Open-Label Extension Study of BBP-418 (Ribitol) for LGMD2I/R9

Conditions

• Limb-Girdle Muscular Dystrophy Type 2I (LGMD2I)

Eligibility Criteria

Sex

ALL

Age

N/A - N/A

Healthy Volunteers

Not accepted

Interventions

• BBP-418 (ribitol) — DRUG
  Drug: BBP-418. Single arm. Participants who completed Study MLB-01-005 and meet eligibility criteria will receive BBP-418 (ribitol) taken orally twice daily at 9 or 12g (based on body weight measured) for up to 36 months.

Study Details

This is an open-label extension (rollover) study designed to evaluate the long-term safety and efficacy of BBP-418 (ribitol) in participants with limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9) who have previously participated in Study MLB-01-005 (Fortify). Participants will receive BBP-418 administered orally at protocol-defined doses and schedules. The study will assess long-term safety through monitoring of adverse events, clinical laboratory evaluations, and other safety assessments. Efficacy will be evaluated using functional measures and other clinical endpoints relevant to LGMD2I/R9. Participants will be followed for up to 36 months, with a final safety follow-up assessment conducted approximately 30 days after the last dose of study drug.

Key Dates

First listed

Jul 1, 2026

Start date

Jun 5, 2026

Status verified

Jun 2026

Primary completion

Oct 31, 2030

Completion

Oct 31, 2030

Study Design

Enrollment

107 participants (estimated)

Allocation

NA

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Arms

• Experimental: BBP-418
  Drug: BBP-418. Single arm.

Primary Outcome Measure

Frequency and severity of treatment-emergent adverse events to assess long-term safety of BBP-418. [ Time Frame: 36 months ]

Locations (12)

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