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NCT Number: NCT05708677

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)

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Key information

Conditions

RDEB

Age range

6 year and older

Sex eligibility

All sexes

Study type

Observational

Primary location

Stanford University, Redwood City, California, United States

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About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

EB-101

Biological

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

Other names: pz-cel

Primary outcomes

  1. Wound Closure

    Time frame: 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.

  2. Pain Severity Scale (Wong-Baker FACES)

    Time frame: 5 years

    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.

  3. Itch Severity Scale

    Time frame: 5 years

    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.

  4. Zarit Burden Interview Scale

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

  5. Quality of Life in Epidermolysis Bullosa

    Time frame: 5 years

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

  6. Caregiver Global Impression of Change for Pain Scale

    Time frame: 5 years

    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.

  7. Wound Infection and Adverse Events

    Time frame: 5 years

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

  8. The number of treatment-emergent adverse events.

    Time frame: 5 years

    The number of treatment-emergent adverse events.

  9. Serious Adverse Events

    Time frame: 5 years

    The number of participants and wounds that result in hospitalization (serious adverse event [SAE]).

  10. Incidence of squamous cell carcinoma.

    Time frame: 5 years

    Incidence of squamous cell carcinoma.

  11. Replication-competent retrovirus (RCR) status

    Time frame: 5 years

    Replication-competent retrovirus (RCR) status

Sponsors and collaborators

Lead sponsor

Abeona Therapeutics, Inc

Industry

Registry information

Official study title

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

Important dates

Study start
2021
Primary completion
2036
Study completion
2036
First posted
Feb 1, 2023
Registry last updated
Jul 17, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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