Muscular Atrophy, Spinal Clinical Trials

43 clinical trials for Muscular Atrophy, Spinal across international registries, including 7 currently recruiting participants. Compare status, phase, eligibility criteria and locations.

• A Study to Learn About Salanersen's (BIIB115) Effects on Movement and Its Safety When Given Before Symptoms Appear in Babies With Genetically Diagnosed Spinal Muscular Atrophy (SMA) — RECRUITING · PHASE3
• A Study to Learn About the Safety and Effects of Salanersen (BIIB115) When Given to Babies With Spinal Muscular Atrophy (SMA) Who Were Previously Treated With Onasemnogene Abeparvovec — RECRUITING · PHASE3
• 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 — RECRUITING · PHASE4
• A Study Evaluating the Effectiveness and Safety of Risdiplam Administered as an Early Intervention in Pediatric Participants With Spinal Muscular Atrophy After Gene Therapy — RECRUITING · PHASE4
• 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) — RECRUITING · PHASE1
• 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 — RECRUITING
• Natural History of SMA — RECRUITING
• A Study to Learn About the Long-Term Safety of Higher Doses of Nusinersen (BIIB058) Given as Injections to Participants With Spinal Muscular Atrophy (SMA) Who Took Part in an Earlier Nusinersen Trial (ONWARD) — COMPLETED · PHASE3
• Adult Spinal Muscular Atrophy (SMA) China Registry — ACTIVE_NOT_RECRUITING
• Observational, Postmarketing Surveillance Study of Spinraza Injection (Nusinersen Sodium) — ACTIVE_NOT_RECRUITING
• A Study of Risdiplam in Infants With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy — ACTIVE_NOT_RECRUITING · PHASE2
• 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 — ACTIVE_NOT_RECRUITING · PHASE1
• A Study to Investigate the Pharmacokinetics and Safety of Risdiplam in Infants With Spinal Muscular Atrophy — COMPLETED · PHASE2
• A Real-World Study of Clinical Use of Available Treatments for Pediatric Patients With Spinal Muscular Atrophy — COMPLETED
• A Real-world Study of Disease-modifying Therapy Treatment Outcomes in Patients With Spinal Muscular Atrophy — COMPLETED
• A Study of Dosing Patterns and Costs in Patients With Spinal Muscular Atrophy Receiving Disease Modifying Therapies — COMPLETED
• Efficacy and Safety of Apitegromab in Patients With Later-Onset Spinal Muscular Atrophy Treated With Nusinersen or Risdiplam — COMPLETED · PHASE3
• Newborn Screening for Spinal Muscular Atrophy — COMPLETED
• A Study to Learn About the Effect of Nusinersen (BIIB058) Given as Injections to Children With Spinal Muscular Atrophy (SMA) Who Were Previously Treated With Onasemnogene Abeparvovec (RESPOND) — COMPLETED · PHASE4
• Pediatric Spinal Muscular Atrophy (SMA) China Registry — ACTIVE_NOT_RECRUITING
• Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy — COMPLETED · PHASE3
• Long-Term Safety & Efficacy of Apitegromab in Patients With SMA Who Completed Previous Trials of Apitegromab — ACTIVE_NOT_RECRUITING · PHASE3
• Two Different Treatment Modalities in Patients With Spinal Muscular Atrophy — COMPLETED · NA
• An Active Treatment Study of SRK-015 in Patients With Type 2 or Type 3 Spinal Muscular Atrophy — COMPLETED · PHASE2
• Safety and Efficacy of Intravenous OAV101 (AVXS-101) in Pediatric Patients With Spinal Muscular Atrophy (SMA) (OFELIA) — COMPLETED · PHASE4
• Investigate Safety, Tolerability, PK, PD and Efficacy of Risdiplam (RO7034067) in Infants With Type1 Spinal Muscular Atrophy — COMPLETED · PHASE2
• A BHI to Increase Hope Level and Stress Level of Parents With a CMC — COMPLETED · NA
• A Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of Risdiplam (RO7034067) in Type 2 and 3 Spinal Muscular Atrophy (SMA) Participants — COMPLETED · PHASE2
• Ultrasound-assisted vs Landmark Based Intrathecal Administration of Nusinersen — COMPLETED · NA
• Non-Interventional, Postmarketing Surveillance Study of Nusinersen Sodium Injection — COMPLETED
• Bioavailability and Bioequivalence of Two Risdiplam Tablets in Healthy Participants — COMPLETED · PHASE1
• Procedural Sedation for Pediatric Patients With Spinal Muscular Atrophy — COMPLETED
• Controlled Trial to Evaluate Amifampridine Phosphate in Spinal Muscular Atrophy Type 3 Patients — COMPLETED · PHASE2
• A Study to Investigate the Effect of Hepatic Impairment on the Pharmacokinetics and Safety and Tolerability of a Single Oral Dose of Risdiplam Compared to Matched Healthy Participants With Normal Hepatic Function — COMPLETED · PHASE1
• An Expanded Access Program for Risdiplam in Participants With Spinal Muscular Atrophy (SMA) — OTHER
• A Pilot Therapeutic Trial Using Hydroxyurea in Type I Spinal Muscular Atrophy Patients — COMPLETED · PHASE1
• A Pilot Therapeutic Trial Using Hydroxyurea in Type II and Type III Spinal Muscular Atrophy Patients — COMPLETED · PHASE1
• A Study to Evaluate Long Term Safety, Tolerability, and Effectiveness of Olesoxime in Patients With Spinal Muscular Atrophy (SMA) — COMPLETED · PHASE2
• International SMA Patient Registry — COMPLETED
• Progressive Strength Training in Spinal Muscular Atrophy — COMPLETED · NA
• A Study of RO6885247 in Adult and Pediatric Patients With Spinal Muscular Atrophy (MOONFISH) — TERMINATED · PHASE1
• A Study to Collect Blood Samples From Patients With Spinal Muscular Atrophy for Biomarker Analysis — COMPLETED
• Pilot Study of Growth Hormon to Treat SMA Typ II and III — COMPLETED · PHASE2

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