Trials / Conditions / Spinal Muscular Atrophy Type I

Spinal Muscular Atrophy Type I

11 registered clinical trials studyying Spinal Muscular Atrophy Type I — 3 currently recruiting.

Status	Trial	Sponsor	Phase
Recruiting	Study of Safety, Tolerability and Efficacy of GB221 in Infants With Spinal Muscular Atrophy Type 1 NCT07070999	Gemma Biotherapeutics	Phase 1 / Phase 2
Not Yet Recruiting	Psychological Evaluation of the Parental Experience of Newborn Screening for Infantile Spinal Muscular Atrophy NCT07208903	University Hospital, Strasbourg, France	—
Completed	Impact of Motor and Oral Motor Function on Quality of Life in Children With SMA NCT06864767	Zeynep HOŞBAY	—
Completed	The Effect of Spinal Orthosis on the Development of Scoliosis and Chest Deformity in Type I Spinal Muscular At NCT05878418	Medipol University	N/A
Completed	Therapeutic Management and Use of Resources and Costs of Spinal Muscular Atrophy in Spain NCT06632730	Novartis	—
Enrolling By Invitation	Clinical Trial to Assess the Safety and Efficacy of EXG001-307 in Patients with Spinal Muscular Atrophy Type 1 NCT05614531	Guangzhou Jiayin Biotech Ltd	Phase 1 / Phase 2
Active Not Recruiting	Long-term Follow-up Study of Patients Receiving Onasemnogene Abeparvovec-xioi NCT04042025	Novartis Gene Therapies	Phase 3
Completed	Single-Dose Gene Replacement Therapy Using for Patients With Spinal Muscular Atrophy Type 1 With One or Two SM NCT03837184	Novartis Gene Therapies	Phase 3
Completed	Body Weight Support Harness System in Spinal Muscular Atrophy NCT05715749	Nationwide Children's Hospital	N/A
Completed	CARNIVAL Type I: Valproic Acid and Carnitine in Infants With Spinal Muscular Atrophy (SMA) Type I NCT00661453	University of Utah	Phase 1 / Phase 2
Terminated	Clinical Trial of Sodium Phenylbutyrate in Children With Spinal Muscular Atrophy Type I NCT00439218	Westat	Phase 1 / Phase 2