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Study on the Effects of Dimethyl Fumarate for Patients with Friedreich's Ataxia

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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 Friedreich's Ataxia, a rare genetic disease that affects the nervous system and movement. The trial will test the effectiveness, safety, and tolerability of a medication called Dimethyl Fumarate, also known by its code names BG00012 and FP 187. Participants in the study will receive either the medication or a placebo, which looks like the medication but does not contain the active ingredient.

The purpose of the study is to understand how Dimethyl Fumarate affects the production of certain proteins in the body that are important for people with Friedreich's Ataxia. The study will last for several months, during which participants will take the medication or placebo orally in the form of gastro-resistant tablets. These tablets are designed to pass through the stomach and dissolve in the intestines, which helps to protect the active ingredient until it reaches the right part of the body.

Throughout the study, researchers will monitor participants to see how the medication affects their health and any symptoms of Friedreich's Ataxia. This includes looking at changes in specific proteins and genes, as well as assessing physical abilities and overall well-being. The study aims to provide valuable information that could lead to better treatments for this condition in the future.

The research process

The trial runs in 5 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Joining the trial

    Upon joining the trial, you will be randomly assigned to one of two groups. One group will receive the actual medication, dimethyl fumarate, and the other group will receive a placebo, which looks like the medication but does not contain the active ingredient.

    This process is called a double-blind study, meaning neither you nor the researchers will know which group you are in. This helps ensure the results are unbiased.

  2. Step 2

    Medication administration

    If you are in the group receiving the actual medication, you will take Skilarence 120 mg gastro-resistant tablets orally. The dosage and frequency will be determined by the study protocol, and you will be informed of these details at the start of the trial.

    The core phase of the trial lasts for 12 weeks, during which you will continue taking the medication as instructed.

  3. Step 3

    Monitoring and assessments

    Throughout the trial, you will undergo regular assessments to monitor your health and the effects of the medication. These assessments may include physical exams, blood tests, and other evaluations to measure the impact on your condition.

    Specific tests will be conducted to evaluate changes in gene expression and protein levels related to Friedreich's Ataxia, as well as other health indicators.

  4. Step 4

    End of core phase

    At the end of the 12-week core phase, the primary outcomes will be assessed. This includes evaluating the effect of the medication on specific genetic and protein markers.

    You may be asked to continue in an extension phase of the study, where further data will be collected to assess long-term effects.

  5. Step 5

    Completion of trial

    Upon completion of the trial, you will have a final assessment to evaluate your overall health and any changes observed during the study.

    The results of the trial will contribute to understanding the efficacy and safety of dimethyl fumarate in treating Friedreich's Ataxia.

Who can join the trial?

4 criteria

  • Molecular diagnosis of Friedreich's Ataxia with a homozygous GAA expansion (a specific genetic change related to the condition).
  • Age 12 years or older.
  • Body weight of 30 kg (about 66 pounds) or more.
  • Ability to read and sign the informed consent (a document that explains the study and confirms your agreement to participate).

Who cannot join the trial?

7 criteria

  • Patients who do not have Friedreich's Ataxia cannot participate. This is a specific condition being studied.
  • Patients who are not within the age range specified for the study cannot participate. The study includes certain age groups only.
  • Patients who are not willing or able to follow the study procedures cannot participate. This means they must be able to attend all appointments and follow instructions.
  • Patients who have other medical conditions that might interfere with the study cannot participate. This is to ensure the study results are accurate.
  • Patients who are taking medications that might affect the study results cannot participate. This is to avoid any interference with the study's findings.
  • Patients who are pregnant or breastfeeding cannot participate. This is to ensure the safety of both the mother and the child.
  • Patients who have participated in another clinical trial recently cannot participate. This is to prevent any overlap that might affect the study results.
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Investigated drugs

Dimethyl Fumarate is a medication being tested in this clinical trial for its potential benefits in treating Friedreich Ataxia, a rare genetic disorder. The main goal of using Dimethyl Fumarate in this study is to see if it can help increase the production of a protein called frataxin, which is typically low in people with this condition. By boosting frataxin levels, the medication might help improve symptoms or slow the progression of the disease. Participants in the trial will receive this medication to evaluate its safety, effectiveness, and how well it is tolerated by patients with Friedreich Ataxia.

What is already known about the treatment

Dimethyl Fumarate – Dimethyl Fumarate is administered orally in the form of capsules. It is currently being studied in clinical trials for its potential use in treating Friedreich's Ataxia, a rare genetic disorder. The main therapeutic indication for this medication is to improve the production of frataxin protein, which is deficient in patients with Friedreich's Ataxia. At the molecular level, Dimethyl Fumarate works by activating a pathway that helps protect cells from stress and damage. It is classified as an immunomodulatory and neuroprotective agent.

Investigated diseases

Friedreich's Ataxia – Friedreich's Ataxia is a genetic disorder that affects the nervous system and the heart. It is characterized by progressive damage to the spinal cord and peripheral nerves, leading to muscle weakness and loss of coordination. The disease typically begins in childhood or early adulthood and gradually worsens over time. Individuals with Friedreich's Ataxia often experience difficulty walking, loss of sensation in the arms and legs, and impaired speech. As the condition progresses, it can also affect the heart, leading to conditions such as cardiomyopathy. The progression of symptoms varies among individuals, but it generally leads to increasing physical disability.
Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase IITrial ID2022-503016-16-00Protocol codeDMF-FA-201Estimated enrolment40 patientsSponsorUniversita' Degli Studi Di Napoli Federico II

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