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

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

Sponsors

Lead Sponsor: Abeona Therapeutics, Inc

Source Abeona Therapeutics, Inc
Brief Summary

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).

Detailed Description

Recessive dystrophic epidermolysis bullosa (RDEB) is an ultra-rare, severe inherited blistering skin disease caused by the absence of a protein known as type 7 collagen (C7). There is no approved treatment for RDEB. Only supportive care is currently possible.

This open-label, controlled study will evaluate the efficacy and safety of EB-101 for the treatment of large, chronic, RDEB wounds. The study intervention consists of One-time surgical application of approximately 30 autologous, gene-corrected keratinocyte sheets (EB-101) for the treatment of RDEB wound sites in up to approximately 15 participants. A single EB-101 sheet is able to provide healing to a wound area up to approximately 40cm2. Up to 6 (six) EB-101 sheets may be applied to each patient, depending on the area of existing wounds. Treatment effect will be measured as wound healing at 3 months, with follow up to 6 months post treatment, comparing treated versus matched untreated wound sites. Wound healing will be adjudicated by an independent committee not involved in the study conduct. Patient-reported outcomes and safety will also be collected throughout the study.

The primary analysis for efficacy will be assessed when all patients reach Week 12. Safety and efficacy assessments will be conducted at regular intervals and completed when last patient reaches Week 24 post-treatment.

Upon completion of the 6-month study period, patients will be monitored annually as per standard of care for up to 15 years.

Overall Status Recruiting
Start Date January 10, 2020
Completion Date March 2021
Primary Completion Date September 2020
Phase Phase 3
Study Type Interventional
Primary Outcome
Measure Time Frame
Wound Healing 12 weeks post-treatment
Enrollment 15
Condition
Intervention

Intervention Type: Biological

Intervention Name: EB-101

Description: autologous RDEB keratinocytes isolated from skin biopsies and transduced with a recombinant retrovirus containing a full-length COL7A1 expression cassette for C7

Arm Group Label: EB-101

Other Name: LZRSE-Col7A1 Engineered Autologous Epidermal Sheets [LEAES]

Eligibility

Criteria:

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.

Gender: All

Minimum Age: 6 Years

Maximum Age: N/A

Healthy Volunteers: No

Overall Official
Last Name Role Affiliation
Jean Tang, MD Principal Investigator Stanford University
Overall Contact

Last Name: Abeona Clinical Operations

Phone: +1-646-813-4701

Email: [email protected]

Location
Facility: Status: Contact: Investigator: Stanford University Irene Bailey 650-721-7149 [email protected] Jean Tang, MD Principal Investigator Peter Marinkovich, MD Sub-Investigator
Location Countries

United States

Verification Date

December 2019

Responsible Party

Type: Sponsor

Keywords
Has Expanded Access No
Condition Browse
Number Of Arms 1
Arm Group

Label: EB-101

Type: Experimental

Description: One-time surgical application of EB-101 on up to 6 chronic, RDEB wounds

Patient Data No
Study Design Info

Allocation: N/A

Intervention Model: Single Group Assignment

Primary Purpose: Treatment

Masking: None (Open Label)

Source: ClinicalTrials.gov