Ribitol for Limb-Girdle Muscular Dystrophy

(Fortify Trial)

Recruiting at 28 trial locations

Overseen By Doris Leung, MD

Age: < 65

Sex: Any

Trial Phase: Phase 3

Sponsor: ML Bio Solutions, Inc.

Must not be taking: Ribose, Sugar alcohols, Corticosteroids, others

Disqualifiers: Renal impairment, Pregnancy, Suicidal ideation, others

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

Duration: Long-term

81 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial will test the safety and effectiveness of BBP-418 in patients aged 12 to 60 who have a specific type of muscular dystrophy called LGMD2I/R9. The goal is to see if this new treatment can help improve their condition over several months.

Will I have to stop taking my current medications?

The trial does not specify if you need to stop taking your current medications, but you cannot use ribose or other sugar alcohol supplements, systemic corticosteroids for muscular dystrophy, or be on experimental therapies within 90 days before the screening. It's best to discuss your current medications with the study team.

Is ribitol safe for use in humans?

Ribitol has been tested in animal models and clinical trials in humans without severe side effects, although some gastrointestinal issues like bloating and loose stool were observed at higher doses. ^{1 2 3 4 5}

What makes the drug Ribitol unique for treating Limb-Girdle Muscular Dystrophy?

Ribitol is unique because it works by increasing the levels of a specific sugar molecule called matriglycan, which is important for muscle function, and it compensates for the decreased function of a mutated protein in the disease. Unlike other treatments, it is a simple sugar alcohol that can be administered orally and has shown to improve muscle function and lifespan in animal models with minimal side effects. ^{1 2 3 4 6}

What data supports the effectiveness of the drug BBP-418 (Ribitol) for treating Limb-Girdle Muscular Dystrophy?

Research shows that ribitol can improve muscle function and increase lifespan in mouse models of Limb-Girdle Muscular Dystrophy by enhancing the expression of a protein called matriglycan, which is important for muscle health. Additionally, combining ribitol with gene therapy has shown promising results in improving muscle pathology in similar conditions. ^{1 2 3 4 5}

Are You a Good Fit for This Trial?

This trial is for individuals aged 12-60 with genetically confirmed LGMD2I/R9, weighing over 30 kg. They must understand and consent to study procedures, use effective contraception if of childbearing or reproductive potential, and be able to complete all study tasks like biopsies. Exclusions include significant other diseases, severe kidney issues, recent surgeries affecting assessments, pregnancy/breastfeeding intentions within the study period through 12 weeks after last dose.

Inclusion Criteria

I have a genetic diagnosis of LGMD2I/R9 and show symptoms.

The participant (or parent/guardian) who signs the ICF understands the study procedures and agrees to participate in the study

I am willing to use effective birth control during the study.

Exclusion Criteria

Participants must not have a score of zero on any one or more of the primary or key secondary endpoints at the time of screening

I haven't used ribose or sugar alcohol supplements in the last 90 days.

Participants must not have active suicidal ideation

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive BBP-418 or placebo for long-term administration

Long-term

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• BBP-418 (Ribitol)

Trial Overview The trial tests BBP-418 (Ribitol) for safety and effectiveness in treating Limb Girdle Muscular Dystrophy type 2I (LGMD2I). Participants will receive either the investigational drug or a placebo over a long-term period to see how well it works compared to not receiving the active treatment.

How Is the Trial Designed?

Group I: BBP-418 Active Control 2 Interventions

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.

Group II: Placebo to Match BBP-418 Placebo Group 2 Interventions

The placebo will be identical to the BBP-418 Granules for Oral Solution in appearance, packaging, labeling, and storage conditions.

Who Is Running the Clinical Trial?

ML Bio Solutions, Inc.

Lead Sponsor

Trials

3

Recruited

200+

Citations

1. United States pubmed.ncbi.nlm.nih.gov

Ribitol dose-dependently enhances matriglycan expression and improves muscle function with prolonged life span in limb girdle muscular dystrophy 2I mouse model. [2023]

2. England pubmed.ncbi.nlm.nih.gov

CDP-ribitol prodrug treatment ameliorates ISPD-deficient muscular dystrophy mouse model. [2023]

3. United States pubmed.ncbi.nlm.nih.gov

Improved efficacy of FKRP AAV gene therapy by combination with ribitol treatment for LGMD2I. [2023]

4. England pubmed.ncbi.nlm.nih.gov

Cytidine Diphosphate-Ribitol Analysis for Diagnostics and Treatment Monitoring of Cytidine Diphosphate-l-Ribitol Pyrophosphorylase A Muscular Dystrophy. [2020]

5. United States pubmed.ncbi.nlm.nih.gov

ISPD Overexpression Enhances Ribitol-Induced Glycosylation of α-Dystroglycan in Dystrophic FKRP Mutant Mice. [2020]

6. England pubmed.ncbi.nlm.nih.gov

Ribitol restores functionally glycosylated α-dystroglycan and improves muscle function in dystrophic FKRP-mutant mice. [2020]