Lamellar Ichthyosis MedDRA version: 8.1 Level: LLT Classification code 10023686 Term: Lamellar ichthyosis
Conditions
Interventions
Product Name: Liarozole
Product Code: R085246
Pharmaceutical Form: Film-coated tablet
INN or Proposed INN: Liarozole fumarate
CAS Number: 145858-52-2
Concentration unit: mg milligram(s)
Concentration
Sponsors
Barrier Therapeutics, Inc.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Subjects of either sex aged 18 years or older 2. Clinical diagnosis of lamellar ichthyosis with at visits 1 and 2 an Investigator’s Global Assessment of at least 3 when defined as follows: Investigator’s global assessment of the overall severity of subject’s lamellar ichthyosis at a particular time point, taking into consideration the two individual characteristics of ichthyosis (scaling and/or erythema), measured by the following 5-point Likert scale (0 = clear; 1 = almost clear; 2 = mild; 3 = moderate; 4 = severe). 3. Women of childbearing potential are asked to use simultaneously 2 forms of effective contraception during the trial. The first one must be a hormonal contraceptive containing an association of progestin and estrogen and must be taken for at least 1 month (full cycle) before the start of treatment (visit 2). The second one must be a barier method with spermicide and must be used at least from visit 1. Both forms must then be used during treatment and until one month after the end of the treatment period. 4. Women of childbearing potential should have a negative pregnancy test at screening visit (Visit 1), confirmed by another pregnancy test at Visit 2 before randomization. 5. Subjects wearing contact lenses must agree not to wear their lenses and to use glasses when necessary for the entire treatment period. 6. Subjects are, except for their lamellar ichthyosis, in good general health and free of any disease state or physical condition that, in the investigator’s opinion, might impair evaluation of their lamellar ichthyosis. 7. Subjects signed informed consent. Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: 1. Concurrent inflammatory skin disease unrelated to ichthyosis. 2. Subject is receiving topical (except emollient) or UV treatment for ichthyosis in the 2 weeks prior to the start of treatment (Visit 2). 3. Subject is receiving systemic therapy for ichthyosis in the 4 weeks prior to the start of treatment (Visit 2). 4. Subject is pregnant or breast feeding. 5. History or suspicion of alcohol or drug abuse. 6. Significant co-existing hepatic, renal, immune disease, chronic pancreatitis, osteoporosis or a history indicating adrenal cortex dysfunction. 7. Heart disorder requiring treatment, or a myocardial infarction within the previous 24 weeks or a history of heart-failure or clinically relevant cardiac arrhythmia. 8. Clinically significant abnormal electrocardiogram-intervals or morphology of the electrocardiogram; QT or QTc >470 ms in women or >450 ms in men. 9. Subjects with risk factors for a QT/QTc prolongation (cardiac insufficiency, hypokalemia, a family history of long QT syndrome). 10. History of hypersensitivity to retinoids. 11. History of hypersensitivity to any of the ingredients in the trial medication. 12. Clinically relevant laboratory abnormalities at screening, especially levels of blood triglycerides, cholesterol and liver function parameters. 13. Use of Vitamin A supplements in the 4 weeks prior to the start of treatment or during the trial. 14. Use of disallowed drugs, mainly drugs metabolized by the CYP450 system during the treatment period. 15. Use of immune-suppressive drugs including topical or systemic corticosteroids. 16. Participation in an investigational trial 30 days prior to the start of the trial (Visit 1).
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To demonstrate the efficacy of oral liarozole, given at a dose of 75 mg o.d. or 150 mg o.d. vs. placebo in subjects with lamellar ichthyosis, as measered by the Investigator's Global Assessment at the end of 12 weeks of treatment.;Secondary Objective: · Investigator’s Global Assessment at 8 weeks of treatment · Overall Scaling Score · Individual severity scores for erythema and pruritus · Quality of Life (QOL): generic (SF-36) and disease specific (DLQI) questionnaires · Use of emollients and mechanical scale removal · Safety and tolerability · Pharmacokinetics · Photographs;Primary end point(s): The primary efficacy endpoint is the percentage of responders to treatment. A subject is considered a responder if the Investigator's Global Assessment is decreased with at least 2 points on the 5-point Likert scale by the end of the treatment period. | — |
Countries
Germany, Italy, Sweden
Outcome results
None listed