NCT04800874

Study of BBP-418 in Patients With LGMD2I

An Open Label Phase 2 Study of BBP-418 in Patients With Limb Girdle Muscular Dystrophy Type 2I (MLB-01-003)

• Status: Active Not Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 14 (actual)
• Sponsor: ML Bio Solutions, Inc. · Industry
• Sex: All
• Age: 12 Years – 55 Years
• Healthy volunteers: Not accepted

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.

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

Conditions

• LGMD2I

Interventions

Type	Name	Description
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 α-dystroglycan. The BBP-418 drug product will be packaged in sachets and provided in a carton for in-clinic and at home use.

Timeline

• Start date: 2021-02-18
• Primary completion: 2026-11-01
• Completion: 2026-11-01
• First posted: 2021-03-16
• Last updated: 2025-05-08

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study