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

Abeona Therapeutics

Status: Invitation-only

Conditions: RDEB

Treatments: Biological: EB-101

Study type: Observational

Funder types: Industry

Identifiers

NCT05708677

EB-101-LT-001

Details and patient eligibility

About

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

Full 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.

Enrollment: 22 estimated patients

Sex: All

Ages: 6+ years old

Volunteers: No Healthy Volunteers

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

Trial design

22 participants in 1 patient group

Long-Term Extension

Description:

This study will follow one group of participants who were enrolled in a previous EB-101 study.

Treatment:

Biological: EB-101