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 range
12 Months
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
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.
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
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)
. 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.
. Current evidence or a history of SCC in the area that will undergo EB-101 application.
. Active drug or alcohol addiction.
. Hypersensitivity to vancomycin or amikacin.
. Receipt of chemical or biological investigational therapy for the specific treatment of DEB in the 3 months prior to EB-101 application.