X-linked adrenoleukodystrophy (X-ALD), AMN phenotype.
Conditions
Interventions
Bezafibrate 400 mg 1 pd during three months, followed by bezafibrate 400 mg 2 pd during three months.
Sponsors
University of Amsterdam, Academic Medical Center, Department of Neurology and Department of Genetic Metabolic Diseases.
Eligibility
Inclusion criteria
Inclusion criteria: 1. An age of 18 years or older; 2. Capable of giving informed consent and physically capable of visiting the hospital for follow-up visits; 3. No contra-indications for the use of bezafibrate, e.g. kidney- and/or liver disease; 4. Confirmed X-ALD, AMN phenotype (confirmed by VLCFA analysis and mutation analysis of the ABCD1 gene).
Exclusion criteria
Exclusion criteria: 1. Use of medication that lowers cholesterol and/or triglycerides (e.g. statins); 2. Liver disease or and increase in serum CK of more than 3 times the baseline level; 3. Treatment with Lorenzo’s oil in the 8 weeks preceding the trial.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| 1. Cholesterol (total-, LDL-, and HDL-cholesterol) and triglycerides in plasma; 2. VLCFA (C22:0, C24:0 and C26:0) in plasma, leukocytes and erythrocytes; 3. C26:0-lysophosphatidylcholine in blood. | — |
Secondary
| Measure | Time frame |
|---|---|
| Side effects reported by patients and abnormalities in the safety lab. | — |
Contacts
Public ContactM. Engelen
Academic Medical Center (AMC), Department of Neurology, P.O. Box 22660.
Outcome results
None listed