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

Phase 3

Completed

• Conditions: Recessive Dystrophic Epidermolysis Bullosa; Epidermolysis Bullosa
• Interventions: Biological: EB-101

• Registration Number: NCT04227106
• Lead Sponsor: 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.
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Basic Information
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Recruitment & Eligibility
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Study & Design
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Trial Locations
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Study Timeline

• Study Start: 2020-01-10
• Study First Submitted: 2020-01-10
• Study First Submitted QC: 2020-01-10
• Study First Posted: 2020-01-13
• Status Verified: 2022-04
• Primary Completion: 2022-10-18
• Study Completion: 2022-10-18
• Last Update Submitted: 2022-12-02
• Last Update Posted: 2022-12-05

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 11

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

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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.

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

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

Primary Outcome Measures

Name	Time	Method
Pain Reduction	24 weeks post-treatment	Associated with wound dressing change assessed by the mean differences in scores of the Wong-Baker FACES scale between treated and untreated wounds
Wound Healing	24 weeks post-treatment	Proportion of RDEB wound sites with ≥50% healing from Baseline in treated versus untreated wounds

Trial Locations

Locations (2)

University of Massachusetts Medical School; 🇺🇸 Worcester, Massachusetts, United States

Stanford University; 🇺🇸 Redwood City, California, United States