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RO7239361 · 1 trial · 1 indication
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.
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).
| Arm | Type | Description |
|---|---|---|
| RO7239361 Low Dose | EXPERIMENTAL | Participants received low dose RO7239361 SC on specified days of the 48-week DB period. Following the DB period participants received low dose RO7239361 on specified days for up to 192 weeks during the open-label period followed by 24 weeks of follow-up. |
| RO7239361 High Dose | EXPERIMENTAL | Participants received high dose RO7239361 SC on specified days of the 48-week DB period. Following the DB period participants received high dose RO7239361 on specified days for up to 192 weeks during the open-label period followed by 24 weeks of follow-up. |
| Placebo | PLACEBO_COMPARATOR | Participants received matching placebo solution subcutaneously (SC) on specified days of the 48-week double-blind (DB) period. Following the DB period participants received low dose or high dose RO7239361 on specified days for up to 192 weeks during the open-label period followed by 24 weeks of follow-up. |
| Name | Type | Description |
|---|---|---|
| RO7239361 | DRUG | Take RO7239361 subcutaneously on specified days over a 48 week blinded period |
| Placebo for RO7239361 | DRUG | Take placebo subcutaneously on specified days over a 48 week blinded period |
Inclusion Criteria: * Diagnosed with DMD by confirmed medical history and genetic testing * Able to walk without assistance * Minimum North Star Ambulatory Assessment score of 15 at screening * Able to walk up 4 stairs in 8 seconds or less * Weigh at least 15 kg (33 lbs) * Taking corticosteroids fo...
RO7239361 is an investigational small molecule developed by Roche Holding AG for Duchenne Muscular Dystrophy, a genetic muscle-wasting disorder. It was studied in a Phase 2 clinical trial in ambulatory boys with the condition. It is not an approved therapy and remains in clinical development.
RO7239361 was developed as a treatment for Duchenne Muscular Dystrophy, a neuromuscular disorder that causes progressive muscle weakness. The Phase 2 program enrolled ambulatory boys aged 6 years and older with the condition. The trial has been completed, and the drug is not approved for any use.
RO7239361 is developed by Roche Holding AG, the Swiss pharmaceutical company whose shares trade over the counter in the United States under the ticker RHHBY. Roche sponsored the Phase 2 clinical trial evaluating the compound in Duchenne Muscular Dystrophy.
RO7239361 is in Phase 2 development. One Phase 2 trial has been completed, and no trials are currently active. The study was randomized, double-blind, and placebo-controlled, enrolling 166 participants. RO7239361 is investigational and has not been approved by the FDA.
RO7239361 was evaluated in one completed Phase 2 trial, NCT03039686, titled a clinical trial to evaluate the efficacy, safety, and tolerability of RO7239361 in ambulatory boys with Duchenne Muscular Dystrophy. It enrolled 166 male participants aged 6 years and older across the United States, Europe, Japan, Canada, Australia, and Argentina.