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

A Randomized, Double Blind, Placebo-Controlled, Study to Assess the Efficacy, Safety, and Tolerability of RO7239361 in Ambulatory Boys with Duchenne Muscular Dystrophy

Status

Active, not recruiting

Phases

Phase 3

Study type

Interventional

Source

JPRN

Registry ID

JPRN-jRCT2080223587

Enrollment

159

Registered

2017-07-13

Start date

2017-11-30

Completion date

Unknown

Last updated

2026-06-29

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Duchenne Muscular Dystrophy

Interventions

investigational material(s) Generic name etc : RO7239361, dose 1 INN of investigational material : - Therapeutic category code : 49- Other agents affecting cellular function Dosage and Administration

Eligibility

Sex/Gender

Male

Inclusion criteria

Inclusion criteria: Diagnosed with DMD by a blood test Able to walk without assistance Able to walk up 4 stairs in 8 seconds or less Weigh at least 15 kg (33 lbs) Taking corticosteroids for DMD Other protocol defined Inclusion/Exclusion Criteria could apply.

Exclusion criteria

Exclusion criteria: Any behavior or mental issue that will affect the ability to complete the required study procedures Previously or currently taking medications like androgens or human growth hormone Use of a ventilator during the day Unable to have blood samples collected or receive an injection under the skin Other protocol defined Inclusion/Exclusion Criteria could apply.

Design outcomes

Primary

Measure	Time frame
efficacy The change from baseline in the North Star Ambulatory Assessment (NSAA) total score at Week 48 in RO723936-treated participants compared to placebo-treated participants.	—

Secondary

Measure	Time frame
efficacy pharmacokinetics pharmacodynamics safety Change from baseline at Week 48 in RO7239361-treated participants compared to placebo-treated participants in the following - 4 stair climb velocity (4SCV) - Stand from supine velocity - 10 M walk/run velocity - PODCI transfers and basic mobility subscale - Proximal lower extremity flexor (knee extension and knee flexion) strength, measured usingmanual myometry - 6-Minute Walk Distance (6MWD) - Clinical Global Impression of Change (CGI-C) - 95th percentile stride velocity, as recorded with ActiMyo in a subset of the overall study population Tabulations of the numbers of unique participants with new or worsening laboratory abnormalities, SAEs and AEs leading to discontinuation, in RO7239361 arms compared to the placebo arm	—

Measure

Time frame

efficacy pharmacokinetics pharmacodynamics safety Change from baseline at Week 48 in RO7239361-treated participants compared to placebo-treated participants in the following - 4 stair climb velocity (4SCV) - Stand from supine velocity - 10 M walk/run velocity - PODCI transfers and basic mobility subscale - Proximal lower extremity flexor (knee extension and knee flexion) strength, measured usingmanual myometry - 6-Minute Walk Distance (6MWD) - Clinical Global Impression of Change (CGI-C) - 95th percentile stride velocity, as recorded with ActiMyo in a subset of the overall study population Tabulations of the numbers of unique participants with new or worsening laboratory abnormalities, SAEs and AEs leading to discontinuation, in RO7239361 arms compared to the placebo arm

—

Countries

Europe, Japan, North America, Oceania, South America

Outcome results

None listed

Source: JPRN (via WHO ICTRP) · Data processed: Jul 3, 2026