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Clinical Trial Details — Status: Active, not recruiting

Administrative data

NCT number NCT04800874
Other study ID # MLB-01-003
Secondary ID
Status Active, not recruiting
Phase Phase 2
First received
Last updated
Start date February 18, 2021
Est. completion date November 1, 2026

Study information

Verified date April 2024
Source ML Bio Solutions, Inc.
Contact n/a
Is FDA regulated No
Health authority
Study type Interventional

Clinical Trial 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 levels of BBP-418 in the treatment of ambulatory and non-ambulatory patients with LGMD2I for which no approved therapy currently exists.


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 safety and tolerability of ascending dose levels of BBP-418 in those subjects.


Recruitment information / eligibility

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.

Study Design


Related Conditions & MeSH terms


Intervention

Drug:
BBP-418
BBP-418 is being developed for the treatment of patients with Limb-Girdle Muscular Dystrophy Type 2I (LGMD2I) for which no approved therapy currently exists. It targets the molecular defect at the source by supplying excess substrate to the mutant enzyme thus boosting glycosylation of muscle a-dystroglycan. The BBP-418 drug product will be packaged in sachets and provided in a carton for in-clinic and at home use.

Locations

Country Name City State
United States Virginia Commonwealth University Richmond Virginia

Sponsors (1)

Lead Sponsor Collaborator
ML Bio Solutions, Inc.

Country where clinical trial is conducted

United States, 

Outcome

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
See also
  Status Clinical Trial Phase
Recruiting NCT05989620 - Long-Term Development of Muscular Dystrophy Outcome Assessments
Recruiting NCT04001595 - Global FKRP Registry
Recruiting NCT05230459 - A Study to Evaluate the Safety of AB-1003 (Previously LION-101) in Subjects With Genetic Confirmation of LGMD2I/R9 (Part1) Phase 1/Phase 2
Completed NCT02841267 - A Trial of PF-06252616 in Ambulatory Participants With LGMD2I Phase 1/Phase 2
Active, not recruiting NCT03842878 - Natural History Study of Patients With Limb-Girdle Muscular Dystrophy 2I