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NCT05115110 · ClinicalTrials.gov registry record · Phase 2
A Study to Investigate the Safety and Efficacy of RO7204239 in Combination With Risdiplam (RO7034067) in Participants With Spinal Muscular Atrophy
A Phase 2 study of Spinal Muscular Atrophy (SMA), sponsored by Hoffmann-La Roche.
- Active
- Registry status
- Phase 2
- Development phase
- 259
- Enrollment target
- 20
- Study locations
NCT05115110: Active Phase 2 study of Spinal Muscular Atrophy (SMA), sponsored by Hoffmann-La Roche.
NCT05115110 is a Phase 2 study of Spinal Muscular Atrophy (SMA) that is active but no longer recruiting, run by Hoffmann-La Roche. The registered enrollment target is 259 participants, above the 231-participant average among 5 other Spinal Muscular Atrophy (SMA) trials with a reported enrollment target (12% higher). The trial reports 20 study locations across 13 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT05115110, a Phase 2 study of Spinal Muscular Atrophy (SMA), is active but no longer recruiting, sponsored by Hoffmann-La Roche.
- ACTIVE NOT RECRUITING
- Registry status
- Phase 2
- Development phase
- 259 participants
- Enrollment target
- 20
- Study locations
Study 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 Studied
Interventions
- DRUG Placebo
- DRUG Risdiplam
- DRUG RO7204239
Study Locations (20)
Other
- UZ Gent - Ghent
- Chr de La Citadelle - Liège
- Clinical Hospital Centre Zagreb - Zagreb
- Kobe University Hospital - Hyōgo
- Kagoshima University Hospital - Kagoshima
- National Center for Global Health and Medicine - Tokyo
Lazio
- Ospedale Pediatrico Bambino Gesù - Rome
- Policlinico Agostino Gemelli - Rome
Lombardy
- Fondazione IRCCS Istituto Neurologico ?Carlo Besta? - Milan
- Asst Grande Ospedale Metropolitano Niguarda - Milan
Florida
- Nemours Children's Hospital - Orlando
Massachusetts
- Boston Childrens Hospital - Boston
New York
- Columbia University Medical Center - New York
Texas
- Neurology & Neuromuscular Care Center - Flower Mound
New South Wales
- Sydney Children's Hospital - Randwick
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 259 participants |
| Start Date | 2022-06-02 |
| Est. Completion | 2029-02-27 |
| Phase | Phase 2 |
What the registry record for NCT05115110 still lists
NCT05115110 is an interventional study that assigns participants to a tested intervention. The registered 259 participants enrollment target is mid-sized for trials with a published cap, above the 231-participant average among 5 other Spinal Muscular Atrophy (SMA) trials with a reported enrollment target (12% higher).
The record links to 1 condition, with Spinal Muscular Atrophy (SMA) appearing as the primary indexed condition, and to 3 interventions - of which Placebo is the first listed.
NCT05115110 names 20 study sites across 13 states, led by Other, Lazio, Lombardy.
Frequently Asked Questions
What is clinical trial NCT05115110 about?
NCT05115110 is a clinical study titled "A Study to Investigate the Safety and Efficacy of RO7204239 in Combination With Risdiplam (RO7034067) in Participants With Spinal Muscular Atrophy". 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 i...
What is the current status of trial NCT05115110?
This trial is currently active not recruiting. It is a Phase 2 study. The enrollment target is 259 participants. The study started on 2022-06-02. Estimated completion is 2029-02-27.
What conditions does trial NCT05115110 study?
This clinical trial studies the following conditions: Spinal Muscular Atrophy (SMA).
What interventions are being tested in trial NCT05115110?
The interventions under investigation include: Placebo (DRUG), Risdiplam (DRUG), RO7204239 (DRUG).
Who is sponsoring clinical trial NCT05115110?
This trial is sponsored by Hoffmann-La Roche, which has 909 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT05115110 being conducted?
This trial has 20 study locations across Florida, Massachusetts, New York, Texas, New South Wales. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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