Infantile Spinal Muscular Atrophy

Clinical trial pipeline · Data from ClinicalTrials.gov

See which Infantile Spinal Muscular Atrophy trials you may qualify for

Total trials

Actively recruiting

Phase 3 trials

Completed

Trial phase breakdown

Trial status

Actively recruiting — 5 trials

RecruitingNot Applicable

NCT06562283

Evaluation of the Reproducibility of a Fatigability Test Fitted to Patients With Spinal Muscular Atrophy

Spinal muscular atrophy (SMA) is an autosomal recessive neuromuscular disease caused by the degeneration of motor neurons in the anterior horn of the spinal cor…

France80 participantsStarted 2024-12-06

Treatment: Thumb test, Grip test

RecruitingPhase 1/2

NCT07070999

Study of Safety, Tolerability and Efficacy of GB221 in Infants With Spinal Muscular Atrophy Type 1

GB221 is a gene therapy that delivers a working SMN1 gene to the motor neurons of people with spinal muscular atrophy (SMA) Type 1. This study will evaluate the…

Brazil22 participantsStarted 2026-01-06

Treatment: GB221

RecruitingNot Applicable

NCT06152302

Test of Aquatic Mobility of SMA Infants

Spinal muscular atrophy (SMA) is a genetic neurodegenerative disease impacting spinal cord motor neurons, leading to motor and respiratory issues and, ultimatel…

France15 participantsStarted 2025-06-26

Treatment: Mobility study in dry conditions, Mobility study in water conditions

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

RecruitingNot Applicable

NCT05954455

Exploring Bulbar Function, Speech And Communication Development in SMA Type 1

5q-spinal muscular atrophy (5q-SMA) is a rare, autosomal recessive neuromuscular disease characterized by degeneration of motor neurons in the spinal cord and l…

United Kingdom30 participantsStarted 2024-03-21

All recent trials (36 shown)

Not Yet RecruitingPhase 1/2

NCT07554924

A Phase I/II Clinical Study to Evaluate SKG0201 Injection in Subjects With Spinal Muscular Atrophy Type I

This is a phase I/II clinical study to evaluate the safety, preliminary efficacy and immunogenicity of SKG0201 injection in subjects with Spinal Muscular Atroph…

China11 participantsStarted 2026-05-15

Treatment: SKG0201 Injection

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

Infantile Spinal Muscular Atrophy

Trial phase breakdown

Trial status

Actively recruiting — 5 trials

Evaluation of the Reproducibility of a Fatigability Test Fitted to Patients With Spinal Muscular Atrophy

Study of Safety, Tolerability and Efficacy of GB221 in Infants With Spinal Muscular Atrophy Type 1

Test of Aquatic Mobility of SMA Infants

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

Exploring Bulbar Function, Speech And Communication Development in SMA Type 1

All recent trials (36 shown)

A Phase I/II Clinical Study to Evaluate SKG0201 Injection in Subjects With Spinal Muscular Atrophy Type I

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

Trial phase breakdown

Trial status

Actively recruiting — 5 trials

Evaluation of the Reproducibility of a Fatigability Test Fitted to Patients With Spinal Muscular Atrophy

Study of Safety, Tolerability and Efficacy of GB221 in Infants With Spinal Muscular Atrophy Type 1

Test of Aquatic Mobility of SMA Infants

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

Exploring Bulbar Function, Speech And Communication Development in SMA Type 1

All recent trials (36 shown)

A Phase I/II Clinical Study to Evaluate SKG0201 Injection in Subjects With Spinal Muscular Atrophy Type I

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

A Clinical Study Evaluating the Safety and Efficacy of SKG0201 Injection in Patients With Spinal Muscular Atrophy Type 1

Psychological Evaluation of the Parental Experience of Newborn Screening for Infantile Spinal Muscular Atrophy in the Gr…

Palliative Care in Spinal Muscular Atrophy (SMA) 1

The Use of Assistive Gait Devices Can Reduce the Risk of Falls in Patients With Neuromuscular Diseases Following a Train…

Long-Term Follow-up Study for Patients From AVXS-101-CL-101

Long-term Follow-up Study of Patients Receiving Onasemnogene Abeparvovec-xioi

Impact of Motor and Oral Motor Function on Quality of Life in Children With SMA

Therapeutic Management and Use of Resources and Costs of Spinal Muscular Atrophy in Spain

The Effect of Spinal Orthosis on the Development of Scoliosis and Chest Deformity in Type I Spinal Muscular Atrophy

Clinical Trial to Assess the Safety and Efficacy of EXG001-307 in Patients with Spinal Muscular Atrophy Type 1

Clinical Trial to Evaluate the Safety and Efficacy of EXG001-307 in Patients With Spinal Muscular Atrophy Type I

Body Weight Support Harness System in Spinal Muscular Atrophy

Longitudinal Data Collection in Pediatric and Adult Patients With Spinal Muscular Atrophy in Latin America

Sleep Spindles Organization as an Early Neural Marker of Neuromotor Outcome

Gene Transfer Clinical Trial for Spinal Muscular Atrophy Type 1

Trial Evaluating the Interest of Noninvasive Ventilation in NAVA Mode in Respiratory Decompensations Children With Infan…

European Registry of Patients With Infantile-onset Spinal Muscular Atrophy

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