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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT01165060
Recruitment Status : Completed
First Posted : July 19, 2010
Last Update Posted : August 10, 2011
The Stop ALD Foundation
Information provided by:
Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA)

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 lab).

Condition or disease Intervention/treatment Phase
X-linked Adrenoleukodystrophy Adrenomyeloneuropathy Drug: Bezafibrate Not Applicable

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 10 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Effect of Bezafibrate on Very Long Chain Fatty Acid Metabolism in Men With X-linked Adrenoleukodystrophy (X-ALD)
Study Start Date : July 2010
Actual Primary Completion Date : August 2011
Actual Study Completion Date : August 2011

Arm Intervention/treatment
Experimental: 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.
Drug: Bezafibrate
Week 0 to 12: 400 mg once daily 1 tablet. Week 13 to 24: 400 mg once daily 2 tablets.
Other Name: Bezalip retard.

Primary Outcome Measures :
  1. Very long chain fatty acids (VLCFA; C22:0, C24:0 and C26:0) in plasma, lymphocytes and erythrocytes. [ Time Frame: 24 weeks ]

Secondary Outcome Measures :
  1. Side effects [ Time Frame: At 4, 8, 12, 16, 20 and 24 weeks. ]
  2. Cholesterol (total-, HDL, and LDL-cholesterol) in plasma. [ Time Frame: 24 weeks ]

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT01165060

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Academisch Medisch Centrum
Amsterdam, NH, Netherlands, 1100 DD
Sponsors and Collaborators
Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA)
The Stop ALD Foundation
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Principal Investigator: Bwee Tien Poll - The, MD, PhD Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA)

Publications automatically indexed to this study by Identifier (NCT Number):
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Responsible Party: Department of (pediatric) neurology, Academisch Medisch Centrum, Amsterdam, The Netherlands Identifier: NCT01165060     History of Changes
Other Study ID Numbers: MEC 09/278
First Posted: July 19, 2010    Key Record Dates
Last Update Posted: August 10, 2011
Last Verified: July 2010

Keywords provided by Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA):
adrenomyeloneuropathy phenotype

Additional relevant MeSH terms:
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Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Hereditary Central Nervous System Demyelinating Diseases
Demyelinating Diseases
Mental Retardation, X-Linked
Intellectual Disability
Neurobehavioral Manifestations
Neurologic Manifestations
Genetic Diseases, X-Linked
Genetic Diseases, Inborn
Heredodegenerative Disorders, Nervous System
Metabolism, Inborn Errors
Peroxisomal Disorders
Metabolic Diseases
Adrenal Insufficiency
Adrenal Gland Diseases
Endocrine System Diseases
Hypolipidemic Agents
Molecular Mechanisms of Pharmacological Action
Lipid Regulating Agents