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

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 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.

  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.

  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.

  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.

  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.

  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