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

Primary Purpose

Ichthyosis, Lamellar

Status
Completed
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
Liarozole
Sponsored by
Stiefel, a GSK Company
About
Eligibility
Locations
Outcomes
Full info

About this trial

This is an interventional treatment trial for Ichthyosis, Lamellar focused on measuring Lamellar ichthyosis, Liarozole, Investigator's Global Assessment, Scaling

Eligibility Criteria

14 Years - undefined (Child, Adult, Older Adult)All SexesDoes not accept 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.

Sites / Locations

  • Academisch Ziekenhuis Vrije Universiteit Brussel
  • Geel
  • Hôpital Saint-Justine
  • Newlab Clinical Research Inc.
  • Instituto Dermatologico
  • Hôtel Dieu CHU
  • Tomesa Fachklinik
  • Dueren
  • Otto-von-Guericke-Universität
  • University Hospital Muenster
  • Fondazione Policlinico Mangiagalli e Regina Elena
  • Istituto Dermopatico dell'Immacolata
  • Academisch Ziekenhuis Maastricht
  • University Hospital Rotterdam
  • Rikshospitalet Universitetsklinikk
  • Uppsala University Hospital

Outcomes

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

Full Information

First Posted
January 20, 2006
Last Updated
September 23, 2011
Sponsor
Stiefel, a GSK Company
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1. Study Identification

Unique Protocol Identification Number
NCT00282724
Brief Title
Efficacy and Safety of Two Doses of Liarozole vs. Placebo for the Treatment of Lamellar Ichthyosis
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 Type
Interventional

2. Study Status

Record Verification Date
September 2011
Overall Recruitment Status
Completed
Study Start Date
January 2006 (undefined)
Primary Completion Date
April 2007 (Actual)
Study Completion Date
April 2007 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Stiefel, a GSK Company

4. Oversight

5. 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.
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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Ichthyosis, Lamellar
Keywords
Lamellar ichthyosis, Liarozole, Investigator's Global Assessment, Scaling

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2, Phase 3
Interventional Study Model
Parallel Assignment
Masking
ParticipantInvestigator
Allocation
Randomized
Enrollment
98 (Actual)

8. Arms, Groups, and Interventions

Intervention Type
Drug
Intervention Name(s)
Liarozole
Primary Outcome Measure Information:
Title
Efficacy: Investigator's Global Assessment
Secondary Outcome Measure Information:
Title
Overall Scaling Score
Title
Severity scores of other symptoms
Title
Quality of Life
Title
Safety and tolerability
Title
Pharmacokinetics

10. Eligibility

Sex
All
Minimum Age & Unit of Time
14 Years
Accepts Healthy Volunteers
No
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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Koen van Rossem, MD, PhD
Organizational Affiliation
Barrier Therapeutics/ Stiefel, a GSK Company
Official's Role
Study Director
Facility Information:
Facility Name
Academisch Ziekenhuis Vrije Universiteit Brussel
City
Brussels
Country
Belgium
Facility Name
Geel
City
Geel
Country
Belgium
Facility Name
Hôpital Saint-Justine
City
Montreal
Country
Canada
Facility Name
Newlab Clinical Research Inc.
City
St John
Country
Canada
Facility Name
Instituto Dermatologico
City
Santo Domingo
Country
Dominican Republic
Facility Name
Hôtel Dieu CHU
City
Nantes
Country
France
Facility Name
Tomesa Fachklinik
City
Bad Salzschlirf
Country
Germany
Facility Name
Dueren
City
Dueren
Country
Germany
Facility Name
Otto-von-Guericke-Universität
City
Magdeburg
Country
Germany
Facility Name
University Hospital Muenster
City
Muenster
Country
Germany
Facility Name
Fondazione Policlinico Mangiagalli e Regina Elena
City
Milano
Country
Italy
Facility Name
Istituto Dermopatico dell'Immacolata
City
Rome
Country
Italy
Facility Name
Academisch Ziekenhuis Maastricht
City
Maastricht
Country
Netherlands
Facility Name
University Hospital Rotterdam
City
Rotterdam
Country
Netherlands
Facility Name
Rikshospitalet Universitetsklinikk
City
Oslo
Country
Norway
Facility Name
Uppsala University Hospital
City
Uppsala
Country
Sweden

12. IPD Sharing Statement

Citations:
PubMed Identifier
2299598
Citation
Van Wauwe JP, Coene MC, Goossens J, Cools W, Monbaliu J. Effects of cytochrome P-450 inhibitors on the in vivo metabolism of all-trans-retinoic acid in rats. J Pharmacol Exp Ther. 1990 Jan;252(1):365-9.
Results Reference
background
PubMed Identifier
1374473
Citation
Van Wauwe J, Van Nyen G, Coene MC, Stoppie P, Cools W, Goossens J, Borghgraef P, Janssen PA. Liarozole, an inhibitor of retinoic acid metabolism, exerts retinoid-mimetic effects in vivo. J Pharmacol Exp Ther. 1992 May;261(2):773-9.
Results Reference
background
PubMed Identifier
8129749
Citation
Van Wauwe J, Coene MC, Cools W, Goossens J, Lauwers W, Le Jeune L, Van Hove C, Van Nyen G. Liarozole fumarate inhibits the metabolism of 4-keto-all-trans-retinoic acid. Biochem Pharmacol. 1994 Feb 11;47(4):737-41. doi: 10.1016/0006-2952(94)90137-6.
Results Reference
background
PubMed Identifier
8757760
Citation
Kang S, Duell EA, Kim KJ, Voorhees JJ. Liarozole inhibits human epidermal retinoic acid 4-hydroxylase activity and differentially augments human skin responses to retinoic acid and retinol in vivo. J Invest Dermatol. 1996 Aug;107(2):183-7. doi: 10.1111/1523-1747.ep12329579.
Results Reference
background
PubMed Identifier
8546999
Citation
Dockx P, Decree J, Degreef H. Inhibition of the metabolism of endogenous retinoic acid as treatment for severe psoriasis: an open study with oral liarozole. Br J Dermatol. 1995 Sep;133(3):426-32. doi: 10.1111/j.1365-2133.1995.tb02672.x.
Results Reference
background
PubMed Identifier
11122017
Citation
Berth-Jones J, Todd G, Hutchinson PE, Thestrup-Pedersen K, Vanhoutte FP. Treatment of psoriasis with oral liarozole: a dose-ranging study. Br J Dermatol. 2000 Dec;143(6):1170-6. doi: 10.1046/j.1365-2133.2000.03884.x.
Results Reference
background
PubMed Identifier
11703279
Citation
Bhushan M, Burden AD, McElhone K, James R, Vanhoutte FP, Griffiths CE. Oral liarozole in the treatment of palmoplantar pustular psoriasis: a randomized, double-blind, placebo-controlled study. Br J Dermatol. 2001 Oct;145(4):546-53. doi: 10.1046/j.1365-2133.2001.04411.x.
Results Reference
background
PubMed Identifier
9039298
Citation
Lucker GP, Heremans AM, Boegheim PJ, van de Kerkhof PC, Steijlen PM. Oral treatment of ichthyosis by the cytochrome P-450 inhibitor liarozole. Br J Dermatol. 1997 Jan;136(1):71-5.
Results Reference
background
PubMed Identifier
24102348
Citation
Vahlquist A, Blockhuys S, Steijlen P, van Rossem K, Didona B, Blanco D, Traupe H. Oral liarozole in the treatment of patients with moderate/severe lamellar ichthyosis: results of a randomized, double-blind, multinational, placebo-controlled phase II/III trial. Br J Dermatol. 2014 Jan;170(1):173-81. doi: 10.1111/bjd.12626.
Results Reference
derived

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

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