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

• Conditions: RDEB
• Interventions: Biological: EB-101

• Registration Number: NCT05708677
• Lead Sponsor: Abeona Therapeutics, Inc

Brief Summary

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

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 Timeline

• Study Start: 2021-02-09
• Study First Submitted: 2023-01-23
• Study First Submitted QC: 2023-01-23
• Study First Posted: 2023-02-01
• Status Verified: 2024-06
• Last Update Submitted: 2024-06-26
• Last Update Posted: 2024-06-27
• Primary Completion: 2036-02
• Study Completion: 2036-08

Recruitment & Eligibility

• Status: ENROLLING_BY_INVITATION
• Sex: All
• Target Recruitment: 22

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 & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
Long-Term Extension	EB-101	This study will follow one group of participants who were enrolled in a previous EB-101 study.

Primary Outcome Measures

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

Trial Locations

Locations (2)

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

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