NCT05115110

A Study to Investigate the Safety and Efficacy of RO7204239 in Combination With Risdiplam (RO7034067) in Participants With Spinal Muscular Atrophy

A Two-Part, Seamless, Multi-Center, Randomized, Placebo-Controlled, Double-Blind Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of RO7204239 in Combination With Risdiplam (RO7034067) in Patients With Spinal Muscular Atrophy

• Status: Active Not Recruiting
• Phase: Phase 2 / Phase 3
• Study type: Interventional
• Enrollment: 259 (estimated)
• Sponsor: Hoffmann-La Roche · Industry
• Sex: All
• Age: 2 Years – 25 Years
• Healthy volunteers: Not accepted

Summary

Risdiplam works by helping the body produce more survival motor neuron (SMN) protein throughout the body. This means fewer motor neurons - nerve cells that pass impulses from nerves to muscles to cause movement - are lost, which may improve how well muscles work in people with SMA. RO7204239 is an investigational anti-myostatin antibody that is designed to target myostatin. Myostatin plays an important role in the regulation of skeletal muscle size by controlling growth. Inhibiting myostatin may help muscles grow in size and strength. RO7204239 in combination with risdiplam, which is designed to increase the amount of SMN protein throughout the body, has the potential to further improve motor function and clinical outcomes for people living with SMA. This trial will study the safety and efficacy of RO7204239 in combination with risdiplam in patients with spinal muscular atrophy (SMA). The trial has two parts; Part 1 is the dose-finding part in SMA patients that are either ambulant (aged 2-10 years) or non-ambulant (aged 5-10 years) within separate cohorts, and Part 2 is the pivotal part in SMA patients aged 2-25 years that are ambulant.

Conditions

• Spinal Muscular Atrophy (SMA)

Interventions

Type	Name	Description
DRUG	RO7204239	RO7204239 will administered every 4 weeks (Q4W) by subcutaneous (SC) injection into the abdomen. RO7204239 will be investigated at low- and high-dose in Part 1.
DRUG	Placebo	Placebo will be administered Q4W by SC injection into the abdomen.
DRUG	Risdiplam	Risdiplam will be administered orally once daily (QD) for the duration of the study.

Timeline

• Start date: 2022-06-02
• Primary completion: 2029-02-27
• Completion: 2029-02-27
• First posted: 2021-11-10
• Last updated: 2026-03-13

Locations

35 sites across 12 countries: United States, Australia, Belgium, Canada, Croatia, Italy, Japan, Netherlands, Poland, Portugal, Spain, United Kingdom

Regulatory

• FDA-regulated drug study