Education and Training
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.
Stanford Investigator(s):
Intervention(s):
- biological: EB-101
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
I'm interested