Open-Label Extension Study of BBP-418 (Ribitol) for LGMD2I/R9

June 30, 2026 updated by: ML Bio Solutions, Inc.

An Open-Label Extension Study to Evaluate the Long-Term Safety and Efficacy of BBP-418 (Ribitol) in Participants With Limb-Girdle Muscular Dystrophy 2I/R9 (LGMD2I/R9)

This is an open-label extension (rollover) study designed to evaluate the long-term safety and efficacy of BBP-418 (ribitol) in participants with limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9) who have previously participated in Study MLB-01-005 (Fortify).

Participants will receive BBP-418 administered orally at protocol-defined doses and schedules. The study will assess long-term safety through monitoring of adverse events, clinical laboratory evaluations, and other safety assessments. Efficacy will be evaluated using functional measures and other clinical endpoints relevant to LGMD2I/R9.

Participants will be followed for up to 36 months, with a final safety follow-up assessment conducted approximately 30 days after the last dose of study drug.

Study Overview

Status

Active, not recruiting

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Estimated)

107

Phase

  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Australia
      • Brisbane, Australia
        • Royal Brisbane and Women's Hospital
  • Denmark
      • Copenhagen, Denmark
        • Rigshospitalet, Neuromuscular Clinic and Research Unit
  • Germany
      • Berlin, Germany
        • Charité Universitätsmedizin Berlin and Max Delbrück Center
  • Italy
      • Milan, Italy
        • Fondazione IRCCS Cà Granda Ospedale Maggiore Policlinico
  • Netherlands
      • Leiden, Netherlands
        • Leids Universitair Medisch Centrum
  • Norway
      • Tromsø, Norway
        • Universitetssykehuset Nord-Norge, Department of Neurology
  • United Kingdom
      • London, United Kingdom
        • UCL Great Ormond Street Hospital
      • Newcastle upon Tyne, United Kingdom
        • Newcastle University - John Walton Muscular Dystrophy Research Centre
  • United States
    • Arkansas
      • Little Rock, Arkansas, United States, 72202
        • Arkansas Children's Hospital
    • California
      • Orange, California, United States, 92868
        • University of California Irvine Medical Center
    • Colorado
      • Aurora, Colorado, United States, 80045
        • University of Colorado Hospital (UCH) - Anschutz Medical Campus
    • Florida
      • Gainesville, Florida, United States, 32608
        • University of Florida Health Center for Pediatric Neuromuscular and Rare Diseases
    • Iowa
      • Iowa City, Iowa, United States, 52242
        • University of Iowa Stead Family Children's Hospital
    • Kansas
      • Kansas City, Kansas, United States, 66160
        • University of Kansas Medical Center (KUMC)
    • Maryland
      • Baltimore, Maryland, United States, 21205
        • Kennedy Krieger Institute
    • Minnesota
      • Minneapolis, Minnesota, United States, 55455
        • University of Minnesota Medical School
    • Missouri
      • St Louis, Missouri, United States, 63110
        • Washington University School of Medicine
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19104
        • University of Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19104
        • The Children's Hospital of Philadelphia
    • Virginia
      • Norfolk, Virginia, United States, 23510
        • Children's Hospital of the Kings Daughters

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: BBP-418
Drug: BBP-418. Single arm.
Drug: BBP-418 (ribitol)
Drug: BBP-418. Single arm. Participants who completed Study MLB-01-005 and meet eligibility criteria will receive BBP-418 (ribitol) taken orally twice daily at 9 or 12g (based on body weight measured) for up to 36 months.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Frequency and severity of treatment-emergent adverse events to assess long-term safety of BBP-418.
Time Frame: 36 months
36 months
Change from baseline in North Star Assessment for LGMD2I/R9 to assess long-term efficacy of BBP-418.
Time Frame: 36 months
36 months

Secondary Outcome Measures

Outcome Measure
Time Frame
Change from baseline in 10MWT (10-meter walk test) (m/s) for LGMD2I/R9 to assess long-term efficacy of BBP-418.
Time Frame: 36 months
36 months
Change from baseline FVC (forced vital capacity) (percent predicted, performed in a sitting position) for LGMD2I/R9 to assess long-term efficacy of BBP-418.
Time Frame: 36 months
36 months
Change from baseline in PUL 2.0 (performance of the upper limb) total score for LGMD2I/R9 to assess long-term efficacy of BBP-418.
Time Frame: 36 months
36 months
Change from baseline in 100MTT (100-meter timed test) (m/s) for LGMD2I/R9 to assess long-term efficacy of BBP-418.
Time Frame: 36 months
36 months
Change from baseline in Serum CK (creatine kinase) for LGMD2I/R9 to assess long-term biomarker changes in participants.
Time Frame: 36 months
36 months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

ML Bio Solutions, Inc.

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

June 5, 2026

Primary Completion (Estimated)

October 1, 2030

Study Completion (Estimated)

October 1, 2030

Study Registration Dates

First Submitted

June 25, 2026

First Submitted That Met QC Criteria

June 25, 2026

First Posted (Actual)

July 1, 2026

Study Record Updates

Last Update Posted (Actual)

July 2, 2026

Last Update Submitted That Met QC Criteria

June 30, 2026

Last Verified

June 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • MLB-01-007

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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