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

X-linked Adrenoleukodystrophy Clinical Trial

— BEZA  

Official title:

Effect of Bezafibrate on Very Long Chain Fatty Acid Metabolism in Men With X-linked Adrenoleukodystrophy (X-ALD)

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01165060
• Other study ID #: MEC 09/278
• Status: Completed
• Phase: N/A
• First received: July 13, 2010
• Last updated: August 9, 2011
• Start date: July 2010
• Est. completion date: August 2011

Study information

• Verified date: July 2010
• Source: Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA)
• Contact: n/a
• Is FDA regulated: No
• Health authority: Netherlands: The Central Committee on Research Involving Human Subjects (CCMO)
• Study type: Interventional

Clinical Trial 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 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).

Recruitment information / eligibility

• Status: Completed
• Enrollment: 10
• Est. completion date: August 2011
• Est. primary completion date: August 2011
• Accepts healthy volunteers: No
• Gender: Male
• Age group: 18 Years and older

Eligibility

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

Study Design

Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Adrenoleukodystrophy
• Adrenomyeloneuropathy
• X-linked Adrenoleukodystrophy

Intervention

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

Locations

Country	Name	City	State
Netherlands	Academisch Medisch Centrum	Amsterdam	NH

Sponsors (2)

Lead Sponsor	Collaborator
Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA)	The Stop ALD Foundation

Country where clinical trial is conducted

Netherlands, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Very long chain fatty acids (VLCFA; C22:0, C24:0 and C26:0) in plasma, lymphocytes and erythrocytes.		24 weeks	No
Secondary	Side effects		At 4, 8, 12, 16, 20 and 24 weeks.	Yes
Secondary	Cholesterol (total-, HDL, and LDL-cholesterol) in plasma.		24 weeks	No