Status:
ACTIVE_NOT_RECRUITING
Study of BBP-418 in Patients With LGMD2I
Lead Sponsor:
ML Bio Solutions, Inc.
Conditions:
LGMD2I
Eligibility:
All Genders
12-55 years
Phase:
PHASE2
Brief Summary
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 le...
Detailed Description
This is an open label study in ambulatory and non-ambulatory subjects with LGMD2I (also known as LGMD R9) previously enrolled in the natural history Study MLB-01-001. This is a study to determine the ...
Eligibility Criteria
Inclusion
- 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
- 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.
Key Trial Info
Start Date :
February 18 2021
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
November 1 2026
Estimated Enrollment :
14 Patients enrolled
Trial Details
Trial ID
NCT04800874
Start Date
February 18 2021
End Date
November 1 2026
Last Update
May 8 2025
Active Locations (1)
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1
Virginia Commonwealth University
Richmond, Virginia, United States, 23219