CLINICAL TRIAL
This is a single institution trial to determine the effectiveness of stem cells from closely matched related and unrelated donors to replace missing or defective proteins responsible for severe forms of epidermolysis bullosa.
Eligibility Criteria
1. Patients aged < 25 years of age
2. Diagnosis of a severe form of EB characterized by collagen, laminin, integrin or plakin deficiency
3. Adequate organ function
4. HLA matched related or unrelated marrow donor or partially HLA matched unrelated umbilical cord blood donor
Ineligibility Criteria
1. Active infection
2. History of HIV infection
3. Evidence of squamous cell carcinoma
If you or your child may meet the above criteria, you may be eligible to come to the University of Minnesota for the proposed treatment. Prior to evaluation and screening to confirm eligibility, the following information is requested:
1. Clinical summary from a treating physician, including history of prior surgeries, transfusions, skin grafts, medications, allergies, infections
2. DNA mutation report if available
3. Skin biopsy report (including immunofluorescent staining)
4. HLA typing on child or adult with severe EB and unaffected siblings (instructions will be provided by our team)
SCREENING VISIT
The screening visit will involve skin biopsies, blood tests, photographs, assessment of skin fragility, and genetic testing. In addition, the visit will entail review of the clinical history and detailed to review of what is known and unknown regarding the proposed treatment procedure using stem cell populations from an allogeneic donor.
Based on this information, you or your child may need additional evaluations (e.g., dermatology, surgery, gastroenterology).
If you would like more information or have questions regarding the study, please contact John E. Wagner, M.D. at 612-626-2961.
Dr. Wagner’s email address is
Wagne002@umn.edu
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