Trial Review

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Trial registered on ANZCTR


Registration number
ACTRN12606000320550
Ethics application status
Approved
Date submitted
19/07/2006
Date registered
26/07/2006
Date last updated
26/07/2006
Type of registration
Prospectively registered

Titles & IDs
Public title
Zileuton therapy for Sjogren-Larsson syndrome
Scientific title
Randomised crossover trial to evaluate the effects of Zileuton in patients with Sjogren-Larsson syndrome assessing potential benefit in terms of symptoms (itch), biochemical and neuropsychological indices
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Sjogren-Larsson syndrome 1290 0
Condition category
Condition code
Human Genetics and Inherited Disorders 1380 1380 0 0
Other human genetics and inherited disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Zileuton treatment, orally, dosage by weight:
12.5-18.75 kg: 300 mg twice a day
18.75-25 kg: 300 mg three times a day
25-37.5 kg: 300 mg four times a day
37.5-50 kg: 600 mg hree times a day
>50 kg: 600 mg qds
Patients provide their own internal control as crossover trial with treatment/placebo periods of 3 months separated by a one month wash out period.
Intervention code [1] 1214 0
None
Comparator / control treatment
No comparator.
Control group
Placebo

Outcomes
Primary outcome [1] 1881 0
Itch scores
Timepoint [1] 1881 0
At O, 3, and 7 months (crossover period), and 19 months (open label)
Primary outcome [2] 1882 0
Clinical neuropsychological evaluation
Timepoint [2] 1882 0
At O, 3, and 7 months (crossover period), and 19 months (open label)
Primary outcome [3] 1883 0
EEG
Timepoint [3] 1883 0
At O, 3, and 7 months (crossover period), and 19 months (open label)
Primary outcome [4] 1884 0
MRI/MRS
Timepoint [4] 1884 0
At O, 3, and 7 months (crossover period), and 19 months (open label)
Secondary outcome [1] 3324 0
Lack of adverse side effects of treatment.
Timepoint [1] 3324 0
At 7 months or 19 months if choose participate in the open label extension.

Eligibility
Key inclusion criteria
Paediatric patients with a clinical diagnosis and biochemically confirmed Sjogren-Larsson syndrome.
Minimum age
Not stated
Maximum age
16 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
Adult patients.

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Central randomisation by computer
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Simple randomisation by computer software
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?



Intervention assignment
Crossover
Other design features
Phase
Type of endpoint/s
Safety/efficacy
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Date of first participant enrolment
Anticipated
1/08/2006
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
3
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)

Funding & Sponsors
Funding source category [1] 1513 0
Hospital
Name [1] 1513 0
Sydney Children Hospital
Country [1] 1513 0
Australia
Funding source category [2] 1514 0
Commercial sector/Industry
Name [2] 1514 0
Critical Therapeutics Inc
Country [2] 1514 0
United States of America
Primary sponsor type
Government body
Name
South East Sydney Area Health Service
Address
Country
Australia
Secondary sponsor category [1] 1329 0
Hospital
Name [1] 1329 0
Sydney Children's Hospital
Address [1] 1329 0
Country [1] 1329 0
Australia

Ethics approval
Ethics application status
Approved
Ethics committee name [1] 2937 0
Sydney Children's Hospital, part of South East Sydney and Illawarra Area Health Service
Ethics committee address [1] 2937 0
Ethics committee country [1] 2937 0
Australia
Date submitted for ethics approval [1] 2937 0
Approval date [1] 2937 0
Ethics approval number [1] 2937 0
02/071

Summary
Brief summary
The purpose of this study is find out whether zileuton helps to improve the symptoms of children with SLS in terms of skin symptoms (itch), neurology and motor function.
patients, parents, investigators and asessors blinded - only central pharmacy will be aware of whether the patient is on placebo or treatment at the time (open label period excepted).
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 36088 0
Address 36088 0
Country 36088 0
Phone 36088 0
Fax 36088 0
Email 36088 0
Contact person for public queries
Name 10403 0
Dr Edwin Kirk
Address 10403 0
Department of Medical Genetics
Sydney Children's Hospital
High Street
Randwick NSW 2031
Country 10403 0
Australia
Phone 10403 0
+61 2 93821704
Fax 10403 0
Email 10403 0
[email protected]
Contact person for scientific queries
Name 1331 0
Dr Jason Pinner
Address 1331 0
Department of Medical Genetics
Sydney Children's Hospital
High Street
Randwick NSW 2031
Country 1331 0
Australia
Phone 1331 0
+61 2 93821704
Fax 1331 0
Email 1331 0
[email protected]

No information has been provided regarding IPD availability


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

Documents added manually
No documents have been uploaded by study researchers.

Documents added automatically
No additional documents have been identified.