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 range0 Years – 55 Years
SexALL
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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;
✕. 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;
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])