Limb Girdle Muscular Dystrophy Clinical Trial
Official title:
GRASP-01-003: Trial Readiness and Endpoint Assessment in LGMD R1
This is a 24-month, observational study of 100 participants with Limb Girdle Muscular Dystrophy type R1, also known as CAPN3.
Limb girdle muscular dystrophies (LGMD) are a group of over 30 heterogenous genetic disorders which have in common a pattern of weakness affecting proximal muscles of the shoulders and hips. LGMD type R1 (LGMDR1; also LGMD2A) is due to loss of function of the muscle structural gene calpain 3 (CAPN3) and causes progressive weakness and muscle wasting, which can lead to loss of ambulation or the ability to maintain a job. LGMDR1 is one of the most common LGMDs in the United States and has no FDA approved therapies but is amenable to gene replacement strategies, regenerative medicine approaches, or myostatin based approaches. There have been rapid advances in gene delivery therapies for Duchenne Muscular Dystrophy and for LGMDR4 that have set the stage for targeted therapeutic development for all LGMDs, and LGMDR1 in particular is at a crossroads: the pace of therapeutic development has outstripped the efforts at clinical trial preparedness. There is a need for a more rigorous natural history study to assist in the design of clinical trials; in particular, identifying biomarkers for early phase development and clinical outcome assessments (COAs) for drug approval studies. This study will enroll 100 subjects across participating sites in the GRASP-LGMD Research Consortium. No treatment will be administered as part of this study. A subset of 80 patients will undergo MR scans at selected imaging sites. Study visits will occur at Baseline Day 1, Baseline Day 2, Month 12, and Month 24. ;
Status | Clinical Trial | Phase | |
---|---|---|---|
Recruiting |
NCT04001595 -
Global FKRP Registry
|
||
Recruiting |
NCT05409079 -
Schulze Muscular Dystrophy Ability Clinical Study
|
N/A | |
Active, not recruiting |
NCT05906251 -
A Gene Transfer Study to Evaluate the Safety, Tolerability and Efficacy of SRP-6004 in Ambulatory Participants With Limb Girdle Muscular Dystrophy, Type 2B/R2 (LGMD2B/R2, Dysferlin [DYSF] Related)
|
Phase 1 | |
Recruiting |
NCT05230459 -
A Study to Evaluate the Safety of AB-1003 (Previously LION-101) in Subjects With Genetic Confirmation of LGMD2I/R9 (Part1)
|
Phase 1/Phase 2 | |
Recruiting |
NCT03981289 -
Defining Clinical Endpoints in Limb Girdle Muscular Dystrophy (LGMD)
|
||
Completed |
NCT02836418 -
Study to Evaluate the Long-Term Safety, Tolerability, and Biological Activity of ATYR1940 in Participants With Limb Girdle and Facioscapulohumeral Muscular Dystrophy (FSHD)
|
Phase 1/Phase 2 | |
Active, not recruiting |
NCT03930628 -
Limb-Girdle Muscular Dystrophy Type 2I in Norway
|
||
Completed |
NCT01066455 -
Cardiac Outcome Measures in Children With Muscular Dystrophy
|
N/A | |
Active, not recruiting |
NCT05206617 -
3 Year Follow up on ANO5 Patients
|
||
Completed |
NCT01126697 -
Clinical Trial of Coenzyme Q10 and Lisinopril in Muscular Dystrophies
|
Phase 2/Phase 3 | |
Withdrawn |
NCT02245711 -
Cell Therapy in Limb Girdle Muscular Dystrophy
|
Phase 1 | |
Withdrawn |
NCT02050776 -
Stem Cell Therapy in Limb Girdle Muscular Dystrophy
|
Phase 1 | |
Active, not recruiting |
NCT05876780 -
A Gene Transfer Single Dose Study to Evaluate the Safety, Tolerability and Efficacy of SRP-9003 in Non-Ambulatory and Ambulatory Participants With Limb Girdle Muscular Dystrophy, Type 2E/R4 (Beta-Sarcoglycan [β-SG] Deficiency)
|
Phase 1 | |
Recruiting |
NCT06378203 -
Rehabilitation in Muscular Dystrophies From the Hospital Facility to the Home: Pilot Project [RIMUDI]
|
N/A | |
Completed |
NCT04202627 -
Biomarker Development in LGMD2i
|
||
Not yet recruiting |
NCT06390566 -
Evolution of the Functional and Muscular State of Patients With Muscular Dystrophy 2A Belts
|
||
Completed |
NCT01081080 -
Cardiac Magnetic Resonance in Children With Muscular Dystrophy
|
N/A | |
Recruiting |
NCT05102799 -
MRI-phenotyping of Patients With Pathogenic Anoctamin 5 Variants
|