Efficacy and Safety of Two Doses of Liarozole vs. Placebo for the Treatment of Lamellar Ichthyosis

Sponsor

Stiefel, a GSK Company (Industry)

Overall Status

Completed

CT.gov ID

NCT00282724

Collaborator

(none)

Enrollment

Locations

14.9

Duration (Months)

6.1

Patients Per Site

0.4

Patients Per Site Per Month

Study Details

Study Description

Brief Summary

Lamellar ichthyosis is a congenital disease of the skin with a generalized scaling. The primary activity of liarozole is considered to be the inhibition of the degradation of a substance called retinoic acid, which is the principal endogenous regulator of growth and differentiation of epithelial tissues in mammals. The current study intends to evaluate the efficacy and safety in patients with lamellar ichthyosis.

Condition or Disease	Intervention/Treatment	Phase
Ichthyosis, Lamellar	Drug: Liarozole	Phase 2/Phase 3

Detailed Description

Lamellar ichthyosis is an autosomal recessive disorder that is apparent at birth and is present throughout life. Although the disorder is not life threatening, it is quite disfiguring and causes considerable psychological stress to affected patients. Prevalence is less than 1 case per 300,000 individuals. Treatment is mainly symptomatic i.e. emollients with or without keratolytic agents. Treatment with systemic retinoids is reserved for those patients, refractory to conventional therapy, because of the long-term adverse effects and teratogenicity of systemic retinoids.

Liarozole may provide a new concept for the treatment of this condition. Because of its mechanism of action, retinoic acid (RA) levels will only be increased in tissues that are targets for RA production.

The proposed Phase II/III study intends to evaluate the efficacy of liarozole compared with placebo, in patients with lamellar ichthyosis.

Study Design

Study Type:

Interventional

Actual Enrollment :

98 participants

Allocation:

Randomized

Intervention Model:

Parallel Assignment

Masking:

Double (Participant, Investigator)

Primary Purpose:

Treatment

Official Title:

A Randomized, Double-blind, Placebo-controlled Phase II/III Trial to Evaluate the Efficacy and Safety of 2 Doses of Oral Liarozole (75 mg od and 150 mg od) Given During 12 Weeks in Lamellar Ichthyosis

Study Start Date :

Jan 1, 2006

Actual Primary Completion Date :

Apr 1, 2007

Actual Study Completion Date :

Apr 1, 2007

Outcome Measures

Primary Outcome Measures

Efficacy: Investigator's Global Assessment []

Secondary Outcome Measures

Overall Scaling Score []
Severity scores of other symptoms []
Quality of Life []
Safety and tolerability []
Pharmacokinetics []

Eligibility Criteria

Criteria

Ages Eligible for Study:

14 Years and Older

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

Inclusion Criteria:

Subjects of either sex aged 14 years or older.
Clinical diagnosis of lamellar ichthyosis
Women of childbearing potential should use appropriate contraception
Women of childbearing potential should have a negative pregnancy test at screening visit.
Subjects are, except for their lamellar ichthyosis, in good general health.
Subjects and legal representative(s), if applicable, signed informed consent.

Exclusion Criteria:

Subject is receiving topical (except emollient), UV treatment or systemic treatment for ichthyosis.
Subject is pregnant or breast feeding.
History or suspicion of alcohol or drug abuse.
Significant co-existing diseases.
Clinically significant abnormal ECG
History of hypersensitivity to retinoids or any of the ingredients in the trial medication.
Clinically relevant laboratory abnormalities at screening.
Use of immune-suppressive drugs including topical or systemic corticosteroids.
Participation in an investigational trial 30 days prior to the start of the trial.

Contacts and Locations

Locations

	Site	City	Country
1	Academisch Ziekenhuis Vrije Universiteit Brussel	Brussels	Belgium
2	Geel	Geel	Belgium
3	Hôpital Saint-Justine	Montreal	Canada
4	Newlab Clinical Research Inc.	St John	Canada
5	Instituto Dermatologico	Santo Domingo	Dominican Republic
6	Hôtel Dieu CHU	Nantes	France
7	Tomesa Fachklinik	Bad Salzschlirf	Germany
8	Dueren	Dueren	Germany
9	Otto-von-Guericke-Universität	Magdeburg	Germany
10	University Hospital Muenster	Muenster	Germany
11	Fondazione Policlinico Mangiagalli e Regina Elena	Milano	Italy
12	Istituto Dermopatico dell'Immacolata	Rome	Italy
13	Academisch Ziekenhuis Maastricht	Maastricht	Netherlands
14	University Hospital Rotterdam	Rotterdam	Netherlands
15	Rikshospitalet Universitetsklinikk	Oslo	Norway
16	Uppsala University Hospital	Uppsala	Sweden