Study of BBP-418 in Patients With LGMD2I

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)

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT04800874
• Other study ID #: MLB-01-003
• Status: Active, not recruiting
• Phase: Phase 2
• 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
• 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.

Related Conditions & MeSH terms

• LGMD2I

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