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Study on the Effects of Ribitol (BBP-418) in Patients with Limb Girdle Muscular Dystrophy Type 2I

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What is this trial about?

A plain-language summary of the goals, design and what participants do

This clinical trial is focused on studying a condition called Limb Girdle Muscular Dystrophy 2I (LGMD2I), which is a genetic disorder that causes muscle weakness, particularly around the hips and shoulders. The study is testing a treatment called BBP-418, also known as ribitol, which is taken as granules dissolved in a liquid and consumed orally. The purpose of the study is to evaluate how effective and safe this treatment is for patients with LGMD2I.

Participants in the study will be randomly assigned to receive either the BBP-418 treatment or a placebo, which looks like the treatment but does not contain the active substance. The study is designed to be double-blind, meaning neither the participants nor the researchers will know who is receiving the actual treatment or the placebo. This helps ensure that the results are not influenced by expectations. The study will last for a period of 36 months, during which participants will undergo regular assessments to monitor their health and the effects of the treatment.

Throughout the study, participants will have various health checks, including physical examinations and laboratory tests, to track any changes in their condition. The main focus will be on changes in muscle strength and function, as well as any side effects that may occur. The study aims to provide valuable information on whether BBP-418 can help improve the symptoms of Limb Girdle Muscular Dystrophy 2I and enhance the quality of life for those affected by this condition.

The research process

The trial runs in 6 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Initial assessment

    Upon joining the study, an initial assessment is conducted to confirm eligibility. This includes a review of genetic testing records to verify a diagnosis of limb girdle muscular dystrophy 2I (LGMD2I).

    A physical examination is performed to assess clinical weakness in either a limb-girdle pattern or in a distal extremity.

  2. Step 2

    Medication administration

    Participants receive either ribitol or a placebo. The medication is provided in the form of granules for oral solution.

    The dosage and frequency of administration are determined by the study protocol and are followed throughout the trial duration.

  3. Step 3

    Regular monitoring

    Participants undergo regular monitoring to assess the effects of the medication. This includes physical examinations, vital signs checks, and laboratory analyses of blood chemistry and hematology.

    An electrocardiogram (ECG) is performed to monitor heart activity, including QTc intervals.

  4. Step 4

    Evaluation of primary outcomes

    The primary outcomes are evaluated at 36 months. These include changes from baseline in the North Star Ambulatory Assessment for Dysferlinopathy (NSAD) and the frequency and severity of treatment-emergent adverse events (TEAEs) and serious adverse events (TESAEs).

  5. Step 5

    Evaluation of secondary outcomes

    Secondary outcomes are also assessed at 36 months. These include changes in walking speed measured by the 10-meter walk test (10MWT), pulmonary function as measured by forced vital capacity (FVC), and other functional assessments.

    Additional laboratory tests are conducted to measure changes in glycosylated alpha-dystroglycan (αDG) expression and serum creatine kinase (CK) levels.

  6. Step 6

    Completion of study

    The study is estimated to conclude by September 2027. Participants complete all study procedures, including any final assessments and evaluations, as outlined in the study schedule.

Who can join the trial?

6 criteria

  • Have a genetically confirmed diagnosis of **Limb Girdle Muscular Dystrophy 2I/R9**. This means that a genetic test has shown you have this specific type of muscle condition, and you show signs of muscle weakness either around the hips and shoulders or in the arms and legs.
  • Be a male or female between the ages of 18 and 60.
  • Have a body weight greater than 30 kilograms (about 66 pounds).
  • Understand the study procedures and agree to participate by signing an informed consent form. This means you know what the study involves and agree to take part.
  • If you are a woman who can have children or a man who can father children, you must agree to use a very effective form of birth control from the time you agree to join the study until 12 weeks after the last dose of the study medication.
  • Be willing and able to complete all study procedures, including biopsies. A biopsy is a small sample of tissue taken from the body for testing.

Who cannot join the trial?

8 criteria

  • Patients with any other serious health condition that could interfere with the study.
  • Patients who are pregnant or breastfeeding.
  • Patients who have participated in another clinical trial within the last 30 days.
  • Patients who have a known allergy to the study medication or its ingredients.
  • Patients who are unable to follow the study procedures or attend study visits.
  • Patients with a history of drug or alcohol abuse that could affect the study results.
  • Patients who have received certain medications that might interfere with the study drug.
  • Patients with a history of certain heart conditions that could pose a risk during the study.
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Investigated drugs

BBP-418 (ribitol) is being studied for its potential to help patients with Limb Girdle Muscular Dystrophy 2I (LGMD2I). This medication is being tested to see if it can improve muscle strength and function in people with this condition. The trial aims to determine how effective and safe BBP-418 is for treating LGMD2I.

What is already known about the treatment

BBP-418 (ribitol) – This medication is administered orally and is currently being studied in a Phase 3 clinical trial for its effectiveness and safety in treating Limb Girdle Muscular Dystrophy 2I (LGMD2I). It is not yet widely used in medical practice, as it is still under investigation. The main therapeutic indication for BBP-418 is to improve muscle function in patients with LGMD2I. At the molecular level, ribitol is believed to work by enhancing the glycosylation of alpha-dystroglycan, a protein important for muscle integrity. It is classified pharmacologically as a potential therapeutic agent for muscular dystrophies.

Investigated diseases

Limb Girdle Muscular Dystrophy 2I/R9 – This is a genetic disorder characterized by progressive muscle weakness, primarily affecting the muscles around the hips and shoulders. Over time, individuals may experience difficulty with activities such as climbing stairs, lifting objects, or rising from a seated position. The disease progresses at varying rates, with some individuals experiencing a slow decline in muscle function, while others may see a more rapid progression. Muscle wasting and weakness can lead to challenges in mobility and daily activities. The condition is caused by mutations in specific genes that affect muscle protein production. It is classified as a rare disease, meaning it affects a small percentage of the population.
Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase IIITrial ID2023-503379-33-01Protocol codeMLB-01-005Estimated enrolment107 patientsSponsorML Bio Solutions Inc.

sourced from the EU Clinical Trials Register and site verification

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