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Trial record 1 of 1 for:    NCT02515669
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Study of an Investigational Drug, RO7239361 (BMS-986089), in Ambulatory Boys With DMD

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02515669
Recruitment Status : Terminated (A pre-planned futility analysis indicated lack of efficacy.)
First Posted : August 5, 2015
Results First Posted : May 8, 2019
Last Update Posted : June 9, 2020
Sponsor:
Information provided by (Responsible Party):
Hoffmann-La Roche

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor);   Primary Purpose: Basic Science
Condition Muscular Dystrophy (DMD)
Interventions Drug: RO7239361
Drug: Placebo
Enrollment 43
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.
Pre-assignment 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.
Arm/Group Title Placebo Panel 1 RO7239361 Panel 2 RO7239361 Panel 3 and Expansion Panel RO7239361
Hide Arm/Group Description Placebo subcutaneous injections on specified days 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). 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 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
Period Title: 24 Week Double-Blind Phase
Started 11 7 6 19
Completed 11 7 6 19
Not Completed 0 0 0 0
Period Title: 48 Week Open-Label Phase
Started [1] 0 [2] 9 [3] 8 [4] 26 [5]
Completed 0 9 7 26
Not Completed 0 0 1 0
Reason Not Completed
Other             0             0             1             0
[1]
Participants assigned to placebo in the double blind phase switched to RO for the open-label phase
[2]
Double-blind (DB) placebo participants switched to RO7239361 (RO) for the open label phase.
[3]
2 DB placebo participants switched to the Panel 1 RO cohort for the open-label phase.
[4]
2 DB placebo participants switched to the Panel 2 RO cohort for the open-label phase.
[5]
7 DB placebo participants switched to the Panel 3/Expansion RO cohort for the open-label phase.
Arm/Group Title Placebo Panel 1 RO7239361 Panel 2 RO7239361 Panel 3 and Expansion Panel RO7239361 Total
Hide Arm/Group Description Placebo subcutaneous injections on specified days 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). 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 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 Total of all reporting groups
Overall Number of Baseline Participants 11 7 6 19 43
Hide Baseline Analysis Population Description
Doses tested in this study were based upon achieving and maintaining moderate (> 50% suppression), high (> 85%), and near complete (> 95%) suppression of serum free myostatin levels for Panels 1, 2, and 3 and Expansion Panel, respectively. Panel 3 was expanded after at least 2 weeks of dosing had been complete by at least 6 participants.
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 11 participants 7 participants 6 participants 19 participants 43 participants
8.8  (1.33) 8.0  (2.24) 8.0  (1.79) 8.1  (1.81) 8.2  (1.74)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 11 participants 7 participants 6 participants 19 participants 43 participants
Female
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Male
11
 100.0%
7
 100.0%
6
 100.0%
19
 100.0%
43
 100.0%
Ethnicity (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 11 participants 7 participants 6 participants 19 participants 43 participants
Hispanic or Latino
1
   9.1%
0
   0.0%
1
  16.7%
2
  10.5%
4
   9.3%
Not Hispanic or Latino
9
  81.8%
7
 100.0%
5
  83.3%
13
  68.4%
34
  79.1%
Unknown or Not Reported
1
   9.1%
0
   0.0%
0
   0.0%
4
  21.1%
5
  11.6%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 11 participants 7 participants 6 participants 19 participants 43 participants
American Indian or Alaska Native
0
   0.0%
1
  14.3%
0
   0.0%
0
   0.0%
1
   2.3%
Asian
2
  18.2%
0
   0.0%
2
  33.3%
5
  26.3%
9
  20.9%
Native Hawaiian or Other Pacific Islander
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Black or African American
0
   0.0%
0
   0.0%
1
  16.7%
0
   0.0%
1
   2.3%
White
9
  81.8%
6
  85.7%
3
  50.0%
13
  68.4%
31
  72.1%
More than one race
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Unknown or Not Reported
0
   0.0%
0
   0.0%
0
   0.0%
1
   5.3%
1
   2.3%
1.Primary Outcome
Title Safety Summary for the 24 Week Double-Blind Phase
Hide 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.

Time Frame 24 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Placebo Panel 1 RO7239361 Panel 2 RO7239361 Panel 3 and Expansion RO7239361
Hide Arm/Group Description:
Placebo subcutaneous injections on specified days
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).
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
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
Overall Number of Participants Analyzed 11 7 6 19
Measure Type: Number
Unit of Measure: Percent of Participants
Death 0 0 0 0
Adverse Events (AEs) 81.8 71.4 100.0 94.7
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
2.Primary Outcome
Title Safety Summary for the Whole Study
Hide Description

Percentage of participants with fatalities, adverse event (AEs) and serious adverse events (SAEs) for the whole study.

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.

Time Frame Baseline to 72 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Panel 1 RO7239361 Panel 2 RO7239361 Panel 3 and Expansion Panel RO7239361
Hide Arm/Group 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).
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
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
Overall Number of Participants Analyzed 9 8 26
Measure Type: Number
Unit of Measure: Percent of Participants
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 3.8
Related AEs 77.8 62.5 50.0
3.Secondary Outcome
Title Maximum Observed Serum Concentrations (Cmax) of RO7239361 at Steady State for 4 mg QW, 12.5 mg QW and 35 mg QW Doses.
Hide 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.

Time Frame Baseline to Week 24
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Panel 1 RO7239361 Panel 2 RO7239361 Panel 3 and Expansion Panel RO7239361
Hide Arm/Group 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).
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
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
Overall Number of Participants Analyzed 7 6 19
Geometric Mean (Geometric Coefficient of Variation)
Unit of Measure: ng/ml
3217
(15.8%)
8490
(21.5%)
24242
(26.3%)
4.Secondary Outcome
Title Maximum Observed Serum Concentrations (Cmax) of RO7239361 at Steady State for 50 mg QW Dose.
Hide 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.

Time Frame Baseline to Week 24
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Panel 3 and Expansion Panel RO7239361
Hide Arm/Group 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
Overall Number of Participants Analyzed 19
Geometric Mean (Geometric Coefficient of Variation)
Unit of Measure: ng/mL
23297 [1] 
(NA%)
[1]
Geometric Coefficient of Variation not evaluable as only one participant was included at this dose.
5.Secondary Outcome
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.
Hide 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.

Time Frame Baseline to Week 24
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Panel 1 RO7239361 Panel 2 RO7239361 Panel 3 and Expansion Panel RO7239361
Hide Arm/Group 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).
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
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
Overall Number of Participants Analyzed 7 6 18
Median (Full Range)
Unit of Measure: hour
28
(12 to 48)
24
(11 to 47)
30
(11 to 65)
6.Secondary Outcome
Title Time of Maximum Observed Serum Concentrations (Tmax) of RO7239361 at Steady State for 50 mg QW Dose.
Hide 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.

Time Frame Baseline to Week 24
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Panel 3 and Expansion Panel RO7239361
Hide Arm/Group 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
Overall Number of Participants Analyzed 1
Median (Full Range)
Unit of Measure: hour
44 [1] 
(NA to NA)
[1]
Geometric Coefficient of Variation not evaluable as only one participant was included at this dose.
7.Secondary Outcome
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.
Hide 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.

Time Frame Baseline to Week 24
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Panel 1 RO7239361 Panel 2 RO7239361 Panel 3 and Expansion Panel RO7239361
Hide Arm/Group 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).
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
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
Overall Number of Participants Analyzed 7 6 18
Geometric Mean (Geometric Coefficient of Variation)
Unit of Measure: ng•day/mL
18676
(23%)
51461
(18%)
150609
(24%)
8.Secondary Outcome
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.
Hide 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.

Time Frame Baseline to Week 24
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Panel 3 and Expansion Panel RO7239361
Hide Arm/Group 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
Overall Number of Participants Analyzed 1
Geometric Mean (Geometric Coefficient of Variation)
Unit of Measure: ng•day/mL
151000 [1] 
(NA%)
[1]
Geometric Coefficient of Variation not evaluable as only one participant was included at this dose.
9.Secondary Outcome
Title RO7239361 Trough Concentrations
Hide 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.

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.

Time Frame Baseline to Week 24
Hide Outcome Measure Data
Hide Analysis Population Description
Pharmacokinetic population. Where the number of participants analyzed is 0 for the Panel 2 20mg dose, it is due to that dose not being administered at that visit.
Arm/Group Title Panel 1 RO7239361 Panel 2 RO7239361 12.5mg Panel 2 RO7239361 20mg Panel 3 RO7239361 35mg Expansion Panel RO7239361 35mg Expansion Panel RO7239361 50mg
Hide Arm/Group 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).
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

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.

The dose was not administered at Days 1, 15, 22, 29 and 84.

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
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
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
Overall Number of Participants Analyzed 9 7 1 7 16 1
Geometric Mean (Geometric Coefficient of Variation)
Unit of Measure: ng/mL
Day 1 Number Analyzed 7 participants 6 participants 0 participants 5 participants 12 participants 1 participants
825.343
(23%)
2115.951
(20%)
6559.364
(26%)
6017.978
(23%)
5020.000 [1] 
(NA%)
Day 15 Number Analyzed 7 participants 6 participants 0 participants 6 participants 12 participants 1 participants
1258.746
(35%)
3697.590
(20%)
9957.352
(34%)
9944.183
(24%)
9980.000 [1] 
(NA%)
Day 22 Number Analyzed 5 participants 6 participants 0 participants 5 participants 11 participants 1 participants
1640.052
(15%)
4220.280
(32%)
12383.747
(30%)
13215.188
(24%)
NA [2] 
(NA%)
Day 29 Number Analyzed 7 participants 6 participants 0 participants 6 participants 12 participants 1 participants
2015.695
(32%)
5107.668
(25%)
14110.005
(34%)
15094.455
(19%)
NA [2] 
(NA%)
Day 84 Number Analyzed 8 participants 6 participants 0 participants 7 participants 16 participants 1 participants
2179.385
(26%)
6484.502
(19%)
19332.421
(20%)
18789.382
(18%)
27800.000 [1] 
(NA%)
Day 168 Number Analyzed 9 participants 7 participants 1 participants 7 participants 14 participants 1 participants
2438.595
(16%)
6100.315
(25%)
6340.000 [1] 
(NA%)
19923.182
(41%)
12580.299
(41%)
26200.000 [1] 
(NA%)
[1]
Geometric Coefficient of Variation not evaluable as only one participant was included at this dose and therefore there was no dispersion.
[2]
Not evaluable, no sample taken
10.Secondary Outcome
Title Frequency of Subjects With Positive Anti-RO7239361 Antibodies (ADA) Assessment, Double-Blind Phase
Hide 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.

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.

Time Frame Day 8 through Week 24, baseline and on-study information represented in table.
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Placebo Panel 1 RO7239361 Panel 2 RO7239361 Panel 3 and Expansion Panel RO7239361
Hide Arm/Group Description:
Placebo subcutaneous injections on specified days
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).
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
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
Overall Number of Participants Analyzed 11 7 6 19
Measure Type: Number
Unit of Measure: Percentage of Participants
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
11.Secondary Outcome
Title Frequency of Subjects With Positive Anti-RO7239361 Antibodies (ADA) Assessment, Whole Study
Hide 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.

Time Frame Day 8 through Week 72, baseline and on-study information represented in table.
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Panel 1 RO7239361 Panel 2 RO7239361 Panel 3 and Expansion Panel RO7239361
Hide Arm/Group 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).
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
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
Overall Number of Participants Analyzed 9 8 26
Measure Type: Number
Unit of Measure: Percentage of Participants
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
12.Secondary Outcome
Title Serum Concentration of Free Myostatin in the Double-blind Phase
Hide 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.
Time Frame Baseline through week 24
Hide Outcome Measure Data
Hide Analysis Population Description
The pharmacodynamics (PD) data set includes all available data from subjects for whom PD measurements are available at baseline and at least one other timepoint.
Arm/Group Title Placebo Panel 1 RO7239361 Panel 2 RO7239361 Panel 3 and Expansion Panel RO7239361
Hide Arm/Group Description:
Placebo subcutaneous injections on specified days
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).
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
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
Overall Number of Participants Analyzed 11 7 6 19
Mean (Standard Deviation)
Unit of Measure: pg/mL
Baseline Number Analyzed 11 participants 6 participants 6 participants 16 participants
1405.1  (809.57) 815.3  (155.14) 1391.3  (1084.97) 1121.8  (526.45)
Day 4 Number Analyzed 8 participants 5 participants 4 participants 18 participants
1356.6  (641.27) 421.4  (70.60) 321.3  (165.91) 58.3  (33.20)
Day 5 Number Analyzed 9 participants 7 participants 6 participants 18 participants
1438.2  (809.18) 446.6  (108.81) 357.8  (166.27) 66.1  (39.07)
Day 8 Number Analyzed 10 participants 6 participants 6 participants 19 participants
1014.2  (549.26) 522.7  (131.51) 479.2  (319.13) 206.8  (419.21)
Day 15 Number Analyzed 11 participants 7 participants 6 participants 19 participants
1266.9  (611.27) 478.4  (111.44) 280.8  (148.53) 54.8  (61.85)
Day 22 Number Analyzed 11 participants 7 participants 6 participants 17 participants
1317.1  (728.48) 382.4  (82.58) 284.8  (324.16) 35.3  (51.45)
Day 29 Number Analyzed 11 participants 7 participants 6 participants 17 participants
1214.0  (676.80) 372.3  (79.97) 179.0  (137.39) 24.8  (27.24)
Day 33 Number Analyzed 11 participants 7 participants 6 participants 18 participants
1141.4  (606.99) 291.1  (77.55) 115.7  (84.49) 24.3  (30.64)
Week 12 Number Analyzed 10 participants 6 participants 4 participants 18 participants
1366.5  (631.28) 307.8  (97.84) 110.8  (49.80) 16.2  (11.60)
Week 24 Number Analyzed 11 participants 7 participants 6 participants 18 participants
1194.0  (599.59) 297.1  (74.62) 160.8  (122.75) 23.4  (25.34)
13.Secondary Outcome
Title Percent Inhibition of Free Myostatin
Hide 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.
Time Frame Baseline through week 24
Hide Outcome Measure Data
Hide Analysis Population Description
The pharmacodynamics (PD) data set includes all available data from subjects for whom PD measurements are available at baseline and at least one other timepoint.
Arm/Group Title Placebo Panel 1 RO7239361 Panel 2 RO7239361 Panel 3 and Expansion Panel RO7239361
Hide Arm/Group Description:
Placebo subcutaneous injections on specified days
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).
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
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
Overall Number of Participants Analyzed 11 7 6 19
Mean (Standard Deviation)
Unit of Measure: Percent
Day 4 Number Analyzed 8 participants 5 participants 4 participants 18 participants
0.595  (23.6952) 49.689  (11.7394) 73.533  (9.8637) 95.334  (2.4477)
Day 5 Number Analyzed 9 participants 7 participants 6 participants 18 participants
3.114  (20.3395) 46.506  (9.0917) 69.700  (10.3411) 94.545  (2.7608)
Day 8 Number Analyzed 10 participants 6 participants 6 participants 19 participants
23.917  (31.1378) 38.411  (7.0320) 62.524  (9.7074) 84.797  (22.1958)
Day 15 Number Analyzed 11 participants 7 participants 6 participants 19 participants
5.744  (18.6190) 40.763  (10.0071) 77.090  (6.2939) 96.488  (1.9665)
Day 22 Number Analyzed 11 participants 7 participants 6 participants 17 participants
5.561  (11.7425) 51.836  (9.3488) 81.693  (5.7392) 97.927  (1.1259)
Day 29 Number Analyzed 11 participants 7 participants 6 participants 17 participants
12.599  (16.1591) 52.926  (6.6001) 86.894  (2.9590) 98.202  (1.4590)
Day 33 Number Analyzed 11 participants 7 participants 6 participants 18 participants
15.662  (17.3640) 61.796  (4.5760) 91.557  (2.6444) 97.042  (5.4443)
Week 12 Number Analyzed 10 participants 6 participants 4 participants 18 participants
5.289  (15.5082) 60.254  (9.4283) 92.454  (1.7243) 98.415  (1.2882)
Week 24 Number Analyzed 11 participants 7 participants 6 participants 18 participants
9.910  (17.8109) 63.790  (5.7031) 88.392  (5.1669) 97.921  (2.2143)
14.Secondary Outcome
Title Serum Concentration of Drug-myostatin Complex
Hide 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.
Time Frame Baseline through week 24
Hide Outcome Measure Data
Hide Analysis Population Description
The pharmacodynamics (PD) data set includes all available data from subjects for whom PD measurements are available at baseline and at least one other timepoint.
Arm/Group Title Placebo Panel 1 RO7239361 Panel 2 RO7239361 Panel 3 and Expansion Panel RO7239361
Hide Arm/Group Description:
Placebo subcutaneous injections on specified days
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).
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
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
Overall Number of Participants Analyzed 11 7 6 19
Mean (Standard Deviation)
Unit of Measure: ng/mL
Day 4 Number Analyzed 9 participants 3 participants 4 participants 6 participants
0.750  (0.9061) 2.147  (0.4356) 3.460  (1.1139) 4.116  (0.6420)
Day 5 Number Analyzed 8 participants 5 participants 6 participants 7 participants
1.084  (1.3095) 1.984  (0.5813) 3.782  (0.8241) 4.990  (1.1641)
Day 8 Number Analyzed 10 participants 6 participants 6 participants 7 participants
1.008  (2.0898) 2.562  (0.7098) 5.032  (0.8312) 5.459  (2.3499)
Day 15 Number Analyzed 11 participants 7 participants 6 participants 6 participants
0.275  (0.1718) 3.600  (0.8242) 8.252  (2.8423) 11.174  (2.8970)
Day 22 Number Analyzed 10 participants 7 participants 6 participants 7 participants
0.557  (0.3906) 4.536  (1.5801) 8.243  (4.7497) 13.305  (4.2587)
Day 29 Number Analyzed 11 participants 7 participants 6 participants 7 participants
0.602  (0.7697) 5.249  (1.2965) 9.088  (4.8965) 15.817  (4.3606)
Day 33 Number Analyzed 9 participants 7 participants 4 participants 18 participants
0.372  (0.2090) 5.849  (1.5351) 12.838  (7.3075) 18.093  (5.8485)
Week 12 Number Analyzed 10 participants 6 participants 5 participants 14 participants
13.416  (34.0016) 9.252  (4.0709) 15.894  (4.9462) 23.339  (5.0080)
Week 24 Number Analyzed 8 participants 7 participants 5 participants 7 participants
0.746  (1.2056) 7.074  (1.3202) 11.990  (4.3473) 21.303  (5.4669)
15.Secondary Outcome
Title Fold Change From Baseline in Contractile Versus Non-contractile Content for Muscles in the Right Thigh
Hide 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.

Time Frame Baseline through Week 24
Hide Outcome Measure Data
Hide Analysis Population Description
The pharmacodynamics (PD) data set includes all available data from subjects for whom PD measurements are available at baseline and at least one other timepoint.
Arm/Group Title Placebo Panel 1 RO7239361 Panel 2 RO7239361 Panel 3 RO7239361 Expansion Panel RO7239361
Hide Arm/Group Description:
Placebo subcutaneous injections on specified days
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).
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
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
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
Overall Number of Participants Analyzed 11 7 6 6 13
Mean (Standard Error)
Unit of Measure: Fold Change
W12: ADDUCTOR LONGUS Number Analyzed 7 participants 3 participants 5 participants 4 participants 11 participants
1.04  (0.291) 0.95  (0.088) 1.16  (0.330) 2.27  (1.122) 2.36  (0.875)
W12: ADDUCTOR MAGNUS Number Analyzed 8 participants 5 participants 4 participants 5 participants 11 participants
0.69  (0.104) 0.88  (0.283) 1.12  (0.281) 0.93  (0.255) 1.04  (0.101)
W12: BICEPS FEMORIS LONG Number Analyzed 7 participants 4 participants 6 participants 4 participants 11 participants
0.78  (0.221) 0.94  (0.304) 1.57  (0.556) 1.10  (0.280) 1.26  (0.237)
W12: BICEPS FEMORIS SHORT Number Analyzed 7 participants 4 participants 4 participants 5 participants 11 participants
0.92  (0.269) 2.77  (1.144) 2.03  (1.564) 0.79  (0.162) 1.31  (0.301)
W12: GRACILIS Number Analyzed 6 participants 4 participants 4 participants 4 participants 11 participants
1.13  (0.318) 7.57  (5.372) 0.98  (0.176) 3.41  (2.474) 5.42  (3.871)
W12:RECTUS FEMORIS Number Analyzed 7 participants 6 participants 6 participants 4 participants 11 participants
0.74  (0.185) 0.55  (0.134) 4.49  (2.729) 0.99  (0.345) 2.02  (0.764)
W12: SARTORIUS Number Analyzed 6 participants 4 participants 5 participants 4 participants 10 participants
1.78  (0.771) 0.69  (0.307) 1.04  (0.443) 0.90  (0.396) 2.00  (0.683)
W12: SEMIMEMBRANOSUS Number Analyzed 8 participants 5 participants 6 participants 6 participants 13 participants
0.82  (0.230) 0.82  (0.284) 1.47  (0.777) 0.86  (0.276) 1.31  (0.194)
W12: SEMITENDINOSUS Number Analyzed 8 participants 4 participants 5 participants 4 participants 11 participants
0.78  (0.262) 2.95  (1.016) 1.31  (0.260) 1.42  (0.317) 1.17  (0.160)
W12: VASTUS INTERMEDIUS Number Analyzed 7 participants 3 participants 4 participants 4 participants 10 participants
2.59  (1.749) 1.11  (0.292) 0.62  (0.258) 0.86  (0.157) 1.64  (0.377)
W12: VASTUS LATERALIS Number Analyzed 7 participants 5 participants 6 participants 5 participants 12 participants
0.66  (0.160) 0.89  (0.272) 1.10  (0.333) 1.78  (0.455) 1.70  (0.334)
W12: VASTUS MEDIALIS Number Analyzed 6 participants 4 participants 6 participants 4 participants 12 participants
1.08  (0.269) 1.26  (0.112) 2.97  (1.981) 0.85  (0.280) 5.96  (3.376)
W24: ADDUCTOR LONGUS Number Analyzed 9 participants 4 participants 4 participants 5 participants 11 participants
0.81  (0.290) 1.14  (0.269) 1.11  (0.471) 1.36  (0.718) 2.41  (0.813)
W24: ADDUCTOR MAGNUS Number Analyzed 10 participants 6 participants 4 participants 5 participants 11 participants
0.66  (0.112) 0.94  (0.296) 0.59  (0.144) 1.61  (0.697) 1.14  (0.146)
W24: BICEPS FEMORIS LONG Number Analyzed 8 participants 5 participants 5 participants 4 participants 12 participants
1.07  (0.511) 1.13  (0.315) 1.00  (0.349) 1.29  (0.238) 2.10  (1.044)
W24: BICEPS FEMORIS SHORT Number Analyzed 7 participants 5 participants 4 participants 5 participants 12 participants
0.91  (0.195) 2.24  (1.027) 2.15  (1.003) 1.24  (0.411) 2.02  (0.416)
W24: GRACILIS Number Analyzed 9 participants 3 participants 3 participants 4 participants 9 participants
1.43  (0.686) 3.03  (2.564) 1.37  (0.465) 1.45  (0.748) 5.17  (3.540)
W24:RECTUS FEMORIS Number Analyzed 8 participants 6 participants 5 participants 4 participants 11 participants
0.94  (0.256) 0.84  (0.220) 7.43  (6.216) 0.80  (0.262) 3.01  (1.612)
W24: SARTORIUS Number Analyzed 7 participants 5 participants 4 participants 4 participants 10 participants
1.90  (0.872) 0.73  (0.207) 0.78  (0.375) 3.24  (1.850) 2.45  (0.842)
W24: SEMIMEMBRANOSUS Number Analyzed 10 participants 6 participants 5 participants 4 participants 12 participants
0.66  (0.126) 0.90  (0.361) 0.72  (0.157) 0.94  (0.250) 1.39  (0.256)
W24: SEMITENDINOSUS Number Analyzed 9 participants 5 participants 4 participants 6 participants 11 participants
0.56  (0.119) 1.59  (0.520) 1.43  (0.524) 1.04  (0.254) 1.22  (0.221)
W24: VASTUS INTERMEDIUS Number Analyzed 7 participants 4 participants 3 participants 4 participants 10 participants
0.95  (0.412) 1.47  (0.446) 0.63  (0.355) 0.96  (0.095) 1.47  (0.318)
W24: VASTUS LATERALIS Number Analyzed 10 participants 5 participants 5 participants 4 participants 12 participants
0.78  (0.159) 1.88  (0.905) 1.45  (0.596) 1.43  (0.256) 2.03  (0.484)
W24: VASTUS MEDIALIS Number Analyzed 8 participants 5 participants 4 participants 4 participants 12 participants
2.14  (0.889) 5.16  (3.580) 6.01  (5.478) 1.29  (0.474) 3.50  (1.390)
16.Secondary Outcome
Title Change From Baseline in Thigh Muscle Maximal Cross Sectional Area (CSAmax)
Hide 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.

Time Frame Baseline through Week 24
Hide Outcome Measure Data
Hide Analysis Population Description
The pharmacodynamics (PD) data set includes all available data from subjects for whom PD measurements are available at baseline and at least one other timepoint.
Arm/Group Title Placebo Panel 1 RO7239361 Panel 2 RO7239361 Panel 3RO7239361 Expansion Panel RO7239361
Hide Arm/Group Description:
Placebo subcutaneous injections on specified days
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).
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
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
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
Overall Number of Participants Analyzed 11 7 6 6 13
Mean (Standard Error)
Unit of Measure: Percentage Change
W12: ADDUCTOR LONGUS Number Analyzed 9 participants 6 participants 6 participants 6 participants 12 participants
7.29  (16.559) 8.31  (11.767) 5.85  (11.074) 14.61  (9.755) 29.98  (8.530)
W12: ADDUCTOR MAGNUS Number Analyzed 9 participants 6 participants 6 participants 6 participants 12 participants
31.01  (24.708) 24.12  (11.804) -3.73  (29.037) 53.20  (25.328) 4.01  (54.787)
W12: BICEPS FEMORIS LONG Number Analyzed 9 participants 6 participants 6 participants 6 participants 12 participants
34.24  (18.328) 33.43  (35.009) 16.72  (17.806) 19.22  (9.802) 23.93  (11.289)
W12: BICEPS FEMORIS SHORT Number Analyzed 9 participants 6 participants 6 participants 6 participants 12 participants
14.10  (7.724) 7.45  (11.592) 2.69  (13.733) 18.68  (7.479) 9.84  (3.882)
W12: GRACILIS Number Analyzed 9 participants 6 participants 6 participants 6 participants 12 participants
20.14  (15.489) 43.00  (28.539) 15.28  (8.693) 27.02  (5.686) 23.22  (5.440)
W12:RECTUS FEMORIS Number Analyzed 9 participants 6 participants 6 participants 6 participants 12 participants
-7.86  (14.391) 10.33  (11.927) -3.56  (14.175) 34.91  (6.037) 16.01  (9.888)
W12: SARTORIUS Number Analyzed 9 participants 6 participants 6 participants 6 participants 12 participants
11.26  (6.584) 6.59  (11.952) -0.85  (6.383) 10.81  (11.017) 18.20  (5.519)
W12: SEMIMEMBRANOSUS Number Analyzed 9 participants 6 participants 6 participants 6 participants 12 participants
39.11  (15.041) 32.57  (19.133) 16.56  (19.998) 46.70  (16.480) 31.01  (10.564)
W12: SEMITENDINOSUS Number Analyzed 9 participants 6 participants 6 participants 6 participants 12 participants
25.03  (9.409) -1.24  (10.973) 33.00  (26.730) 36.69  (14.802) 40.10  (10.180)
W12: VASTUS INTERMEDIUS Number Analyzed 9 participants 6 participants 6 participants 6 participants 12 participants
10.47  (12.160) 23.35  (12.704) -52.03  (71.421) -0.81  (19.353) 10.44  (7.344)
W12: VASTUS LATERALIS Number Analyzed 9 participants 6 participants 6 participants 6 participants 12 participants
31.47  (39.343) 38.32  (19.570) 14.08  (27.902) 57.86  (34.026) 22.73  (15.138)
W12: VASTUS MEDIALIS Number Analyzed 9 participants 6 participants 6 participants 6 participants 12 participants
-17.89  (19.358) 15.65  (8.463) -11.60  (15.849) 21.14  (9.746) 17.89  (6.788)
W24: ADDUCTOR LONGUS Number Analyzed 11 participants 7 participants 5 participants 6 participants 12 participants
11.69  (15.626) 23.41  (13.199) 12.81  (16.393) 17.15  (15.484) 44.12  (13.323)
W24: ADDUCTOR MAGNUS Number Analyzed 11 participants 7 participants 5 participants 6 participants 12 participants
34.17  (24.918) 21.90  (12.345) -6.71  (37.311) 45.05  (27.520) 4.41  (52.393)
W24: BICEPS FEMORIS LONG Number Analyzed 11 participants 7 participants 5 participants 6 participants 12 participants
39.21  (19.760) 42.84  (22.086) -5.32  (29.140) 33.70  (12.073) 31.33  (13.370)
W24: BICEPS FEMORIS SHORT Number Analyzed 11 participants 7 participants 5 participants 6 participants 12 participants
20.09  (9.818) 11.83  (13.013) -0.44  (16.183) 30.01  (13.494) 18.60  (5.394)
W24: GRACILIS Number Analyzed 11 participants 7 participants 5 participants 6 participants 12 participants
8.14  (7.148) 18.72  (16.757) 11.21  (5.461) 48.95  (17.064) 40.15  (8.382)
W24:RECTUS FEMORIS Number Analyzed 11 participants 7 participants 5 participants 6 participants 12 participants
8.97  (10.214) 7.68  (13.082) -10.01  (16.121) 55.65  (27.535) 38.40  (14.293)
W24: SARTORIUS Number Analyzed 11 participants 7 participants 5 participants 6 participants 12 participants
10.75  (7.136) 0.81  (8.143) -14.18  (9.481) 16.63  (4.476) 25.51  (6.404)
W24: SEMIMEMBRANOSUS Number Analyzed 11 participants 7 participants 5 participants 6 participants 12 participants
45.83  (14.896) 30.01  (18.631) -11.48  (24.468) 61.03  (25.524) 49.49  (12.011)
W24: SEMITENDINOSUS Number Analyzed 11 participants 7 participants 5 participants 6 participants 12 participants
32.00  (16.102) 36.17  (23.285) 24.16  (23.403) 73.68  (17.594) 62.55  (11.073)
W24: VASTUS INTERMEDIUS Number Analyzed 11 participants 7 participants 5 participants 6 participants 12 participants
14.86  (15.072) 43.01  (11.506) -68.05  (86.362) 41.63  (34.926) 22.55  (8.284)
W24: VASTUS LATERALIS Number Analyzed 11 participants 7 participants 5 participants 6 participants 12 participants
12.95  (35.465) 47.79  (26.178) 32.64  (30.136) 45.41  (43.774) 46.87  (20.919)
W24: VASTUS MEDIALIS Number Analyzed 11 participants 7 participants 5 participants 6 participants 12 participants
-9.09  (11.856) 9.82  (6.965) -13.74  (29.664) 10.46  (12.093) 23.94  (11.321)
Time Frame Baseline up to 26 months
Adverse Event Reporting 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.
 
Arm/Group Title 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
Hide Arm/Group Description Placebo subcutaneous injections on specified days RO7239361 subcutaneous injections on specified days RO7239361 subcutaneous injections on specified days RO7239361 subcutaneous injections on specified days RO7239361 subcutaneous injections on specified days RO7239361 subcutaneous injections on specified days RO7239361 subcutaneous injections on specified days
All-Cause Mortality
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
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   0/11 (0.00%)   0/7 (0.00%)   0/6 (0.00%)   0/19 (0.00%)   0/9 (0.00%)   0/8 (0.00%)   0/26 (0.00%) 
Hide 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
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   1/11 (9.09%)   0/7 (0.00%)   0/6 (0.00%)   1/19 (5.26%)   0/9 (0.00%)   3/8 (37.50%)   1/26 (3.85%) 
Infections and infestations               
INFLUENZA * 1  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%) 
Injury, poisoning and procedural complications               
FEMUR FRACTURE * 1  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%) 
SPINAL COMPRESSION FRACTURE * 1  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%) 
SKULL FRACTURE * 1  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%) 
Musculoskeletal and connective tissue disorders               
RHABDOMYOLYSIS * 1  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%) 
Psychiatric disorders               
CONVERSION DISORDER * 1  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%) 
1
Term from vocabulary, MedDRA 20.1
*
Indicates events were collected by non-systematic assessment
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
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
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   9/11 (81.82%)   5/7 (71.43%)   6/6 (100.00%)   18/19 (94.74%)   9/9 (100.00%)   8/8 (100.00%)   25/26 (96.15%) 
Cardiac disorders               
SINUS TACHYCARDIA * 1  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%) 
Congenital, familial and genetic disorders               
DERMOID CYST * 1  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%) 
Ear and labyrinth disorders               
EAR PAIN * 1  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%) 
TYMPANIC MEMBRANE PERFORATION * 1  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%) 
Gastrointestinal disorders               
DIARRHOEA * 1  2/11 (18.18%)  1/7 (14.29%)  1/6 (16.67%)  2/19 (10.53%)  1/9 (11.11%)  1/8 (12.50%)  6/26 (23.08%) 
VOMITING * 1  1/11 (9.09%)  1/7 (14.29%)  0/6 (0.00%)  4/19 (21.05%)  3/9 (33.33%)  0/8 (0.00%)  3/26 (11.54%) 
ABDOMINAL PAIN UPPER * 1  1/11 (9.09%)  1/7 (14.29%)  0/6 (0.00%)  2/19 (10.53%)  1/9 (11.11%)  0/8 (0.00%)  2/26 (7.69%) 
ABDOMINAL PAIN * 1  1/11 (9.09%)  0/7 (0.00%)  0/6 (0.00%)  1/19 (5.26%)  0/9 (0.00%)  0/8 (0.00%)  0/26 (0.00%) 
ABDOMINAL DISTENSION * 1  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%) 
BREATH ODOUR * 1  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%) 
CONSTIPATION * 1  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%) 
DYSPEPSIA * 1  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%) 
LIP SWELLING * 1  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%) 
MOUTH ULCERATION * 1  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%) 
NAUSEA * 1  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%) 
GASTRIC ULCER * 1  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%) 
General disorders               
PYREXIA * 1  3/11 (27.27%)  0/7 (0.00%)  1/6 (16.67%)  4/19 (21.05%)  1/9 (11.11%)  1/8 (12.50%)  3/26 (11.54%) 
INJECTION SITE BRUISING * 1  1/11 (9.09%)  0/7 (0.00%)  2/6 (33.33%)  3/19 (15.79%)  1/9 (11.11%)  0/8 (0.00%)  5/26 (19.23%) 
INJECTION SITE RASH * 1  1/11 (9.09%)  1/7 (14.29%)  1/6 (16.67%)  0/19 (0.00%)  0/9 (0.00%)  1/8 (12.50%)  0/26 (0.00%) 
CHILLS * 1  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%) 
INJECTION SITE HAEMORRHAGE * 1  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%) 
INJECTION SITE IRRITATION * 1  0/11 (0.00%)  1/7 (14.29%)  0/6 (0.00%)  0/19 (0.00%)  4/9 (44.44%)  0/8 (0.00%)  0/26 (0.00%) 
PERIPHERAL SWELLING * 1  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%) 
INJECTION SITE PAIN * 1  0/11 (0.00%)  0/7 (0.00%)  0/6 (0.00%)  2/19 (10.53%)  0/9 (0.00%)  1/8 (12.50%)  0/26 (0.00%) 
FATIGUE * 1  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%) 
INJECTION SITE DISCOMFORT * 1  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%) 
INJECTION SITE ERYTHEMA * 1  0/11 (0.00%)  0/7 (0.00%)  0/6 (0.00%)  1/19 (5.26%)  1/9 (11.11%)  0/8 (0.00%)  5/26 (19.23%) 
INJECTION SITE SWELLING * 1  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%) 
INJECTION SITE REACTION * 1  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%) 
Immune system disorders               
SEASONAL ALLERGY * 1  0/11 (0.00%)  0/7 (0.00%)  0/6 (0.00%)  0/19 (0.00%)  0/9 (0.00%)  1/8 (12.50%)  1/26 (3.85%) 
Infections and infestations               
UPPER RESPIRATORY TRACT INFECTION * 1  2/11 (18.18%)  0/7 (0.00%)  2/6 (33.33%)  4/19 (21.05%)  0/9 (0.00%)  1/8 (12.50%)  4/26 (15.38%) 
NASOPHARYNGITIS * 1  1/11 (9.09%)  1/7 (14.29%)  1/6 (16.67%)  4/19 (21.05%)  2/9 (22.22%)  1/8 (12.50%)  6/26 (23.08%) 
EAR INFECTION * 1  1/11 (9.09%)  0/7 (0.00%)  1/6 (16.67%)  0/19 (0.00%)  2/9 (22.22%)  1/8 (12.50%)  1/26 (3.85%) 
PHARYNGITIS STREPTOCOCCAL * 1  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%) 
SINUSITIS * 1  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%) 
NAIL INFECTION * 1  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%) 
INFLUENZA * 1  0/11 (0.00%)  0/7 (0.00%)  0/6 (0.00%)  0/19 (0.00%)  1/9 (11.11%)  0/8 (0.00%)  2/26 (7.69%) 
PHARYNGITIS * 1  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%) 
GASTROENTERITIS VIRAL * 1  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%) 
Injury, poisoning and procedural complications               
ARTHROPOD BITE * 1  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%) 
CONTUSION * 1  1/11 (9.09%)  1/7 (14.29%)  0/6 (0.00%)  0/19 (0.00%)  3/9 (33.33%)  0/8 (0.00%)  3/26 (11.54%) 
FALL * 1  0/11 (0.00%)  1/7 (14.29%)  0/6 (0.00%)  1/19 (5.26%)  4/9 (44.44%)  0/8 (0.00%)  2/26 (7.69%) 
LIGAMENT SPRAIN * 1  0/11 (0.00%)  1/7 (14.29%)  0/6 (0.00%)  1/19 (5.26%)  0/9 (0.00%)  1/8 (12.50%)  2/26 (7.69%) 
SKIN ABRASION * 1  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%) 
EYELID CONTUSION * 1  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%) 
FOOT FRACTURE * 1  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%) 
POST-TRAUMATIC PAIN * 1  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%) 
THERMAL BURN * 1  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%) 
EYE CONTUSION * 1  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%) 
HEAD INJURY * 1  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%) 
UPPER LIMB FRACTURE * 1  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%) 
Investigations               
BLOOD CREATINE PHOSPHOKINASE INCREASED * 1  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%) 
LYMPH NODE PALPABLE * 1  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%) 
Metabolism and nutrition disorders               
DECREASED APPETITE * 1  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%) 
Musculoskeletal and connective tissue disorders               
PAIN IN EXTREMITY * 1  0/11 (0.00%)  1/7 (14.29%)  2/6 (33.33%)  0/19 (0.00%)  1/9 (11.11%)  0/8 (0.00%)  1/26 (3.85%) 
BACK PAIN * 1  1/11 (9.09%)  0/7 (0.00%)  0/6 (0.00%)  1/19 (5.26%)  2/9 (22.22%)  0/8 (0.00%)  1/26 (3.85%) 
JOINT SWELLING * 1  0/11 (0.00%)  1/7 (14.29%)  0/6 (0.00%)  1/19 (5.26%)  0/9 (0.00%)  0/8 (0.00%)  0/26 (0.00%) 
MUSCLE SPASMS * 1  1/11 (9.09%)  0/7 (0.00%)  1/6 (16.67%)  0/19 (0.00%)  0/9 (0.00%)  0/8 (0.00%)  1/26 (3.85%) 
NECK PAIN * 1  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%) 
ARTHRALGIA * 1  0/11 (0.00%)  0/7 (0.00%)  0/6 (0.00%)  1/19 (5.26%)  2/9 (22.22%)  0/8 (0.00%)  1/26 (3.85%) 
MYALGIA * 1  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%) 
JOINT CONTRACTURE * 1  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%) 
OSTEOPOROSIS * 1  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%) 
MUSCULAR WEAKNESS * 1  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%) 
Nervous system disorders               
HEADACHE * 1  2/11 (18.18%)  2/7 (28.57%)  0/6 (0.00%)  7/19 (36.84%)  2/9 (22.22%)  0/8 (0.00%)  3/26 (11.54%) 
DIZZINESS * 1  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%) 
MOTOR DYSFUNCTION * 1  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%) 
SINUS HEADACHE * 1  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%) 
Psychiatric disorders               
BRUXISM * 1  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%) 
Renal and urinary disorders               
POLYURIA * 1  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%) 
Respiratory, thoracic and mediastinal disorders               
COUGH * 1  2/11 (18.18%)  0/7 (0.00%)  0/6 (0.00%)  2/19 (10.53%)  1/9 (11.11%)  4/8 (50.00%)  4/26 (15.38%) 
EPISTAXIS * 1  1/11 (9.09%)  0/7 (0.00%)  1/6 (16.67%)  1/19 (5.26%)  0/9 (0.00%)  0/8 (0.00%)  1/26 (3.85%) 
NASAL CONGESTION * 1  1/11 (9.09%)  0/7 (0.00%)  0/6 (0.00%)  1/19 (5.26%)  1/9 (11.11%)  0/8 (0.00%)  4/26 (15.38%) 
PRODUCTIVE COUGH * 1  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%) 
ASTHMA * 1  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%) 
HICCUPS * 1  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%) 
NASAL DISCHARGE DISCOLOURATION * 1  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%) 
PARANASAL SINUS HYPERSECRETION * 1  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%) 
OROPHARYNGEAL PAIN * 1  0/11 (0.00%)  0/7 (0.00%)  0/6 (0.00%)  2/19 (10.53%)  0/9 (0.00%)  0/8 (0.00%)  2/26 (7.69%) 
RHINORRHOEA * 1  0/11 (0.00%)  0/7 (0.00%)  0/6 (0.00%)  2/19 (10.53%)  0/9 (0.00%)  0/8 (0.00%)  1/26 (3.85%) 
BRONCHOSPASM * 1  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%) 
Skin and subcutaneous tissue disorders               
RASH * 1  0/11 (0.00%)  0/7 (0.00%)  3/6 (50.00%)  1/19 (5.26%)  0/9 (0.00%)  0/8 (0.00%)  1/26 (3.85%) 
DERMATITIS CONTACT * 1  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%) 
SKIN ULCER * 1  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%) 
DRY SKIN * 1  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%) 
ERYTHEMA * 1  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%) 
INGROWING NAIL * 1  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%) 
Vascular disorders               
FLUSHING * 1  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%) 
HAEMATOMA * 1  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%) 
1
Term from vocabulary, MedDRA 20.1
*
Indicates events were collected by non-systematic assessment
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
The Study being conducted under this Agreement is part of the Overall Study. Investigator is free to publish in reputable journals or to present at professional conferences the results of the Study, but only after the first publication or presentation that involves the Overall Study. The Sponsor may request that Confidential Information be deleted and/or the publication be postponed in order to protect the Sponsor's intellectual property rights.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Medical Communications
Organization: Hoffmann- LaRoche
Phone: 1-800-821-8590
EMail: genentech@druginfo.com
Layout table for additonal information
Responsible Party: Hoffmann-La Roche
ClinicalTrials.gov Identifier: NCT02515669    
Other Study ID Numbers: CN001-006
WN40226 ( Other Identifier: Hoffman-La Roche )
First Submitted: July 29, 2015
First Posted: August 5, 2015
Results First Submitted: February 7, 2019
Results First Posted: May 8, 2019
Last Update Posted: June 9, 2020