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