The Effect of Bezafibrate on the Level of Very Long Chain Fatty Acids (VLCFA) in X-linked Adrenoleukodystrophy (X-ALD)

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

NA

Total Enrollment

10 participants

Study Classification

INTERVENTIONAL

Study Start Date

2010-07-31

Study Completion Date

2011-08-31

Brief Summary

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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 triglycerides in plasma, VLCFA levels in plasma, leukocytes and erythrocytes and also C26:0-lyso-PC in bloodspots.

Secondary study parameters/outcome of the study (if applicable):

Secondary outcome parameters are side-effects (subjective and abnormalities in the safety lab).

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Detailed Description

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Conditions

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X-linked Adrenoleukodystrophy Adrenomyeloneuropathy

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Bezafibrate

All patients in the trial will use bezafibrate 400 mg once daily until week 12, and subsequently use 800 mg onde daily until week 24.

Group Type EXPERIMENTAL

Bezafibrate

Intervention Type DRUG

Week 0 to 12: 400 mg once daily 1 tablet. Week 13 to 24: 400 mg once daily 2 tablets.

Interventions

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Bezafibrate

Week 0 to 12: 400 mg once daily 1 tablet. Week 13 to 24: 400 mg once daily 2 tablets.

Intervention Type DRUG

Other Intervention Names

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Bezalip retard.

Eligibility Criteria

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Inclusion Criteria

* an age of 18 years or older
* capable of giving informed consent and capable of visiting the hospital for follow-up visits
* no contra-indications for the use of bezafibrate, e.g. kidney- and/or liver disease.
* confirmed X-ALD, AMN phenotype (confirmed by VLCFA analysis or analysis of the ABCD1 gene)

Exclusion Criteria

* use of medication that lowers cholesterol and/or triglycerides (e.g. statins)
* liver disease or and increase in serum CK of more than 3 times the baseline level
* treatment with Lorenzo's oil in the 8 weeks preceding the trial

Minimum Eligible Age

18 Years

Eligible Sex

MALE

Accepts Healthy Volunteers

No