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Study on the Effects of Dimethyl Fumarate for Patients with Adrenomyeloneuropathy

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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 the effects of a medication called dimethyl fumarate on patients with a condition known as adrenomyeloneuropathy (AMN). AMN is a rare genetic disorder that affects the nervous system and adrenal glands, leading to symptoms such as difficulty walking and muscle weakness. The trial aims to determine if dimethyl fumarate can improve the clinical condition of patients with AMN compared to a placebo. Dimethyl fumarate is administered in the form of gastro-resistant tablets, which are designed to withstand stomach acid and dissolve in the intestines.

Participants in the study will receive either dimethyl fumarate or a placebo over a period of 36 months. The study will monitor changes in the participants' ability to walk, using tests like the 2 Minute Walk Test (2MWT) and the 6 Minute Walk Test (6MWT). These tests help assess improvements in walking ability and overall mobility. Other assessments will include tests for balance, strength, and the ability to climb stairs, as well as questionnaires about urinary and bowel function. The study will also involve neuroimaging techniques, such as MRI, to observe changes in the brain, and blood tests to measure markers of nerve damage and inflammation.

In addition to dimethyl fumarate, the study will also involve the use of magnesium stearate and microcrystalline cellulose, which are common substances used in tablet formulations. These substances are included in the study to ensure the tablets are properly formed and effective. The trial is designed to provide valuable information on the potential benefits of dimethyl fumarate for individuals with AMN, with the hope of improving their quality of life and managing the symptoms of this challenging 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

    Joining the study

    Upon joining the study, eligibility is confirmed based on specific criteria such as age, medical condition, and ability to participate in evaluations.

    Written informed consent is required to participate in the study.

  2. Step 2

    Initial assessment

    An initial assessment is conducted to evaluate clinical signs of adrenomyeloneuropathy (AMN) and other health parameters.

    This includes a brain MRI and evaluation of walking ability using the Expanded Disability Status Scale (EDSS).

  3. Step 3

    Medication administration

    The medication, dimethyl fumarate, is administered orally in the form of Skilarence 120 mg gastro-resistant tablets.

    The dosage is 480 mg per day.

  4. Step 4

    Ongoing assessments

    Regular assessments are conducted to monitor progress and response to the medication.

    These include the 2 Minute Walk Test (2MWT) and other evaluations at months 6, 12, 24, and 36.

  5. Step 5

    Secondary evaluations

    Additional tests such as the 6 Minute Walk Test (6MWT), Time to walk 25 feet (TW25), and postural sway are performed.

    Questionnaires about urinary and fecal function, as well as neuroimaging and biological markers, are also part of the evaluation.

  6. Step 6

    Completion of study

    The study is expected to conclude by December 18, 2026.

    Final assessments are conducted to evaluate the overall effectiveness of the treatment.

Who can join the trial?

7 criteria

  • Men and women aged 18 to 65 years old who have **Adrenomyeloneuropathy (AMN)**. This includes having high levels of certain fats in the blood called **VLCFA** and a specific change in the **ABCD1 gene**.
  • Must show signs of AMN, such as problems with movement in the lower limbs and difficulty walking. This is measured by a score called **EDSS**, which should be between 2.0 and 6.5.
  • Must have a normal brain scan (**MRI**) or one that shows changes typical in AMN patients without severe brain damage. The **Loes score**, which measures brain changes, should be 4 or less, or if there is stable brain damage, the score should be 12 or less.
  • If the patient has **adrenal insufficiency** (a condition where the body doesn't produce enough hormones), they should be receiving the right treatment with steroids.
  • Women who can have children should use a reliable method of birth control to prevent pregnancy during the study. If using birth control pills, it's recommended to also use another method like condoms.
  • The patient should be able to attend all planned check-ups and complete all necessary study activities.
  • The patient must provide a signed and dated written agreement to participate in the study, following local rules.

Who cannot join the trial?

3 criteria

  • Patients who have a different condition than Adrenomyeloneuropathy cannot participate. Adrenomyeloneuropathy is a rare genetic disorder that affects the nervous system and muscles.
  • Patients who are not within the specified age range for the study cannot participate. The age range is not specified in the provided data.
  • Patients who are part of a vulnerable population cannot participate. A vulnerable population includes groups like children, pregnant women, or those unable to give consent.
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Investigated drugs

Dimethyl Fumarate (DMF) is a medication being tested in this clinical trial for its potential to improve the condition of patients with adrenomyeloneuropathy (AMN). The trial aims to determine if DMF can provide better clinical outcomes compared to not receiving the active treatment. DMF is already used in other conditions, and this study is exploring its effectiveness and safety for AMN.

What is already known about the treatment

Dimethyl Fumarate – This medication is administered orally in the form of capsules. It is currently being studied in a phase IIb/III clinical trial for its effectiveness in treating adrenomyeloneuropathy, a rare neurological disorder. The main therapeutic indication for dimethyl fumarate in this trial is to improve clinical outcomes in patients with this condition. At the molecular level, dimethyl fumarate works by activating the Nrf2 pathway, which helps protect cells from oxidative stress. It is classified as an immunomodulatory agent.

Investigated diseases

Adrenomyeloneuropathy – Adrenomyeloneuropathy is a genetic disorder that primarily affects the spinal cord and peripheral nerves. It is a form of X-linked adrenoleukodystrophy, which is caused by mutations in the ABCD1 gene. This disease typically manifests in adulthood and is characterized by progressive stiffness and weakness in the legs, leading to difficulty walking. Over time, individuals may experience problems with balance and coordination, as well as bladder and bowel dysfunction. The condition can also affect the adrenal glands, leading to hormonal imbalances. Symptoms and progression can vary widely among individuals.
Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase IVTrial ID2023-506795-27-00Estimated enrolment40 patientsSponsorFundacio Institut D'Investigacio Biomedica De Bellvitge IDIBELL

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