Using computational methods to find new treatments for epidermolysis bullosa
Computational Drug Repurposing for All Epidermolysis Bullosa Simplex (EBS) Cases
This study is trying to find new treatments for people with epidermolysis bullosa by looking at skin samples to see if existing medications can help heal their wounds and ease their pain.
Quick facts
| Study type | Observational |
|---|---|
| Enrollment | 60 (estimated) |
| Ages | 0 Years and up |
| Sex | All |
| Sponsor | Stanford University Academic / other |
| Locations | 1 site (Palo Alto, California) |
| Trial ID | NCT03269474 on ClinicalTrials.gov |
What this trial studies
This observational study aims to compare gene expression differences between blistered and non-blistered skin from individuals with all subtypes of epidermolysis bullosa (EB) and healthy individuals. By utilizing advanced computational analysis, researchers will identify existing drugs that have been approved for other conditions and may be repurposed to improve wound healing and reduce pain in EB patients. The study will characterize molecular changes in EB to uncover new therapeutic targets, ultimately aiming to provide rapid access to effective treatments for patients. The first year will focus on identifying ten promising drugs for further clinical testing.
Who should consider this trial
Good fit: Ideal candidates include individuals of all ages diagnosed with any subtype of epidermolysis bullosa, as well as healthy non-EB subjects for comparison.
Not a fit: Patients who are pregnant, breastfeeding, or have serious concurrent medical illnesses that could complicate tissue collection may not benefit from this study.
Why it matters
Potential benefit: If successful, this approach could lead to new, effective treatments for patients suffering from epidermolysis bullosa, significantly improving their quality of life.
How similar studies have performed: While drug repurposing is a well-established concept, this specific computational approach for epidermolysis bullosa is novel and has not been extensively tested in prior studies.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * Subjects of all ages * Diagnosis of all subtypes of EB subjects * Healthy, non-EB subjects * Ability to complete study visit to collect tissue and blood specimen Exclusion Criteria: * 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 tissue collection from subjects
Where this trial is running
Palo Alto, California
- Pediatric Dermatology Clinic at Stanford Children's Hospital — Palo Alto, California, United States (Recruiting)
Study contacts
- Principal investigator: Joyce M Teng, MD, PhD — Stanford University
- Study coordinator: Monica Martin
- Email: momartin@stanford.edu
- Phone: 650-723-0636
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.