Dimethyl Fumarate in Adrenomyeloneuropathy (NCT06513533) | Clinical Trial Compass
Active — Not RecruitingPhase 2/3
Dimethyl Fumarate in Adrenomyeloneuropathy
Spain40 participantsStarted 2024-04-23
Plain-language summary
The goal of this clinical trial is to determine if dimethyl fumarate is effective in treating motor problems in adults with Adrenomyeloneuropathy. The trial will also assess the safety of dimethyl fumarate and explore the molecular mechanisms underlying the disease. The primary questions it aims to answer are:
* Does dimethyl fumarate improve motor problems in participants?
* What medical issues do participants experience while taking dimethyl fumarate? Researchers will compare the effects of dimethyl fumarate to a placebo (a substance that looks like the drug but contains no active ingredients) to evaluate its effectiveness in treating Adrenomyeloneuropathy.
Participants will:
* Take either dimethyl fumarate or a placebo daily for 36 months.
* Visit the clinic at the start of the trial, then at 3 months, 6 months, and every 6 months thereafter for checkups and tests.
Who can participate
Age range
18 Years – 65 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion Criteria:
* Men and women of 18 to 65 years old at the time of the inclusion, suffering from AMN with:
* elevated plasma VLCFA
* ABCD1 gene mutation identified
* Clinical signs of AMN with at least pyramidal signs in the lower limbs and difficulties to walk (EDSS score ≥ 2.0 and ≤ 6.5). EDSS score will also be re-evaluated at M12, M24 and M36.
* Normal brain MRI or brain MRI showing:
* abnormalities that can be observed in AMN patients without cerebral demyelination with a maximum Loes score of 4
* and/or stable (≥ 6 months) cerebral demyelination without gadolinium enhancement with a Loes score ≤ 12
* Appropriate steroid replacement if adrenal insufficiency is present
* Potential childbearing women should use an adequate method of contraception to avoid pregnancy throughout the study to minimize the risk of pregnancy. If oral contraceptives are used, the use of an alternative barrier method is recommended.
* Likely to be able to participate in all scheduled evaluations and complete all required study procedures
* Signed and dated written informed consent to participate in the study in accordance with local regulations
Exclusion Criteria:
* Any progressive neurological disease other than AMN
* Leukopenia below 3.0x109/L, lymphopenia below 0.5x109/L or other pathological results in the complete blood count
* Suspected or confirmed progressive multifocal leukoencephalopathy (PML)
* Severe gastrointestinal disease
* Uncontrolled hepatic, renal or cardiovas…
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Postural sway test
Timeframe: Interim analysis 1 (at 12 months of treatment)
2
Postural sway test
Timeframe: Final analysis 1 (at 24 months of treatment)
3
Postural sway test
Timeframe: Final analysis 2 (at 36 months of treatment)