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Effect of dimethyl fumarate administered to patients with adrenomyeloneuropathy: a multicenter, placebo controlled, phase IIb/III trial
EU CTIS ID: 2023-506795-27-00
What this study is testing
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.
- Phase II and Phase III (Integrated)
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- 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
You likely can't join if
- 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
See the full eligibility 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
- 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
The study team makes the final eligibility decision.
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 18-64 years. The study team makes the final eligibility decision.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.