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A Long-Term Extension Study for Participants Previously Treated with EB-101 for the Treatment of Recessive Dystrophic Epidermolysis Bullosa (RDEB)
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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Long-Term Extension | This study will follow one group of participants who were enrolled in a previous EB-101 study. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| EB-101 | Biological | This is non-interventional and follows patients treated with EB-101 |
|
| Measure | Description | Time Frame |
|---|---|---|
| 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. |
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Inclusion Criteria:
Exclusion Criteria:
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Patient with previous EB-101 treatment
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| Name | Affiliation | Role |
|---|---|---|
| Angela Iheanacho, MS | Abeona Therapeutics, Inc | Study Director |
| Sarah Abdelwahab, MD | Abeona Therapeutics, Inc | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Stanford University | Redwood City | California | 94063 | United States | ||
| University of Massachusetts Medical School |
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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 |
| Worcester |
| Massachusetts |
| 01605 |
| United States |