AN OPEN LABEL PHASE 2 STUDY OF BBP 418 IN PATIENTS WITH LIMB GIRDLE MUSCULAR DYSTROPHY TYPE 2I (MLB 01 003)

Phase 1

• Conditions: imb Girdle Muscular Dystrophy 2I/R9; Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]

• Registration Number: EUCTR2024-000202-16-Outside-EU/EEA
• Lead Sponsor: ML Bio Solutions

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2024-05-31
• Last Update Posted: 3 June 2024

Recruitment & Eligibility

• Status: A
• Sex: All
• Target Recruitment: Not specified

Inclusion Criteria

1. Be a male or female patient, ages 12 to 55 years of age (inclusive)
2. Have a body weight >30 kg
3. 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)
4. Able to complete the 10MWT in =12 seconds unaided (moderate disease”) or are with severe disease”/non-ambulatory as defined by being unable to complete the 10MWT unaided in >12 seconds.
Are the trial subjects under 18? yes
Number of subjects for this age range: 6
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 8
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

1. Evidence of clinically significant concomitant disease, including:
a. Any history of a gastrointestinal condition, including surgeries, which may affect absorption after oral administration
b. Any significant concomitant medical condition, including cardiac, pulmonary, renal, hepatic or endocrine disease other than that associated with LGMD2I
c. 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)
d. Any other laboratory, vital sign, ECG abnormality, or clinical history or finding that, in the Investigator’s opinion, is likely to unfavourably alter the risk-benefit of study participation, confound study results, or interfere with study conduct or compliance

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified