Clinical Trial: The Effect of Bezafibrate on the Level of Very Long Chain Fatty Acids (VLCFA) in X-linked Adrenoleukodystrophy (X-ALD)
Study Status: Completed
Recruit Status: Completed
Study Type: Interventional
Official Title: Effect of Bezafibrate on Very Long Chain Fatty Acid Metabolism in Men With X-linked Adrenoleukodystrophy (X-ALD)
Brief Summary:
X-linked adrenoleukodystrophy (X-ALD) is an inherited metabolic disorder characterised by accumulation of very long chain fatty acids (VLCFA) in plasma and tissue. Presumably this accumulation is responsible for tissue damage. The disease can cause severe demyelinisation of the central nervous system usually causing death in childhood or progressive ambulatory problems in adults caused by a progressive myelopathy. For the latter category of patients no curative treatment is currently available. Recent investigations in human fibroblasts and mice identified bezafibrate as an agent that might reduce VLCFA in patients with X-ALD.
Objective of the study:
The trial is designed as an open-label pilot study. The main goal is to investigate if bezafibrate can reduce VLCFA in vivo in patient with X-ALD. If there is indeed a biochemical effect, a large follow-up study will be initiated with clinical outcome parameters.
Study design:
10 men with X-ALD will use bezafibrate during a period of 6 months (in combination with a low fat diet). On 6 different time points the participants will undergo a venipuncture for detecting possible side effects and to determine the biochemical outcome parameters.
Study population:
Adult men with X-linked adrenoleukodystrophy.
Intervention (if applicable):
Bezafibrate.
Primary study parameters/outcome of the study:
The primary outcome parameters are cholesterol levels (total-, LDL, and HDL) and levels of trigly
Detailed Summary:
Sponsor: Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA)
Current Primary Outcome: Very long chain fatty acids (VLCFA; C22:0, C24:0 and C26:0) in plasma, lymphocytes and erythrocytes. [ Time Frame: 24 weeks ]
Original Primary Outcome: Same as current
Current Secondary Outcome:
- Side effects [ Time Frame: At 4, 8, 12, 16, 20 and 24 weeks. ]
- Cholesterol (total-, HDL, and LDL-cholesterol) in plasma. [ Time Frame: 24 weeks ]
Original Secondary Outcome: Same as current
Information By: Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA)
Dates:
Date Received: July 13, 2010
Date Started: July 2010
Date Completion:
Last Updated: August 9, 2011
Last Verified: July 2010
