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RECRUITING

NCT06513533

PHASE2/PHASE3

Dimethyl Fumarate in Adrenomyeloneuropathy

Sponsor: Pujol, Aurora, M.D.

View on ClinicalTrials.gov

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.

Official title: Effect of Dimethyl Fumarate Administered to Patients With Adrenomyeloneuropathy: a Multicenter, Placebo Controlled, Phase IIb/III Trial

Key Details

Gender

All

Age Range

18 Years - 65 Years

Study Type

INTERVENTIONAL

Enrollment

Start Date

2024-04-23

Completion Date

2028-06-30

Last Updated

2024-07-25

Healthy Volunteers

Conditions

Adrenomyeloneuropathy Without Cerebral Involvement

Interventions

OTHER

Placebo

1 tablet twice daily (one in the morning and one in the evening) for the first 7 days as a starter dose followed by 2 tablets (240 mg) twice daily

DRUG

Dimethyl fumarate

1 tablet twice daily (one in the morning and one in the evening) for the first 7 days as a starter dose followed by 2 tablets (240 mg) twice daily

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

Locations (3)

Bellvitge University Hospital

L'Hospitalet de Llobregat, Barcelona, Spain

Donostia University Hospital

Donostia / San Sebastian, Spain

University Hospital 12 de Octubre

Madrid, Spain