Infantile Spinal Muscular Atrophy of Type 2 or 3

Clinical trial pipeline · Data from ClinicalTrials.gov

See which Infantile Spinal Muscular Atrophy of Type 2 or 3 trials you may qualify for

Total trials

Actively recruiting

Phase 3 trials

Completed

Trial phase breakdown

Trial status

Actively recruiting — 1 trial

RecruitingNot Applicable

NCT01793168

Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford

CoRDS, or the Coordination of Rare Diseases at Sanford, is based at Sanford Research in Sioux Falls, South Dakota. It provides researchers with a centralized, i…

United States, Australia20,000 participantsStarted 2010-07

All recent trials (3 shown)

CompletedPhase 3

NCT03837184

Single-Dose Gene Replacement Therapy Using for Patients With Spinal Muscular Atrophy Type 1 With One or Two SMN2 Copies

This is a Phase 3, open-label, single-arm, single-dose, trial of onasemnogene abeparvovec-xioi (gene replacement therapy) in participants with spinal muscular a…

Japan, South Korea, Taiwan2 participantsStarted 2019-05-31

Treatment: Onasemnogene Abeparvovec-xioi

CompletedNot Applicable

NCT02061189

Pilot Study of an Innovative Physiotherapy in Patients With Infantile Spinal Muscular Atrophy (SMA)

This clinical trial aims to test a new physio-therapeutic approach tailored to type 2 and 3 Spinal Muscular Atrophy patients, based on physical training in swim…

France19 participantsStarted 2014-03

Treatment: Physical exercise in a swimming pool during 6 months