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

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
GlaxoSmithKline ( Stiefel, a GSK Company )
ClinicalTrials.gov Identifier:
NCT00282724
First received: January 20, 2006
Last updated: September 23, 2011
Last verified: September 2011
  Purpose

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 Intervention Phase
Ichthyosis, Lamellar
Drug: Liarozole
Phase 2
Phase 3

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, 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

Resource links provided by NLM:


Further study details as provided by GlaxoSmithKline:

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

Enrollment: 98
Study Start Date: January 2006
Study Completion Date: April 2007
Primary Completion Date: April 2007 (Final data collection date for primary outcome measure)
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.

  Eligibility

Ages Eligible for Study:   14 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
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.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00282724

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

Publications:

Additional publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: GlaxoSmithKline ( Stiefel, a GSK Company )
ClinicalTrials.gov Identifier: NCT00282724     History of Changes
Other Study ID Numbers: BT0500INT001
Study First Received: January 20, 2006
Last Updated: September 23, 2011
Health Authority: Germany: Federal Institute for Drugs and Medical Devices
France: Afssaps - Agence française de sécurité sanitaire des produits de santé (Saint-Denis)
Netherlands: Medicines Evaluation Board (MEB)
Italy: The Italian Medicines Agency
Sweden: Medical Products Agency
Norway: Norwegian Medicines Agency
Canada: Health Canada

Keywords provided by GlaxoSmithKline:
Lamellar ichthyosis
Liarozole
Investigator's Global Assessment
Scaling

Additional relevant MeSH terms:
Ichthyosiform Erythroderma, Congenital
Ichthyosis
Ichthyosis, Lamellar
Skin Abnormalities
Congenital Abnormalities
Infant, Newborn, Diseases
Keratosis
Skin Diseases
Skin Diseases, Genetic
Genetic Diseases, Inborn
Liarozole
Androgen Antagonists
Hormone Antagonists
Hormones, Hormone Substitutes, and Hormone Antagonists
Physiological Effects of Drugs
Pharmacologic Actions
Dermatologic Agents
Therapeutic Uses
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action
Antineoplastic Agents, Hormonal
Antineoplastic Agents

ClinicalTrials.gov processed this record on September 16, 2014