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

Phase 2

Not yet recruiting

• 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: 2024-05-06

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