2 active not recruiting, 2 completed, 1 not yet recruiting, 1 recruiting. 2 Not applicable, 2 Phase 1, 1 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Not applicable | 2 | Active not recruiting 1, Completed 1 |
| Phase 1 | 2 | Completed 1, Recruiting 1 |
| Phase 2 | 1 | Recruiting 1 |
Follow Spinal Muscular Atrophy 1: new trials by email Literature Review of Spinal Muscular Atrophy 1 trials Research Report on Spinal Muscular Atrophy 1
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 Phase I/II Clinical Study to Evaluate SKG0201 Injection in Subjects With Spinal Muscular Atrophy Type I SKG0201 Injection - Spinal Muscular Atrophy 1 | Phase 1 / Phase 2 | Recruiting | Lanyue Biotech (Hangzhou) Co., Ltd. | 11 | 2026-07-15 | 2026-10-14 |
| Exploring Lived Experiences of Families of Children With Spinal Muscular Atrophy(SMA) Type 1 Regarding Feeding and Communication Spinal Muscular Atrophy 1 | Not yet recruiting | Guy's and St Thomas' NHS Foundation Trust | 15 | 2026-06-19 | 2026-06-02 | |
| A Clinical Study Evaluating the Safety and Efficacy of SKG0201 Injection in Patients With Spinal Muscular Atrophy Type 1 SKG0201 Injection - Spinal Muscular Atrophy 1 | Not applicable | Active not recruiting | Kun Sun | 12 | 2023-06-25 | 2025-11-21 |
| 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 |
| Palliative Care in Spinal Muscular Atrophy (SMA) 1 Follow-up diary and questionnaire - Spinal Muscular Atrophy 1 | Not applicable | Completed | Assistance Publique - Hôpitaux de Paris | 39 | 2012-06-01 | 2025-09-11 |
Spinal Muscular Atrophies of Childhood (6) · Communication (1) · Deglutition Disorders (1) · Language (1) · Muscular Atrophy, Spinal (1) · Speech (1)
Central Nervous System Diseases (6) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (6) · Genetic Diseases, Inborn (6) · Heredodegenerative Disorders, Nervous System (6) · Motor Neuron Disease (6) · Nervous System Diseases (6) · Neurodegenerative Diseases (6) · Neuromuscular Diseases (6) · Spinal Cord Diseases (6) · Muscular Atrophy, Spinal (5) · Behavior (1) · Digestive System Diseases (1) · Esophageal Diseases (1) · Gastrointestinal Diseases (1) · Otorhinolaryngologic Diseases (1)
Zolgensma (2) · Palliative Care (1) · Surveys and Questionnaires (1)
Novartis Gene Therapies (2) · Assistance Publique - Hôpitaux de Paris (1) · Guy's and St Thomas' NHS Foundation Trust (1) · Kun Sun (1) · Lanyue Biotech (Hangzhou) Co., Ltd. (1)
China (2) · United States (2) · France (1) · United Kingdom (1)
Isabelle Desguerre, MD, PhD (1) · Jerry R Mendell, MD (1) · Kun Sun, Doctor (1) · Yongguo Yu, Doctor (1)
PMID 34383289, PMID 42016922, PMID 29091557, PMID 30548438, PMID 30879249, PMID 31277975, PMID 32133329, PMID 33999158