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| Sponsor: | Stanford University |
|---|---|
| Collaborator: |
National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS) |
| Information provided by (Responsible Party): | Alfred Lane, Stanford University |
| ClinicalTrials.gov Identifier: | NCT01263379 |
Purpose
Recessive dystrophic epidermolysis bullosa (RDEB) is a severe inherited blistering skin disease caused by absence of a protein known as type VII collagen. Patients with RDEB develop large, severely painful blisters and open wounds from minor trauma to their skin. This trial will create a graft, which the investigators call "LEAES," of the patient's own skin that has been genetically engineered in the investigators lab to express this missing protein. The purpose of this study is to achieve proof-of-concept for this general approach to cell-based gene therapy in humans and to set the stage for further therapeutic extension in RDEB. The investigators will basically take a subject's own cells, correct them in culture, and then transplant the corrected cells back onto them.
| Condition | Intervention | Phase |
|---|---|---|
|
Epidermolysis Bullosa Dystrophica Epidermolysis Bullosa |
Genetic: LZRSE-Col7A1 Engineered Autologous Epidermal Sheets (LEAES) |
Phase 1 |
| Study Type: | Interventional |
| Study Design: | Allocation: Non-Randomized Endpoint Classification: Safety/Efficacy Study Intervention Model: Single Group Assignment Masking: Open Label Primary Purpose: Treatment |
| Official Title: | A Phase 1 Single Center Trial of Gene Transfer for Recessive Dystrophic Epidermolysis Bullosa (RDEB) Using the Drug LZRSE-Col7A1 Engineered Autologous Epidermal Sheets (LEAES) |
| Estimated Enrollment: | 12 |
| Study Start Date: | December 2010 |
| Estimated Study Completion Date: | December 2015 |
| Estimated Primary Completion Date: | December 2015 (Final data collection date for primary outcome measure) |
Eligibility| Ages Eligible for Study: | 18 Years and older |
| Genders Eligible for Study: | Both |
| Accepts Healthy Volunteers: | No |
Inclusion Criteria:
Exclusion Criteria:
Clinically significant abnormalities (Grade 2 or higher on the National Cancer Institute [NCI] toxicity scale) on laboratory tests performed prior to grafting, except for the following specific exclusionary laboratory threshold results, subject to approval or exemption by the EB physician:
Clinically significant abnormalities (Grade 2 or higher on the NCI toxicity scale) identified through medical history and physical examination on Day 0, with the following exceptions:
Contacts and Locations| Contact: Emily S Gorell, MS | (650) 721-7166 | egorell@stanford.edu |
| United States, California | |
| Stanford University School of Medicine | Recruiting |
| Stanford, California, United States, 94305 | |
| Contact: Emily S Gorell, MS 650-721-7166 egorell@stanford.edu | |
| Principal Investigator: M. Peter Marinkovich M.D. | |
| Principal Investigator: Paul A. Khavari | |
| Principal Investigator: Alfred T Lane | |
| Principal Investigator: Phuong Khuu, MD | |
| Sub-Investigator: Peter Lorenz, MD | |
| Sub-Investigator: Louise Furukawa, MD | |
| Principal Investigator: | Alfred T Lane | Stanford University |
More Information
| Responsible Party: | Alfred Lane, Professor of Dermatology and Pediatrics, Stanford University |
| ClinicalTrials.gov Identifier: | NCT01263379 History of Changes |
| Other Study ID Numbers: | SU-10202010-7130, Basic Biosafety #698, IND# 13708, R01AR055914, RAC Protocol # 0701-827, eProtocol 14563 |
| Study First Received: | December 15, 2010 |
| Last Updated: | January 29, 2012 |
| Health Authority: | United States: Food and Drug Administration |
|
gene transfer |
|
Epidermolysis Bullosa Epidermolysis Bullosa Dystrophica Skin Abnormalities Congenital Abnormalities Skin Diseases, Genetic |
Genetic Diseases, Inborn Skin Diseases Skin Diseases, Vesiculobullous Collagen Diseases Connective Tissue Diseases |