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

NCT03016715 · Status: UNKNOWN · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 8

Last updated 2017-10-24

No results posted yet for this study

Summary

: Epidermolysis bullosa (EB) simplex is a rare orphan disease caused by a mutation in DNA leading to abnormal dominant keratins in the skin. Patients with EB simplex develop lifelong painful thick soles on their feet, and current standard of care is supportive. This pilot study will target the dominant mutant keratin proteins in the skin to ameliorate the severity of EB simplex. The purpose is to improve the function of EB simplex feet with an application of topical sirolimus, 2%. The investigators plan on inhibiting the mTOR pathway to down regulate the translation of defective keratin proteins and work through anti proliferative pathways.

Conditions

  • Epidermolysis Bullosa Simplex
  • Epidermolysis Bullosa Simplex Kobner
  • Weber-Cockayne Syndrome

Interventions

DRUG

Sirolimus 2%

DRUG

Vehicle

Sponsors & Collaborators

  • Premier Specialists, Australia

    lead OTHER

Principal Investigators

  • Dedee F Murrell, MD · University of New South Wales

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
TRIPLE
Model
CROSSOVER

Eligibility

Min Age
5 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2016-05-31
Primary Completion
2019-06-30

Countries

  • Australia

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT03016715 on ClinicalTrials.gov