ACTIVE_NOT_RECRUITING

A Phase 3b Study for the Treatment of Dystrophic Epidermolysis Bullosa (DEB) in New and Previously EB-101 Treated Patients

Conditions

Epidermolysis Bullosa Recessive Dystrophic Epidermolysis Bullosa RDEB Chronic wounds

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

To evaluate and further characterize the safety of EB-101 (LZRSE-Col7A1 gene-corrected keratinocyte sheets with type VII collagen \[C7\] expression) for the treatment of large, chronic DEB wounds in new and previously EB-101 treated patients 12 months and older.

Official Title

A Phase 3b Study for the Treatment of Dystrophic Epidermolysis Bullosa (DEB) in New and Previously EB-101 Treated Patients

Quick Facts

Study Start:2023-04-02

Study Completion:2025-09-30

Study Type:Not specified

Phase:Not Applicable

Enrollment:Not specified

Status:ACTIVE_NOT_RECRUITING

Study ID

NCT05725018

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:12 Months

Sexes Eligible for Study:ALL

Accepts Healthy Volunteers:No

Standard Ages:CHILD, ADULT, OLDER_ADULT

Inclusion Criteria	Exclusion Criteria
1. Clinical diagnosis of DEB. 2. Age 12 months and older. 3. Willing and able to give consent/assent; if under the age of 18 years, guardian(s) is/are willing and able to give consent. 4. (This inclusion criterion was deleted as of Amendment 1.) 5. Confirmation of DEB diagnosis (either dominant \[DDEB\] or recessive \[RDEB\] forms) by genetic testing. 6. Able to undergo adequate anesthesia during EB-101 treatment. 7. All women of childbearing potential must have a negative urine pregnancy test and use a reliable birth control method throughout the duration of the study. 8. On stable pain medication regimen for at least 30 days prior to Screening (and through Baseline). 9. Must have at least one wound site that meets all of the following criteria: 1. An area ≥20 cm2, 2. Present for ≥6 months, and 3. Stage 2 wound defined as an open skin wound with partial thickness loss of dermis that has not extended through the dermis into subcutaneous tissue. 10. For patients with RDEB, they must have had Vyjuvek exposure by the time of screening or positive anti-C7 antibodies at baseline. 11. Patients must be willing to discontinue Vyjuvek and Filsuvez use on EB-101 treated sites until completion of the Week 24 visit.	1. Medical instability limiting ability to travel to the study site or undergo EB-101 treatment. 2. 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. 3. (This exclusion criterion was deleted as of Amendment 3.) 4. Evidence of systemic infection. 5. Current evidence or a history of SCC in the area that will undergo EB-101 application. 6. Active drug or alcohol addiction. 7. Hypersensitivity to vancomycin or amikacin. 8. Receipt of chemical or biological investigational therapy for the specific treatment of DEB in the 3 months prior to EB-101 application. 9. Breast-feeding. 10. Inability to properly follow protocol assessments and protect keratinocyte sheet sites as determined by the PI. 11. Grade 3 clinical event or laboratory abnormality at Day 0. Abnormalities such as esophageal strictures, anemia, low albumin, and pain/itch are expected in severe DEB patients, and these abnormalities will not exclude a patient. 12. Unwillingness or inability to provide 4 skin biopsies, or patient's keratinocytes cannot be manufactured for use in EB-101 application. 13. Any other circumstance where the PI believes that the patient may not be appropriate for participation in the study.

Inclusion Criteria

Exclusion Criteria

1. Clinical diagnosis of DEB.
2. Age 12 months and older.
3. Willing and able to give consent/assent; if under the age of 18 years, guardian(s) is/are willing and able to give consent.
4. (This inclusion criterion was deleted as of Amendment 1.)
5. Confirmation of DEB diagnosis (either dominant \[DDEB\] or recessive \[RDEB\] forms) by genetic testing.
6. Able to undergo adequate anesthesia during EB-101 treatment.
7. All women of childbearing potential must have a negative urine pregnancy test and use a reliable birth control method throughout the duration of the study.
8. On stable pain medication regimen for at least 30 days prior to Screening (and through Baseline).
9. Must have at least one wound site that meets all of the following criteria:
1. An area ≥20 cm2,
2. Present for ≥6 months, and
3. Stage 2 wound defined as an open skin wound with partial thickness loss of dermis that has not extended through the dermis into subcutaneous tissue.
10. For patients with RDEB, they must have had Vyjuvek exposure by the time of screening or positive anti-C7 antibodies at baseline.
11. Patients must be willing to discontinue Vyjuvek and Filsuvez use on EB-101 treated sites until completion of the Week 24 visit.

1. Medical instability limiting ability to travel to the study site or undergo EB-101 treatment.
2. 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.
3. (This exclusion criterion was deleted as of Amendment 3.)
4. Evidence of systemic infection.
5. Current evidence or a history of SCC in the area that will undergo EB-101 application.
6. Active drug or alcohol addiction.
7. Hypersensitivity to vancomycin or amikacin.
8. Receipt of chemical or biological investigational therapy for the specific treatment of DEB in the 3 months prior to EB-101 application.
9. Breast-feeding.
10. Inability to properly follow protocol assessments and protect keratinocyte sheet sites as determined by the PI.
11. Grade 3 clinical event or laboratory abnormality at Day 0. Abnormalities such as esophageal strictures, anemia, low albumin, and pain/itch are expected in severe DEB patients, and these abnormalities will not exclude a patient.
12. Unwillingness or inability to provide 4 skin biopsies, or patient's keratinocytes cannot be manufactured for use in EB-101 application.
13. Any other circumstance where the PI believes that the patient may not be appropriate for participation in the study.

Contacts and Locations

Principal Investigator

Angela Iheanacho, MS

STUDY_DIRECTOR

Abeona Therapeutics, Inc

Sarah Abdelwahab, MD

STUDY_DIRECTOR

Abeona Therapeutics, Inc

Study Locations (Sites)

Stanford University

Redwood City, California, 94063

United States

University of Massachusetts Medical School

Worcester, Massachusetts, 01605

United States

Collaborators and Investigators

Sponsor: Abeona Therapeutics, Inc

Angela Iheanacho, MS, STUDY_DIRECTOR, Abeona Therapeutics, Inc
Sarah Abdelwahab, MD, STUDY_DIRECTOR, Abeona Therapeutics, Inc

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2023-04-02

Study Completion Date2025-09-30

Study Record Updates

Study Start Date2023-04-02

Study Completion Date2025-09-30

Terms related to this study

Keywords Provided by Researchers

Chronic wounds

Additional Relevant MeSH Terms

Epidermolysis Bullosa
Recessive Dystrophic Epidermolysis Bullosa
RDEB