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

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

Sponsors

Lead Sponsor: Stiefel, a GSK Company

Source GlaxoSmithKline
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.

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.

Overall Status Completed
Start Date January 2006
Completion Date April 2007
Primary Completion Date April 2007
Phase Phase 2/Phase 3
Study Type Interventional
Primary Outcome
Measure Time Frame
Efficacy: Investigator's Global Assessment
Secondary Outcome
Measure Time Frame
Overall Scaling Score
Severity scores of other symptoms
Quality of Life
Safety and tolerability
Pharmacokinetics
Enrollment 98
Condition
Intervention

Intervention Type: Drug

Intervention Name: Liarozole

Eligibility

Criteria:

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.

Gender: All

Minimum Age: 14 Years

Maximum Age: N/A

Healthy Volunteers: No

Overall Official
Last Name Role Affiliation
Koen van Rossem, MD, PhD Study Director Barrier Therapeutics/ Stiefel, a GSK Company
Location
Facility:
Academisch Ziekenhuis Vrije Universiteit Brussel | Brussels, Belgium
Geel | Geel, Belgium
Hôpital Saint-Justine | Montreal, Canada
Newlab Clinical Research Inc. | St John, Canada
Instituto Dermatologico | Santo Domingo, Dominican Republic
Hôtel Dieu CHU | Nantes, France
Tomesa Fachklinik | Bad Salzschlirf, Germany
Dueren | Dueren, Germany
Otto-von-Guericke-Universität | Magdeburg, Germany
University Hospital Muenster | Muenster, Germany
Fondazione Policlinico Mangiagalli e Regina Elena | Milano, Italy
Istituto Dermopatico dell'Immacolata | Rome, Italy
Academisch Ziekenhuis Maastricht | Maastricht, Netherlands
University Hospital Rotterdam | Rotterdam, Netherlands
Rikshospitalet Universitetsklinikk | Oslo, Norway
Uppsala University Hospital | Uppsala, Sweden
Location Countries

Belgium

Canada

Dominican Republic

France

Germany

Italy

Netherlands

Norway

Sweden

Verification Date

September 2011

Responsible Party

Type: Sponsor

Keywords
Has Expanded Access No
Condition Browse
Study Design Info

Allocation: Randomized

Intervention Model: Parallel Assignment

Primary Purpose: Treatment

Masking: Double (Participant, Investigator)

Source: ClinicalTrials.gov