Hope for muscle strength: experimental drug tested for Long-Term use in rare muscular dystrophy
NCT ID NCT07678775
First seen Jul 01, 2026 · Last updated Jul 02, 2026 · Updated 1 time
Summary
This study tests the long-term safety and effectiveness of an experimental drug called BBP-418 (ribitol) in people with limb-girdle muscular dystrophy type 2I/R9, a rare genetic muscle-weakening disease. Participants who completed a previous study will take BBP-418 orally twice daily for up to 36 months. Researchers will monitor side effects and measure changes in muscle function, walking speed, and breathing capacity.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- BBP-418 (ribitol)
- What this could lead to
- If successful, this could provide a long-term treatment option to slow disease progression and improve muscle function in people with LGMD2I/R9.
- What could go wrong
- This is an extension study, so participants already know the drug is safe enough to continue. However, long-term side effects are still being monitored, and the drug may not work for everyone.
This is an AI summary of the original study and may miss details. Read our disclaimer.
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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About 107 people
The number the study aims to enrol. It can still change while the study runs.
- Started
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Jun 2026
- Expected to finish
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Oct 2030
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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Children (under 18), adults (18 to 64) and older adults (65 and over)
- 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.
Inclusion Criteria: * Completed Study MLB-01-005 on study drug through the final clinic visit (Month 36 or another qualifying end-of-study visit as determined by the Sponsor). * The participant (or parent/guardian) who signs the ICF understands the study procedures and agrees to participate in the study by giving informed consent (or assent, if \<18 years of age). * Is willing and able to complete all study procedures according to the Schedule of Assessments. * A WOCBP or a nonsterile male participant must be willing to use an acceptable method of contraception from the time of consent through 30 days after the last dose of study drug in this study. Exclusion Criteria: * Has developed clinically significant concomitant disease that would, in the Investigator's opinion, be likely to unfavorably impact study participation, including: * Any significant concomitant medical condition, including psychiatric, cardiac, renal, pulmonary, hepatic, or endocrine disease other than that associated with LGMD2I/R9 * Any other significant laboratory, vital sign, ECG abnormality, clinical history, or finding * Is pregnant (based on the Baseline / Day 1 pregnancy test result) and/or breastfeeding or planning to conceive children within the projected duration of the study through 30 days after the last dose of study drug in this study. * Has active suicidal ideation, defined as having a 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) on the C-SSRS at Baseline / Day 1.
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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, 23510, United States
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Fondazione IRCCS Cà Granda Ospedale Maggiore Policlinico
Milan, Italy
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Kennedy Krieger Institute
Baltimore, Maryland, 21205, United States
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Leids Universitair Medisch Centrum
Leiden, Netherlands
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Newcastle University - John Walton Muscular Dystrophy Research Centre
Newcastle upon Tyne, United Kingdom
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Rigshospitalet, Neuromuscular Clinic and Research Unit
Copenhagen, Denmark
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Royal Brisbane and Women's Hospital
Brisbane, Australia
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The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States
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UCL Great Ormond Street Hospital
London, United Kingdom
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Universitetssykehuset Nord-Norge, Department of Neurology
Tromsø, Norway
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University of California Irvine Medical Center
Orange, California, 92868, United States
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University of Colorado Hospital (UCH) - Anschutz Medical Campus
Aurora, Colorado, 80045, United States
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University of Florida Health Center for Pediatric Neuromuscular and Rare Diseases
Gainesville, Florida, 32608, United States
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University of Iowa Stead Family Children's Hospital
Iowa City, Iowa, 52242, United States
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University of Kansas Medical Center (KUMC)
Kansas City, Kansas, 66160, United States
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University of Minnesota Medical School
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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