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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register.
Register: ClinicalTrials.gov
Last refreshed on: 16 December 2017
Main ID:  NCT03016715
Date of registration: 09/01/2017
Prospective Registration: No
Primary sponsor: Premier Specialists, Australia
Public title: Using Topical Sirolimus 2% for Patients With Epidermolysis Bullous Simplex (EBS) Study
Scientific title: A Prospective, Double-Blind, Cross-Over, Pilot Study to Assess Safety and Efficacy of Topical Sirolimus 2% in the Treatment of Plantar Blistering in Patients With Epidermolysis Bullous Simplex (EBS)
Date of first enrolment: May 2016
Target sample size: 8
Recruitment status: Recruiting
URL:  https://clinicaltrials.gov/show/NCT03016715
Study type:  Interventional
Study design:  Allocation: Randomized. Intervention model: Crossover Assignment. Primary purpose: Treatment. Masking: Triple (Participant, Care Provider, Investigator).  
Phase:  Phase 2
Countries of recruitment
Australia
Contacts
Name:     Dedee F Murrell, MD
Address: 
Telephone:
Email: d.murrell@unsw.edu.au
Affiliation: 
Name:     Charmaine Peras
Address: 
Telephone: 61 02 9598 5800
Email: premierspecnurse@gmail.com
Affiliation: 
Name:     Dedee F Murrell, MD
Address: 
Telephone:
Email:
Affiliation:  University of New South Wales
Key inclusion & exclusion criteria

Inclusion Criteria:

Subjects must:

- Be capable of understanding the purpose and risks of the study and sign a written
Informed Consent Form (ICF); Legally authorized representative of subjects willing and
able to give consent for children 5-18 yo

- Be male or female with a diagnosis of EBS

- Minimum EBDASI feet activity score of 2/10

- Age - 5 years or older

- Ability to complete 12 study visits within a 40-week period, each for approximately
30-60 minutes.

Anticipated life expectancy =52 weeks

- Males and females of childbearing potential should be using an effective means of
contraception.

- Laboratory values within the range of normal for the participating institution unless
the PI feels they are not clinically relevant

- Be able to comply with all study requirements

Exclusion Criteria:

- Allergy to sirolimus or components of the vehicle ointment

- Pregnancy, breast feeding

- Prior history of liver disease

- Serious known concurrent medical illness or infection, which could potentially present
a safety risk and/or prevent compliance with the requirements of the treatment
program.

- Known immunodeficiency virus or syndrome including those with:

- Acquired Immunodeficiency Syndrome (AIDS)

- Human Immunodeficiency Virus (HIV)

- Hepatitis B

- Prior history of grafting surgeries or other surgeries in the dermatologic treatment
area

- History of significant condition in the dermatologic treatment area such as trauma,
which could impair evaluation for the treatment of EBS or non-healing chronic wound.

- Use of other investigational drugs within 30 days of the screening visit and/or has
not recovered from any side effects of prior investigational drugs or procedure in the
affected area (e.g., a biopsy).

- Use of acitretin within the last 1 month

- Use of Roaccutane within last 3 months

- Botox injections to the feet within the last 6 months.

- Participant is planning extra physical activities within the next 3 months.

- Amputated foot



Age minimum: 5 Years
Age maximum: N/A
Gender: All
Health Condition(s) or Problem(s) studied
Weber-Cockayne Syndrome
Epidermolysis Bullosa Simplex
Epidermolysis Bullosa Simplex Kobner
Intervention(s)
Drug: Sirolimus 2%
Drug: Vehicle
Primary Outcome(s)
Foot Health Status Questionnaire [Time Frame: Change from Baseline to End of Treatment completion at 32 Week]
Secondary Outcome(s)
Plantar defect size using 3D Photography [Time Frame: Change in total defect area from Baseline, clinical visits at Week 4, Week 12, Week 16, Week 28, through study treatment completion at 32-weeks]
Child Dermatological Quality of Life Questionnaire [Time Frame: Baseline through study treatment completion at 32 weeks]
The Epidermolysis Bullosa Disease Activity and Scarring Index (EBDASI) Disease Severity Scale [Time Frame: Baseline and through study treatment completion at 32-weeks]
FitBitĀ® / pedometer [Time Frame: Baseline and through study treatment completion at 32-weeks]]
Secondary ID(s)
2016-05-413
Source(s) of Monetary Support
Please refer to primary and secondary sponsors
Secondary Sponsor(s)
Ethics review
Results
Results available:
Date Posted:
Date Completed:
URL:
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