- active not recruitingPHASE1
A Study to Learn About the Safety of BIIB115 Injections and How BIIB115 is Processed in the Bodies of Healthy Adult Male Volunteers and of Pediatric Participants With Spinal Muscular Atrophy Who Previously Took Onasemnogene Abeparvovec
This study looks at a new medicine called BIIB115 for spinal muscular atrophy (SMA). It first tests the medicine's safety in healthy men, then in children with SMA. Researchers want to understand how safe BIIB115 is and how the body handles it.
Belgium · Canada · France - active not recruitingPHASE3
Long-Term Safety & Efficacy of Apitegromab in Patients With SMA Who Completed Previous Trials of Apitegromab
This study looks at the long-term safety and effects of a medicine called apitegromab for people with Spinal Muscular Atrophy (SMA) types 2 and 3. It's for those who have already completed earlier studies with this drug.
United States · Belgium · France - recruitingPHASE2
A Study to Investigate the Pharmacokinetics and Safety of Risdiplam in Infants With Spinal Muscular Atrophy
This study is looking into a medicine called risdiplam for babies born with spinal muscular atrophy (SMA). It aims to understand how the medicine works in their bodies and if it's safe for very young infants (under 20 days old) at their first dose.
United States · Belgium · Canada - recruitingPHASE4
A Study Evaluating the Effectiveness and Safety of Risdiplam Administered in Pediatric Patients With Spinal Muscular Atrophy Who Experienced a Plateau or Decline in Function After Gene Therapy
This study looks at a medicine called risdiplam for young children (under 2) with a muscle condition called spinal muscular atrophy (SMA). It's for those who had gene therapy but aren't getting better or are getting worse. The study checks if risdiplam is safe and helps them.
United States · Germany · Israel - recruitingPHASE1
A Study to Find Out How Nusinersen is Processed in the Body When Given Through the ThecaFlex DRx™ System in Adult and Pediatric Participants With Spinal Muscular Atrophy (PIERRE-PK)
This study looks at how a medicine (nusinersen) for Spinal Muscular Atrophy (SMA) travels through the body when given in two ways: a standard spinal injection or using a new implantable system called ThecaFlex DRx. It aims to see which method delivers the medicine best.
United States · France · Germany - recruitingPHASE4
A Study Evaluating the Effectiveness and Safety of Risdiplam Administered as an Early Intervention in Pediatric Participants With Spinal Muscular Atrophy After Gene Therapy
This study looks at giving Risdiplam to young children with Spinal Muscular Atrophy (SMA) who have already had gene therapy. It wants to see if this combination helps them even more and if it's safe. It's for children under 2 years old with a specific type of SMA.
United States · Germany · Poland - recruiting
A Study to Learn How Nusinersen (Spinraza) Affects Participants With Spinal Muscular Atrophy (SMA) Who Took it Before or During Pregnancy And About The Health of Their Babies
This study looks at pregnant women with Spinal Muscular Atrophy (SMA) who have taken the drug nusinersen (Spinraza). Researchers want to understand how the drug affects their pregnancy and the health of their babies, as there's not enough information yet.
United States · United Kingdom