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

Study Overview
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Brief Summary
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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
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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.

Official Title
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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

Conditions
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Ichthyosis, Lamellar

Intervention / Treatment
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* Drug: Liarozole


Participation Criteria
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Eligibility Criteria
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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.

Ages Eligible for Study
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Minimum Age: 14 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double


Arms and Interventions

| Intervention/Treatment |
| --- |
|Drug: Liarozole|nan|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Efficacy: Investigator's Global Assessment |  |  | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Overall Scaling Score |  |  | 
| Severity scores of other symptoms |  |  | 
| Quality of Life |  |  | 
| Safety and tolerability |  |  | 
| Pharmacokinetics |  |  | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Lamellar ichthyosis, Liarozole, Investigator's Global Assessment, Scaling