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Ribitol

A groundbreaking Phase 3 clinical trial is underway to assess the effectiveness and safety of Ribitol (BBP-418) in patients with Limb Girdle Muscular Dystrophy Type 2I (LGMD2I). This study aims to provide valuable insights into a potential treatment for this rare genetic disorder, offering hope to those affected by LGMD2I. The trial involves long-term administration of Ribitol to patients aged 12 to 60, covering a significant portion of diagnosed individuals based on the disease’s epidemiology.

Table of Contents

What is RIBITOL?

RIBITOL, also known as BBP-418, is a new drug being studied for the treatment of a specific type of muscular dystrophy. It is currently undergoing clinical trials to evaluate its safety and effectiveness in patients with Limb-Girdle Muscular Dystrophy Type 2I (LGMD2I), also referred to as LGMD2I/R9.[1]

What condition does RIBITOL target?

RIBITOL is specifically designed to treat Limb-Girdle Muscular Dystrophy Type 2I (LGMD2I). This is a rare genetic disorder that causes progressive muscle weakness, primarily affecting the muscles around the shoulders and hips. The condition can vary in severity and age of onset, but it typically leads to difficulty with mobility and daily activities over time.[1]

How does RIBITOL work?

While the exact mechanism of action is not fully described in the provided information, RIBITOL is believed to work by addressing the underlying genetic defect in LGMD2I. The drug appears to target a protein called alpha-dystroglycan, which plays a crucial role in muscle function. By potentially increasing the glycosylation (a process of adding sugar molecules) of alpha-dystroglycan, RIBITOL may help improve muscle strength and function in patients with LGMD2I.[1]

Current Clinical Trial

RIBITOL is currently being evaluated in a Phase 3 clinical trial. This is an advanced stage of research that aims to confirm the drug’s effectiveness and safety in a larger group of patients. Here are some key points about the trial:

  • It is a randomized, placebo-controlled, double-blind study. This means that participants are randomly assigned to receive either RIBITOL or a placebo, and neither the patients nor the researchers know who is receiving which treatment. This design helps ensure unbiased results.[1]
  • The study includes patients aged 12 to 60 years old, covering the majority of diagnosed LGMD2I patients.[1]
  • Participants are divided into groups based on their specific genetic mutation, age, and other factors to ensure a balanced evaluation.[1]

How is RIBITOL administered?

RIBITOL is provided as granules for oral solution. Here’s what patients should know about its administration:

  • The drug comes in sachets (small packets) containing granules.[1]
  • These granules are mixed with water to create a solution that can be taken by mouth.[1]
  • The dose is determined based on the patient’s weight, with options of 9 grams or 12 grams, taken twice daily.[1]

How is RIBITOL’s effectiveness measured?

The clinical trial is using several methods to assess how well RIBITOL works. These include:

  1. North Star Assessment for Limb Girdle Muscular Dystrophy: This is the primary measure, looking at changes in muscle function over 36 months of treatment.[1]
  2. 10-meter walk test: This measures how quickly patients can walk a short distance.[1]
  3. Pulmonary function tests: These assess how well the lungs are working.[1]
  4. Performance of Upper Limb scale: This evaluates arm and hand function.[1]
  5. Biomarker tests: These look at changes in specific proteins (alpha-dystroglycan) in the body that are related to LGMD2I.[1]

Safety Monitoring

As with any new medication, monitoring for side effects is crucial. The study is carefully tracking the frequency and severity of any adverse events that occur during the 36-month treatment period. This information will help determine the overall safety profile of RIBITOL.[1]

Aspect Details
Study Type Phase 3, randomized, placebo-controlled, double-blind clinical trial
Drug Ribitol (BBP-418)
Condition Limb Girdle Muscular Dystrophy Type 2I (LGMD2I)
Participant Age Range 12 to 60 years (18 to 60 years in EU region)
Treatment Duration 36 months
Primary Outcomes Change in North Star Assessment, Safety assessment
Secondary Outcomes 10-meter walk test, Pulmonary function, Upper limb performance
Other Outcomes Changes in glycosylated Alpha dystroglycan levels
Randomization 2:1 ratio (drug:placebo)

FAQ

  • What is Ribitol (BBP-418) and how is it administered in the trial? Ribitol (BBP-418) is a drug being studied for the treatment of Limb Girdle Muscular Dystrophy Type 2I (LGMD2I). In the trial, it's provided as granules for oral solution in sachets. The granules are mixed with water and taken by mouth twice daily, with the dose (9g or 12g) determined by the participant's weight.
  • Who can participate in this clinical trial? The trial includes patients aged 12 to 60 years old with LGMD2I/R9. However, it's important to note that in the EU region, only participants aged 18 to 60 are being enrolled.
  • How long does the trial last? The primary outcomes of the trial are measured after 36 months of treatment, suggesting that the main phase of the study lasts for 3 years.
  • What are the main outcomes being measured in this trial? The primary outcomes include changes in the North Star Assessment for Limb Girdle Muscular Dystrophy to measure efficacy, and the frequency and severity of treatment-related side effects to assess safety. Secondary outcomes include changes in walking speed, lung function, and upper limb performance.
  • Is there a placebo group in this study? Yes, this is a placebo-controlled study. Some participants will receive a placebo that looks identical to the Ribitol granules. The study uses a 2:1 randomization, meaning more participants will receive the actual drug than the placebo.

Ongoing Clinical Trials on Ribitol

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

    Not recruiting

    1 1
    Investigated diseases:
    Investigated drugs:
    Denmark Germany Italy The Netherlands Norway

Glossary

  • Limb Girdle Muscular Dystrophy Type 2I (LGMD2I): A rare genetic disorder that causes progressive muscle weakness, primarily affecting the muscles around the shoulders and hips.
  • Ribitol (BBP-418): The drug being tested in this clinical trial as a potential treatment for LGMD2I.
  • Phase 3 Clinical Trial: A large-scale study to confirm the effectiveness and monitor side effects of a new drug in a larger group of people, typically the last phase before seeking approval for general use.
  • Placebo: A substance that looks identical to the study drug but contains no active ingredient, used to compare the effects of the real drug.
  • Randomization: The process of randomly assigning participants to different treatment groups in a clinical trial to reduce bias.
  • North Star Assessment: A standardized test used to measure physical function and disease progression in muscular dystrophies.
  • FVC (Forced Vital Capacity): A measure of lung function that shows the amount of air a person can forcefully exhale after taking a deep breath.
  • Performance of Upper Limb scale: A test designed to measure upper limb function in patients with neuromuscular disorders.
  • Alpha dystroglycan: A protein important for muscle function, which is often affected in LGMD2I.
  • Biomarker: A measurable indicator of a biological state or condition, used here to assess the effectiveness of the treatment.

References