= Fully recruited
= Not yet recruiting
= Enrolling by invitation
= Completed / Terminated
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.
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.
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.
An Open-Label Study of Risdiplam in Infants With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy
This is a Phase 2 trial that looks at the safety, efficacy, and tolerability of Risdiplam in infants aged from birth to 6 weeks old. This study involves infants who have been diagnosed with SMA, but do not yet show any symptoms (pre-symptomatic). Risdiplam will be given orally (by mouth) to each participant, once daily, for 2 years. After this, participants will continue taking Risdiplam in an open-label extension for 3 years with follow-up, resulting in at least 5 years of treatment for each participant.
A Long Term Follow up Safety Study of Patients in the AVXS-101-CL-101 Gene Replacement Therapy Clinical Trial for Spinal Muscular Atrophy Type 1 Delivering AVXS 101
This is a long-term follow-up study for participants who have received the AVXS-101 (gene replacement therapy) infusion in a previous clinical trial. The study will investigate the long-term safety and effects of AVXS-101 in these participants. The participants will have annual face-to-face follow-up meetings for 5 years, and then annual phone-call meetings for 10 years.
A Global Study of a Single, One-Time Dose of AVXS-101 Delivered to Infants With Genetically Diagnosed and Pre-symptomatic Spinal Muscular Atrophy With Multiple Copies of SMN2
This is a Phase 3 trial that studies a single dose of AVXS-101 (gene replacement therapy) in pre-symptomatic infants with SMA. The study involves one intravenous (IV) infusion followed by post-treatment monitoring of safety and efficacy. Depending on the number of SMN2 copies that the participant has, the study will involve 18 months (SMN2 = 2 copies) or 24 months (SMN2 = 3) of post-treatment monitoring. After this visit, eligible patients will be asked to rollover into a long-term follow-up study.
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