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 · Unknown
International FKRP patient-registry record designed to characterize the population, support standards of care work, and help identify participants for future studies. ClinicalTrials.gov currently lists the study status as Unknown because the record has not been verified since January 2024.
Why it matters: The Global FKRP Registry remains important public research infrastructure, but its ClinicalTrials.gov status is Unknown; readers should not interpret the last known Recruiting status as confirmation that enrollment is currently open.
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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