Clinical efficacy and safety of R0002 cream in the initial and maintenance therapies of lamellar ichthyosis (LI)
- Conditions
- amellar ichthyosis (LI)Skin and Connective Tissue DiseasesLamellar ichthyosis (LI)
- Registration Number
- ISRCTN62315004
- Lead Sponsor
- Orfagen (France)
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Completed
- Sex
- All
- Target Recruitment
- 80
1. Male or female patients of at least 9 years old
2. Patients with a documented diagnosis of LI based on clinical signs, histopathology and/or genotype and, when possible, pedigree analysis
3. Patients requiring topical treatment by keratolytics either as monotherapy or as alternate therapy to oral retinoids
4. Patients with a global score of at least 3 for each parameter scaling and roughness, according to the scale used for primary efficacy parameter
5. Patients or patient?s parents/guardians able to understand and follow the study procedures
6. Written informed consent (personally signed and dated) from the patients and/or parent(s)/ guardian(s) (according to local legislation)
7. Patients or patients? parents/guardians affiliated to a healthcare security system (when applicable in the national regulation)
1. Patients under 9 years of age
2. Pregnant or lactating women
3. 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
3.1. [For german centers only] Young girls (9-17 years old) who are already of child bearing potential but not already taking a medical contraception (oral contraceptive, intra uterine contraceptive device) beforehand to the clinical trial, and unwilling to use condoms up to 8 weeks after the last test product application
4. Women of childbearing potential with a positive systemic pregnancy test at baseline
5. Patients with congenital ichthyoses other than LI
6. Patients with an erythrodermic component of LI (EARLI)
7. Patients with LI of overall severity < 3 for scaling or roughness, according to the scale used for primary efficacy parameter
8. Patients with lesional superinfection
9. Patients with skin or other disease likely to interfere with the study conduct or the evaluation parameters
10. Patients with excessive pruritus, burning, skin redness or peeling, not fully recovered at baseline
11. Patients with inherent sensitivity to sunlight
12. Patients with a known contact allergy to one of the ingredients contained in the test products
13. Patients treated with topicals (e.g. vitamin A analogues, vitamin D analogues) within 14 days prior to baseline
14. Patients treated with tazarotene gel within 28 days prior to baseline
15. Patients treated with keratolytics (e.g. urea, hydroxy-acids) or moisturisers other than the standard moisturiser within 7 days prior to baseline
16. Patients treated with concomitant dermatologic medications and cosmetics that have a strong drying effect within 7 days prior to baseline
17. 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
18. Patients treated with drugs known to be photosensitisers (e.g. thiazides, tetracyclines, quinolones, phenothiazines, sulfonamides, hydrochlorates, chlorpromazine, psoralen, amiodarone, tar) within 14 days prior to baseline
19. 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
20. Patients treated with ultraviolet (UV) therapy or patients medically exposed to UV within 28 days prior to baseline
21. Patients having significant sun exposure due to their occupation
22. Patients who participated in a study within the 3 months prior to study entry
23. Patients or patients? parents/guardians who are unable to understand and/or to follow the study procedures and patient instructions
24
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Response to test treatment at the end of initial therapy (D84): comparison between treatment groups of severity of the lesions (scaling and roughness), according to a severity scale for all periods, by an independent physician
- Secondary Outcome Measures
Name Time Method