Healthy Clinical Trial
Official title:
Computational Drug Repurposing for All Epidermolysis Bullosa Simplex (EBS) Cases
NCT number | NCT03269474 |
Other study ID # | 41142 |
Secondary ID | |
Status | Recruiting |
Phase | |
First received | |
Last updated | |
Start date | November 28, 2017 |
Est. completion date | December 31, 2024 |
The study will compare gene expression differences between blistered and non-blistered skin from individuals with all subtypes of EB, as well as normal skin from non-EB subjects. State of the art computational analysis will be performed to help identify new drugs that might help all EB 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 EB. 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 all EB patients rapid access to treatments, thus improving their quality of life.
Status | Recruiting |
Enrollment | 60 |
Est. completion date | December 31, 2024 |
Est. primary completion date | December 30, 2024 |
Accepts healthy volunteers | Accepts Healthy Volunteers |
Gender | All |
Age group | 0 Years and older |
Eligibility | 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 |
Country | Name | City | State |
---|---|---|---|
United States | Pediatric Dermatology Clinic at Stanford Children's Hospital | Palo Alto | California |
Lead Sponsor | Collaborator |
---|---|
Joyce Teng |
United States,
Bchetnia M, Tremblay ML, Leclerc G, Duperee A, Powell J, McCuaig C, Morin C, Legendre-Guillemin V, Laprise C. Expression signature of epidermolysis bullosa simplex. Hum Genet. 2012 Mar;131(3):393-406. doi: 10.1007/s00439-011-1077-7. Epub 2011 Aug 30. — View Citation
Bray NL, Pimentel H, Melsted P, Pachter L. Near-optimal probabilistic RNA-seq quantification. Nat Biotechnol. 2016 May;34(5):525-7. doi: 10.1038/nbt.3519. Epub 2016 Apr 4. Erratum In: Nat Biotechnol. 2016 Aug 9;34(8):888. — View Citation
Cohn HI, Teng JM. Advancement in management of epidermolysis bullosa. Curr Opin Pediatr. 2016 Aug;28(4):507-16. doi: 10.1097/MOP.0000000000000380. — View Citation
Law V, Knox C, Djoumbou Y, Jewison T, Guo AC, Liu Y, Maciejewski A, Arndt D, Wilson M, Neveu V, Tang A, Gabriel G, Ly C, Adamjee S, Dame ZT, Han B, Zhou Y, Wishart DS. DrugBank 4.0: shedding new light on drug metabolism. Nucleic Acids Res. 2014 Jan;42(Database issue):D1091-7. doi: 10.1093/nar/gkt1068. Epub 2013 Nov 6. — View Citation
Lee B, Geyfman M, Andersen B, Dai X. Analysis of gene expression in skin using laser capture microdissection. Methods Mol Biol. 2013;989:109-17. doi: 10.1007/978-1-62703-330-5_10. — View Citation
Li J, Zheng S, Chen B, Butte AJ, Swamidass SJ, Lu Z. A survey of current trends in computational drug repositioning. Brief Bioinform. 2016 Jan;17(1):2-12. doi: 10.1093/bib/bbv020. Epub 2015 Mar 31. — View Citation
Lovendorf MB, Mitsui H, Zibert JR, Ropke MA, Hafner M, Dyring-Andersen B, Bonefeld CM, Krueger JG, Skov L. Laser capture microdissection followed by next-generation sequencing identifies disease-related microRNAs in psoriatic skin that reflect systemic mi — View Citation
Low YS, Daugherty AC, Schroeder EA, Chen W, Seto T, Weber S, Lim M, Hastie T, Mathur M, Desai M, Farrington C, Radin AA, Sirota M, Kenkare P, Thompson CA, Yu PP, Gomez SL, Sledge GW Jr, Kurian AW, Shah NH. Synergistic drug combinations from electronic hea — View Citation
McLaren PJ, Mayne M, Rosser S, Moffatt T, Becker KG, Plummer FA, Fowke KR. Antigen-specific gene expression profiles of peripheral blood mononuclear cells do not reflect those of T-lymphocyte subsets. Clin Diagn Lab Immunol. 2004 Sep;11(5):977-82. doi: 10.1128/CDLI.11.5.977-982.2004. — View Citation
Nystrom A, Thriene K, Mittapalli V, Kern JS, Kiritsi D, Dengjel J, Bruckner-Tuderman L. Losartan ameliorates dystrophic epidermolysis bullosa and uncovers new disease mechanisms. EMBO Mol Med. 2015 Sep;7(9):1211-28. doi: 10.15252/emmm.201505061. — View Citation
Robinson MD, McCarthy DJ, Smyth GK. edgeR: a Bioconductor package for differential expression analysis of digital gene expression data. Bioinformatics. 2010 Jan 1;26(1):139-40. doi: 10.1093/bioinformatics/btp616. Epub 2009 Nov 11. — View Citation
Roth W, Reuter U, Wohlenberg C, Bruckner-Tuderman L, Magin TM. Cytokines as genetic modifiers in K5-/- mice and in human epidermolysis bullosa simplex. Hum Mutat. 2009 May;30(5):832-41. doi: 10.1002/humu.20981. — View Citation
Sleasman JW, Leon BH, Aleixo LF, Rojas M, Goodenow MM. Immunomagnetic selection of purified monocyte and lymphocyte populations from peripheral blood mononuclear cells following cryopreservation. Clin Diagn Lab Immunol. 1997 Nov;4(6):653-8. doi: 10.1128/cdli.4.6.653-658.1997. — View Citation
Subramanian A, Kuehn H, Gould J, Tamayo P, Mesirov JP. GSEA-P: a desktop application for Gene Set Enrichment Analysis. Bioinformatics. 2007 Dec 1;23(23):3251-3. doi: 10.1093/bioinformatics/btm369. Epub 2007 Jul 20. — View Citation
Sugaya N, Kanai S, Furuya T. Dr. PIAS 2.0: an update of a database of predicted druggable protein-protein interactions. Database (Oxford). 2012 Oct 10;2012:bas034. doi: 10.1093/database/bas034. Print 2012. — View Citation
Uitto J, Bruckner-Tuderman L, Christiano AM, McGrath JA, Has C, South AP, Kopelan B, Robinson EC. Progress toward Treatment and Cure of Epidermolysis Bullosa: Summary of the DEBRA International Research Symposium EB2015. J Invest Dermatol. 2016 Feb;136(2) — View Citation
Wally V, Kitzmueller S, Lagler F, Moder A, Hitzl W, Wolkersdorfer M, Hofbauer P, Felder TK, Dornauer M, Diem A, Eiler N, Bauer JW. Topical diacerein for epidermolysis bullosa: a randomized controlled pilot study. Orphanet J Rare Dis. 2013 May 7;8:69. doi: — View Citation
* Note: There are 17 references in all — Click here to view all references
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Characterize gene expression changes in EB using RNA sequencing (RNA-seq) and Computational Profiling Potential Drug Targets | 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. | Through the completion of study in 1 year. |
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