8 completed, 3 active not recruiting, 2 recruiting, 1 approved for marketing. 8 Phase 3, 2 Phase 1, 1 Phase 4.
| Phase | Trials | Status |
|---|---|---|
| Phase 3 | 8 | Completed 6, Active not recruiting 2 |
| Phase 1 | 2 | Completed 1, Terminated 1 |
| Phase 4 | 1 | Completed 1 |
Follow Zolgensma: new trials by email Literature Review of Zolgensma trials Research Report on Zolgensma
Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.
| Trial | Phase | Status | Sponsor | Enrollment | Start | Updated |
|---|---|---|---|---|---|---|
| A Long-term Safety Study in Brazilian Patients With a Diagnosis of Spinal Muscular Atrophy Treated With Zolgensma Onasemnogene Abeparvovec - Spinal Muscular Atrophies | Recruiting | Novartis Pharmaceuticals | 50 | 2023-11-22 | 2025-01-15 | |
| 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 Risdiplam OAV101 - Spinal Muscular Atrophy | Phase 3 | Completed | Novartis Pharmaceuticals | 27 | 2023-01-12 | 2026-01-13 |
| Long-term Follow-up of Patients With Spinal Muscular Atrophy Treated With OAV101 in Clinical Trials onasemnogene abeparvovec - Spinal Muscular Atrophy (SMA) | Phase 3 | Active not recruiting | Novartis Pharmaceuticals | 20 | 2022-12-19 | 2026-07-09 |
| Efficacy and Safety of Intrathecal OAV101 (AVXS-101) in Pediatric Patients With Type 2 Spinal Muscular Atrophy (SMA) OAV101, Sham control - Type 2 Spinal Muscular Atrophy | Phase 3 | Completed | Novartis Pharmaceuticals | 126 | 2022-02-01 | 2026-01-13 |
| Safety and Efficacy of Intravenous OAV101 (AVXS-101) in Pediatric Patients With Spinal Muscular Atrophy (SMA) (OFELIA) OAV101 - Muscular Atrophy, Spinal | Phase 4 | Completed | Novartis Pharmaceuticals | 16 | 2021-11-04 | 2024-10-09 |
| Long-term Follow-up Study of Patients Receiving Onasemnogene Abeparvovec-xioi Onasemnogene Abeparvovec-xioi - Spinal Muscular Atrophy Type I, Spinal Muscular Atrophy Type II | Phase 3 | Active not recruiting | Novartis Gene Therapies | 85 | 2020-02-10 | 2026-09-24 |
| Single-Dose Gene Replacement Therapy Using for Patients With Spinal Muscular Atrophy Type 1 With One or Two SMN2 Copies Onasemnogene Abeparvovec-xioi - Spinal Muscular Atrophy Type I | Phase 3 | Completed | Novartis Gene Therapies | 2 | 2019-05-31 | 2026-01-26 |
| Registry of Patients With a Diagnosis of Spinal Muscular Atrophy (SMA) Prospective observational registry, Zolgensma - Spinal Muscular Atrophy (SMA) | Recruiting | Novartis Pharmaceuticals | 700 | 2018-09-25 | 2025-01-31 | |
| Single-Dose Gene Replacement Therapy Clinical Trial for Participants With Spinal Muscular Atrophy Type 1 Onasemnogene Abeparvovec-xioi - SMA | Phase 3 | Completed | Novartis Gene Therapies | 33 | 2018-08-16 | 2026-01-26 |
| Pre-Symptomatic Study of Intravenous Onasemnogene Abeparvovec-xioi in Spinal Muscular Atrophy (SMA) for Patients With Multiple Copies of SMN2 onasemnogene abeparvovec-xioi - Spinal Muscular Atrophy | Phase 3 | Completed | Novartis Gene Therapies | 30 | 2018-04-02 | 2026-01-26 |
| Study of Intrathecal Administration of Onasemnogene Abeparvovec-xioi for Spinal Muscular Atrophy Onasemnogene Abeparvovec-xioi - Spinal Muscular Atrophy | Phase 1 | Terminated | Novartis Gene Therapies | 32 | 2017-12-14 | 2026-01-26 |
| Gene Replacement Therapy Clinical Trial for Participants With Spinal Muscular Atrophy Type 1 Onasemnogene Abeparvovec-xioi - SMA - Spinal Muscular Atrophy, Gene Therapy | Phase 3 | Completed | Novartis Gene Therapies | 22 | 2017-10-24 | 2026-01-26 |
| Long-Term Follow-up Study for Patients From AVXS-101-CL-101 Onasemnogene Abeparvovec-xioi - Spinal Muscular Atrophy 1 | Active not recruiting | Novartis Gene Therapies | 13 | 2017-09-21 | 2026-09-22 | |
| Gene Transfer Clinical Trial for Spinal Muscular Atrophy Type 1 AVXS-101 - Spinal Muscular Atrophy 1 | Phase 1 | Completed | Novartis Gene Therapies | 15 | 2014-05-05 | 2022-09-15 |
| AveXis Managed Access Program Cohort for Access to AVXS-101 AVXS-101 - Spinal Muscular Atrophy | Approved for marketing | United BioSource, LLC | 2020-08-28 |
Muscular Atrophy, Spinal (10) · Spinal Muscular Atrophies of Childhood (5) · Muscular Atrophy (4) · Muscular Diseases (4) · Bulbo-Spinal Atrophy, X-Linked (3)
Novartis Gene Therapies (8) · Novartis Pharmaceuticals (6) · United BioSource, LLC (1)
United States (11) · Japan (6) · Belgium (5) · Taiwan (5) · Australia (4) · Brazil (4) · Canada (4) · France (4) · Italy (4) · United Kingdom (4) · China (2) · Denmark (2) · Malaysia (2) · Netherlands (2) · Saudi Arabia (2)
AveXis Medinfo (1) · Jerry R Mendell, MD (1) · Sitra Tauscher-Wisniewski, MD (1)
PMID 34383289, PMID 42016922, PMID 29091557, PMID 30548438, PMID 30879249, PMID 31277975, PMID 33743238, PMID 33999158, PMID 34536405, PMID 35715566, PMID 35715567, PMID 41217888, PMID 41360993, PMID 41360995, PMID 42265796
2017 (2), 2019 (1), 2023 (1)