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

NCT05708677 | Enrolling By Invitation FDA Drug

• 1 other identifier: EB-101-LT-001
• Study Type: observational
• Target: 22
• Locations: 1 country
• Sites: 2

Brief Summary

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

Conditions

RDEB

Outcome Measures

Primary Outcomes (11)

• 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

Study Arms (1)

Long-Term Extension

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

Biological: EB-101

Interventions

EB-101 BIOLOGICAL

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

Also known as: pz-cel

Long-Term Extension

Eligibility Criteria

• Age: 6 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Patient with previous EB-101 treatment

You may qualify if:

• 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.

You may not qualify if:

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

Sponsors & Collaborators

• Abeona Therapeutics, Inc: lead

Study Sites (2)

Stanford University

Redwood City, California, 94063, United States

Location

University of Massachusetts Medical School

Worcester, Massachusetts, 01605, United States

Location

Study Officials

• Angela Iheanacho, MS

  Abeona Therapeutics, Inc

  STUDY DIRECTOR

• Sarah Abdelwahab, MD

  Abeona Therapeutics, Inc

  STUDY DIRECTOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 23, 2023
• First Posted: February 1, 2023
• Study Start: February 9, 2021
• Primary Completion (Estimated): February 1, 2036
• Study Completion (Estimated): August 1, 2036
• Last Updated: June 27, 2024

Record last verified: 2024-06

Locations

US (2)