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Long-Term Safety and Efficacy of Oral Ribitol in Participants with Limb-Girdle Muscular Dystrophy Type 2I (LGMD2I/R9)

Fast replyInvestigationalNo placebo
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What is this trial about?

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

The study looks at people with Limb Girdle Muscular Dystrophy type 2I, also called LGMD2I/R9. This is a rare condition that slowly weakens the muscles around the hips and shoulders. The treatment being tested is an oral medication made of granules that contain Ribitol, referred to in the study as BBP-418.

The purpose of the study is to assess the long‑term safety and clinical benefit of the medication. Participants will take the granules by mouth each day for an extended period and will attend regular visits where doctors will check how they are feeling and perform simple tests.

During the visits, several simple assessments are done: a muscle‑function questionnaire called NSAD, a walking speed test over ten meters known as the 10MWT, a breathing test that measures how much air can be exhaled called FVC, an upper‑limb function test named PUL 2.0, a walking distance test over one hundred meters called 100MTT, and a blood test that measures a protein called CK which indicates muscle damage. These checks help researchers understand how the medication affects muscle strength, movement, and overall health over time.

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

    Enrollment confirmation

    After signing the consent form, a confirmation is provided that participation in the study has begun.

    The study identifier and the period of participation, from september 2026 to september 2029, are recorded.

  2. Step 2

    Baseline visit

    A first clinic visit is scheduled to collect baseline information.

    Measurements include a physical exam, functional tests such as the 10‑meter walk test, forced vital capacity, and muscle strength assessments, as well as a blood sample for serum ck.

    The results form the reference point for later comparisons of safety and efficacy.

  3. Step 3

    Start of medication

    The study drug ribitol is provided in granules for oral solution.

    The prescribed dose is 24 g per administration, taken by mixing the granules with water as instructed.

    The medication is taken according to the schedule defined in the study protocol.

  4. Step 4

    Regular monitoring visits

    Periodic clinic visits are arranged throughout the study period.

    At each visit, health status is reviewed, any side effects are recorded, and blood tests may be performed to monitor safety.

    The study team records the frequency and severity of any treatment‑emergent adverse events.

  5. Step 5

    Efficacy assessments

    Functional assessments that were done at baseline are repeated at scheduled times to evaluate changes.

    These include the 10‑meter walk test, forced vital capacity, the performance of upper limb test for non‑ambulatory participants, the 100‑meter timed test, and serum ck levels.

    Changes from baseline are used to assess the long‑term clinical benefit of the treatment.

  6. Step 6

    Final visit and study completion

    At the end of the study period in september 2029, a final visit is conducted.

    All final safety and efficacy measurements are taken, and the study medication is discontinued according to protocol.

    The collected data are compiled for analysis of long‑term safety and efficacy of ribitol.

Who can join the trial?

6 criteria

  • You must have finished a previous study called MLB-01-005 and attended the final clinic visit (Month 36 or another qualifying end‑of‑study visit).
  • You (or your parent/guardian if you are under 18) must sign the informed consent form (ICF) and understand what the study will involve.
  • You need to be willing and able to complete all study visits, tests, and other procedures that are listed in the schedule of assessments.
  • If you are a woman who could become pregnant (WOCBP) or a male who is not sterile, you must agree to use an acceptable method of birth control (contraception) from the time you sign the consent until 30 days after the last dose of the study drug.
  • You must have a diagnosis of Limb Girdle Muscular Dystrophy (LGMD) Type 2I.
  • The study accepts both male and female participants, including children; you must be at least 3 years old.

Who cannot join the trial?

3 criteria

  • Clinically significant additional illness: If you have another serious health problem (such as mental health, heart, kidney, lung, liver, or hormone disorders) that is not part of LGMD2I/R9, or if recent lab tests, vital signs, or heart‑electrocardiogram (ECG) results show concerning abnormalities, you cannot join the study.
  • Pregnancy or breastfeeding: If you are currently pregnant, are nursing a baby, or plan to become pregnant during the study period (including up to 30 days after the last dose), you are not eligible.
  • Active suicidal thoughts: If you are experiencing strong thoughts of suicide, as measured by a score of 4 or 5 on the Columbia‑Suicide Severity Rating Scale (C‑SSRS) at the start of the study, you cannot participate.
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Investigated drugs

Ribitol (BBP-418) is an oral medication that comes as granules that can be mixed with water to make a solution you drink. It is being studied as a potential treatment for people with Limb‑Girdle Muscular Dystrophy type 2I/R9, a condition that weakens the muscles around the hips and shoulders. In the trial, Ribitol is given to see if it can safely improve muscle function over a long period of time. The drug works by providing a building block that may help the body make a missing protein needed for healthy muscle cells, which could slow down the loss of strength and improve daily activities.

What is already known about the treatment

Ribitol - Ribitol is taken as granules that are dissolved in water and swallowed orally; it is being studied as an orphan drug for the rare muscle disease Limb‑Girdle Muscular Dystrophy type 2I/R9. The substance is still in clinical trials and has not yet been approved for routine medical use. It is intended to treat LGMD2I/R9 by providing a building block that helps the body produce a sugar‑derived compound needed for normal muscle cell function. Ribitol works by increasing the levels of this compound, supporting muscle protein stability, and it is classified as a sugar‑alcohol metabolic supplement.

Investigated diseases

Limb Girdle Muscular Dystrophy (LGMD) Type 2I - A genetic condition that causes muscle weakness primarily around the hips and shoulders. The weakness usually appears in childhood or early teenage years and gets gradually stronger over time. As the disease progresses, walking may become difficult, climbing stairs can be challenging, and rising from a seated position may require assistance. The condition continues to develop slowly, affecting daily activities as the muscles become less able to contract.
Trial detailsLast updated 2 Oct 2026
Age18+ yearsPhasePhase IIITrial ID2025-524371-23-00Protocol codeMLB-01-007Estimated enrolment107 patientsSponsorML Bio Solutions Inc.

sourced from the EU Clinical Trials Register and site verification

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