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

N/AEnrolling By InvitationNCT05708677

Abeona Therapeutics, Inc22 enrolled

Overview

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

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

Conditions

RDEB

Interventions

EB-101BIOLOGICAL

This is non-interventional and follows patients treated with EB-101

Eligibility

Sex: ALLMin age: 6 Years

Medical Language ↔ Plain English

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

Locations (2)

Stanford University

Redwood City, California, United States

University of Massachusetts Medical School

Worcester, Massachusetts, United States

Outcomes

Primary Outcomes

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.

Time frame: 5 years

Pain Severity Scale (Wong-Baker FACES)

Change in pain assessed by Wong-Baker FACES scale at all clinic visits up to Month 54. The minimum score value is 0 and the maximum score value is 10, with the higher scores being the worse outcome.

Time frame: 5 years

Itch Severity Scale

Longitudinal change of Itch severity scores assessed at all clinic visits up to Week 54. The minimum score value is 0 and the maximum score value is 10, with the higher scores being the worse outcome.

Time frame: 5 years

Zarit Burden Interview Scale

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. The minimum score value is 0 and the maximum score value is 4, with the higher scores being the worse outcome.

Time frame: 5 years

Quality of Life in Epidermolysis Bullosa

Quality of Life in Epidermolysis Bullosa (QOLEB) responses will be assessed at all clinic visits up to Month 54.

Time frame: 5 years

Caregiver Global Impression of Change for Pain Scale

Caregiver Global Impression of Change for Pain (CrGI-Pain) scores assessed at all clinic visits up to Month 54. The minimum score value is 1 and the maximum score value is 7, with the higher scores being the worse outcome.

Time frame: 5 years

Wound Infection and Adverse Events

The number of participants and wounds that have an infection or any related adverse event.

Data from ClinicalTrials.gov

This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.