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…
ID
Source
Brief title
Condition
- Metabolic and nutritional disorders congenital
- Spinal cord and nerve root disorders
Synonym
Research involving
Sponsors and support
Intervention
Outcome measures
Primary outcome
The primary outcome parameters are cholesterol levels (total-, LDL, and HDL)
and triglycerid levels in plasma, VLCFA levels in plasma, leukocytes and
erythrocytes and also C26:0-lyso-PC in bloodspots.
Secondary outcome
Secondary outcome parameters are side-effects (subjective and abnormalities in
the safety lab).
Background 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.
Study objective
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
12 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 vanapuncture for detecting possible side effects and to
determine the biochemical outcome parameters.
Intervention
Bezafibrate.
Study burden and risks
In rare cases bezafibrate can cause a (reversible) increase in liver
transaminases and/or rhabdomyolysis. This is a rare side effect. Patients will
also be required to visit the hospital 6 times for a venapuncture and will be
on study medication (bezafibrate) for a period of 6 months.
Meibergdreef 9
1100DD Amsterdam
NL
Meibergdreef 9
1100DD Amsterdam
NL
Listed location countries
Age
Inclusion criteria
- 18 years of age or older
- mentally competent and able to give informed consent and able to visit the hospital for follow-up
- no contra-indications for the use of the trial medication (kidney- and/or liver failure)
- confirmed diagnosis of X-ALD (by ABCD1 mutation analysis or abnormal plasma VLCFA)
Exclusion criteria
- Use of other medication that influences plasma cholesterol and/or triglycerid levels (for instance drugs of the statin class)
- Liver disease or increased CK (> 3 times upper limit of normal)
- Treament with VLCFA lowering agent (i.e. Lorenzo's oil).
- Unable to give informed consent
Design
Recruitment
Medical products/devices used
Followed up by the following (possibly more current) registration
No registrations found.
Other (possibly less up-to-date) registrations in this register
No registrations found.
In other registers
| Register | ID |
|---|---|
| EudraCT | EUCTR2009-015732-15-NL |
| CCMO | NL30249.018.09 |