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Also known as GYM329
RO7204239 · 4 trials · 6 indications
| Arm | Type | Description |
|---|---|---|
| Placebo + Tirzepatide | ACTIVE_COMPARATOR | Participants will receive RO7204239 matching placebo via subcutaneous (SC) injection every 4 weeks (Q4W) for the core treatment period of 48 weeks and the treatment extension period of 24 weeks. Participants will also receive tirzepatide, as a background therapy, up-titrated according to the approved tirzepatide prescribing information, SC, every week (QW) during the core treatment period of 48 weeks. |
| RO7204239 low dose + Tirzepatide | EXPERIMENTAL | Participants will receive RO7204239, low dose, SC, Q4W for the core treatment period of 48 weeks. During the treatment extension period participants will receive placebo for the period of 24 weeks. Participants will also receive tirzepatide, as a background therapy, up-titrated according to the approved tirzepatide prescribing information, SC, QW during the core treatment period of 48 weeks. |
| RO7204239 medium dose + Tirzepatide | EXPERIMENTAL | Participants will receive RO7204239, medium dose, SC, Q4W for the core treatment period of 48 weeks. During the treatment extension period participants will receive placebo for the period of 24 weeks. Participants will also receive tirzepatide, as a background therapy, up-titrated according to the approved tirzepatide prescribing information, SC, QW during the core treatment period of 48 weeks. |
| RO7204239 high dose + Tirzepatide | EXPERIMENTAL | Participants will receive RO7204239, high dose, SC, Q4W along with tirzepatide, given as a background therapy, up-titrated according to the approved tirzepatide prescribing information, SC, QW during the core treatment period of 48 weeks. During the treatment extension period participants will be randomized to receive RO7204239 or matching placebo, SC, Q4W for 24 weeks. |
| Placebo | PLACEBO_COMPARATOR | Participants will complete a 4-week pre-treatment period to collect baseline movement data with a wearable device, then receive subcutaneous (SC) placebo every 4 weeks for 52 weeks. After the treatment period, participants will have the option to receive RO7204239 for an additional 52 weeks. |
| RO7204239 | EXPERIMENTAL | Participants will complete a 4-week pre-treatment period to collect baseline movement data with a wearable device, then receive SC RO7204239 every 4 weeks for 52 weeks. After the treatment period, participants will have the option to receive RO7204239 for an additional 52 weeks. |
| RO7204239 + Risdiplam | EXPERIMENTAL | Participants who have not previously been treated with risdiplam will receive risdiplam for at least 8 weeks prior to randomization into a treatment group (Part 1 only). Participants that have been treated with risdiplam for at least 8 continuous weeks immediately prior to joining the study may be immediately randomized to combination therapy, or join the study run-in period (the period between screening and randomization to a treatment group) where they will continue to receive risdiplam monotherapy until randomization. Participants enrolled in Part 1 will receive RO7204239 (low or high dose) + risdiplam for 24 weeks, followed by RO7204239 + risdiplam for 72 weeks. Participants enrolled in Part 2 will receive risdiplam for 8 weeks and then treatment with RO7204239 + risdiplam for 72 weeks. Once the treatment period has completed (Part 1 or Part 2), participants will have the option of treatment with RO7204239 + risdiplam for 2 additional years. |
| Placebo + Risdiplam | ACTIVE_COMPARATOR | Participants who have not previously been treated with risdiplam will receive risdiplam for at least 8 weeks prior to randomization into a treatment group (Part 1 only). Participants that have been treated with risdiplam for at least 8 continuous weeks immediately prior to joining the study may be immediately randomized to combination therapy, or join the study run-in period (the period between screening and randomization to a treatment group) where they will continue to receive risdiplam monotherapy until randomization. Participants enrolled in Part 1 will receive placebo (low or high dose-matched) + risdiplam for 24 weeks, followed by RO7204239 + risdiplam for 72 weeks. Participants enrolled in Part 2 will receive risdiplam for 8 weeks and then treatment with placebo + risdiplam for 72 weeks. Once the treatment period has completed (Part 1 or Part 2), participants will have the option of treatment with RO7204239 + risdiplam for 2 additional years. |
| Name | Type | Description |
|---|---|---|
| RO7204239 | DRUG | RO7204239 or matching placebo will be administered as per the schedule specified in the arms. |
| RO7204239 Matching Placebo | DRUG | RO7204239 matching placebo will be administered as per the schedule specified in the arm. |
| Tirzepatide | DRUG | Tirzepatide will be administered as per the schedule specified in the arms. |
| Placebo | DRUG | Participants will receive subcutaneous (SC) placebo every 4 weeks (Q4W) |
| Risdiplam | DRUG | Risdiplam will be administered orally once daily (QD) for the duration of the study. |
Inclusion Criteria: * BMI ≥ 30.0 kilograms per square meter (kg/m²) (additional weight-related comorbidities are not required for inclusion) * BMI ≥ 27.0 kg/m² and \< 30.0 kg/m² with at least one weight-related comorbidity such as: hypertension, dyslipidemia, obstructive sleep apnea and any cardiov...
RO7204239 is an investigational small molecule developed by Roche Holding AG. It is being studied in neurology and metabolic conditions, including facioscapulohumeral muscular dystrophy (FSHD), spinal muscular atrophy (SMA), obesity, and type 2 diabetes mellitus. It is in Phase 2 clinical development.
RO7204239 targets myostatin, a protein that regulates muscle growth. By acting on myostatin, the small molecule is designed to influence muscle mass and composition, which is the rationale for studying it in muscle-wasting and metabolic conditions such as FSHD, SMA, and obesity.
RO7204239 is developed by Roche Holding AG, which trades under the ticker RHHBY. Roche is running the clinical program for the drug across several indications, including facioscapulohumeral muscular dystrophy, spinal muscular atrophy, obesity, and type 2 diabetes mellitus.
RO7204239 is in Phase 2 clinical development. It is investigational and is being evaluated in placebo-controlled, randomized, double-blind studies. Its trial program spans FSHD, SMA, obesity, and type 2 diabetes mellitus, with a mix of completed and active-not-recruiting studies.
RO7204239 has been studied in NCT05548556, a completed Phase 2 trial in facioscapulohumeral muscular dystrophy, and NCT05115110, a Phase 2 trial in spinal muscular atrophy combining it with risdiplam. It is also in NCT06965413, a Phase 2 obesity trial with tirzepatide, and NCT07137585, a Phase 1 trial in type 2 diabetes and obesity.
RO7204239 is being investigated as a treatment for facioscapulohumeral muscular dystrophy (FSHD). A completed Phase 2 study, NCT05548556, evaluated the drug in participants with FSHD across the United States, Denmark, Italy, and the United Kingdom. The drug remains investigational for this indication.