Bezafibrate in X-linked adrenoleukodystrophy (X-ALD).
Recruiting
- Conditions
- X-linked adrenoleukodystrophy (X-ALD), AMN phenotype.
- Registration Number
- NL-OMON24772
- Lead Sponsor
- niversity of Amsterdam, Academic Medical Center, Department of Neurology and Department of Genetic Metabolic Diseases.
- Brief Summary
/A
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Recruiting
- Sex
- Not specified
- Target Recruitment
- 10
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;
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;
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method 1. Cholesterol (total-, LDL-, and HDL-cholesterol) and triglycerides in plasma;<br /><br>2. VLCFA (C22:0, C24:0 and C26:0) in plasma, leukocytes and erythrocytes;<br /><br>3. C26:0-lysophosphatidylcholine in blood.
- Secondary Outcome Measures
Name Time Method Side effects reported by patients and abnormalities in the safety lab.