Trial Search Results

Phase 3, Open-label Clinical Trial of EB-101 for the Treatment of Recessive Dystrophic Epidermolysis Bullosa (RDEB)

The purpose of this trial is to evaluate safety and efficacy of surgical application of EB-101 (autologous, gene-corrected keratinocyte sheets) as a treatment of recessive dystrophic epidermolysis bullosa (RDEB).

Stanford is currently accepting patients for this trial.

Lead Sponsor:

Abeona Therapeutics, Inc

Stanford Investigator(s):

Intervention(s):

  • Biological: EB-101

Phase:

Phase 3

Eligibility


Inclusion Criteria:

   - Clinical diagnosis of RDEB;

   - Age 6 years or older, willing and able to give consent/assent;

   - If under the age of 18, guardian(s) is/are willing and able to give consent;

   - Positive expression of the non-collagenous region 1 of the type 7 collagen protein
   (NC1+) in the skin;

   - Two confirmed RDEB C7 mutations with recessive inheritance patterns (or confirmation
   that parents don't have any evidence of dominant disease);

   - At least 40 cm2 areas of chronically wounded area on the trunk and/or extremities
   suitable for EB-101 application (open erosions);

   - Able to undergo adequate anesthesia during EB-101 application;

   - Must have at least two matched, eligible wound sites (one pair);

      - Wound sites must:

         - Have an area ≥20 cm2,

         - Present for ≥6 months, and

         - Stage 2 wound;

   - Women of childbearing potential must use a reliable birth control method throughout
   the duration of the study and for 6 months post treatment;

   - Negative pregnancy test;

   - Must be on stable pain medication regimen at least 30 days prior to Screening

Exclusion Criteria:

   - Medical instability limiting ability to travel to the study site;

   - The presence of medical illness expected to complicate participation and/or compromise
   the safety of this technique, such as active infection with human immunodeficiency
   virus (HIV), hepatitis B or hepatitis C;

   - Evidence of immune response to C7 by indirect immunofluorescence (IIF);

   - Evidence of systemic infection;

   - Current evidence or a history of squamous cell carcinoma (SCC) in the area that will
   undergo EB-101 application;

   - 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 3 months;

   - Positive pregnancy test or breast-feeding;

   - Clinically significant medical or laboratory abnormalities as determined by the
   Principal Investigator;

   - Inability to properly follow protocol and protect keratinocyte sheet sites, as
   determined by the Principal Investigator;

   - Grade 3 clinical event or laboratory abnormality at Day 0. Abnormalities such as
   esophageal strictures, anemia, low albumin, and pain/itch are expected in RDEB
   patients. These abnormalities will not exclude a participant; and

   - Inability to culture participant's keratinocytes.

Ages Eligible for Study

6 Years - N/A

Genders Eligible for Study

All

Now accepting new patients

Contact Information

Stanford University
School of Medicine
300 Pasteur Drive
Stanford, CA 94305
Irene Bailey
650-721-7149
Recruiting