A study to assess the efficacy of a vitamin D-containing ointment applied on the skin to improve wound healing in "butterfly children"

• Register: EUCTR
• Last refreshed on: 10 December 2018
• Main ID: EUCTR2016-001967-35-AT
• Date of registration: 28/06/2016
• Prospective Registration: Yes
• Primary sponsor: Universitätsklinik für Dermatologie, Paracelsus medizinische Privatuniversität Salzburg
• Public title: A study to assess the efficacy of a vitamin D-containing ointment applied on the skin to improve wound healing in "butterfly children"
• Scientific title: A double-blind, placebo-controlled cross-over study to assess the efficacy of topical calcipotriol (Psorcutan®-ointment containing 0.05 µg/g calcipotriol) to improve wound healing in dystrophic epidermolysis bullosa (DEB) - Topical calcipotriol in DEB
• Date of first enrolment: 19/07/2016
• Target sample size: 15
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2016-001967-35
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: yes; Randomised: yes; Open: no; Single blind: no; Double blind: yes; Parallel group: no; Cross over: yes; Other: no; If controlled, specify comparator, Other Medicinial Product: no; Placebo: yes; Other: no; Number of treatment arms in the trial: 2
• Phase: Human pharmacology (Phase I): no Therapeutic exploratory (Phase II): yes Therapeutic confirmatory - (Phase III): no Therapeutic use (Phase IV): no

Countries of recruitment

Austria

Contacts

• Name: EB-Haus Austria
• Affiliation: Universitätslinik für Dermatologie

• Name: EB-Haus Austria
• Affiliation: Universitätslinik für Dermatologie

Key inclusion & exclusion criteria

Inclusion criteria:
1. Signed Written Informed Consent
a) Subjects or legal guardians for minor children must have signed and dated an IRB/IEC approved written informed consent form in accordance with regulatory and institutional guidelines. This must be obtained before the performance of any protocol related procedures that are not part of normal subject care.

b) Subjects must be willing and able to comply with scheduled visits, treatment schedule, laboratory testing, and other requirements of the study.

2. Target Population
a) Subjects with dystrophic epidermolysis bullosa (DEB) with a known mutation in the type VII collagen gene.

b) Subjects must have at least 2 wounds with a minimum size of 6 cm2 per wound.

3. Age and Reproductive Status
a) Children = 6 years, men and women.

b) Pregnant women are excluded from the study.

c) Women of childbearing potential (not surgically sterilized and between menarche and 1 year postmenopause) must agree to use appropriate medically accepted highly effective methods of contraception (failure rate of less than 1% per year). Medically accepted highly effective methods of contraception are: 3-monthly contraceptive injection, combined oral hormonal contraceptive pills containing ethinyl estradiol and progestogens, oral hormonal contraception with Cerazette®, vaginal ring (e.g. NuvaRing®), contraceptive transdermal patch (e.g. Evra®), contraceptive implant (e.g. Implanon®) and gestagen-containing intrauterine device.
Negative Serum ß-Human Chorionic Gonadotrophin (ß-HCG) pregnancy test at screening and crossover.

d) Surgically sterilized female patients with documentation of prior
hysterectomy, tubal ligation or complete bilateral oophorectomy and postmenopausal women do not require contraception.

e) Women must not be breastfeeding.

Are the trial subjects under 18? yes
Number of subjects for this age range: 6
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 9
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion criteria:
1. Medical History and Concurrent Diseases
a) Simultaneous participation in another clinical trial.

b) Any serious or uncontrolled medical disorder that, in the opinion of the investigator, may impair the ability of the subject to receive protocol therapy, or interfere with the interpretation of study results.

c) Subjects with a condition requiring systemic treatment with either corticosteroids (>10 mg daily prednisone equivalents) or other immunosuppressive medications or antibiotics within14 days of study drug administration.

d) Subjects with known severely impaired kidney-or liver function.

e) Subjects with known disorders of calcium metabolism.

2. Allergies and Adverse Drug Reactions
a) History of a severe hypersensitivity reaction to calcipotriol or other study drug components.

3. Sex and Reproductive Status
a) Women of childbearing potential who are pregnant or breastfeeding.

b) Women with a positive pregnancy test at screening or crossover.



Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

Subjects with dystrophic epidermolysis bullosa (DEB) with a known mutation in the type VII collagen gene.
MedDRA version: 19.0 Level: PT Classification code 10014989 Term: Epidermolysis bullosa System Organ Class: 10010331 - Congenital, familial and genetic disorders

Intervention(s)

Trade Name: Psorcutan-Ointment
Pharmaceutical Form: Ointment
INN or Proposed INN: CALCIPOTRIOL
CAS Number: 112828-00-9
Concentration unit: µg microgram(s)
Concentration type: equal
Concentration number: 0.05-
Pharmaceutical form of the placebo: Ointment
Route of administration of the placebo: Topical use (Noncurrent)

Primary Outcome(s)

Timepoint(s) of evaluation of this end point: At screening (V1) and cross-over (V4) 2 target wounds (minimum size of 6 cm2 per wound) should be identified and photographically documented using a square grid.
Target wounds should also be photographically documented using a square grid at visits V2 (2 weeks after initiation of treatment), V3 (4 weeks after initiation of treatment), V5 (2 weeks after initiation of treatment after cross-over) and V6 (4 weeks after initiation of treatment after cross-over) and evaluated for changes in wound area.

Primary end point(s): 40% reduction of wound area in the treatment group compared to placebo after 4 weeks of treatment.

Main Objective: To establish the superiority of topical calcipotriol (Psorcutan®-ointment containing 0.05 µg/g calcipotriol) therapy over placebo with respect to improved wound-healing (40% reduction of wound area in the treatment group compared to placebo after 4 weeks of treatment) for subjects with DEB.

Secondary Objective: To evaluate and compare topical calcipotriol (Psorcutan®-ointment containing 0.05 µg/g calcipotriol) versus placebo with respect to bacterial wound colonization and with respect to pruritus/pain and to characterize the safety of topical calcipotriol (Psorcutan®-ointment containing 0.05 µg/g calcipotriol) therapy.

Secondary Outcome(s)

Timepoint(s) of evaluation of this end point: Bacterial swabs will be taken from the 2 target wounds, itch and pain of the 2 target wounds will be evaluated by using a visual analogue scale ranging from 0 (no itch/pain) to 10 (maximum itch/pain) as well as the use of itch/pain medication including drug name, dose, frequency and route of administration will be recorded at each study visit.
V1 (Screening visit)
V2 (2 weeks after initiation of treatment)
V3 (4 weeks after initiation of treatment)
V4 (Cross-over visit)
V5 (2 weeks after initiation of treatment after cross-over)
V6 (4 weeks after initiation of treatment after cross-over)

Adverse events will be documented at V2, V3 , V5 and V6.

Secondary end point(s): Evalution of bacterial wound colonization of the 2 target wound areas in the treatment group compared to placebo after 4 weeks of treatment.
Evaluation of pruritus/pain of the 2 target wounds in the treatment group compared to placebo after 4 weeks of treatment.
Characterization of the safety of topical calcipotriol therapy after 4 weeks of treatment.

Secondary ID(s)

CALCIDEB2016

Source(s) of Monetary Support

EB-Haus Austria