Clinical trials: Limb-girdle Muscular Dystrophy
Limb Girdle Muscular Dystrophy
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)
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-girdle muscular dystrophy, type 2E/R4 (LGMD2E/R4).
- Trial name
- NCT05876780
- Testing
- SRP-9003
- Where
- 2 sites in United States
- Volunteers
- 6 people
- Sponsor
- Sarepta Therapeutics, Inc.
Migraine
Safety and Tolerability of Frovatriptan to Prevention of Menstrually Associated Migraine (MAM) Headaches
In this open-label, multi-center study, patients were treated with frovatriptan for a maximum of 12 perimenstrual periods (PMPs).
- Trial name
- MAM (NCT01035983)
- Testing
- Frovatriptan 2.5 mg
- Where
- 5 sites in Germany, Hungary and 3 more
- Volunteers
- 550 people
- Sponsor
- Endo USA Inc., a Keenova Therapeutics Company
Limb-Girdle Muscular Dystrophy ; Subtypes 2A, 2B, and 2I
Limb-Girdle Video Assessment
The purpose of this study is to develop a new remote-based video assessment outcome measure for Limb-Girdle Muscular Dystrophy (LGMD) trials. The overall objectives for this study are: 1.
- Trial name
- NCT05528757
- Where
- United States
- Volunteers
- 45 people
- Sponsor
- The Emmes Company, LLC
Muscular Dystrophies, Limb-Girdle
A Study of SRP-9005 in Limb Girdle Muscular Dystrophy Type 2C/R5 Pediatric and Adult Participants
This is a study of a single systemic dose of SRP-9005 in pediatric and adult participants with limb girdle muscular dystrophy type 2C/R5 (LGMD2C/R5).
- Trial name
- COMPASS (NCT06952686)
- Testing
- SRP-9005, Corticosteroid
- Where
- United States
- Sponsor
- Sarepta Therapeutics, Inc.
Becker Muscular Dystrophy, Facioscapulohumeral Muscular Dystrophy
Study Evaluating MYO-029 in Adult Muscular Dystrophy
The purpose of this phase I/II, multicenter, safety trial is to study MYO-029 in adult patients with muscular dystrophy.
- Conditions
- Becker Muscular Dystrophy, Facioscapulohumeral Muscular Dystrophy, Limb-Girdle Muscular Dystrophy
- Trial name
- NCT00104078
- Testing
- MYO-029
- Where
- 9 sites in United States
- Volunteers
- 108 people
- Sponsor
- Wyeth is now a wholly owned subsidiary of Pfizer
Limb-Girdle Muscular Dystrophy Type 2I (LGMD2I)
Open-Label Extension Study of BBP-418 (Ribitol) for LGMD2I/R9
This is an open-label extension (rollover) study designed to evaluate the long-term safety and efficacy of BBP-418 (ribitol) in participants with limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9) who have previously…
- Trial name
- NCT07678775
- Testing
- BBP-418 (ribitol)
- Where
- 20 sites in United States, Australia and 6 more
- Volunteers
- 107 people
- Sponsor
- ML Bio Solutions, Inc.
LGMD2I
Study of BBP-418 in Patients With LGMD2I
BBP-418 is being developed for the treatment of patients with Limb-Girdle Muscular Dystrophy Type 2I (LGMD2I). This is an open label study to determine the safety and tolerability of ascending dose levels of BBP-418 in the…
- Trial name
- NCT04800874
- Testing
- BBP-418
- Where
- United States
- Volunteers
- 14 people
- Sponsor
- ML Bio Solutions, Inc.
Limb-Girdle Muscular Dystrophies, Facioscapulohumeral Muscular Dystrophy
Evaluate Safety and Biological Activity of ATYR1940 in Participants With Limb Girdle Muscular Dystrophy 2B (LGMD2B) and Facioscapulohumeral Muscular Dystrophy (FSHD)
The purpose of this study is to assess the safety and biological activity of ATYR1940 in participants with LGMD2B and FSHD.
- Trial name
- FSHD (NCT02579239)
- Testing
- ATYR1940
- Where
- 6 sites in United States, Denmark and 1 more
- Volunteers
- 18 people
- Sponsor
- aTyr Pharma, Inc.