RDEB
Conditions
Brief summary
A Long-Term Extension Study for Participants Previously Treated with EB-101 for the Treatment of Recessive Dystrophic Epidermolysis Bullosa (RDEB)
Detailed 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.
Interventions
BIOLOGICALEB-101
This is non-interventional and follows patients treated with EB-101
Sponsors
Abeona Therapeutics, Inc
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
6 Years to No maximum
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Wound Closure | 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. |
| Pain Severity Scale (Wong-Baker FACES) | 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. |
| Itch Severity Scale | 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. |
| Zarit Burden Interview Scale | 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. |
| Quality of Life in Epidermolysis Bullosa | 5 years | Quality of Life in Epidermolysis Bullosa (QOLEB) responses will be assessed at all clinic visits up to Month 54. |
| Caregiver Global Impression of Change for Pain Scale | 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. |
| Wound Infection and Adverse Events | 5 years | The number of participants and wounds that have an infection or any related adverse event. |
| The number of treatment-emergent adverse events. | 5 years | The number of treatment-emergent adverse events. |
| Serious Adverse Events | 5 years | The number of participants and wounds that result in hospitalization (serious adverse event \[SAE\]). |
| Incidence of squamous cell carcinoma. | 5 years | Incidence of squamous cell carcinoma. |
| Replication-competent retrovirus (RCR) status | 5 years | Replication-competent retrovirus (RCR) status |
Countries
United States
Contacts
STUDY_DIRECTORAngela Iheanacho, MS
Abeona Therapeutics, Inc
STUDY_DIRECTORSarah Abdelwahab, MD
Abeona Therapeutics, Inc
Outcome results
None listed