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

NCT02960997 · Status: COMPLETED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 8

Last updated 2022-03-09

Study results available
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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

Principal Investigators

  • Joyce M Teng, MD, PhD · Stanford School of Medicine

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
TRIPLE
Model
CROSSOVER

Eligibility

Min Age
4 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2016-05-31
Primary Completion
2018-12-14
Completion
2021-12-01

Countries

  • United States

Study Locations

More Related Trials

Entities

Companies

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 NCT02960997 on ClinicalTrials.gov