Actively Recruiting
Dimethyl Fumarate in Adrenomyeloneuropathy
Led by Pujol, Aurora, M.D. · Updated on 2024-07-25
40
Participants Needed
3
Research Sites
218 weeks
Total Duration
On this page
Sponsors
P
Pujol, Aurora, M.D.
Lead Sponsor
I
Instituto de Salud Carlos III
Collaborating Sponsor
AI-Summary
What this Trial Is About
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.
CONDITIONS
Official Title
Dimethyl Fumarate in Adrenomyeloneuropathy
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Men and women aged 18 to 65 years
- Diagnosed with Adrenomyeloneuropathy (AMN) confirmed by elevated plasma VLCFA and ABCD1 gene mutation
- Clinical signs of AMN including pyramidal signs in the lower limbs and walking difficulties with EDSS score between 2.0 and 6.5
- Normal brain MRI or brain MRI showing AMN-related abnormalities with Loes score up to 4, or stable cerebral demyelination with Loes score up to 12
- Receiving appropriate steroid replacement if adrenal insufficiency is present
- Women of childbearing potential must use adequate contraception during the study
- Able to participate in all scheduled evaluations and complete required procedures
- Provided signed informed consent according to local regulations
You will not qualify if you...
- Progressive neurological diseases other than AMN
- Leukopenia below 3.0x10^9/L, lymphopenia below 0.5x10^9/L, or other abnormal blood counts
- Suspected or confirmed progressive multifocal leukoencephalopathy (PML)
- Severe gastrointestinal disease
- Uncontrolled liver, kidney, or heart disease, or any active cancer
- Pregnancy, breastfeeding, or women unable or unwilling to use acceptable contraception
- New AMN medication started less than three months before inclusion
- Contraindications for MRI such as having metal implants or devices like pacemakers
- Participation in another clinical trial for ALD
- Not easily reachable by the investigator or unable to contact in an emergency
AI-Screening
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Trial Site Locations
Total: 3 locations
1
Bellvitge University Hospital
L'Hospitalet de Llobregat, Barcelona, Spain, 08907
Actively Recruiting
2
Donostia University Hospital
Donostia / San Sebastian, Spain, 20014
Not Yet Recruiting
3
University Hospital 12 de Octubre
Madrid, Spain, 28041
Actively Recruiting
Research Team
A
Aurora Pujol, MD, PhD
CONTACT
S
Stéphane Fourcade, PhD
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
TRIPLE
Allocation
RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
2
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