SMA Trials
Location Trial Status:
= Fully recruited
= Recruiting
= Not yet recruiting
= Enrolling by invitation
= Completed / Terminated
= Fully recruited
= Recruiting
= Not yet recruiting
= Enrolling by invitation
= Completed / Terminated
An Open-Label Study to Assess the Efficacy, Safety, Tolerability, and Pharmacokinetics of Multiple Doses of ISIS 396443 Delivered Intrathecally to Subjects With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy.
This is a Phase 2 clinical trial to test the safety and efficacy of Nusinersen in infants with genetically diagnosed and presymptomatic SMA. This study also aims to examine the effects of Nusinersen in infants.
Escalating Dose and Randomized, Controlled Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy
This is a Phase 2 and 3 clinical trial that studies the safety, efficacy, and tolerability of nusinersen at higher doses in participants with SMA. The study is comprised of 3 parts. Part A investigates a high dose of nusinersen (28mg) in nusinersen-naiive participants with late-onset SMA. Part B investigates two different doses of nusinersen (12mg or 50mg) in nusinersen-naiive participants with infantile or late-onset SMA. Part C investigates participants who have previously taken 12mg of nusinersen for 1 year prior to the study; it involves giving an initial dose of 50mg of nusinersen at the start of the study, and subsequently 28mg of nusinersen at later visits in the study.
An Open-Label Extension Study for Patients With Spinal Muscular Atrophy Who Previously Participated in Investigational Studies of Nusinersen (ISIS 396443)
This is a Phase 3 study that evaluates the long-term safety, efficacy, and tolerability of Nusinersen in participants with SMA who have previously participated in other Nusinersen studies.
A Phase IIIb, Open-label, Single-arm, Single-dose, Multicenter Study to Evaluate the Safety, Tolerability and Efficacy of Gene Replacement Therapy With Intravenous OAV101 (AVXS-101) in Pediatric Patients With Spinal Muscular Atrophy (SMA)
This is a gene replacement therapy trial where participants with SMA will receive a single intravenous dose of OAV101 (AVXS-101). This study will investigate the safety, tolerability, and effectiveness of OAV101 (AVXS-101). The participants will be monitored for 48 hours after the dose to ensure that no serious side effects have taken place. They will then will be asked to return for follow-up appointments over the next 12 months. After the study is complete, participants will be invited to enroll into a long-term follow-up study to collect additional safety and efficacy data.
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