- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT00533572
Recessive Dystrophic Epidermolysis Bullosa Screening for Possible Gene Transfer
May 22, 2015 updated by: Alfred Lane, Stanford University
Recessive Dystrophic Epidermolysis Bullosa (RDEB) is a severe inherited blistering skin disease caused by absence of type VII collagen.
Patients with RDEB develop large, severly painful blisters and open wounds from minor trauma to their skin.
In the future, we hope to start a gene transfer study on a specific group of RDEB subjects and we are screening subjects for that potential trial now.
Study Overview
Status
Terminated
Conditions
Detailed Description
This study is a preparation for a gene transfer trial.
In the gene transfer trial, we will transfect autologous RDEB keratinocytes with a type VII collagen containing retrovirus vector.
We then plan to graft the genetically engineered and corrected autologous keratinocytes back on to wounds of the RDEB subject.
We expect the grafts to attach as normal keratinocytes and replace the damaged skin with skin that is able to make type VII collagen and anchor normally to the dermis.
We are trying to define the number of subjects 18 or older who meet the criteria for the gene transfer trial.
We are offering the subjects the opportunity to be evaluated for the potential trial.
Study Type
Observational
Enrollment (Actual)
1
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Locations
-
-
California
-
Stanford, California, United States, 94305
- Stanford University School of Medicine
-
-
Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
18 years and older (ADULT, OLDER_ADULT)
Accepts Healthy Volunteers
No
Genders Eligible for Study
All
Sampling Method
Non-Probability Sample
Study Population
Adults with RDEB
Description
Inclusion Criteria:1.
Clinical diagnosis of RDEB by local dermatologist 2. 18 years of age or more and willing to give consent 3.
Estimated to have at least 100 to 200 sq.
cm.
areas of open erosions on the trunk or extremities suitable for skin grafting 4. Ability to undergo adequate anesthesia to allow grafting procedures to take place 5. Parents are alive, do not have EB, and they are willing to give consent for genetic testing Exclusion Criteria:1.
Medical instability limiting ability to travel to Stanford University Medical Center 2. Participation in another clinical trial without prior approval 3. The presence of medical illness expected to complicate participation and/or compromise the safety of this technique
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Disease documentation
Time Frame: 1 year
|
Documentation of disease
|
1 year
|
documentation of disease
Time Frame: 1 year
|
documentation of disease
|
1 year
|
Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Sponsor
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start
August 1, 2007
Primary Completion (ACTUAL)
August 1, 2008
Study Completion (ACTUAL)
August 1, 2008
Study Registration Dates
First Submitted
September 20, 2007
First Submitted That Met QC Criteria
September 20, 2007
First Posted (ESTIMATE)
September 21, 2007
Study Record Updates
Last Update Posted (ESTIMATE)
May 27, 2015
Last Update Submitted That Met QC Criteria
May 22, 2015
Last Verified
May 1, 2015
More Information
Terms related to this study
Additional Relevant MeSH Terms
Other Study ID Numbers
- 97822
- 8557
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
Clinical Trials on Epidermolysis Bullosa Dystrophica
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Phoenicis TherapeuticsTerminatedEpidermolysis Bullosa Dystrophica, Recessive | Epidermolysis Bullosa Dystrophica, DominantUnited States, Spain, France
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Castle Creek Biosciences, LLC.TerminatedEpidermolysis Bullosa Dystrophica, RecessiveUnited States
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Instituto de Investigación Hospital Universitario...Instituto de Salud Carlos III; Universidad Carlos III Madrid (TERMeG); St John... and other collaboratorsUnknownEpidermolysis Bullosa Dystrophica, RecessiveSpain
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Castle Creek Pharmaceuticals, LLCCompletedDystrophic Epidermolysis Bullosa | Epidermolysis Bullosa Simplex | Junctional Epidermolysis Bullosa | Epidermolysis Bullosa (EB)United States
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Krystal Biotech, Inc.CompletedDystrophic Epidermolysis Bullosa | Recessive Dystrophic Epidermolysis Bullosa | Dominant Dystrophic Epidermolysis BullosaUnited States
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Lenus Therapeutics, LLCTerminatedDystrophic Epidermolysis Bullosa | Junctional Epidermolysis BullosaUnited States
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Krystal Biotech, Inc.CompletedDystrophic Epidermolysis Bullosa | Recessive Dystrophic Epidermolysis Bullosa | Dominant Dystrophic Epidermolysis Bullosa | DEB - Dystrophic Epidermolysis BullosaUnited States
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Thomas Jefferson UniversityOnconova Therapeutics, Inc.RecruitingRecessive Dystrophic Epidermolysis BullosaUnited States