Novartis: Muscular Atrophy, Spinal trials

The registry lists 18 Muscular Atrophy, Spinal studies with Novartis as lead sponsor. 4 are open to participants now and 9 have results posted.

Phase 1 2Phase 2 1Phase 3 7Phase 4 2No phase 7(a study in two phases counts in both)

Studies

Open to participants first, then studies with results posted, then the rest; newest registration first within each.

• A Study of the Safety and Effectiveness of Onasemnogene Abeparvovec (Zolgensma) Intrathecal Injection in Spinal Muscular Atrophy PatientsRecruitingNCT07767890
• A Real-World Medical Chart Review of Spinal Muscular Atrophy Patients Treated With Onasemnogene Abeparvovec in Saudi ArabiaNot yet recruitingNCT07737756
• ASsessing The REAl-world Safety & Effectiveness of Spinal Muscular Atrophy Participants Treated With Intrathecal Onasemnogene Abeparvovec-brve (OAV101B) (ITVISMA®): A U.S. Pragmatic Multicenter Study (STREAM)RecruitingNCT07448610Phase 4
• Registry of Patients With a Diagnosis of Spinal Muscular Atrophy (SMA)RecruitingNCT04174157
• Phase IIIb, Open-label, Multi-center Study to Evaluate Safety, Tolerability and Efficacy of OAV101 Administered Intrathecally to Participants With SMA Who Discontinued Treatment With Nusinersen or RisdiplamCompletedNCT05386680Phase 3Results posted
• Efficacy and Safety of Intrathecal OAV101 (AVXS-101) in Pediatric Patients With Type 2 Spinal Muscular Atrophy (SMA)CompletedNCT05089656Phase 3Results posted
• Safety and Efficacy of Intravenous OAV101 (AVXS-101) in Pediatric Patients With Spinal Muscular Atrophy (SMA) (OFELIA)CompletedNCT05073133Phase 4Results posted
• Safety and Efficacy of Intravenous OAV101 (AVXS-101) in Pediatric Patients With Spinal Muscular Atrophy (SMA)CompletedNCT04851873Phase 3Results posted
• Single-Dose Gene Replacement Therapy Using for Patients With Spinal Muscular Atrophy Type 1 With One or Two SMN2 CopiesCompletedNCT03837184Phase 3Results posted
• Pre-Symptomatic Study of Intravenous Onasemnogene Abeparvovec-xioi in Spinal Muscular Atrophy (SMA) for Patients With Multiple Copies of SMN2CompletedNCT03505099Phase 3Results posted
• Study of Intrathecal Administration of Onasemnogene Abeparvovec-xioi for Spinal Muscular AtrophyTerminatedNCT03381729Phase 1Results posted
• Gene Replacement Therapy Clinical Trial for Participants With Spinal Muscular Atrophy Type 1CompletedNCT03306277Phase 3Results posted
• An Open Label Study of LMI070 (Branaplam) in Type 1 Spinal Muscular Atrophy (SMA)CompletedNCT02268552Phase 1/2Results posted
• A Real-World Study of Clinical Use of Available Treatments for Pediatric Patients With Spinal Muscular AtrophyCompletedNCT07474311
• A Real-world Study of Disease-modifying Therapy Treatment Outcomes in Patients With Spinal Muscular AtrophyCompletedNCT07403214
• A Study of Dosing Patterns and Costs in Patients With Spinal Muscular Atrophy Receiving Disease Modifying TherapiesCompletedNCT07378943
• Real-World Use of Novel Treatments in Patients With Spinal Muscular Atrophy (SMA): A Multi-Site Retrospective Chart Review of Pediatric SMA Patients Outside of the United StatesCompletedNCT06178393
• Long-term Follow-up of Patients With Spinal Muscular Atrophy Treated With OAV101 in Clinical TrialsActive, not recruitingNCT05335876Phase 3