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Can a common MS drug help people with a rare nerve disease walk better?

NCT ID NCT06513533

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing This study
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

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

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

Apr 2024

Expected to finish

Jun 2028

An estimate. End dates often move.

Lead sponsor

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

18 to 65 years

Sex

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

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

  • Bellvitge University Hospital

    L'Hospitalet de Llobregat, Barcelona, 08907, Spain

  • Donostia University Hospital

    Donostia / San Sebastian, Donostia, 20014, Spain

  • University Hospital 12 de Octubre

    Madrid, Madrid, 28041, Spain