Effect of bezafibrate on very long chain fatty acid (VLCFA) metabolism in men with X-linked adrenoleukodystrophy.

Phase 2

• Conditions: 10027424; Schilder's disease; X-linked adrenoleukodystrofie; 10041543

• Registration Number: NL-OMON32731
• Lead Sponsor: Academisch Medisch Centrum

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Last Update Posted: 6 May 2024

Recruitment & Eligibility

• Status: Pending
• Sex: Not specified
• Target Recruitment: 8

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

- 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

Study & Design

• Study Type: Interventional
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
<p>The primary outcome parameters are cholesterol levels (total-, LDL, and HDL)<br /><br>and triglycerid levels in plasma, VLCFA levels in plasma, leukocytes and<br /><br>erythrocytes and also C26:0-lyso-PC in bloodspots.</p><br>

Secondary Outcome Measures

Name	Time	Method
<p>Secondary outcome parameters are side-effects (subjective and abnormalities in<br /><br>the safety lab).</p><br>