Effect of dimethyl fumarate administered to patients with adrenomyeloneuropathy: a multicenter, placebo controlled, phase IIb/III trial

Phase 1

Recruiting

Conditions: Adrenomyeloneuropathy
Therapeutic area: Diseases [C] - Nervous System Diseases [C10]

Registration Number: CTIS2023-506795-27-00

Lead Sponsor: Fundacio Institut D Investigacio Biomedica De Bellvitge IDIBE

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: Recruiting

Sex: All

Target Recruitment: 40

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, Not easily contactable by the investigator in case of emergency or not able to call the investigator, 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

Study & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: The primary objective of the trial is to demonstrate the superiority of DMF at 480 mg/day over placebo in the clinical improvement of patients with AMN.;Secondary Objective: The secondary objective of the trial is the assessment of safety of DMF in AMN patients.;Primary end point(s): The primary endpoint is the mean change in 2 Minute Walk Test (2MWT) between M0 and M24. This criterion will also be evaluated at M6, M12 and at the end of the extension phase (M36). Details about the 2MWT are given in chapter 3.7.

Secondary Outcome Measures