LGMD2E

Clinical trial pipeline · Data from ClinicalTrials.gov

See which LGMD2E trials you may qualify for

Total trials

Actively recruiting

Phase 3 trials

Completed

Trial phase breakdown

Trial status

Actively recruiting — 1 trial

RecruitingNot Applicable

NCT05989620

Long-Term Development of Muscular Dystrophy Outcome Assessments

This is a 24-month, observational study of up to 1000 participants with Limb Girdle Muscular Dystrophy (LGMD), Myotonic Dystrophy Type 2 (DM2), and late onset P…

United States1,000 participantsStarted 2023-10-18

All recent trials (9 shown)

Active — Not RecruitingPhase 3

NCT06246513

A Trial to Learn More About an Experimental Gene Therapy Called Bidridistrogene Xeboparvovec (SRP-9003) as a Possible Tr…

This is a multicenter, global study of the effects of a single systemic dose of SRP-9003 on beta-sarcoglycan (β-SG) gene expression in participants with limb-gi…

United States, Belgium17 participantsStarted 2024-01-15

Treatment: SRP-9003, Glucocorticoid

Active — Not RecruitingPhase 1

NCT05876780

A Gene Transfer Single Dose Study to Evaluate the Safety, Tolerability and Efficacy of SRP-9003 in Non-Ambulatory and Am…

The primary purpose of this study is to evaluate the safety of SRP-9003 and to quantify expression of β-SG in the skeletal muscle of participants with limb-gird…

United States6 participantsStarted 2022-12-19

Treatment: SRP-9003

LGMD2E

Trial phase breakdown

Trial status

Actively recruiting — 1 trial

Long-Term Development of Muscular Dystrophy Outcome Assessments

All recent trials (9 shown)

A Trial to Learn More About an Experimental Gene Therapy Called Bidridistrogene Xeboparvovec (SRP-9003) as a Possible Tr…

A Gene Transfer Single Dose Study to Evaluate the Safety, Tolerability and Efficacy of SRP-9003 in Non-Ambulatory and Am…

Trial phase breakdown

Trial status

Actively recruiting — 1 trial

Long-Term Development of Muscular Dystrophy Outcome Assessments

All recent trials (9 shown)

A Trial to Learn More About an Experimental Gene Therapy Called Bidridistrogene Xeboparvovec (SRP-9003) as a Possible Tr…

A Gene Transfer Single Dose Study to Evaluate the Safety, Tolerability and Efficacy of SRP-9003 in Non-Ambulatory and Am…

Gene Delivery Clinical Trial of SRP-9003 (Bidridistrogene Xeboparvovec) for Participants With Limb-Girdle Muscular Dystr…

A Study of the Natural History of Participants With LGMD2E/R4, LGMD2D/R3, LGMD2C/R5, and LGMD2A/R1 ≥ 4 Years of Age, Who…

Natural History of Limb Girdle Muscular Dystrophy Type 2A and Type 2E

Prospective, Non-interventional, Multi-center, Post-authorization Safety Study of Riociguat for Pulmonary Arterial Hyper…

Limb Girdle Muscular Dystrophy Type 2E Recruitment Study

Clinical Determinants of Disease Progression in Patients With Limb Girdle Muscular Distrophy Type 2E