Gene Transfer for Recessive Dystrophic Epidermolysis Bullosa (NCT01263379) | Clinical Trial Compass
CompletedPhase 1/2
Gene Transfer for Recessive Dystrophic Epidermolysis Bullosa
United States12 participantsStarted 2010-10-05
Plain-language 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 VII collagen. The corrected cells will be transplanted back to the patient.
Who can participate
Age range13 Years
SexALL
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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
✓. 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 criteria
✕. 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
What they're measuring
1
Number of Wounds by Healing Category Per Investigator Visual Assessment