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 1/2 placebo-controlled gene-therapy study in adults with genetically confirmed FKRP-related LGMD2I/R9.
Why it matters: The current registry record lists nine actual participants and active follow-up without recruitment. It does not establish access, eligibility, safety, or benefit outside the protocol.
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 Phase 1 record describes six participants in an FKRP gene-transfer study with long-term follow-up. It is listed as active, not recruiting; it does not establish clinical benefit or offer enrollment.
Trial · Active, not recruiting
Phase 3 randomized ribitol trial in FKRP-related LGMD2I/LGMDR9, with trial details and separately dated sponsor-reported regulatory updates.
Why it matters: FORTIFY is listed as active, not recruiting. The sponsor has reported interim results and regulatory review, but a trial record and an application under review do not establish treatment availability.
Observational study · Recruiting in registry record
Prospective University of Iowa natural-history study collecting clinical, functional, respiratory, quality-of-life, and selected biospecimen data across dystroglycanopathies.
Why it matters: This long-running observational study underlies several FKRP publications. The registry says recruiting, but status and participation must be confirmed with the current study source.
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: ClinicalTrials.gov lists this registry as Unknown, which does not establish whether registration is open or closed. Ask the Newcastle registry team about current participation.
Observational study · Recruiting in registry record
A 24-month observational study evaluating clinical and patient-reported outcome measures across selected muscular dystrophies, including LGMD subtypes.
Why it matters: This study evaluates outcome measures across several muscular-dystrophy groups. It is not an FKRP treatment study, and the registry listing does not establish current site availability or individual eligibility.
Observational study · Published cohort study
Norwegian observational study describing FKRP-related disease epidemiology and clinical data from 101 people, published in 2023.
Why it matters: This study describes respiratory, cardiac, and mobility patterns in a Norwegian cohort. It cannot predict an individual's disease course.
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: This open-label Phase 2 record describes 14 participants and remains active, not recruiting. It provides early-development context for BBP-418 without establishing definitive treatment benefit.
No study records matched that search.