A Long-Term Extension Study for Participants Previously Treated With EB-101 for the Treatment of RDEB

A Long-Term Extension Study for Participants Previously Treated With EB-101 for the Treatment of Recessive Dystrophic Epidermolysis Bullosa (RDEB)

A Long-Term Extension Study for Participants Previously Treated with EB-101 for the Treatment of Recessive Dystrophic Epidermolysis Bullosa (RDEB)

Study Overview

• Status: Enrolling by invitation
• Conditions: RDEB
• Intervention / Treatment: Biological: EB-101

Detailed Description

This is an open-label, long-term, follow-up study in participants from prior interventional trials involving surgical application of gene-corrected keratinocyte sheets (EB-101) for the treatment of RDEB wound sites. Up to 22 participants will be enrolled in this study, with follow-up through at least 5 years post treatment. Patient-reported outcomes and safety will be assessed throughout the study, and where applicable, compared to Baseline of the preceding interventional EB-101 trial. Upon completion, participants will transfer to an annual monitoring program for additional 10 years of phone visits.

• Study Type: Observational
• Enrollment (Estimated): 22

Contacts and Locations

Study Locations

• United States
  • California
    • Redwood City, California, United States, 94063
      • Stanford University
  • Massachusetts
    • Worcester, Massachusetts, United States, 01605
      • University of Massachusetts Medical School

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 6 years and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

Patient with previous EB-101 treatment

Description

Inclusion Criteria:

• Willing and able to give consent/assent;
• If under the age of 18, guardian(s) is/are willing and able to give consent;
• Prior study treatment with EB-101.

Exclusion Criteria:

• Inability to properly follow protocol as determined by the Principal Investigator

Study Plan

How is the study designed?

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
Long-Term Extension; This study will follow one group of participants who were enrolled in a previous EB-101 study.	Biological: EB-101; This is non-interventional and follows patients treated with EB-101; Other Names: pz-cel

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Wound Closure	Proportion of RDEB wounds with healing ≥50%, ≥75%, and 100% at all clinic visits up to Month 54 as determined by direct investigator assessment.	5 years
Pain Reduction	Pain reduction assessed by Wong-Baker FACES scale at all clinic visits up to Month 54.	5 years
Itch Severity	Longitudinal change of Itch severity scores assessed at all clinic visits up to Week 54.	5 years
Zarit Burden Interview	Longitudinal change of Zarit Burden Interview Short Form (ZBI-12) for caregiver scores related to wound care, assessed at all clinic visits up to Month 54.	5 years
Quality of Life	Quality of Life in Epidermolysis Bullosa (QOLEB) scores assessed at all clinic visits up to Month 54.	5 years
Caregiver Global impression of Pain	Caregiver Global impression of Pain (CrGI-Pain) scores assessed at all clinic visits up to Month 54.	5 years
Wound Infection and Adverse Events	The number of participants and wounds that have an infection or any related adverse event.	5 years
The number of treatment-emergent adverse events.	The number of treatment-emergent adverse events.	5 years
Serious Adverse Events	The number of participants and wounds that result in hospitalization (serious adverse event [SAE]).	5 years
Incidence of squamous cell carcinoma.	Incidence of squamous cell carcinoma.	5 years
Replication-competent retrovirus (RCR) status	Replication-competent retrovirus (RCR) status	5 years

Collaborators and Investigators

• Sponsor: Abeona Therapeutics, Inc
• Investigators: Study Director: Angela Iheanacho, MS, Abeona Therapeutics, Inc; Study Director: Sarah Abdelwahab, MD, Abeona Therapeutics, Inc

Study record dates

Study Major Dates

• Study Start (Actual): February 9, 2021
• Primary Completion (Estimated): February 1, 2036
• Study Completion (Estimated): August 1, 2036

Study Registration Dates

• First Submitted: January 23, 2023
• First Submitted That Met QC Criteria: January 23, 2023
• First Posted (Actual): February 1, 2023

Study Record Updates

• Last Update Posted (Actual): June 27, 2024
• Last Update Submitted That Met QC Criteria: June 26, 2024
• Last Verified: June 1, 2024

More Information

Terms related to this study

• Other Study ID Numbers: EB-101-LT-001

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: UNDECIDED

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No