Ribitol for Limb-Girdle Muscular Dystrophy

(Fortify Trial)

Not currently recruiting at 31 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

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests the safety and effectiveness of BBP-418 (Ribitol), a new treatment for Limb-Girdle Muscular Dystrophy Type 2I/R9. The study compares BBP-418 to a placebo (an inactive substance) to determine if it can improve muscle strength and function in individuals with this condition. Those diagnosed with LGMD2I/R9 and experiencing muscle weakness may be eligible to join. Participants must be comfortable with all study procedures, including muscle biopsies, and must not have any serious health issues unrelated to their muscular dystrophy. As a Phase 3 trial, this study represents the final step before potential FDA approval, offering participants a chance to contribute to a treatment nearing widespread availability.

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 there any evidence suggesting that this treatment is likely to be safe for humans?

Earlier studies have shown that BBP-418, also known as Ribitol, is safe. Research indicates that patients tolerate BBP-418 well. In one study with 93 healthy volunteers, researchers tested the treatment to assess its safety and how the body processes it. The results were positive, with no major safety issues identified.

Further research is ongoing to confirm these findings in people with limb-girdle muscular dystrophy (LGMD2I/R9). This ongoing study aims to understand how BBP-418 works in a larger group over a longer period. While more data will provide a clearer picture, early signs suggest that BBP-418 is safe for human use. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for muscular dystrophy?

Researchers are excited about BBP-418 (Ribitol) for Limb-Girdle Muscular Dystrophy (LGMD) because it offers a novel approach compared to traditional treatments that mainly focus on symptom management. Unlike standard therapies, which do not address the root cause of the disease, BBP-418 is designed to target the underlying metabolic pathway involved in LGMD. Ribitol works by increasing the production of glycosylation in muscle cells, which can potentially enhance muscle function and strength. This innovative mechanism of action provides hope for improved outcomes in patients with LGMD, setting it apart from existing treatment options.

What evidence suggests that BBP-418 (Ribitol) might be an effective treatment for Limb-Girdle Muscular Dystrophy?

Research has shown that BBP-418, also known as Ribitol, may help treat Limb-Girdle Muscular Dystrophy type 2I/R9. In earlier studies, patients who took BBP-418 showed significant improvements in all main health measures after 12 months. Specifically, previous research found about a 70% drop in creatine kinase levels, which indicate muscle damage, after three months of treatment. Additionally, the treatment increased levels of a protein important for muscle function, suggesting better muscle health. These findings support the potential effectiveness of BBP-418 in managing this condition. Participants in this trial will receive either BBP-418 or a placebo to further evaluate its effectiveness. ^{1 3 6 7 8}

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. investor.bridgebio.com investor.bridgebio.com/news/news-details/2025/BridgeBio-Reports-Positive-Phase-3-Results-for-Small-Molecule-BBP-418-in-LGMD2IR9-FORTIFY-Study/default.aspx

BridgeBio Reports Positive Phase 3 Results for Small ... - BBP-418 treated individuals had statistically significant, clinically meaningful improvements at 12 months in all key clinical endpoints ...

2. clinicaltrials.gov clinicaltrials.gov/study/NCT05775848

Study to Evaluate the Efficacy and Safety of BBP-418 ... This study will evaluate the safety and efficacy of long-term administration of BBP-418 in patients with LGMD2I/R9.

3. mdaconference.org mdaconference.org/abstract-library/preliminary-results-from-mlb-01-003-an-open-label-phase-2-study-of-bbp-418-in-patients-with-limb-girdle-muscular-dystrophy-type-2i/

An Open Label Phase 2 Study of BBP-418 in Patients ... All 12 patients showed declines in creatine kinase (CK) (mean ~70% decrease) from baseline assessment after 3 months of treatment with BBP-418 in Part 1. No ...

4. sciencedirect.com sciencedirect.com/science/article/abs/pii/S0960896623006685

an open label phase 2 study of BBP-418 in patients with ... Participants showed increased levels of glycosylated αDG after 90 days of dosing, median 33.4% of normal with BBP-418 treatment compared to a baseline of 7.4%, ...

5. bridgebio.com bridgebio.com/lgmd2i-r9/bbp-418/

BBP-418 | Glycosylation Substrate Pro-Drug For LGMD2I/R9 If approved, BBP-418 has the potential to be the first disease-modifying oral therapy available for people with LGMD2I/R9. Learn more today.

6. sciencedirect.com sciencedirect.com/science/article/abs/pii/S0960896624003390

a phase 3 study to evaluate efficacy & safety of BBP-418 in ... The FORTIFY study will assess the safety, tolerability, and efficacy of BBP-418 in LGMD2I/R9. In addition to evaluating the effect of BBP-418 on motor ...

7. mdaconference.org mdaconference.org/abstract-library/fortify-a-phase-3-study-to-evaluate-efficacy-safety-of-bbp-418-in-individuals-with-limb-girdle-muscular-dystrophy-2i-lgmd2i-lgmdr9-fkrp-related/

A Phase 3 Study to Evaluate Efficacy & Safety of BBP-418 ... FORTIFY is a Phase 3 randomized, placebo-controlled study that will assess the safety, tolerability, and efficacy of BBP-418 in LGMD2I/R9.

8. musculardystrophynews.com musculardystrophynews.com/bbp-418-ribitol-muscular-dystrophy/

BBP-418 for limb-girdle muscular dystrophy Phase 1 trial. BBP-418's safety and pharmacological properties were assessed in a Phase 1 clinical study that involved 93 healthy volunteers.