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Computational Drug Repurposing for EBS

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ClinicalTrials.gov Identifier: NCT03269474
Recruitment Status : Active, not recruiting
First Posted : August 31, 2017
Last Update Posted : September 6, 2018
Sponsor:
Information provided by (Responsible Party):
Joyce Teng, Stanford University

Study Description
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Brief Summary:
The study will compare gene expression differences between blistered and non-blistered skin from individuals with EBS, as well as normal skin from non-EBS subjects. State of the art computational analysis will be performed to help identify new drugs that might help EBS wound healing and reduce pain. Researchers will focus on drugs that have already been approved for treatment of other dermatologic or non-dermatologic diseases, and therefore be repurposed for treatment of EBS. Drug development is a very expensive process taking decades for execution. Drug repurposing on the other hand, significantly reduces the cost and shortens the amount of time that is needed to bring effective treatments to clinical use. To date, there is no specific treatment targeting the physiology and immunologic response in EB patients during wound healing. Market availability of repurposed medications will provide EBS patients rapid access to treatments, thus improving their quality of life.

Condition or disease Intervention/treatment
Epidermolysis Bullosa Simplex Healthy Genetic Skin Disease Procedure: Experimental Group

Detailed Description:
Although gene, cell, and protein-based therapies are in development for patients suffering from epidermolysis bullosa simplex (EBS), new pharmacological treatments are in dire need. Characterizing molecular changes in EBS, including gene expression, can identify new therapeutic targets and drugs that modulate those targets. However, sifting through gene expression information to identify the most promising drug targets is a complex data challenge. The goal of the study will identify a computational approach to evaluate and identify existing drugs approved for other diseases that can be repurposed for EBS patients. The study will perform an unprecedented characterization of gene expression changes in EBS patients compared to healthy, non-EBS individuals across multiple tissues. Using a validated computational drug discovery platform, researchers will analyze gene expression and drug data using unique algorithms. In the first year, a list of ten, safety drugs more probable to treat the EBS disease state will be identified. The most promising drugs discovered will then be tested in the clinic setting.

Study Design
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Study Type : Observational
Actual Enrollment : 16 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: Computational Drug Repurposing for Epidermolysis Bullosa Simplex
Actual Study Start Date : November 28, 2017
Estimated Primary Completion Date : September 30, 2018
Estimated Study Completion Date : December 31, 2018

Resource links provided by the National Library of Medicine


Groups and Cohorts
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Group/Cohort Intervention/treatment
Experimental Group
Blood and tissue specimen will be collected from subjects with an EBS diagnosis. Tissue specimen will be collected from blistered and nonblistered skin.
 Procedure: Experimental Group
Subjects with EBS diagnosis
Control Group
Blood and tissue specimen will be collected from healthy subjects with non-EBS. Tissue specimen will be collected from an inconspicuous skin area.
 Procedure: Experimental Group
Subjects with EBS diagnosis


Outcome Measures
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Primary Outcome Measures :
  1. Characterize gene expression changes in EBS using RNA sequencing (RNA-seq) and Computational Profiling Potential Drug Targets [ Time Frame: Through the completion of study in 1 year. ]
    Using bioinformatic algorithms to identify changes in gene expression and review of over 2000 FDA-approved drugs based on predicted modulation of gene expression changes using a computational evolutionary algorithm system.


Biospecimen Retention:   Samples With DNA
Punch biopsies of non-blistering and blistering skin, and peripheral blood mononuclear cells (PBMCs): CD8+ cytotoxic T cells and CD4+ helper T cells.

Eligibility Criteria
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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   Yes
Sampling Method:   Probability Sample
Study Population
Subject of all ages with either 1) a diagnosis of EBS subjects or 2) healthy, non-EBS subjects
Criteria

Inclusion Criteria:

  • Subjects of all ages
  • Diagnosis of EBS subjects
  • Healthy, non-EBS 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
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03269474


Locations
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United States, California
Pediatric Dermatology Clinic at Stanford Children's Hospital
Palo Alto, California, United States, 94304
Sponsors and Collaborators
Joyce Teng
Investigators
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Principal Investigator: Joyce M Teng, MD, PhD Stanford University
More Information
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Publications of Results:

Other Publications:

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Responsible Party: Joyce Teng, Director of Pediatric Dermatology, Stanford University
ClinicalTrials.gov Identifier: NCT03269474     History of Changes
Other Study ID Numbers: 41142
First Posted: August 31, 2017    Key Record Dates
Last Update Posted: September 6, 2018
Last Verified: September 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No
Plan Description: As of now, there are no plans to share the data with other researchers. Once the outcome measures have been accomplished, the research team will publish results for the entire clinicaltrials.gov community and researchers for this vulnerable population study.

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Joyce Teng, Stanford University:
epidermolysis bullosa simplex
genetic expression
drug repurposing
computational approaches
drug discovery

Additional relevant MeSH terms:
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Skin Diseases
Epidermolysis Bullosa
Epidermolysis Bullosa Simplex
Skin Diseases, Genetic
 Skin Abnormalities
Congenital Abnormalities
Genetic Diseases, Inborn
Skin Diseases, Vesiculobullous