Status:
RECRUITING
Dimethyl Fumarate in Adrenomyeloneuropathy
Lead Sponsor:
Pujol, Aurora, M.D.
Collaborating Sponsors:
Instituto de Salud Carlos III
Spanish Clinical Research Network - SCReN
Conditions:
Adrenomyeloneuropathy Without Cerebral Involvement
Eligibility:
All Genders
18-65 years
Phase:
PHASE2
PHASE3
Brief 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 fum...
Detailed Description
Adrenoleukodystrophy (X-ALD) is the most prevalent rare genetic disorder affecting the brain's white matter. It is caused by mutations in the ABCD1 gene, which encodes a transporter involved in the de...
Eligibility Criteria
Inclusion
- 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
- 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
Key Trial Info
Start Date :
April 23 2024
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
June 30 2028
Estimated Enrollment :
40 Patients enrolled
Trial Details
Trial ID
NCT06513533
Start Date
April 23 2024
End Date
June 30 2028
Last Update
July 25 2024
Active Locations (3)
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1
Bellvitge University Hospital
L'Hospitalet de Llobregat, Barcelona, Spain, 08907
2
Donostia University Hospital
Donostia / San Sebastian, Spain, 20014
3
University Hospital 12 de Octubre
Madrid, Spain, 28041