Condition category
Skin and Connective Tissue Diseases
Date applied
30/03/2011
Date assigned
17/06/2011
Last edited
14/03/2014
Prospective/Retrospective
Retrospectively registered
Overall trial status
Completed
Recruitment status
No longer recruiting

Plain English Summary

Not provided at time of registration

Trial website

Contact information

Type

Scientific

Primary contact

Prof Ruzicka Thomas

ORCID ID

Contact details

Department of Dermatology and Allergies
Ludwig-Maximilian-University Munich
Frauenlobstraße 9-11
Munich
80337
Germany

Additional identifiers

EudraCT number

ClinicalTrials.gov number

Protocol/serial number

R00002 CR 301 (ORF)

Study information

Scientific title

Clinical efficacy and safety of R0002 cream in the initial and maintenance therapies of lamellar ichthyosis (LI): a randomised controlled trial

Acronym

Study hypothesis

1. To demonstrate the long-term clinical efficacy and safety of R0002 cream in LI patients in real-life setting conditions
2. To assess the durability of remission (relapse / prevention) under maintenance treatment
3. To assess the relapse/rebound after the end of the maintenance treatment.
4. To evaluate benefit and life quality outcome of the patients
5. 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
6. To monitor laboratory values during normal conditions of product use
7. To evaluate the overall acceptability of the product by the patients

On 04/03/2014 the following changes were made to the trial record:
1. The anticipated start date was changed from 01/04/2011 to 31/05/2011
2. The anticipated end date was changed from 31/08/2012 to 03/12/2013

On 10/03/2014 Morocco and Netherlands were removed from the countries of recruitment field.

Ethics approval

Ethics Committee of Hospital Necker, France approved on 13/12/2010. All other centres will seek ethics approval before recruitment of the first participant.

Study design

Period I: randomised double-blind two-parallel group comparative
Period II: open-labelled non comparative
Period III: randomised double-blind vehicle-controlled

Primary study design

Interventional

Secondary study design

Randomised controlled trial

Trial setting

Hospitals

Trial type

Treatment

Patient information sheet

Not available in web format, please use the contact details below to request a patient information sheet

Condition

Lamellar ichthyosis (LI)

Intervention

Wash-out period: (at least 7 days) observational, treatment free. Followed by:
1. Period I (84 days): R0002 cream every other day and its vehicle every other day (=> one arm of 40 patients) or urea cream 10% (for patients < 12 years old) or 5% (for children < 12 years old) every day (=> second arm of 40 patients)
2. Period II (84 days): R0002 cream every other day (=> all 80 patients)
3. Period III (up to 56 days): R0002 cream every other day (=> one arm of 40 patients) or its vehicle every other day (=> one arm of 40 patients)

Added 10/03/2014:
4. Period IV: open-labelled non comparative
5. Children follow-up: open-labelled, among children between 9 and 17 years old; follow-up without treatment

Intervention type

Other

Phase

Not Applicable

Drug names

Primary outcome measures

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

1. Time-course severity of scaling, roughness and erythema on the treated areas, according to the scale used in the primary efficacy parameter for scaling and roughness and to a severity scale for erythema, during all periods, by an independent physician
2. Assessment of the relapse/rebound of the treated areas, according to the scale defined in the primary efficacy parameter, during Period II and Period III
3. Separate assessment of the overall clinical severity of the lesions on palms and soles during all periods, according to a severity scale, by an independent physician
4. Autoevaluation of the overall severity of the lesions by the patients, during all periods
5. Life quality assessment by the patients, using the generic Short-Form 12 questionnaire (SF-12) for patients from 16 years of age and the Dermatologic Life Quality Index (DLQI) for patients over 16 years of age (i.e. from 17 years of age), or the child DLQI (CDLQI) for patients between 9 years and 16 years old, at Baseline, end of Period I, and end of Period II
6. Global local tolerance made on end of Period I and end of Period II according to a scale
7. Overall acceptability by the patients (efficacy, local tolerance, ease of use) at end of Period I and end of Period II
8. Plasma monitoring of tazarotenic acid at baseline, at end of Period I, end of Period II and end of period III
9. Blood laboratory tests at Baseline, end of Period I), end of Period II, and end of period III
10. Measurement of bone metabolism, using plasmatic biochemical markers at Baseline, end of Period I, end of Period II, and end of period III
11. Physical examination at each visit
12. Adverse events - an independent Data Safety Management Board (DSMB) will be set up to review adverse events during the study and take any decision in the interest of the patient safety
13. Compliance

Added 10/03/2014:
In period IV: all efficacy and safety secondary outcome measures at each visit
In children follow-up: safety outcome measures at each visit

Overall trial start date

31/05/2011

Overall trial end date

03/12/2013

Reason abandoned

Eligibility

Participant inclusion criteria

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)

Participant type

Patient

Age group

Child

Gender

Both

Target number of participants

About 80 patients in about 20 participating centres (2-10 patients/centre)

Participant exclusion criteria

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. Patients or patients’ parents/guardians who are unwilling to give personally signed and dated written informed consent

Recruitment start date

31/05/2011

Recruitment end date

03/12/2013

Locations

Countries of recruitment

Algeria, Austria, France, Germany, Italy, Sweden, Tunisia

Trial participating centre

Department of Dermatology and Allergies
Munich
80337
Germany

Sponsor information

Organisation

Orfagen (France)

Sponsor details

c/o Caroline Miklaszewski
Orfagen
CRDPF - Langlade
3
Avenue Hubert Curien
Cedex 1
Toulouse
31035
France

Sponsor type

Industry

Website

Funders

Funder type

Industry

Funder name

Orfagen (France)

Alternative name(s)

Funding Body Type

Funding Body Subtype

Location

Results and Publications

Publication and dissemination plan

Not provided at time of registration

Intention to publish date

Participant level data

Not provided at time of registration

Results - basic reporting

Publication summary

Publication citations

Additional files

Editorial Notes