A Phase 3b Study for the Treatment of Dystrophic Epidermolysis Bullosa (DEB) in New and Previousl… (NCT05725018) | Clinical Trial Compass
Active — Not RecruitingPhase 3
A Phase 3b Study for the Treatment of Dystrophic Epidermolysis Bullosa (DEB) in New and Previously EB-101 Treated Patients
United States12 participantsStarted 2023-04-02
Plain-language summary
To evaluate and further characterize the safety of EB-101 (LZRSE-Col7A1 gene-corrected keratinocyte sheets with type VII collagen \[C7\] expression) for the treatment of large, chronic DEB wounds in new and previously EB-101 treated patients 12 months and older.
Who can participate
Age range12 Months
SexALL
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Inclusion criteria
✓. Clinical diagnosis of DEB.
✓. Age 12 months and older.
✓. Willing and able to give consent/assent; if under the age of 18 years, guardian(s) is/are willing and able to give consent.
✓. (This inclusion criterion was deleted as of Amendment 1.)
✓. Confirmation of DEB diagnosis (either dominant \[DDEB\] or recessive \[RDEB\] forms) by genetic testing.
✓. Able to undergo adequate anesthesia during EB-101 treatment.
✓. All women of childbearing potential must have a negative urine pregnancy test and use a reliable birth control method throughout the duration of the study.
✓. On stable pain medication regimen for at least 30 days prior to Screening (and through Baseline).
Exclusion criteria
✕. Medical instability limiting ability to travel to the study site or undergo EB-101 treatment.
✕. The presence of medical illness expected to complicate participation and/or compromise the safety of this technique, such as active infection with human immunodeficiency virus (HIV), hepatitis B, or hepatitis C.
✕. (This exclusion criterion was deleted as of Amendment 3.)
✕. Evidence of systemic infection.
What they're measuring
1
Safety Endpoint (number of treatment-related and treatment-emergent adverse events (AEs) and serious adverse events (SAEs)
Timeframe: 6 months
2
Safety Endpoint (number of patients and wounds that have an infection or any related adverse event.)
Timeframe: 6 months
3
Safety Endpoint (number of patients and wounds that result in hospitalization (serious adverse event )
Timeframe: 6 months
4
Safety Endpoint (Incidence of squamous cell carcinoma)