Key Takeaway
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.
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
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.
- The record describes sequential dose cohorts, safety follow-up through 60 months, and pharmacokinetic and biomarker outcomes.
- The July 2026 update lists estimated primary and overall completion on 2026-11-01. These are estimates, not completed milestones.
Eligibility and participation notes
- Specified ages 12 to 55 years, weight above 30 kg, and genetically confirmed, clinically affected LGMD2I.
- Required prior enrollment in natural-history study MLB-01-001 and included ambulatory and non-ambulatory participants.
- Additional medical and treatment exclusions apply; these selected criteria are not an individual eligibility assessment.
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.
- No results are posted in this registry record. That does not mean results have never been reported elsewhere.
Primary Sources
Direct links for verification
Primary registry or publication source used for the structured study summary on this page.
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