Clinical trials: LGMDR9
Muscular Dystrophies, Limb Girdle Muscular Dystrophy
Biomarker Development in LGMD2i
The overall goal of this natural history study is to define the key LGMD2i phenotypes as measured by standard clinical outcome assessments (COAs), and to validate a muscle biomarker for LGMD2i to support therapeutic development.
- Trial name
- MLB-01-001 (NCT04202627)
- Where
- 11 sites in United States, Denmark
- Volunteers
- 101 people
- Sponsor
- ML Bio Solutions, Inc.
Limb-Girdle Muscular Dystrophy
A Study of Deflazacort (Emflaza®) in Participants With Limb-Girdle Muscular Dystrophy 2I (LGMD2I)
This study is designed to evaluate the safety and efficacy of deflazacort in participants with LGMD2I. Most participants enrolled will have a screening visit and 3 additional visits (after 1, 13, and 26 weeks of treatment).
- Trial name
- NCT03783923
- Testing
- Deflazacort
- Where
- 18 sites in United States, Canada and 6 more
- Volunteers
- 11 people
- Sponsor
- PTC Therapeutics
Limb Girdle Muscular Dystrophy, Limb-Girdle Muscular Dystrophy Type 2
A Study to Evaluate the Safety of AB-1003 (Previously LION-101) in Subjects With Genetic Confirmation of LGMD2I/R9 (Part1)
The purpose of this study is to evaluate the safety and tolerability of a single intravenous infusion of AB-1003 in adults diagnosed with limb girdle muscular dystrophy type 2I/R9 (LGMD2I/R9).
- Conditions
- Limb Girdle Muscular Dystrophy, Limb-Girdle Muscular Dystrophy Type 2, LGMD2I, Muscular Dystrophy
- Trial name
- NCT05230459
- Testing
- AB-1003 dose level 1, AB-1003 dose level 2
- Where
- 6 sites in United States
- Volunteers
- 9 people
- Sponsor
- AskBio Inc
LGMDR9
ATA-100 (Formerly GNT0006) Gene Therapy Trial in Patients With LGMDR9
Phase 1 dose escalation study to assess tolerability and safety of ATA-100 with 5-year follow-up
- Trial name
- NCT05224505
- Testing
- ATA-100 (AAV9 encoding FKRP gene)
- Where
- 3 sites in Denmark, France and 1 more
- Volunteers
- 6 people
- Sponsor
- Atamyo Therapeutics
Limb-Girdle Muscular Dystrophy Type 2I (LGMD2I)
Study to Evaluate the Efficacy and Safety of BBP-418 (Ribitol) in Patients With Limb Girdle Muscular Dystrophy 2I (LGMD2I)
This study will evaluate the safety and efficacy of long-term administration of BBP-418 in patients with LGMD2I/R9. The study will include patients ages 12 to 60, consistent with the existing preclinical toxicology profile.
- Trial name
- Fortify (NCT05775848)
- Testing
- BBP-418 (ribitol)
- Where
- 21 sites in United States, Australia and 6 more
- Volunteers
- 112 people
- Sponsor
- ML Bio Solutions, Inc.
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.