Study of BBP-418 in Patients With LGMD2I

Running, not enrolling · Phase 2

Conditions studied: LGMD2I

In brief

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.

Key facts

Study ID

NCT04800874

Run by

ML Bio Solutions, Inc.

People needed

14

Starts

2021-02-18

Expected to finish

2026-11-01

Last updated by the study team

2026-07-21

Who can join

Age: 12 and older, up to 55. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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

You may not qualify if…

• 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.

Where it is running

• Virginia Commonwealth University — Richmond, Virginia, United States

Full record on ClinicalTrials.gov

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