Can a common MS drug help people with a rare nerve disease walk better?
NCT ID NCT06513533
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This clinical trial is testing whether dimethyl fumarate, a drug already used for multiple sclerosis, can improve balance and walking in adults with adrenomyeloneuropathy (AMN), a rare nerve disease. Forty participants will take either the drug or a placebo daily for 36 months, with regular clinic visits to measure motor function and safety. The goal is to see if the drug can ease motor problems and help understand the disease better.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- dimethyl fumarate (also known as Skilarence or Tecfidera)
- What this could lead to
- If it works, this could point toward a treatment that improves balance and walking in people with adrenomyeloneuropathy.
- What could go wrong
- This is a mid-stage trial with only 40 participants, so results may not apply to everyone. The drug is already used for other conditions, but its effectiveness for this specific disease is unproven.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 2/3
Runs two stages together: whether the treatment works, then large-scale confirmation.
- Participants
-
About 40 people
The number the study aims to enrol. It can still change while the study runs.
- Started
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Apr 2024
- Expected to finish
-
Jun 2028
An estimate. End dates often move.
- Lead sponsor
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An individual researcher
The lead sponsor is a named person rather than an organisation.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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18 to 65 years
- Sex
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Anyone
- Healthy volunteers
-
Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
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 cardiovascular disease, or any evolutive malignancy * Pregnancy and breast-feeding in woman and potential childbearing woman unable or unwilling to use an acceptable contraceptive method during the study * Any new medication for AMN initiated less than three months prior to inclusion * Contra-indications for MRI procedure such as subjects with paramagnetic materials in the body as aneurysm clips, pacemakers, intraocular metal or cochlear implants * Inclusion in another therapeutic clinical trial for ALD * Not easily contactable by the investigator in case of emergency or not able to call the investigator
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
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Bellvitge University Hospital
L'Hospitalet de Llobregat, Barcelona, 08907, Spain
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Donostia University Hospital
Donostia / San Sebastian, Donostia, 20014, Spain
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University Hospital 12 de Octubre
Madrid, Madrid, 28041, Spain