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Hope for rare muscle disease: new drug enters final testing phase
NCT ID NCT05775848
First seen Jun 27, 2026 · Last updated Sep 10, 2026 · Updated 2 times
Summary
This study tests a drug called BBP-418 (Ribitol) in 81 people aged 12 to 60 with limb girdle muscular dystrophy type 2I (LGMD2I), a genetic condition that causes progressive muscle weakness. Participants receive either the drug or a placebo for 36 months to see if it slows the disease and is safe. The main goal is to measure changes in muscle function and track any side effects.
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Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 3
Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.
- Participants
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112 people
The number who actually took part.
- Started
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May 2023
- Expected to finish
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Sep 2027
An estimate. End dates often move.
- Lead sponsor
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A company
The lead sponsor is a pharmaceutical, biotech, or medical-device company.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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12 to 60 years
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Participants must meet all the following criteria to be enrolled: 1. 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). 2. Male or female participants 12 to 60 years of age (inclusive). \[NOTE: EU region will only enroll 18 to 60 years of age\] 3. Have a body weight \>30 kg. 4. The participant (or parent/guardian) who signs the ICF understands the study procedures and the participant agrees to participate in the study by giving informed consent (and assent, if \<18 years of age). 5. Female participants of childbearing potential 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. 6. Willing and able to complete all study procedures, including biopsies, according to the Schedule of Assessments (see Appendix 1). Participants must not meet any of the following criteria to be enrolled: 1. Evidence of clinically significant concomitant disease, including: 1. Any significant concomitant medical condition, including mental, cardiac, renal, pulmonary, hepatic, or endocrine disease other than that associated with LGMD2I/R9. 2. Moderate to severe renal impairment (estimated glomerular filtration rate \[eGFR\] of \< 60 mL/min/1.73 m2 based on cystatin C \[CysC\]), as calculated by the central laboratory. 3. 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. 4. 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. 2. 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 enroll in this study provided all inclusion and no other exclusion criteria are met.) 3. 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. 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.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
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Arkansas Children's Hospital
Little Rock, Arkansas, 72202, United States
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Charité Universitätsmedizin Berlin and Max Delbrück Center
Berlin, Germany
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Children's Hospital of the Kings Daughters
Norfolk, Virginia, 23455, United States
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Great Ormond Street Hospital for Children
London, United Kingdom
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IRCCS Ca' Granda Ospedale
Milan, Italy
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International Centre for Life
Newcastle upon Tyne, United Kingdom
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Kennedy Krieger Institute
Baltimore, Maryland, 21205, United States
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Leiden University Medical Center
Leiden, Netherlands
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Oregon Health & Science University (OHSU) - Neurology Clinic - South Waterfront
Portland, Oregon, 97239, United States
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Rigshospitalet, Neuromuscular Clinic and Research Unit
Copenhagen, Denmark
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Royal Brisbane and Women's Hospital,
Brisbane, Queensland, Australia
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The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States
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Universitetssykehuset Nord-Norge, Department of Neurology
Tromsø, Norway
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University of California Irvine
Irvine, California, 92697, United States
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University of Colorado Anschutz Medical Campus
Aurora, Colorado, 80045, United States
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University of Florida
Gainesville, Florida, 32610, United States
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University of Iowa
Iowa City, Iowa, 52242, United States
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University of Kansas Medical Center
Kansas City, Kansas, 66160, United States
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University of Minnesota, Twin Cities
Minneapolis, Minnesota, 55455, United States
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University of Pennsylvania
Philadelphia, Pennsylvania, 19104, United States
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Washington University School of Medicine
St Louis, Missouri, 63110, United States
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