Computational Drug Repurposing for All EBS Cases
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The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. |
ClinicalTrials.gov Identifier: NCT03269474 |
Recruitment Status :
Active, not recruiting
First Posted : August 31, 2017
Last Update Posted : October 27, 2020
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Condition or disease | Intervention/treatment |
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Epidermolysis Bullosa Healthy Genetic Skin Disease Epidermolysis Bullosa Simplex Epidermolysis Bullosa, Junctional Epidermolysis Bullosa Dystrophica | Procedure: Experimental Group |
Study Type : | Observational |
Actual Enrollment : | 40 participants |
Observational Model: | Cohort |
Time Perspective: | Prospective |
Official Title: | Computational Drug Repurposing for All Epidermolysis Bullosa Simplex (EBS) Cases |
Actual Study Start Date : | November 28, 2017 |
Estimated Primary Completion Date : | December 30, 2020 |
Estimated Study Completion Date : | December 31, 2020 |

Group/Cohort | Intervention/treatment |
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Experimental Group
Blood and tissue specimen will be collected from subjects with an EB diagnosis. Tissue specimen will be collected from blistered and nonblistered skin.
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Procedure: Experimental Group
Subjects with EB diagnosis |
Control Group
Blood and tissue specimen will be collected from healthy subjects with non-EB. Tissue specimen will be collected from an inconspicuous skin area.
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Procedure: Experimental Group
Subjects with EB diagnosis |
- Characterize gene expression changes in EB 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

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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 |
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

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
United States, California | |
Pediatric Dermatology Clinic at Stanford Children's Hospital | |
Palo Alto, California, United States, 94304 |
Principal Investigator: | Joyce M Teng, MD, PhD | Stanford University |
Other Publications:
Responsible Party: | Joyce Teng, Director of Pediatric Dermatology, Stanford University |
ClinicalTrials.gov Identifier: | NCT03269474 |
Other Study ID Numbers: |
41142 |
First Posted: | August 31, 2017 Key Record Dates |
Last Update Posted: | October 27, 2020 |
Last Verified: | October 2020 |
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. |
Studies a U.S. FDA-regulated Drug Product: | No |
Studies a U.S. FDA-regulated Device Product: | No |
epidermolysis bullosa genetic expression drug repurposing computational approaches drug discovery |
Epidermolysis Bullosa Epidermolysis Bullosa Simplex Epidermolysis Bullosa Dystrophica Skin Diseases, Genetic Epidermolysis Bullosa, Junctional Skin Diseases |
Skin Abnormalities Congenital Abnormalities Genetic Diseases, Inborn Skin Diseases, Vesiculobullous Collagen Diseases Connective Tissue Diseases |