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RO7239361 · 2 trials · 2 indications
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. |
| Treatment A: RO7239361 | ACTIVE_COMPARATOR | RO7239361 subcutaneous injections on specified days; abdomen |
| Treatment B: RO7239361 | ACTIVE_COMPARATOR | RO7239361 subcutaneous injections on specified days; arm |
| Treatment C: RO7239361 | ACTIVE_COMPARATOR | RO7239361 subcutaneous injections on specified days; thigh |
| 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 being studied for the treatment of Duchenne Muscular Dystrophy (DMD). It was evaluated in a Phase 2 clinical trial in ambulatory boys with DMD. The drug is also being studied in healthy volunteers for bioavailability purposes. It is not yet approved and remains in clinical development.
RO7239361 is a small molecule developed by Roche Holding AG for Duchenne Muscular Dystrophy. Its specific molecular target has not been disclosed in the available clinical trial information. The drug is being investigated for its potential to modify the disease course in DMD, though the exact mechanism of action is not publicly detailed.
RO7239361 is developed by Roche Holding AG, a multinational healthcare company traded under the ticker RHHBY. Roche is conducting clinical trials to evaluate the efficacy, safety, and tolerability of RO7239361 in patients with Duchenne Muscular Dystrophy and in healthy volunteers.
RO7239361 is in Phase 2 clinical development for Duchenne Muscular Dystrophy. A Phase 2 trial (NCT03039686) has been completed, and a Phase 1 trial (NCT03100630) in healthy volunteers has also been completed. The drug is investigational and has not received FDA approval.
RO7239361 has been studied in two completed clinical trials. NCT03039686 was a Phase 2, randomized, double-blind, placebo-controlled trial in 166 ambulatory boys with Duchenne Muscular Dystrophy. NCT03100630 was a Phase 1 trial in 99 healthy volunteers to compare bioavailability after subcutaneous injection.
RO7239361 is the investigational code name used by Roche Holding AG for this drug candidate. No alternative brand names or other designations have been publicly associated with this compound in the clinical trial information. It is referred to solely as RO7239361 in the registered studies.