Using Topical Sirolimus 2% for Patients With Epidermolysis Bullous Simplex (EBS) Study

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT02960997
• Date of registration: 15/06/2016
• Prospective Registration: No
• Primary sponsor: Stanford University
• 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: Active, not recruiting
• URL: https://clinicaltrials.gov/show/NCT02960997
• 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

United States

Contacts

• Name: Joyce M Teng, MD, PhD
• Affiliation: Stanford School of Medicine

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 4-18 yo.

- Be male or female with a diagnosis of EBS

- Minimum EBDASI feet activity score of 2/10

- Age - 4 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: 4 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Epidermolysis Bullosa Simplex

Weber-Cockayne Syndrome

Epidermolysis Bullosa Simplex Kobner

Intervention(s)

Drug: Sirolimus, 2%

Drug: Vehicle

Primary Outcome(s)

Foot Health Status Questionnaire, Foot Function Domain Score [Time Frame: Week 0 and week 12 of the respective treatment period]

Trough Concentration of Sirolimus [Time Frame: Week 12]

Foot Health Status Questionnaire, Physical Activity Domain Score [Time Frame: Week 0 and week 12 of the respective treatment period]

Secondary Outcome(s)

Change in mTOR Pathway Inhibition [Time Frame: Baseline, week 12]

Plantar Defect Size Using 3D Photography [Time Frame: Baseline, week 12]

Epidermolysis Bullosa Disease Activity and Scarring Index (EBDASI) Disease Severity Scale Score [Time Frame: Week 0 and week 12 of the respective treatment period]

Foot Plantar Pressure Measurements [Time Frame: Baseline, week 12]

5-D Pruritus Scale Score [Time Frame: Week 0 and week 12 of the respective treatment period]

Average Steps Per Day Assessed by FitBit® / Pedometer [Time Frame: 12 weeks]

Child Dermatological Quality of Life Questionnaire Score [Time Frame: Week 0 and week 12 of the respective treatment period]

Secondary ID(s)

35498

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 30/01/2020
• URL: https://clinicaltrials.gov/ct2/show/results/NCT02960997