Application of zileuton (Zyflo CR) in patients with Sjögren Larsson Syndrome - Zileuton in Sjögren Larsson Syndrome
Phase 1
- Conditions
- Sjögren Larsson syndrome (SLS) is an autosomal recessive inherited neurometabolic disorder which is characterized by a clinical triad of congenital ichthyosis, spastic di- or tetraplegia and mental retardation. A disturbance of lipid metabolism due to deficiency of the microsomal fatty aldehyde dehydrogenase (FALDH) underlies SLS.
- Registration Number
- EUCTR2009-015895-87-NL
- Lead Sponsor
- MC St Radboud
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- 20
Inclusion Criteria
Biochemical and genetically proven Sjögren Larsson patients.
Age > 6 years.
Parental informed consent.
Are the trial subjects under 18? yes
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range
Exclusion Criteria
Renal and/or hepatic failure
Use of alcohol. Smoking.
Use of theophylline, warfarin and/or propranolol during the study period.
Use of zileuton less than 6 weeks prior to start of the study.
Patients not willing to maintain their dermatological treatment stable during the study period.
Pregnancy and/or lactation.
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Main Objective: To investigate the effect of Zileuton on the clinical parameters pruritus, ichthyosis as well as on the urinary concentrations of LTB4 and its metabolite omega-OH-LTB4.;Secondary Objective: Secondary, we study pharmacokinetic and pharmacodynamic properties of zileuton in humans as well as the dosage-effect relationship.;Primary end point(s): Clinical endpoints: Decrease in severity of pruritus and excoriations as well as improvement of ichthyosis measured by visual analogue scores (VAS), Willemsen score” (both filled out by parents/caregivers) as well as the clinical Sjögren Larsson Area Severity Index (SLASI). Decrease in the amount of topical emollients that is needed by the patients. <br><br>Biochemical endpoints: Significant decrease in urinary LTB4 and 20-OH-LTB4 metabolite concentrations of SLS patients during treatment with zileuton. <br>
- Secondary Outcome Measures
Name Time Method