What side effects are associated with this type of intervention?

Common treatments for Limb-Girdle Muscular Dystrophy (LGMD) include glucocorticoids and disease-modifying therapies. Glucocorticoids, such as prednisone and deflazacort, can cause side effects like weight gain, osteoporosis, hypertension, and increased risk of infections. Disease-modifying therapies, which may include antisense oligonucleotides and other investigational drugs, can have side effects such as injection site reactions, flu-like symptoms, and potential renal or hepatic toxicity. It is important to discuss these potential side effects with a healthcare provider to tailor the treatment plan to the patient's needs.

What prior FDA approvals exist?

As of now, there are limited FDA-approved treatments specifically for Limb-Girdle Muscular Dystrophy (LGMD). Most treatments focus on managing symptoms and improving quality of life rather than modifying the disease itself. Glucocorticoids like deflazacort have been used to improve muscle strength and function in some muscular dystrophies, including LGMD. Additionally, investigational therapies such as gene therapy and enzyme replacement are being explored in clinical trials. BBP-418 (Ribitol) is currently under investigation for its efficacy and safety in treating LGMD2I/R9, but no specific mechanism of action has been detailed in the provided context.

What other types of research exist?

Currently, there are no specific alternatives to BBP-418 mentioned in the provided context for Limb-Girdle Muscular Dystrophy (LGMD). However, patients should consult with their healthcare providers about ongoing clinical trials and emerging therapies that may not be listed here.

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Ribitol for Limb-Girdle Muscular Dystrophy (Fortify Trial)

Phase 3

Waitlist Available

Research Sponsored by ML Bio Solutions, Inc.

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Be younger than 65 years old

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 5 years

Awards & highlights

Pivotal Trial

Summary

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.

Who is the study for?

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.

What is being tested?

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.

What are the potential side effects?

While specific side effects are not listed here, participants may experience adverse reactions related to BBP-418 (Ribitol), which could range from mild symptoms like nausea or headaches to more serious ones depending on individual responses and pre-existing health conditions.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 5 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to 5 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 5 years for reporting.

Treatment Details

Awards & Highlights

Pivotal Trial

The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

Trial Design

2Treatment groups

Active Control

Placebo Group

Group I: BBP-418Active Control2 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-418Placebo Group2 Interventions

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

Do I qualify?Apply below to find out

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

The most common treatments for Limb-Girdle Muscular Dystrophy (LGMD) often involve genetic therapies, such as exon skipping and gene replacement, which aim to correct or bypass the genetic mutations causing the disease. These therapies work by either restoring the production of functional proteins or compensating for the defective ones, thereby improving muscle function and slowing disease progression. For LGMD patients, these treatments are crucial as they target the root cause of muscle degeneration, offering the potential for improved mobility and quality of life. Additionally, supportive treatments like physical therapy and corticosteroids help manage symptoms and maintain muscle strength.