Allogeneic ABCB5-positive Stem Cells for Treatment of Epidermolysis Bullosa (NCT03529877) | Clinical Trial Compass
CompletedPhase 1/2
Allogeneic ABCB5-positive Stem Cells for Treatment of Epidermolysis Bullosa
United States, Austria, France16 participantsStarted 2019-02-16
Plain-language summary
The aim of this clinical trial is to investigate the efficacy (by monitoring overall improvement of EB symptoms) and safety (by monitoring adverse events) of three doses of allo-APZ2-EB administered intravenously to patients with recessive dystrophic epidermolysis bullosa (RDEB).
Who can participate
Age range
0 Years – 55 Years
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
. at least 3 adult patients (safety assessment 2 weeks after last treatment of third patient),
. at least 3 patients ≥12 to \<18 years (safety assessment 2 weeks after first treatment of third patient),
. at least 3 patients ≥5 to \<12 years (safety assessment 2 weeks after first treatment of third patient), and
. at least 3 patients ≥12 months to \<5 years;
. patients 0 to \<12 months (only in the UK);
Exclusion criteria
. Tumor diseases or history of tumor disease;
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
Overall improvement of EB symptoms after 12 weeks (measured by percentage change of a patient's EBDASI score), score), or last available post-baseline measurement if the Week 12 measurement is missing
Timeframe: Week 12 post baseline, or last available post-baseline measurement if the Week 12 measurement is missing (last observation carried forward [LOCF])
. Known positive result for human immunodeficiency virus 1 and/or 2;
. Any known allergies to components of the IMP;
. Evidence of any other medical conditions (such as psychiatric illness or active infection) based on physical examination, or laboratory findings that may interfere with the planned treatment, affect the patient's compliance, or place the patient at high risk of complications related to the treatment; at investigators discretion;
. History of prior thrombosis or patients at risk for thrombosis;
. Clinically significant or unstable concurrent disease or other clinical contraindications (based upon investigator's judgment);
. Patient/legal representative anticipated to be unwilling or unable to comply with the requirements of the protocol;