SMARD1

Clinical trial pipeline · Data from ClinicalTrials.gov

See which SMARD1 trials you may qualify for

Total trials

Actively recruiting

Phase 3 trials

Completed

Trial phase breakdown

Trial status

Actively recruiting — 1 trial

RecruitingNot Applicable

NCT04292574

UK SMA Patient Registry

Spinal muscular atrophy (SMA) is a form of motor neuron disease, most commonly caused by a mutation in the survival motor neuron 1 gene (SMN1) which results in…

United Kingdom800 participantsStarted 2008-07-13

Treatment: Patient Registry

All recent trials (5 shown)

Active — Not RecruitingPhase 1/2

NCT07223632

Treatment of Charcot-Marie-Tooth Disease, Axonal, Type 2S (CMT2S) in an Individual Patient

This is an 'N of 1', open-label, single center study to evaluate the safety of therapy with VCA-894A, an ASO designed to rescue and restore the activity of IGHM…

United States1 participantsStarted 2025-05-13

Treatment: VCA-894A

By InvitationPhase 1/2

NCT05152823

Gene Therapy for IGHMBP2-Related Diseases

Open-label, single intrathecal injection study of a AAV9 vector carrying the IGHMBP2 gene for IGHMBP2-related diseases.

United States10 participantsStarted 2021-11-04

Treatment: Gene Therapy

SMARD1

Trial phase breakdown

Trial status

Actively recruiting — 1 trial

UK SMA Patient Registry

All recent trials (5 shown)

Treatment of Charcot-Marie-Tooth Disease, Axonal, Type 2S (CMT2S) in an Individual Patient

Gene Therapy for IGHMBP2-Related Diseases

Trial phase breakdown

Trial status

Actively recruiting — 1 trial

UK SMA Patient Registry

All recent trials (5 shown)

Treatment of Charcot-Marie-Tooth Disease, Axonal, Type 2S (CMT2S) in an Individual Patient

Gene Therapy for IGHMBP2-Related Diseases

Genetics of Pediatric-Onset Motor Neuron and Neuromuscular Diseases

Muscle Structure, Function and Gait in dHMN