An Open Label Phase 2 Study of BBP-418 in Patients With Limb Girdle Muscular Dystrophy Type 2I (MLB-01-003)

MLB-01-003

BBP-418 is being developed for the treatment of patients with Limb-Girdle Muscular Dystrophy Type 2I (LGMD2I). This is an open label study to determine the safety and tolerability of ascending dose levels of BBP-418 in the treatment of ambulatory and non-ambulatory patients with LGMD2I for which no approved therapy currently exists.

2021-02-18

2026-11-01

2026-11-01

Active (not recruiting)

Early phase I

14

Inclusion Criteria: * Have a body weight \>30 kg * Have a genetically confirmed diagnosis of LGMD2I and be clinically affected (defined as demonstrating clinical weakness on bedside evaluation in either a limb-girdle pattern, or in a distal extremity) * Able to complete the 10-meter walk test in ≤ 12 seconds unaided ("moderate disease") or are with "severe disease"/non-ambulatory as defined by being unable to complete the 10-meter walk unaided in \>12 seconds * Willing to use an adequate method of contraception from time of consent through 12 weeks after last dose * Previous enrolment in the Natural History study MLB-01-001 Exclusion Criteria: * Evidence of clinically significant concomitant disease, including: * Any history of a gastrointestinal condition, including surgeries, which may affect absorption after oral administration * Any significant concomitant medical condition, including cardiac, pulmonary, renal, hepatic or endocrine disease other than that associated with LGMD2I * Any condition other than LGMD2I requiring therapy with prescription medicine (medication for common and mild concomitant conditions may be permitted after consultation with the PI) * Any other laboratory, vital sign, ECG abnormality, or clinical history or finding that, in the investigator's opinion, is likely to unfavorably alter the risk-benefit of study participation, confound study results, or interfere with study conduct or compliance * If pregnant and/or breastfeeding or planning to conceive children within the projected duration of the study through 12 weeks after the last dose of study treatment. * History of drug abuse including alcoholism within 2 years prior to consenting * Use of ribose or other sugar alcohol-containing supplement within 60 days of Day 1 * Use of a corticosteroid within 60 days of Day 1 * Presence of a platelet disorder, bleeding disorder or other contraindication to muscle biopsy * Actively on an experimental therapy or device or was on an experimental therapy or device within 60 days prior to Day 1.

{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE2'], 'designInfo': {'allocation': 'NON_RANDOMIZED', 'interventionModel': 'SEQUENTIAL', 'interventionModelDescription': 'Subjects must have been enrolled in the MLB-01-001 study to participate in this trial. Three ascending dose cohorts of 4-6 subjects each are planned.', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'NONE'}}, 'enrollmentInfo': {'count': 14, 'type': 'ACTUAL'}}

2024-04-22