Trial · NCT03039686

Clinical Trial to Evaluate the Efficacy, Safety, and Tolerability of RO7239361 in Ambulatory Boys With Duchenne Muscular Dystrophy

NCT03039686, Phase 2, Phase 3, Completed, Hoffmann-La Roche; results first posted 2020-12-21

3 sourcestrials-2026-11-12-0.5.0

Cite this record

ConductScience Library. Clinical Trial to Evaluate the Efficacy, Safety, and Tolerability of RO7239361 in Ambulatory Boys With Duchenne Muscular Dystrophy. Trials, record NCT03039686. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03039686

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Trial

As registered on ClinicalTrials.gov, in the graph's capture of the registry.

Title: Clinical Trial to Evaluate the Efficacy, Safety, and Tolerability of RO7239361 in Ambulatory Boys With Duchenne Muscular Dystrophy
NCT number: NCT03039686
Status: Completed
Phases: Phase 2, Phase 3
Enrollment: 166 participants, actual
Lead sponsor: Hoffmann-La Roche
Conditions: Duchenne Muscular Dystrophy
Interventions: RO7239361, Placebo for RO7239361
First posted: 2017-02-01
Last updated: 2020-12-21
Other registries' IDs: eudract:2016-001654-18
Results first posted: 2020-12-21

Source ClinicalTrials.gov

Registered outcomes17

Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.

List	Outcome	Time frame	Description
Primary	Baseline for the North Star Ambulatory Assessment (NSAA) Total Score	Baseline	The NSAA is a functional scale specifically designed for ambulant boys with Duchenne muscular dystrophy (DMD) that can provide information about motor function. The NSAA is a 17-item test of standing, ability to transition from lying to sitting, sitting to standing, and other mobility assessments. Each of the 17 items is evaluated on an ordinal scale of 0-2: 0 = unable to achieve independently, 1 = modified method but achieves goal independent of physical assistance from another, or 2 = normal with no obvious modification of activity. Total score range is 0 to 34. Higher scores reflect better performance.
Primary	Change From Baseline in the North Star Ambulatory Assessment (NSAA) Total Score at Week 48	Baseline, Week 48	The NSAA is a functional scale specifically designed for ambulant boys with Duchenne muscular dystrophy (DMD) that can provide information about motor function. The NSAA is a 17-item test of standing, ability to transition from lying to sitting, sitting to standing, and other mobility assessments. Each of the 17 items is evaluated on an ordinal scale of 0-2: 0 = unable to achieve independently, 1 = modified method but achieves goal independent of physical assistance from another, or 2 = normal with no obvious modification of activity. Total score range is 0 to 34. Higher scores reflect better performance. A positive change from baseline indicates an improvement. Based on the mixed-effect model of repeated measures (MMRM).
Secondary	Baseline Time for 4 Stair Climb	Baseline	The time to complete the 4 stair climb was measured at baseline.
Secondary	Change From Baseline at Week 48 in 4 Stair Climb Velocity (4SCV)	Baseline, Week 48	4SCV was calculated as the ratio of the number of stairs climbed (4) divided by the number of seconds taken to complete the 4-stair climb. The results were converted into velocity (distance/time). A positive change from baseline indicates an improvement. Based on the mixed-effect model of repeated measures (MMRM).
Secondary	Baseline for the Time to Stand From Supine	Baseline	The time required for a participant to stand from supine position. A longer time reflects a worse outcome.
Secondary	Change From Baseline at Week 48 in Stand From Supine Velocity	Baseline, Week 48	The time required for a participant to stand from supine position. A longer time reflects a worse outcome. A negative change from baseline indicates an improvement. Based on the mixed-effect model of repeated measures (MMRM).
Secondary	Baseline Time for 10 Meter Walk/Run	Baseline	The time required for a participant to run or walk a distance of 10 meters as quickly as possible. A longer time reflects a worse outcome.
Secondary	Change From Baseline at Week 48 in 10 M Walk/Run Velocity	Baseline, Week 48	The time required for a participant to run or walk a distance of 10 meters as quickly as possible calculated as velocity (distance/time). A positive change from baseline indicates an improvement. Based on the mixed-effect model of repeated measures (MMRM).
Secondary	Baseline for the Pediatric Outcome Data Collection Instrument (PODCI) Transfer and Basic Mobility Subscale	Baseline	The PODCI is designed to be completed by the parent/guardian of a child who has knowledge of the child's conditions. The Transfer and Basic Mobility scale is one of the subscales of the PODCI. The results are standardized into a scale of 0-100 with a higher score reflecting better performance.
Secondary	Change From Baseline at Week 48 in Pediatric Outcome Data Collection Instrument (PODCI) Transfer and Basic Mobility Subscale	Baseline, Week 48	The PODCI is designed to be completed by the parent/guardian of a child who has knowledge of the child's conditions. The Transfer and Basic Mobility scale is one of the subscales of the PODCI. The results are standardized into a scale of 0-100 with a higher score reflecting better performance. A positive change from baseline indicates an improvement. Based on the mixed-effect model of repeated measures (MMRM).

1 to 10 of 17Source ClinicalTrials.gov

See all 17 registered outcomes

Outcomes17

Each outcome the trial posted results for, in ClinicalTrials.gov's words.

Type	Outcome	Time frame	Population	Units	Measured as
PRIMARY	Baseline for the North Star Ambulatory Assessment (NSAA) Total Score	Baseline	Intent-to-Treat (ITT) population included all enrolled participants who received a randomization treatment assignment.	score on a scale	MEAN
PRIMARY	Change From Baseline in the North Star Ambulatory Assessment (NSAA) Total Score at Week 48	Baseline, Week 48	Intent-to-Treat (ITT) population included all enrolled participants who received a randomization treatment assignment. MMRM analysis included all participants at baseline and the following number of participants by Week 48: Placebo n=30, Low Dose n=29, High Dose n=33.	score on a scale	LEAST_SQUARES_MEAN
SECONDARY	Baseline Time for 4 Stair Climb	Baseline	Intent-to-Treat (ITT) population included all enrolled participants who received a randomization treatment assignment.	seconds (secs)	MEAN
SECONDARY	Change From Baseline at Week 48 in 4 Stair Climb Velocity (4SCV)	Baseline, Week 48	Intent-to-Treat (ITT) population included all enrolled participants who received a randomization treatment assignment. MMRM analysis included all participants at baseline and the following number of participants by Week 48: Placebo n=30, Low Dose n=29, High Dose n=33.	stairs/sec	LEAST_SQUARES_MEAN
SECONDARY	Baseline for the Time to Stand From Supine	Baseline	Intent-to-Treat (ITT) population included all enrolled participants who received a randomization treatment assignment.	secs	MEAN
SECONDARY	Change From Baseline at Week 48 in Stand From Supine Velocity	Baseline, Week 48	Intent-to-Treat (ITT) population included all enrolled participants who received a randomization treatment assignment. MMRM analysis included all participants at baseline and the following number of participants by Week 48: Placebo n=28, Low Dose n=28, High Dose n=32.	1/sec	LEAST_SQUARES_MEAN
SECONDARY	Baseline Time for 10 Meter Walk/Run	Baseline	Intent-to-Treat (ITT) population included all enrolled participants who received a randomization treatment assignment.	secs	MEAN
SECONDARY	Change From Baseline at Week 48 in 10 M Walk/Run Velocity	Baseline, Week 48	Intent-to-Treat (ITT) population included all enrolled participants who received a randomization treatment assignment. MMRM analysis included all participants at baseline and the following number of participants by Week 48: Placebo n=30, Low Dose n=29, High Dose n=31.	m/sec	LEAST_SQUARES_MEAN
SECONDARY	Baseline for the Pediatric Outcome Data Collection Instrument (PODCI) Transfer and Basic Mobility Subscale	Baseline	Intent-to-Treat (ITT) population included all enrolled participants who received a randomization treatment assignment.	score on a scale	MEAN
SECONDARY	Change From Baseline at Week 48 in Pediatric Outcome Data Collection Instrument (PODCI) Transfer and Basic Mobility Subscale	Baseline, Week 48	Intent-to-Treat (ITT) population included all enrolled participants who received a randomization treatment assignment. MMRM analysis included all participants at baseline and the following number of participants by Week 48: Placebo n=31, Low Dose n=29, High Dose n=34.	score on a scale	LEAST_SQUARES_MEAN

1 to 10 of 17Source ClinicalTrials.gov

See all 17 outcomes

Results by group85

Each measurement the trial posted, group by group, in the words and numbers it posted.

Outcome	Group	Value	Spread	Units
Baseline for the North Star Ambulatory Assessment (NSAA) Total Score	Placebo	23.1	Standard Deviation 6.4	score on a scale
Baseline for the North Star Ambulatory Assessment (NSAA) Total Score	RO7239361 Low Dose	24.5	Standard Deviation 5.5	score on a scale
Baseline for the North Star Ambulatory Assessment (NSAA) Total Score	RO7239361 High Dose	22.7	Standard Deviation 6.7	score on a scale
Change From Baseline in the North Star Ambulatory Assessment (NSAA) Total Score at Week 48	Placebo	-2.99	Standard Error 0.65	score on a scale
Change From Baseline in the North Star Ambulatory Assessment (NSAA) Total Score at Week 48	RO7239361 Low Dose	-3.44	Standard Error 0.67	score on a scale
Change From Baseline in the North Star Ambulatory Assessment (NSAA) Total Score at Week 48	RO7239361 High Dose	-2.41	Standard Error 0.64	score on a scale
Baseline Time for 4 Stair Climb	Placebo	3.81	Standard Deviation 1.55	seconds (secs)
Baseline Time for 4 Stair Climb	RO7239361 Low Dose	3.85	Standard Deviation 1.61	seconds (secs)
Baseline Time for 4 Stair Climb	RO7239361 High Dose	3.92	Standard Deviation 1.91	seconds (secs)
Change From Baseline at Week 48 in 4 Stair Climb Velocity (4SCV)	Placebo	-0.15	Standard Error 0.07	stairs/sec

1 to 10 of 85Source ClinicalTrials.gov

See all 85 results by group

Participant flow21

How many started, completed or left each group, period by period.

Period	Milestone	Group	Participants
Double-blind Period	STARTED	Placebo	56
Double-blind Period	STARTED	RO7239361 Low Dose	55
Double-blind Period	STARTED	RO7239361 High Dose	55
Double-blind Period	COMPLETED	Placebo	29
Double-blind Period	COMPLETED	RO7239361 Low Dose	26
Double-blind Period	COMPLETED	RO7239361 High Dose	32
Double-blind Period	NOT COMPLETED	Placebo	27
Double-blind Period	NOT COMPLETED	RO7239361 Low Dose	29
Double-blind Period	NOT COMPLETED	RO7239361 High Dose	23
Open Label Period	STARTED	Placebo	0

1 to 10 of 21Source ClinicalTrials.gov

See all 21 participant flow

Adverse events448

Each adverse event the trial posted, group by group: the participants affected out of those at risk.

Type	Event	Organ system	Group	Affected	At risk	Events
other	Ear pain	Ear and labyrinth disorders	Placebo DB	1	56	1
other	Ear pain	Ear and labyrinth disorders	RO7239361 Low Dose DB	1	55	1
other	Ear pain	Ear and labyrinth disorders	RO7239361 High Dose DB	4	55	7
other	Ear pain	Ear and labyrinth disorders	Placebo, Then RO7239361 Low Dose OL	0	14	0
other	Ear pain	Ear and labyrinth disorders	Placebo, Then RO7239361 High Dose OL	0	13	0
other	Ear pain	Ear and labyrinth disorders	RO7239361 Low Dose, Then RO7239361 Low Dose OL	0	24	0
other	Ear pain	Ear and labyrinth disorders	RO7239361 High Dose, Then RO7239361 High Dose OL	0	29	0
other	Abdominal discomfort	Gastrointestinal disorders	Placebo DB	1	56	2
other	Abdominal discomfort	Gastrointestinal disorders	RO7239361 Low Dose DB	1	55	1
other	Abdominal discomfort	Gastrointestinal disorders	RO7239361 High Dose DB	1	55	1

1 to 10 of 448Source ClinicalTrials.gov

See all 448 adverse events

Adverse event totals21

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	Placebo DB	0	56
Total, all-cause mortality	RO7239361 Low Dose DB	0	55
Total, all-cause mortality	RO7239361 High Dose DB	1	55
Total, all-cause mortality	Placebo, Then RO7239361 Low Dose OL	0	14
Total, all-cause mortality	Placebo, Then RO7239361 High Dose OL	0	13
Total, all-cause mortality	RO7239361 Low Dose, Then RO7239361 Low Dose OL	0	24
Total, all-cause mortality	RO7239361 High Dose, Then RO7239361 High Dose OL	0	29
Total, other adverse events	Placebo DB	43	56
Total, other adverse events	RO7239361 Low Dose DB	43	55
Total, other adverse events	RO7239361 High Dose DB	47	55

1 to 10 of 21Source ClinicalTrials.gov

See all 21 adverse event totals

Papers1

The papers the trial lists on ClinicalTrials.gov.

PMID	Listed as
38190001	DERIVED

Source PubMed

Grants0

The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.

None in this release.

Trial comparisons0

Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.

None in this release.

Tests0

The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.

None in this release.

Data dates

The day each source was copied for this release.

ClinicalTrials.gov: 2026-11-12; ClinicalTrials.gov
Results, from AACT: 2026-03-23; AACT
NIH grant links: 2026-11-13; NIH RePORTER

Sources 3

ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
ClinicalTrials.govNo license stated
NIH RePORTER's clinical studies and project filesPublic domain

Each source's terms

ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
ClinicalTrials.govnone stated
NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)

ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.

For software

Read this trial from software.

The record and each of its lists are JSON, twenty rows a page. No key.

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