Hope for muscle strength: experimental drug tested for Long-Term use in rare muscular dystrophy

NCT ID NCT07678775

Ongoing Disease control Sponsor: ML Bio Solutions, Inc. Source: ClinicalTrials.gov ↗

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.

Disclaimer Read more

This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

Get updates

Get notified about this study

Sign up to get updates when this study changes or when new studies for LIMB-GIRDLE MUSCULAR DYSTROPHY TYPE 2I (LGMD2I) are added.

Our safety recommendation!

By submitting, you agree to our Terms of use

Conditions

The condition(s) this trial relates to.

autosomal recessive limb-girdle muscular dystrophy type 2I

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

LIMB-GIRDLE MUSCULAR DYSTROPHY TYPE 2I (LGMD2I)

More trials for these conditions

Other studies related to the condition(s) this trial covers.

BBP-418 for LGMD2I: Phase 3 Trial (NCT05775848)

Contacts and locations

Show contact details

Locations

Arkansas Children's Hospital

Little Rock, Arkansas, 72202, United States
Charité Universitätsmedizin Berlin and Max Delbrück Center

Berlin, Germany
Children's Hospital of the Kings Daughters

Norfolk, Virginia, 23510, United States
Fondazione IRCCS Cà Granda Ospedale Maggiore Policlinico

Milan, Italy
Kennedy Krieger Institute

Baltimore, Maryland, 21205, United States
Leids Universitair Medisch Centrum

Leiden, Netherlands
Newcastle University - John Walton Muscular Dystrophy Research Centre

Newcastle upon Tyne, United Kingdom
Rigshospitalet, Neuromuscular Clinic and Research Unit

Copenhagen, Denmark
Royal Brisbane and Women's Hospital

Brisbane, Australia
The Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States
UCL Great Ormond Street Hospital

London, United Kingdom
Universitetssykehuset Nord-Norge, Department of Neurology

Tromsø, Norway
University of California Irvine Medical Center

Orange, California, 92868, United States
University of Colorado Hospital (UCH) - Anschutz Medical Campus

Aurora, Colorado, 80045, United States
University of Florida Health Center for Pediatric Neuromuscular and Rare Diseases

Gainesville, Florida, 32608, United States
University of Iowa Stead Family Children's Hospital

Iowa City, Iowa, 52242, United States
University of Kansas Medical Center (KUMC)

Kansas City, Kansas, 66160, United States
University of Minnesota Medical School

Minneapolis, Minnesota, 55455, United States
University of Pennsylvania

Philadelphia, Pennsylvania, 19104, United States
Washington University School of Medicine

St Louis, Missouri, 63110, United States