Phase 3 Study Tracking
Structured FKRP trial, registry, and observational-study records with source links, status notes, and practical relevance.
Trial · Terminated
Phase 3 corticosteroid study record evaluating deflazacort in adults with FKRP-related LGMD2I, ultimately terminated early.
Why it matters: 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 · Active, not recruiting
Phase 1 FKRP gene-transfer trial record for ATA-100, designed to assess safety, pharmacodynamics, and immunogenicity with long-term follow-up.
Why it matters: This is the main currently listed FKRP-targeted gene-therapy trial record and it is important because it shows who was eligible, what safety questions were being studied, and where the field stands clinically.
Trial · Active, not recruiting
Phase 3 randomized ribitol trial in FKRP-related LGMD2I/LGMDR9 and the most advanced current interventional program identified in this FKRP research sweep.
Why it matters: FORTIFY is the main late-stage FKRP-directed ribitol trial in the public record, but its status still needs to be described carefully as ongoing phase 3 work rather than settled treatment evidence.
Registry · Recruiting
International FKRP patient-registry record designed to characterize the population, support standards of care work, and help identify participants for future studies.
Why it matters: The Global FKRP Registry is one of the most important public infrastructure records in this space because it supports natural-history work, care standards, and future trial recruitment.
Observational study · Published cohort study
Published observational cohort describing prevalence, genotype distribution, ventilatory support, and cardiomyopathy patterns in a large Norwegian FKRP population.
Why it matters: This cohort is a strong natural-history reference because it links ventilatory support, wheelchair dependency, and cardiomyopathy patterns to real published follow-up data.
Trial · Active, not recruiting
Phase 2 open-label ribitol study in FKRP-related LGMD2I/LGMDR9 that provides the main early clinical development context for the BBP-418 program.
Why it matters: The phase 2 BBP-418 study is useful because it shows how the ribitol program was framed clinically before the later phase 3 trial, but it should still be read as an ongoing interventional record rather than finished efficacy evidence.
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