Limb Girdle Muscular Dystrophy 2I/R9
Conditions
Interventions
Product Name: BBP-418
Pharmaceutical Form: Oral powder
Sponsors
ML Bio Solutions
Eligibility
Sex/Gender
All
Inclusion criteria
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
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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: • Explore the safety and tolerability of ascending dose levels of BBP-418 in patients with Limb Girdle Muscular Dystrophy (LGMD)2I. ;Secondary Objective: • Explore the dose response of BBP-418 with regard to the glycosylation of aDG. • Explore the feasibility and usefulness of selected clinical efficacy and pharmacodynamic (PD) assessments in patients with LGMD2I receiving ascending doses of BBP-418. • Explore the pharmacokinetics (PK) of BBP-418 in patients with LGMD2I receiving ascending doses of BBP-418. ;Primary end point(s): Incidence of treatment-emergent adverse events (TEAEs) that lead to dose decrease or discontinuation;Timepoint(s) of evaluation of this end point: 15 months | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): • Pharmacokinetic profile of BBP-418 by assessment of maximum concentration (Cmax) • Changes in pharmacodynamic parameters by assessing changes in levels of N-terminal fragment of alpha dystroglycan (a-DG) • Changes in pharmacodynamic parameters by assessing muscle biopsy of the tibialis anterior • Pharmacokinetic profile of BBP-418 by assessment of area under the curve (AUC);Timepoint(s) of evaluation of this end point: 15 months | — |
Countries
United States
Contacts
Public ContactDoug Sproule
ML Bio Solutions
Outcome results
None listed