%)
occurrences all number	0	0	0	0	1	0	0
Endocrine disorders
ADRENAL INSUFFICIENCY
subjects affected / exposed	0 / 11 (0.00%)	0 / 7 (0.00%)	0 / 6 (0.00%)	0 / 19 (0.00%)	0 / 9 (0.00%)	0 / 8 (0.00%)	1 / 26 (3.85%)
occurrences all number	0	0	0	0	0	0	1
CUSHINGOID
subjects affected / exposed	0 / 11 (0.00%)	0 / 7 (0.00%)	0 / 6 (0.00%)	0 / 19 (0.00%)	0 / 9 (0.00%)	0 / 8 (0.00%)	2 / 26 (7.69%)
occurrences all number	0	0	0	0	0	0	2
DELAYED PUBERTY
subjects affected / exposed	0 / 11 (0.00%)	0 / 7 (0.00%)	0 / 6 (0.00%)	0 / 19 (0.00%)	1 / 9 (11.11%)	0 / 8 (0.00%)	0 / 26 (0.00%)
occurrences all number	0	0	0	0	1	0	0
Musculoskeletal and connective tissue disorders
PAIN IN EXTREMITY
subjects affected / exposed	0 / 11 (0.00%)	1 / 7 (14.29%)	2 / 6 (33.33%)	0 / 19 (0.00%)	2 / 9 (22.22%)	0 / 8 (0.00%)	4 / 26 (15.38%)
occurrences all number	0	1	2	0	2	0	4
BACK PAIN
subjects affected / exposed	1 / 11 (9.09%)	0 / 7 (0.00%)	0 / 6 (0.00%)	1 / 19 (5.26%)	2 / 9 (22.22%)	0 / 8 (0.00%)	5 / 26 (19.23%)
occurrences all number	1	0	0	1	2	0	5
JOINT SWELLING
subjects affected / exposed	0 / 11 (0.00%)	1 / 7 (14.29%)	0 / 6 (0.00%)	1 / 19 (5.26%)	0 / 9 (0.00%)	0 / 8 (0.00%)	1 / 26 (3.85%)
occurrences all number	0	1	0	1	0	0	1
MUSCLE SPASMS
subjects affected / exposed	1 / 11 (9.09%)	0 / 7 (0.00%)	1 / 6 (16.67%)	0 / 19 (0.00%)	0 / 9 (0.00%)	0 / 8 (0.00%)	2 / 26 (7.69%)
occurrences all number	1	0	1	0	0	0	2
NECK PAIN
subjects affected / exposed	0 / 11 (0.00%)	0 / 7 (0.00%)	1 / 6 (16.67%)	0 / 19 (0.00%)	0 / 9 (0.00%)	0 / 8 (0.00%)	1 / 26 (3.85%)
occurrences all number	0	0	1	0	0	0	1
ARTHRALGIA
subjects affected / exposed	0 / 11 (0.00%)	0 / 7 (0.00%)	0 / 6 (0.00%)	1 / 19 (5.26%)	3 / 9 (33.33%)	0 / 8 (0.00%)	3 / 26 (11.54%)
occurrences all number	0	0	0	1	3	0	4
MYALGIA
subjects affected / exposed	0 / 11 (0.00%)	0 / 7 (0.00%)	0 / 6 (0.00%)	1 / 19 (5.26%)	1 / 9 (11.11%)	0 / 8 (0.00%)	1 / 26 (3.85%)
occurrences all number	0	0	0	1	1	0	1
JOINT CONTRACTURE
subjects affected / exposed	0 / 11 (0.00%)	0 / 7 (0.00%)	0 / 6 (0.00%)	0 / 19 (0.00%)	1 / 9 (11.11%)	0 / 8 (0.00%)	1 / 26 (3.85%)
occurrences all number	0	0	0	0	1	0	1
OSTEOPOROSIS
subjects affected / exposed	0 / 11 (0.00%)	0 / 7 (0.00%)	0 / 6 (0.00%)	0 / 19 (0.00%)	2 / 9 (22.22%)	0 / 8 (0.00%)	0 / 26 (0.00%)
occurrences all number	0	0	0	0	2	0	0
MUSCULAR WEAKNESS
subjects affected / exposed	0 / 11 (0.00%)	0 / 7 (0.00%)	0 / 6 (0.00%)	0 / 19 (0.00%)	1 / 9 (11.11%)	0 / 8 (0.00%)	0 / 26 (0.00%)
occurrences all number	0	0	0	0	1	0	0
COCCYDYNIA
subjects affected / exposed	0 / 11 (0.00%)	0 / 7 (0.00%)	0 / 6 (0.00%)	0 / 19 (0.00%)	0 / 9 (0.00%)	0 / 8 (0.00%)	1 / 26 (3.85%)
occurrences all number	0	0	0	0	0	0	1
COSTOCHONDRITIS
subjects affected / exposed	0 / 11 (0.00%)	0 / 7 (0.00%)	0 / 6 (0.00%)	0 / 19 (0.00%)	0 / 9 (0.00%)	0 / 8 (0.00%)	1 / 26 (3.85%)
occurrences all number	0	0	0	0	0	0	1
INTERVERTEBRAL DISC COMPRESSION
subjects affected / exposed	0 / 11 (0.00%)	0 / 7 (0.00%)	0 / 6 (0.00%)	0 / 19 (0.00%)	0 / 9 (0.00%)	0 / 8 (0.00%)	1 / 26 (3.85%)
occurrences all number	0	0	0	0	0	0	2
MUSCLE TIGHTNESS
subjects affected / exposed	0 / 11 (0.00%)	0 / 7 (0.00%)	0 / 6 (0.00%)	0 / 19 (0.00%)	1 / 9 (11.11%)	0 / 8 (0.00%)	0 / 26 (0.00%)
occurrences all number	0	0	0	0	1	0	0
PAIN IN JAW
subjects affected / exposed	0 / 11 (0.00%)	0 / 7 (0.00%)	0 / 6 (0.00%)	0 / 19 (0.00%)	0 / 9 (0.00%)	0 / 8 (0.00%)	1 / 26 (3.85%)
occurrences all number	0	0	0	0	0	0	1
SYNOVIAL CYST
subjects affected / exposed	0 / 11 (0.00%)	0 / 7 (0.00%)	0 / 6 (0.00%)	0 / 19 (0.00%)	1 / 9 (11.11%)	0 / 8 (0.00%)	0 / 26 (0.00%)
occurrences all number	0	0	0	0	1	0	0
Infections and infestations
UPPER RESPIRATORY TRACT INFECTION
subjects affected / exposed	2 / 11 (18.18%)	0 / 7 (0.00%)	2 / 6 (33.33%)	4 / 19 (21.05%)	0 / 9 (0.00%)	1 / 8 (12.50%)	11 / 26 (42.31%)
occurrences all number	3	0	2	5	0	1	25
NASOPHARYNGITIS
subjects affected / exposed	1 / 11 (9.09%)	1 / 7 (14.29%)	1 / 6 (16.67%)	4 / 19 (21.05%)	4 / 9 (44.44%)	1 / 8 (12.50%)	7 / 26 (26.92%)
occurrences all number	1	2	1	4	7	2	12
EAR INFECTION
subjects affected / exposed	1 / 11 (9.09%)	0 / 7 (0.00%)	1 / 6 (16.67%)	0 / 19 (0.00%)	2 / 9 (22.22%)	1 / 8 (12.50%)	4 / 26 (15.38%)
occurrences all number	1	0	1	0	2	1	5
PHARYNGITIS STREPTOCOCCAL
subjects affected / exposed	1 / 11 (9.09%)	0 / 7 (0.00%)	0 / 6 (0.00%)	0 / 19 (0.00%)	1 / 9 (11.11%)	1 / 8 (12.50%)	3 / 26 (11.54%)
occurrences all number	1	0	0	0	1	1	4
SINUSITIS
subjects affected / exposed	0 / 11 (0.00%)	0 / 7 (0.00%)	1 / 6 (16.67%)	0 / 19 (0.00%)	0 / 9 (0.00%)	0 / 8 (0.00%)	2 / 26 (7.69%)
occurrences all number	0	0	1	0	0	0	3
NAIL INFECTION
subjects affected / exposed	0 / 11 (0.00%)	0 / 7 (0.00%)	0 / 6 (0.00%)	1 / 19 (5.26%)	0 / 9 (0.00%)	0 / 8 (0.00%)	0 / 26 (0.00%)
occurrences all number	0	0	0	1	0	0	0
INFLUENZA
subjects affected / exposed	0 / 11 (0.00%)	0 / 7 (0.00%)	0 / 6 (0.00%)	0 / 19 (0.00%)	1 / 9 (11.11%)	0 / 8 (0.00%)	3 / 26 (11.54%)
occurrences all number	0	0	0	0	1	0	5
PHARYNGITIS
subjects affected / exposed	0 / 11 (0.00%)	0 / 7 (0.00%)	0 / 6 (0.00%)	0 / 19 (0.00%)	0 / 9 (0.00%)	0 / 8 (0.00%)	2 / 26 (7.69%)
occurrences all number	0	0	0	0	0	0	3
GASTROENTERITIS VIRAL
subjects affected / exposed	0 / 11 (0.00%)	0 / 7 (0.00%)	0 / 6 (0.00%)	0 / 19 (0.00%)	1 / 9 (11.11%)	0 / 8 (0.00%)	0 / 26 (0.00%)
occurrences all number	0	0	0	0	1	0	0
ABSCESS ORAL
subjects affected / exposed	0 / 11 (0.00%)	0 / 7 (0.00%)	0 / 6 (0.00%)	0 / 19 (0.00%)	0 / 9 (0.00%)	0 / 8 (0.00%)	1 / 26 (3.85%)
occurrences all number	0	0	0	0	0	0	1
BODY TINEA
subjects affected / exposed	0 / 11 (0.00%)	0 / 7 (0.00%)	0 / 6 (0.00%)	0 / 19 (0.00%)	0 / 9 (0.00%)	0 / 8 (0.00%)	1 / 26 (3.85%)
occurrences all number	0	0	0	0	0	0	1
CONJUNCTIVITIS
subjects affected / exposed	0 / 11 (0.00%)	0 / 7 (0.00%)	0 / 6 (0.00%)	0 / 19 (0.00%)	0 / 9 (0.00%)	0 / 8 (0.00%)	1 / 26 (3.85%)
occurrences all number	0	0	0	0	0	0	1
FUNGAL SKIN INFECTION
subjects affected / exposed	0 / 11 (0.00%)	0 / 7 (0.00%)	0 / 6 (0.00%)	0 / 19 (0.00%)	0 / 9 (0.00%)	0 / 8 (0.00%)	1 / 26 (3.85%)
occurrences all number	0	0	0	0	0	0	1
LABYRINTHITIS
subjects affected / exposed	0 / 11 (0.00%)	0 / 7 (0.00%)	0 / 6 (0.00%)	0 / 19 (0.00%)	0 / 9 (0.00%)	0 / 8 (0.00%)	1 / 26 (3.85%)
occurrences all number	0	0	0	0	0	0	1
ONYCHOMYCOSIS
subjects affected / exposed	0 / 11 (0.00%)	0 / 7 (0.00%)	0 / 6 (0.00%)	0 / 19 (0.00%)	0 / 9 (0.00%)	1 / 8 (12.50%)	0 / 26 (0.00%)
occurrences all number	0	0	0	0	0	1	0
PNEUMONIA MYCOPLASMAL
subjects affected / exposed	0 / 11 (0.00%)	0 / 7 (0.00%)	0 / 6 (0.00%)	0 / 19 (0.00%)	0 / 9 (0.00%)	0 / 8 (0.00%)	1 / 26 (3.85%)
occurrences all number	0	0	0	0	0	0	1
SCARLET FEVER
subjects affected / exposed	0 / 11 (0.00%)	0 / 7 (0.00%)	0 / 6 (0.00%)	0 / 19 (0.00%)	0 / 9 (0.00%)	0 / 8 (0.00%)	1 / 26 (3.85%)
occurrences all number	0	0	0	0	0	0	1
SKIN CANDIDA
subjects affected / exposed	0 / 11 (0.00%)	0 / 7 (0.00%)	0 / 6 (0.00%)	0 / 19 (0.00%)	1 / 9 (11.11%)	0 / 8 (0.00%)	0 / 26 (0.00%)
occurrences all number	0	0	0	0	1	0	0
STAPHYLOCOCCAL SKIN INFECTION
subjects affected / exposed	0 / 11 (0.00%)	0 / 7 (0.00%)	0 / 6 (0.00%)	0 / 19 (0.00%)	0 / 9 (0.00%)	0 / 8 (0.00%)	1 / 26 (3.85%)
occurrences all number	0	0	0	0	0	0	1
TOOTH ABSCESS
subjects affected / exposed	0 / 11 (0.00%)	0 / 7 (0.00%)	0 / 6 (0.00%)	0 / 19 (0.00%)	0 / 9 (0.00%)	0 / 8 (0.00%)	1 / 26 (3.85%)
occurrences all number	0	0	0	0	0	0	1
VIRAL RHINITIS
subjects affected / exposed	0 / 11 (0.00%)	0 / 7 (0.00%)	0 / 6 (0.00%)	0 / 19 (0.00%)	0 / 9 (0.00%)	0 / 8 (0.00%)	1 / 26 (3.85%)
occurrences all number	0	0	0	0	0	0	1
Metabolism and nutrition disorders
DECREASED APPETITE
subjects affected / exposed	0 / 11 (0.00%)	0 / 7 (0.00%)	1 / 6 (16.67%)	0 / 19 (0.00%)	0 / 9 (0.00%)	0 / 8 (0.00%)	0 / 26 (0.00%)
occurrences all number	0	0	1	0	0	0	0

More information

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Were there any global substantial amendments to the protocol? Yes

18 Sep 2015

Updated schedule and clarified data to be included in the interim analyses. Added additional laboratory assessments for additional safety monitoring and added storage of residual plasma sample remaining after glutamate dehydrogenase (GLDH) analysis for up to five years after the last subject’s last study visit. Clarified the secondary and exploratory endpoints associated with magnetic resonance imaging (MRI) and magnetic resonance spectroscopy (MRS) procedures and clarified that MRS will be conducted at selected sites as training is completed.

09 Dec 2015

Removed ACTIVE assessment from study procedures. Clarified inclusion/exclusion criteria. Updated number of planned interim analyses.

21 Apr 2016

Clarification added after the withdrawal of study drug into an appropriate-sized syringe, the product should be administered SC within 4 hours to align with our current investigational brochure. Updated safety information. Removed the exclusion criteria: baseline 4SC 9 Day -1) more than a 20% or 0.5 second reduction, whichever is greater, from the valid screening 4SC 9 Day -45 to Day -7) used to determine eligibility.

21 Feb 2017

Added open-label extension (OLE) phase to the study. Updated plan for clinical study reports.

21 Aug 2017

Changed Sponsor from Bristol-Myers Squibb to F. Hoffmann-La Roche Ltd. Changed study drug name from BMS-986089 to RO7239361.

14 Oct 2018

Changed investigational medicinal product (IMP) dose in the open-label extension (OLE) phase date stamp. Changed IMP from vial to prefilled syringes (PFS) in the OLE. Changed the study assessments and procedures to add a new clinic visits, pharmacokinetic/pharmacodynamic (PK/PD) and safety assessments after the switch to PFS. Product Development Background and Overall Risk/Benefit sections were updated. Updated monitoring of anti-drug antibodies (ADAs) during 24-week follow-up period Deleted references to BMS study and drug number.

Were there any global interruptions to the trial? Yes

Date	Interruption	Restart date
15 Apr 2020	A pre-planned futility analysis indicated lack of efficacy in study WN40227/2016-001654-18 and led to discontinuation of both ongoing studies in DMD (WN40226/2015-005455 and WN40227/2016-001654-18).	-

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 Multi-Site, Randomized, Placebo-Controlled, Double-Blind, Multiple Ascending Subcutaneous Dose Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of RO7239361 in Ambulatory Boys With Duchenne Muscular Dystrophy

Trial information

Trial information

Subject disposition

Subject disposition

Baseline characteristics

Baseline characteristics

End points

End points

Primary: Safety Summary for the 24 Week Double-Blind Phase

Primary: Safety Summary for the 24 Week Double-Blind Phase

Primary: Safety Summary up to Week 72

Primary: Safety Summary up to Week 72

Secondary: Maximum Observed Serum Concentrations (Cmax) of RO7239361 at Steady State for 4 mg QW, 12.5 mg QW and 35 mg QW doses.

Secondary: Maximum Observed Serum Concentrations (Cmax) of RO7239361 at Steady State for 4 mg QW, 12.5 mg QW and 35 mg QW doses.

Secondary: Maximum Observed Serum Concentrations (Cmax) of RO7239361 at Steady State for 50 mg QW dose.

Secondary: Maximum Observed Serum Concentrations (Cmax) of RO7239361 at Steady State for 50 mg QW dose.

Secondary: Time of Maximum Observed Serum Concentrations (Tmax) of RO7239361 at Steady State for 4 mg QW, 12.5 mg QW and 35 mg QW doses.

Secondary: Time of Maximum Observed Serum Concentrations (Tmax) of RO7239361 at Steady State for 4 mg QW, 12.5 mg QW and 35 mg QW doses.

Secondary: Time of Maximum Observed Serum Concentrations (Tmax) of RO7239361 at Steady State for 50 mg QW dose.

Secondary: Time of Maximum Observed Serum Concentrations (Tmax) of RO7239361 at Steady State for 50 mg QW dose.

Secondary: Area Under the Concentration-Time Curve from Time Zero to Time of Next Dosing (AUCtau) of RO7239361 at Steady State for 4 mg QW, 12.5 mg QW and 35 mg QW doses.

Secondary: Area Under the Concentration-Time Curve from Time Zero to Time of Next Dosing (AUCtau) of RO7239361 at Steady State for 4 mg QW, 12.5 mg QW and 35 mg QW doses.

Secondary: Area Under the Concentration-Time Curve from Time Zero to Time of Next Dosing (AUCtau) of RO7239361 at Steady State for 50 mg QW dose.

Secondary: Area Under the Concentration-Time Curve from Time Zero to Time of Next Dosing (AUCtau) of RO7239361 at Steady State for 50 mg QW dose.

Secondary: RO7239361 Trough Concentrations

Secondary: RO7239361 Trough Concentrations

Secondary: Percentage of Participants with Positive Anti-RO7239361 Antibodies (ADA) Assessment, Double-Blind Phase

Secondary: Percentage of Participants with Positive Anti-RO7239361 Antibodies (ADA) Assessment, Double-Blind Phase

Secondary: Percentage of Participants with Positive Anti-RO7239361 Antibodies (ADA) Assessment up to Week 72

Secondary: Percentage of Participants with Positive Anti-RO7239361 Antibodies (ADA) Assessment up to Week 72

Secondary: Serum Concentration of Free Myostatin in the Double-Blind Phase

Secondary: Serum Concentration of Free Myostatin in the Double-Blind Phase

Secondary: Percent Inhibition of Free Myostatin in the Double-Blind Phase

Secondary: Percent Inhibition of Free Myostatin in the Double-Blind Phase

Secondary: Serum Concentration of Drug-Myostatin Complex in the Double-Blind Phase

Secondary: Serum Concentration of Drug-Myostatin Complex in the Double-Blind Phase

Secondary: Fold Change from Baseline in Contractile Versus Non-contractile Content for Muscles in the Right Thigh in the Double-Blind Phase

Secondary: Fold Change from Baseline in Contractile Versus Non-contractile Content for Muscles in the Right Thigh in the Double-Blind Phase

Secondary: Change from Baseline in Thigh Muscle Maximal Cross Sectional Area (CSAmax) in the Double-Blind Phase

Secondary: Change from Baseline in Thigh Muscle Maximal Cross Sectional Area (CSAmax) in the Double-Blind Phase

Secondary: Percentage of Participants with Positive Anti-RO7239361 Antibodies (ADA) Assessment, Whole Study

Secondary: Percentage of Participants with Positive Anti-RO7239361 Antibodies (ADA) Assessment, Whole Study

Secondary: Serum Concentration of Free Myostatin, Whole Study

Secondary: Serum Concentration of Free Myostatin, Whole Study

Secondary: Percent Inhibition of Free Myostatin, Whole Study

Secondary: Percent Inhibition of Free Myostatin, Whole Study

Secondary: Serum Concentration of Drug-Myostatin Complex, Whole Study

Secondary: Serum Concentration of Drug-Myostatin Complex, Whole Study

Adverse events

Adverse events

More information

Substantial protocol amendments (globally)

Interruptions (globally)

Limitations and caveats

More information

Clinical Trial Results:
A Multi-Site, Randomized, Placebo-Controlled, Double-Blind, Multiple Ascending Subcutaneous Dose Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of RO7239361 in Ambulatory Boys With Duchenne Muscular Dystrophy