Study to Evaluate the Efficacy and Safety of BBP-418 (Ribitol) in Patients With Limb Girdle Muscular Dystrophy 2I (LGMD2I)
NCT ID: NCT05775848
Last Updated: 2025-09-29
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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ACTIVE_NOT_RECRUITING
PHASE3
81 participants
INTERVENTIONAL
2023-05-31
2027-07-31
Brief Summary
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Detailed Description
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Conditions
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Study Design
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RANDOMIZED
PARALLEL
\[NOTE: EU region will only enroll 18 to 60 years of age\]
TREATMENT
QUADRUPLE
Study Groups
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BBP-418
BBP-418 Granules for Oral Solution will be supplied as granules in tri-ply PET/Aluminum/PE sachets for unit dose. The number of sachets to reconstitute will depend on the applicable dose to be delivered, 9 g BID or 12 g BID, as determined by the weight of the participant. The granules will be reconstituted in water for oral administration.
BBP-418 (ribitol)
The BBP-418 drug product is provided as Granules for Oral solution consisting of BBP-418 drug substance and silicon dioxide in a multilaminate sachet with a foil barrier. Silicon dioxide is generally regarded as safe and listed in the FDA IIAD. Silicon dioxide is a compendial excipient and is commonly used in pharmaceutical dosage forms. The BBP-418 Granules for Oral Solution are provided in sachets.The BBP-418 Granules for Oral Solution are reconstituted in water for oral administration.
Placebo
A placebo matched for similar taste and appearance was compounded at the clinical pharmacy with sucralose as a 0.35 mg/mL oral solution in purified water (USP) in a glass container. Sucralose is similar in taste to the compounded drug product.
Placebo to Match BBP-418
The placebo will be identical to the BBP-418 Granules for Oral Solution in appearance, packaging, labeling, and storage conditions.
BBP-418 (ribitol)
The BBP-418 drug product is provided as Granules for Oral solution consisting of BBP-418 drug substance and silicon dioxide in a multilaminate sachet with a foil barrier. Silicon dioxide is generally regarded as safe and listed in the FDA IIAD. Silicon dioxide is a compendial excipient and is commonly used in pharmaceutical dosage forms. The BBP-418 Granules for Oral Solution are provided in sachets.The BBP-418 Granules for Oral Solution are reconstituted in water for oral administration.
Placebo
A placebo matched for similar taste and appearance was compounded at the clinical pharmacy with sucralose as a 0.35 mg/mL oral solution in purified water (USP) in a glass container. Sucralose is similar in taste to the compounded drug product.
Interventions
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BBP-418 (ribitol)
The BBP-418 drug product is provided as Granules for Oral solution consisting of BBP-418 drug substance and silicon dioxide in a multilaminate sachet with a foil barrier. Silicon dioxide is generally regarded as safe and listed in the FDA IIAD. Silicon dioxide is a compendial excipient and is commonly used in pharmaceutical dosage forms. The BBP-418 Granules for Oral Solution are provided in sachets.The BBP-418 Granules for Oral Solution are reconstituted in water for oral administration.
Placebo
A placebo matched for similar taste and appearance was compounded at the clinical pharmacy with sucralose as a 0.35 mg/mL oral solution in purified water (USP) in a glass container. Sucralose is similar in taste to the compounded drug product.
Eligibility Criteria
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Exclusion Criteria
4. Use of ribose or other sugar alcohol-containing supplement within 90 days of the Screening Visit.
5. 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.)
6. Previously received gene therapy to treat LGMD2I/R9.
7. 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).
8. Presence of a platelet disorder, bleeding disorder, or other contraindication to muscle biopsy.
9. Actively on an experimental therapy or device or was on an experimental therapy or device within 90 days of the Screening Visit, or was on BBP-418 at any time.
10. 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.
12 Years
60 Years
ALL
No
Sponsors
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ML Bio Solutions, Inc.
INDUSTRY
Responsible Party
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Locations
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Arkansas Children's Hospital
Little Rock, Arkansas, United States
University of California Irvine
Irvine, California, United States
University of Colorado Anschutz Medical Campus
Aurora, Colorado, United States
University of Florida
Gainsville, Florida, United States
University of Iowa
Iowa City, Iowa, United States
University of Kansas Medical Center
Kansas City, Kansas, United States
Kennedy Krieger Institute
Baltimore, Maryland, United States
University of Minnesota, Twin Cities
Minneapolis, Minnesota, United States
Washington University School of Medicine
St Louis, Missouri, United States
Oregon Health & Science University (OHSU) - Neurology Clinic - South Waterfront
Portland, Oregon, United States
The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
University of Pennsylvania
Philadelphia, Pennsylvania, United States
Children's Hospital of the Kings Daughters
Norfolk, Virginia, United States
Royal Brisbane and Women's Hospital,
Brisbane, Queensland, Australia
Rigshospitalet, Neuromuscular Clinic and Research Unit
Copenhagen, , Denmark
Charité Universitätsmedizin Berlin and Max Delbrück Center
Berlin, , Germany
IRCCS Ca' Granda Ospedale
Milan, , Italy
Leiden University Medical Center
Leiden, , Netherlands
Universitetssykehuset Nord-Norge, Department of Neurology
Tromsø, , Norway
Great Ormond Street Hospital for Children
London, , United Kingdom
International Centre for Life
Newcastle upon Tyne, , United Kingdom
Countries
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Other Identifiers
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MLB-01-005
Identifier Type: -
Identifier Source: org_study_id
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