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

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Brief Title

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

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):


      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

Study Type


Primary Outcome

Very long chain fatty acids (VLCFA; C22:0, C24:0 and C26:0) in plasma, lymphocytes and erythrocytes.

Secondary Outcome

 Side effects


X-linked Adrenoleukodystrophy



Study Arms / Comparison Groups

Description:  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.


* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information

Recruitment Status


Estimated Enrollment


Start Date

July 2010

Completion Date

August 2011

Primary Completion Date

August 2011

Eligibility Criteria

        Inclusion Criteria:

          -  an age of 18 years or older

          -  capable of giving informed consent and capable of visiting the hospital for follow-up

          -  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

        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




18 Years - N/A

Accepts Healthy Volunteers



Bwee Tien Poll - The, MD, PhD, , 

Location Countries


Location Countries


Administrative Informations



Organization ID

MEC 09/278

Study Sponsor

Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA)


 The Stop ALD Foundation

Study Sponsor

Bwee Tien Poll - The, MD, PhD, Principal Investigator, Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA)

Verification Date

July 2010