Trial Search Results

Gene Transfer for Recessive Dystrophic Epidermolysis Bullosa

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.

Stanford is currently not accepting patients for this trial.

Lead Sponsor:

Stanford University

Collaborator: Abeona Therapeutics, Inc

Intervention(s):

  • Biological: LZRSE-Col7A1 Engineered Autologous Epidermal Sheets

Phase:

Phase 1/Phase 2

Eligibility


Inclusion Criteria:

   1. Clinical diagnosis of recessive dystrophic epidermolysis bullosa (RDEB)

   2. 13 years old or older and willing and able to give assent/consent

   3. Confirmation of RDEB diagnosis by immunofluorescence (IF) and electron microscopy (EM)

   4. NC1[+] and mAb LH24 antibody staining negative

   5. RDEB type VII collagen mutations in subject and carrier parents confirmed

   6. At least 100 to 200 cm2 areas of open erosions on the trunk and/or extremities
   suitable for skin grafting

   7. Able to undergo adequate anesthesia to allow grafting procedures to take place.

Exclusion Criteria:

   1. Medical instability limiting ability to travel to Stanford University Medical Center

   2. 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.

   3. Antibodies to type VII collagen associated antigens

   4. Active infection in the area that will undergo grafting

   5. Evidence of systemic infection

   6. Current evidence or a history of squamous cell carcinoma in the area that will undergo
   grafting

   7. Active drug or alcohol addiction

   8. Hypersensitivity to vancomycin or amikacin

   9. Receipt of chemical or biological study product for the specific treatment of RDEB in
   the past six months

10. Positive pregnancy test or breast-feeding

11. 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.

12. 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.

Ages Eligible for Study

13 Years - N/A

Genders Eligible for Study

All

Not currently accepting new patients for this trial

Contact Information

Stanford University
School of Medicine
300 Pasteur Drive
Stanford, CA 94305
Kylie Loutit, MS
650-724-1982
Recruiting