Treatment
Risdiplam clinical trials
We’re tracking 6 recruiting studies that include Risdiplam, each written for real people, not researchers, below.
Recruiting studies
- NCT05861999RecruitingPhase 4
Risdiplam for young children with SMA after gene therapy plateau
This trial studies whether risdiplam is safe and helpful for children under 2 with spinal muscular atrophy (SMA) whose function has leveled off or declined after gene therapy. It may be an option if you saw benefits from gene therapy but then noticed a stop or setback in key abilities.
Little Rock, ArkansasAges 3 months–2 years - NCT05861986RecruitingPhase 4
Risdiplam for very young children after gene therapy for SMA
This trial studies whether risdiplam, given early after gene therapy, is safe and helps children with spinal muscular atrophy (SMA) keep improving or stay stable. It focuses on children under 2 years old who already received gene therapy and need to meet timing and health requirements.
Little Rock, ArkansasAges 3 months–2 years - NCT07531719Recruiting
Risdiplam treatment study for type I and II spinal muscle atrophy
This study follows children with spinal muscular atrophy (SMA)—a genetic condition causing muscle weakness—who are being treated with a medicine called risdiplam. Researchers want to understand how well the treatment works and how it affects daily life in young patients.
CairoAges Up to 5 years - NCT06978985Recruiting
Adult SMA research and clinical hub study
This study creates a registry and clinical hub for adults with 5q spinal muscular atrophy (SMA). It aims to track health outcomes and improve care for people living with SMA.
BirminghamAges 16 years+ - NCT07047144RecruitingPhase 2
Apitegromab for infants with SMA under 2 years old
This study tests a new medicine called apitegromab for babies under 2 years old with spinal muscular atrophy (SMA). It looks at whether the drug can help with muscle strength and reaching motor milestones like sitting or rolling.
Phoenix, ArizonaAges Up to 2 years - NCT07444476RecruitingPhase 3
Study of salanersen for spinal muscular atrophy (ages 15-60)
This trial tests a medicine called salanersen (BIIB115) to see how well it improves movement and how safe it is in people with spinal muscular atrophy (SMA). It includes those who have never taken an SMA drug or are currently on risdiplam and willing to switch.
Norfolk, VirginiaAges 15–60
What Risdiplam is being studied for
Conditions with recruiting Risdiplam studies.
Common questions
- Are there clinical trials studying Risdiplam?
- Yes. Clin2 currently lists 6 recruiting studies that include Risdiplam, drawn from the U.S. registry and rewritten for real people so you can see what each is testing and who it's for.
- How do I know if I qualify for a Risdiplam trial?
- Each study lists its own eligibility criteria — rules about condition, age, and prior treatments. On every Clin2 trial page we explain these in plain language and offer a short, optional pre-screen for a fit read. The study team makes the final decision.
- Is Risdiplam an approved treatment?
- A drug being studied in a trial may be investigational (still being tested) or already approved and being studied for a new use. Each study page explains what phase it's in. Clin2 does not provide medical advice — talk with your doctor about whether Risdiplam or a specific trial is right for you.
Related treatments
Clin2 helps you find and understand clinical trials and does not provide medical advice. Study data comes from ClinicalTrials.gov. A drug appearing here is being studied in a trial and may be investigational. Talk with your doctor about whether a specific treatment or trial is right for you.