The Effect of Bezafibrate on the Level of Very Long Chain Fatty Acids (VLCFA) in X-linked Adrenoleukodystrophy (X-ALD) (BEZA)
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ClinicalTrials.gov Identifier: NCT01165060 |
Recruitment Status
:
Completed
First Posted
: July 19, 2010
Last Update Posted
: August 10, 2011
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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).
Condition or disease | Intervention/treatment | Phase |
---|---|---|
X-linked Adrenoleukodystrophy Adrenomyeloneuropathy | Drug: Bezafibrate | Not Applicable |
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.
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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.
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- Very long chain fatty acids (VLCFA; C22:0, C24:0 and C26:0) in plasma, lymphocytes and erythrocytes. [ Time Frame: 24 weeks ]
- 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 ]

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Ages Eligible for Study: | 18 Years and older (Adult, Senior) |
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

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 ClinicalTrials.gov identifier (NCT number): NCT01165060
Netherlands | |
Academisch Medisch Centrum | |
Amsterdam, NH, Netherlands, 1100 DD |
Principal Investigator: | Bwee Tien Poll - The, MD, PhD | Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA) |
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: | Department of (pediatric) neurology, Academisch Medisch Centrum, Amsterdam, The Netherlands |
ClinicalTrials.gov 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):
X-ALD AMN adrenomyeloneuropathy phenotype |
Additional relevant MeSH terms:
Adrenoleukodystrophy Brain Diseases, Metabolic, Inborn Brain Diseases, Metabolic Brain Diseases Central Nervous System Diseases Nervous System Diseases Hereditary Central Nervous System Demyelinating Diseases Leukoencephalopathies 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 Bezafibrate Hypolipidemic Agents Antimetabolites Molecular Mechanisms of Pharmacological Action Lipid Regulating Agents |