Key Takeaway
The record is useful because it shows that corticosteroids were studied directly in FKRP-related LGMD2I, but the trial ended early and does not establish routine benefit.
Trial and Study Record
A Study of Deflazacort (Emflaza®) in Participants With Limb-Girdle Muscular Dystrophy 2I (LGMD2I)
Phase 3 corticosteroid study record evaluating deflazacort in adults with FKRP-related LGMD2I, ultimately terminated early.
TrialTherapy DevelopmentMonitoringFamilies
Why this record matters
- Helps readers distinguish between supportive-care practices borrowed from other dystrophies and treatment strategies actually studied in FKRP-related disease.
- The source record notes that the study was terminated early because of low enrollment and missing efficacy assessments related to missed COVID-era visits.
- Outcome measures are still valuable for understanding what functional and respiratory endpoints matter in FKRP trials.
Eligibility and participation notes
- Required genetically confirmed LGMD2I due to FKRP mutation and ability to complete the 4-stair climb test.
- Excluded participants with significant cardiomyopathy, full-time ventilator support, or recent continuous corticosteroid exposure.
- Used change in 4-stair climb after 26 weeks as the primary outcome, with pulmonary and strength outcomes also tracked.
Limits and cautions
- Early termination and small enrollment substantially limit interpretation.
- Open-label single-group design after protocol changes reduces the strength of any efficacy signal.
- The enrolled adult population does not cover the full FKRP disease spectrum.
Primary Sources
Direct links for verification
Primary source used for the structured study summary on this page.
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Phase 1 FKRP gene-transfer trial record for ATA-100, designed to assess safety, pharmacodynamics, and immunogenicity with long-term follow-up.