Lamellar Ichthyosis (LI) MedDRA version: 9.1 Level: LLT Classification code 10023686
Conditions
Interventions
Trade Name: Tazorac
Pharmaceutical Form: Cream
INN or Proposed INN: Tazarotene
Concentration unit: % (W/W) percent weight/weight
Concentration type: equal
Concentration number: .05-
Pharmaceutical for
Sponsors
ORFAGEN
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: Male or female patients of at least 9 years old, Patients with a documented diagnosis of LI based on clinical signs, histopathology and/or genotype and, when possible, pedigree analysis, Patients requiring topical treatment by keratolytics either as monotherapy or as alternate therapy to oral retinoids, Patients with a global score of at least 3 for each parameter scaling and roughness, according to the scale used for primary efficacy parameter, Patients or patient’s parents/guardians able to understand and follow the study procedures, Written informed consent from the patients or parents/guardians, Written informed consent (personally signed and dated) from the patients and/or parent(s)/guardian(s) (according to local legislation). Patients or patients’ parents/guardians affiliated to a healthcare security system (when applicable in the national regulation). Are the trial subjects under 18? yes 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: Patients under 9 years of age, Pregnant or lactating women, Women of childbearing potential with no reliable medical contraception (oral contraceptive, intra-uterine contraceptive device), and unwilling to use condoms up to 8 weeks after the last test product application, Women of childbearing potential with a positive systemic pregnancy test at baseline, Patients with congenital ichthyoses other than LI, Patients with an erythrodermic component of LI (EARLI), Patients with LI of overall severity < 3 for scaling or roughness, according to the scale used for primary efficacy parameter, Patients with lesional superinfection, Patients with skin or other disease likely to interfere with the study conduct or the evaluation parameters, Patients with excessive pruritus, burning, skin redness or peeling, not fully recovered at baseline, Patients with inherent sensitivity to sunlight, Patients with a known contact allergy to one of the ingredients contained in the test products, Patients treated with topicals (e.g. vitamin A analogues, vitamin D analogues) within 14 days prior to baseline, Patients treated with tazarotene gel within 28 days prior to baseline, Patients treated with keratolytics (e.g. urea, hydroxy-acids) or moisturizers other than the standard moisturizer within 7 days prior to baseline, Patients treated with concomitant dermatologic medications and cosmetics that have a strong drying effect within 7 days prior to baseline, Patients treated with oral retinoids during the preceding 28 days, or with oral vitamin A supplementation (more than 3000 IU per day) during the preceding 7 days of baseline, Patients treated with drugs known to be photosensitizers (e.g. thiazides, tetracyclines, quinolones, phenothiazines, sulfonamides, hydrochlorates, chlorpromazine, psoralen, amiodarone, tar) within 14 days prior to baseline, Patients with a medical condition that potentially alters bone metabolism (e.g. osteoporosis, thyroid dysfuntion, Cushing syndrome) or treated with a medication interfering with bone activity (e.g. corticosteroids, thyroid hormones, vitamin D analogues, cytotoxics, biphospbonates, calcitonins, tetracyclines, quinolones, thiazides, salicylates in long-term course, heparin, theophylline, barbiturates, colchicines) within the preceding 56 days prior to baseline, Patients treated with UV therapy or patients medically exposed to UV within 28 days prior to baseline, Patients having significant sun exposure due to their occupation, Patients who participated in a study within the 3 months prior to study entry, Patients or patients’ parents/guardians who are unable to understand and/or to follow the study procedures and patient instructions, Patients or patients’ parents/guardians who are unwilling to give personally signed and dated written informed consent.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To demonstrate the long-term clinical efficacy and safety of Tazarotene 0.05% cream in LI patients in real-life setting conditions.;Secondary Objective: - To assess the durability of remission (relapse/prevention) under maintenance treatment. - To assess the relapse/rebound after the end of the maintenance treatment. - To evaluate benefit and life quality outcome of the patients. - To measure the systemic absorption of the active drug in LI patients in the real life condition of product use at the end of initial therapy and maintenance therapy. - To monitor laboratory values during normal conditions of product use. - To evaluate the overall acceptability of the product by the patients.;Primary end point(s): Response to test treatment at the end of initial therapy (Day 84) At each study visit, the overall severity of scaling (visual assessment) and roughness (palpation) will be evaluated by an independent physician not involved in the management of the patients (masked assessment), according to the following 5-point Physician General Assessment (PGA) scale: 0 = Normal skin all over the treated BSA 1 = Lesions of slight intensity involving 30% of the treated BSA 3 = Lesions of moderate intensity involving > 30% of the treated BSA 4 = Lesions of severe intensity involving > 30% of the treated BSA | — |
Countries
Austria, Germany, Italy, Sweden
Outcome results
None listed