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

Trial ID or NCT#

NCT04227106

Status

recruiting iconRECRUITING

Purpose

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).

Official Title

VIITAL: A Phase 3 Study of EB-101 for the Treatment of Recessive Dystrophic Epidermolysis Bullosa (RDEB)

Eligibility Criteria

Ages Eligible for Study: Older than 6 Years
Sexes Eligible for Study: All
Accepts Healthy Volunteers: No
Inclusion Criteria:
  1. - 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:
  1. - 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.

Investigator(s)

M. Peter Marinkovich, MD
M. Peter Marinkovich, MD
Dermatologist, Blistering disease specialist, Psoriasis specialist
Associate Professor of Dermatology
Jean Y. Tang MD PhD
Jean Y. Tang MD PhD
General dermatologist, Dermatologic oncologist, Dermatologist, Cutaneous oncology specialist
Professor of Dermatology

Contact us to find out if this trial is right for you.

Contact

Irene Bailey
650-721-7149