Effects of BBP-418 (ribitol) in Patients with LGMD2I
- Conditions
- imb Girdle Muscular Dystrophy 2I/R9MedDRA version: 20.0Level: PTClassification code: 10028356Term: Muscular dystrophy Class: 100000004850Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
- Registration Number
- CTIS2023-503379-33-01
- Lead Sponsor
- ML Bio Solutions Inc.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Recruiting
- Sex
- All
- Target Recruitment
- 122
Have a genetically confirmed diagnosis of LGMD2I/ R9 (including review of records of previous molecular genetic testing) and be clinically affected (defined as demonstrating clinical weakness on bedside evaluation in either a limb-girdle pattern, or in a distal extremity)., Male or female participants 18 to 60 years of age (inclusive)., Have a body weight >30 kg., The participant who signs the informed consent form (ICF) understands the study procedures and the participant agrees to participate in the study by giving informed consent., Women of childbearing potential (WOCBP) and male participants of reproductive potential must be willing to use a highly effective method of contraception from time of consent through 12 weeks after last dose., Willing and able to complete all study procedures, including biopsies, according to the Schedule of Assessments.
Evidence of clinically significant concomitant disease, including: Any significant concomitant medical condition, including cardiac, renal, pulmonary, hepatic, or endocrine disease other than that associated with LGMD2I/ R9; Moderate to severe renal impairment (eGFR < 60 mL/min/1.73 m2 based on cystatin C[CysC], as calculated by central laboratory; Any other laboratory, vital sign, ECG abnormality, 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; Surgery for scoliosis or other indication that will significantly impact the participant’s ability to execute clinical assessments planned or expected to be required to manage curvature within 12 months following the Screening Visit., Previously received gene therapy to treat LGMD2I/ R9., Participants with active suicidal ideation as measured by Columbia-Suicide Severity Rating Scale during screening with most severe suicide ideation score of 4 (Active Suicidal Ideation with Some Intent to Act, without Specific Plan) or 5 (Active Suicidal Ideation with Specific Plan and Intent)., Presence of a platelet disorder, bleeding disorder, or other contraindication to muscle biopsy., Actively on an experimental therapy or device, was on an experimental therapy or device within 90 days prior to the Screening Visit, or was on BBP-418 at any time, In the judgment of the Investigator or Medical Monitor, has any clinically important ongoing medical condition or laboratory abnormality or condition that might jeopardize the participant’s safety, increase their risk from participation, or interfere with the study. For COVID-19 infections, Investigator should refer to local guidance., A participant with a score of zero on any one or more of the primary or key secondary endpoints at the time of screening. (Participants who previously completed participation in Study MLB-01-001 and would be excluded due to this criterion may enrol in this study provided all inclusion and no other exclusion criteria are met.), 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., Use of ribose or other sugar alcohol-containing supplement within 90 days of the Screening Visit., Use of a systemic corticosteroid for the treatment of muscular dystrophy within 90 days of the Screening Visit. (An inhaled corticosteroid or bronchodilator for reactive airway disease is allowed if the participant is on a stable dose for 30 days prior to study entry.)
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Main Objective: To assess the clinical efficacy and safety of BBP-418 in patients with LGMD2I/ R9;Secondary Objective: To assess the clinical efficacy of BBP-418 in patients with LGMD2I/ R9, To assess biomarkers for clinical efficacy of BBP-418 in patients with LGMD2I/ R9;Primary end point(s): Change from baseline in NSAD at 36 months, Frequency and severity of TEAEs and TESAEs, Results of physical examinations including vital signs, Chemistry and hematology laboratory analyses, 12-lead ECG, including QTc intervals
- Secondary Outcome Measures
Name Time Method Secondary end point(s):Change from baseline in 10MWT (velocity, m/s) at 36 months;Secondary end point(s):Change from baseline in pulmonary function as measured by FVC (percent predicted, performed in a sitting position) at 36 months;Secondary end point(s):Change from baseline in the PUL2.0 at 36 months;Secondary end point(s):Change from baseline in NSAD at 36 months;Secondary end point(s):Change from baseline in total glycosylated aDG expression;Secondary end point(s):Change from baseline in glycosylated aDG/ total aDG ratio;Secondary end point(s):Change from baseline in pre-functional assessment serum CK