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A Phase 3 Randomized, Placebo-controlled, Double-blind Study to Evaluate the Efficacy and Safety of BBP-418 (ribitol) in Patients with Limb Girdle Muscular Dystrophy 21 (LGMD21)

Recruiting

This study will use BBP-418 study drug in patients with LGMD to assess the clinical biomarkers, efficacy and safety of BBP-418 during the 36 months treatment phase.

I'm interested

Male or Female
Not specified
This study is NOT accepting healthy volunteers
Inclusion Criteria:

• 12 to 60 years of age
• genetically confirmed diagnosis of limb girdle muscular dystrophy
• have clinical symptoms of weakness
• weight at least 30 kg (66 lbs.)
• willing to use a highly effective method of birth control until 12 weeks after last dose of study medication
Exclusion Criteria:

• any significant medical or mental health diagnosis including abnormal lab values (study staff will review)
• surgery for scoliosis or other indication planned during the time of the study
• use of ribose or other sugar alcohol-containing supplement within 90 days of staring the study
• use of a systemic corticosteroid for the treatment of muscular dystrophy within 90 days of starting the study

Rare Diseases

Clinics and Surgery Center (CSC), Limb-Girdle Muscular Dystrophy Type 2I (LGMD2I), Muscular Dystrophy

Allison Johnston - joh21779@umn.edu
Peter Kang
PHASE3
STUDY00018570
See this study on ClinicalTrials.gov

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