Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
| NCT ID | Title | Phase | Status | Enrollment | Velocity | Design | Start | Completion | Last Updated | Sites | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT05725018 | A Phase 3b Study for the Treatment of Dystrophic Epidermolysis Bullosa (DEB) in New and Previously EB-101 Treated Patients | PHASE3 | ACTIVE NOT_RECRUITING | 12 | — | — | Apr 2, 2023 | Sep 30, 2025 | Jun 27, 2025 | 2 | United States |
The number of treatment-related and treatment-emergent adverse events (TEAEs), treatment-emergent wound adverse events (TEWAEs), and serious adverse events (SAEs), including systemic and wound-specific adverse events.
The number of patients and wounds that have an infection or any related adverse event (AE).
The number of patients and wounds that result in hospitalization (SAE).
The incidence of squamous cell carcinoma (SCC).
Replication-competent retrovirus (RCR) status.
| Arm | Type | Description |
|---|---|---|
| EB-101 Surgical application of RDEB wounds | EXPERIMENTAL | New or Previously Treated RDEB Patients |
| Name | Type | Description |
|---|---|---|
| EB-101 Surgical application of RDEB wounds | BIOLOGICAL | EB-101 autologous RDEB keratinocytes isolated from skin biopsies and transduced with a recombinant retrovirus containing a full-length COL7A1 expression cassette for C7 Other Names: • LZRSE-Col7A1 Engineered Autologous Epidermal Sheets \[LEAES\] |
Inclusion Criteria: 1. Clinical diagnosis of DEB. 2. Age 12 months and older. 3. Willing and able to give consent/assent; if under the age of 18 years, guardian(s) is/are willing and able to give consent. 4. (This inclusion criterion was deleted as of Amendment 1.) 5. Confirmation of DEB diagnosis ...