LGMD2I Clinical Trial
Official title:
An Open Label Phase 2 Study of BBP-418 in Patients With Limb Girdle Muscular Dystrophy Type 2I (MLB-01-003)
Verified date | April 2024 |
Source | ML Bio Solutions, Inc. |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Interventional |
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.
Status | Active, not recruiting |
Enrollment | 14 |
Est. completion date | November 1, 2026 |
Est. primary completion date | November 1, 2026 |
Accepts healthy volunteers | No |
Gender | All |
Age group | 12 Years to 55 Years |
Eligibility | 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. |
Country | Name | City | State |
---|---|---|---|
United States | Virginia Commonwealth University | Richmond | Virginia |
Lead Sponsor | Collaborator |
---|---|
ML Bio Solutions, Inc. |
United States,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Incidence of treatment-emergent adverse events (TEAEs) that lead to dose decrease or discontinuation | 60 months | ||
Secondary | Pharmacokinetic profile of BBP-418 by assessment of maximum concentration (Cmax) | 24 months | ||
Secondary | Changes in pharmacodynamic parameters by assessing changes in levels of N-terminal fragment of alpha dystroglycan (a-DG) | 24 months | ||
Secondary | Changes in pharmacodynamic parameters by assessing muscle biopsy of the tibialis anterior | 24 months | ||
Secondary | Pharmacokinetic profile of BBP-418 by assessment of area under the curve (AUC) | 24 months |
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