Status:
COMPLETED
Gene Transfer for Recessive Dystrophic Epidermolysis Bullosa
Lead Sponsor:
Abeona Therapeutics, Inc
Collaborating Sponsors:
National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)
Stanford University
Conditions:
Epidermolysis Bullosa Dystrophica
Epidermolysis Bullosa
Eligibility:
All Genders
13+ years
Phase:
PHASE1
PHASE2
Brief Summary
This trial will create a skin graft, which the investigators call "LEAES," using the patient's own skin cells that have been genetically engineered in the lab to express a missing protein called type ...
Detailed Description
The research project involves gene transfer into keratinocytes, which are the majority of the cells in the outer layer of skin. In this gene transfer trial we plan to biopsy some skin tissue, grow the...
Eligibility Criteria
Inclusion
- Clinical diagnosis of recessive dystrophic epidermolysis bullosa (RDEB)
- 13 years old or older and willing and able to give assent/consent
- Confirmation of RDEB diagnosis by immunofluorescence (IF) and electron microscopy (EM)
- NC1\[+\] and mAb LH24 antibody staining negative
- RDEB type VII collagen mutations in subject and carrier parents confirmed
- At least 100 to 200 cm2 areas of open erosions on the trunk and/or extremities suitable for skin grafting
- Able to undergo adequate anesthesia to allow grafting procedures to take place.
Exclusion
- Medical instability limiting ability to travel to Stanford University Medical Center
- The presence of medical illness expected to complicate participation and/or compromise the safety of this technique, such as active infection with HIV, hepatitis B or hepatitis C, as determined by hepatitis B surface antigen screening, detection of hepatitis C antibodies, or positive result of hepatitis C polymerase chain reaction (PCR) analysis.
- Antibodies to type VII collagen associated antigens
- Active infection in the area that will undergo grafting
- Evidence of systemic infection
- Current evidence or a history of squamous cell carcinoma in the area that will undergo grafting
- Active drug or alcohol addiction
- Hypersensitivity to vancomycin or amikacin
- Receipt of chemical or biological study product for the specific treatment of RDEB in the past six months
- Positive pregnancy test or breast-feeding
- 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:
- Albumin \< 2.5 g/dL
- Leukocytes \> 20K/uL
- Hemoglobin \< 7.5 g/dL. Low hemoglobin will be treated at the discretion of the investigators and the EB physician.
- Additional exceptions may be made at the discretion of the investigators and 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:
- Anorexia, can enroll up to Grade 4 (inclusive)
- Constipation, can enroll up to Grade 2 (inclusive)
- Dysphagia, can enroll up to Grade 4 (inclusive)
- Keratitis, can enroll up to Grade 4 (inclusive)
- Bone pain, can enroll up to Grade 2 (inclusive)
- Additional exceptions may be made at the discretion of the investigators and the EB physician.
Key Trial Info
Start Date :
October 5 2010
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
March 9 2022
Estimated Enrollment :
12 Patients enrolled
Trial Details
Trial ID
NCT01263379
Start Date
October 5 2010
End Date
March 9 2022
Last Update
August 22 2023
Active Locations (1)
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1
Stanford University, School of Medicine, Dept of Dermatology
Redwood City, California, United States, 94063