Reference Public rare-disease knowledge base

Trial and Study Record

Study of BBP-418 in Patients With LGMD2I

Phase 2 open-label ribitol study in FKRP-related LGMD2I/LGMDR9 that provides the main early clinical development context for the BBP-418 program.

TrialTherapy DevelopmentTrial ReadinessFamilies

Key Takeaway

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.

Why this record matters

  • Provides important background for readers trying to understand why the ribitol track advanced into late-stage development.
  • Helps the site distinguish between early clinical development and later confirmatory or regulatory phases.
  • Supports careful wording around sponsor-reported updates because the public record remains that of an active study.

Eligibility and participation notes

  • Enrolled adults with genetically confirmed LGMD2I due to FKRP variants.
  • Used an open-label design with sequential dose cohorts rather than a blinded placebo comparison.
  • Focused on safety, pharmacokinetics, and exploratory functional or biomarker endpoints.

Limits and cautions

  • Open-label design limits the strength of efficacy interpretation.
  • Small enrollment and early-phase aims mean the study should not be treated as definitive proof of benefit.
  • Public understanding still depends on later publications and the phase 3 record.

Primary Sources

Direct links for verification

Back to Trials and Studies

Related Content

Related routes inside the site

Study Record

ATA-100 (Formerly GNT0006) Gene Therapy Trial in Patients With LGMDR9

Phase 1 FKRP gene-transfer trial record for ATA-100, designed to assess safety, pharmacodynamics, and immunogenicity with long-term follow-up.

Study Record

BBP-418 (Ribitol) FORTIFY Trial in LGMD2I/LGMDR9

Phase 3 randomized ribitol trial in FKRP-related LGMD2I/LGMDR9 and the most advanced current interventional program identified in this FKRP research sweep.

Study Record

Global FKRP Registry

International FKRP patient-registry record designed to characterize the population, support standards of care work, and help identify participants for future studies.