Schilder's disease X-linked adrenoleukodystrofie
Conditions
Interventions
Bezafibrate.
Sponsors
Academisch Medisch Centrum
Eligibility
Age
18 Years to 99 Years
Inclusion criteria
Inclusion criteria: - 18 years of age or older - mentally competent and able to give informed consent and able to visit the hospital for follow-up - no contra-indications for the use of the trial medication (kidney- and/or liver failure) - confirmed diagnosis of X-ALD (by ABCD1 mutation analysis or abnormal plasma VLCFA)
Exclusion criteria
Exclusion criteria: - Use of other medication that influences plasma cholesterol and/or triglycerid levels (for instance drugs of the statin class) - Liver disease or increased CK (> 3 times upper limit of normal) - Treament with VLCFA lowering agent (i.e. Lorenzo's oil). - Unable to give informed consent
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| The primary outcome parameters are cholesterol levels (total-, LDL, and HDL) and triglycerid levels in plasma, VLCFA levels in plasma, leukocytes and erythrocytes and also C26:0-lyso-PC in bloodspots. | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary outcome parameters are side-effects (subjective and abnormalities in the safety lab). | — |
Countries
Netherlands
Outcome results
None listed