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).
This is an interventional, single arm, non-randomized, open label, phase I/IIa clinical trial to investigate the efficacy and safety of the IMP allo-APZ2-EB in patients with RDEB. Patients will undergo treatment with the IMP (three repeated intravenous applications) and will be followed up for efficacy for 12 weeks. To assess long-term safety of allo-APZ2-EB one follow-up visit at Month 12 and one follow-up visit at Month 24 post IMP applications is included. Determination of the EB linked symptoms and quality of life will be assessed by using the EBDASI score, the iscorEB, the change in pain and itch perception, and patient's quality of life in EB. The wound healing process will be documented by photography.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
16
intravenous infusion of allo-APZ2-EB
University of Minnesota, Masonic Cancer Center and Medical Center
Minneapolis, Minnesota, United States
EB-Haus Austria; Salzburger Landeskliniken (SALK); Paracelsus Medizinische Privatuniversität Salzburg (PMU)
Salzburg, Austria
Hôpital Saint-Louis; Département de dermatologie
Paris, France
Department of Dermatology, Medical Center-University of Freiburg
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
EBDASI: epidermolysis bullosa disease activity and scarring index; measured in percentage change to baseline score
Time frame: Week 12 post baseline, or last available post-baseline measurement if the Week 12 measurement is missing (last observation carried forward [LOCF])
Assessment of adverse event (AE) occurrence
All AEs occurring during the clinical trial will be registered, documented and evaluated.
Time frame: Up to 24 months
Overall improvement of EB symptoms after 12 weeks (measured by percentage change of a patient's EBDASI score)
EBDASI: epidermolysis bullosa disease activity and scarring index; measured in percentage change to baseline score
Time frame: between baseline and week 12 post baseline (without LOCF)
Overall improvement of EB symptoms after 12 weeks (measured by percentage change of patient's iscorEB), or last available post-baseline measurement if the Week 12 measurement is missing
iscorEB: instrument for scoring clinical outcome of research for epidermolysis bullosa; measured in percentage change to baseline score
Time frame: Week 12 post baseline, or last available post-baseline measurement if the Week 12 measurement is missing (LOCF);
Overall improvement of EB symptoms after 12 weeks (measured by percentage change of patient's iscorEB)
iscorEB: instrument for scoring clinical outcome of research for epidermolysis bullosa; measured in percentage change to baseline score
Time frame: between baseline and week 12 post baseline (without LOCF)
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Freiburg im Breisgau, Germany
King's College London; St John's Institute of Dermatology;
London, United Kingdom
Great Ormond Street Hospital; Dermatology Department
London, United Kingdom
Overall improvement of EB symptoms at Day 17 (measured by percentage change of a patient's EBDASI score)
EBDASI: epidermolysis bullosa disease activity and scarring index; measured in percentage change to baseline score
Time frame: between baseline and day 17 post baseline
Overall improvement of EB symptoms at Day 17 (measured by percentage change of a patient's iscorEB)
iscorEB: instrument for scoring clinical outcome of research for epidermolysis bullosa; measured in percentage change to baseline score
Time frame: between baseline and day 17 post baseline
Overall improvement of EB symptoms at Day 35 (measured by percentage change of a patient's EBDASI score)
EBDASI: epidermolysis bullosa disease activity and scarring index; measured in percentage change to baseline score
Time frame: between baseline and day 35 post baseline
Overall improvement of EB symptoms at Day 35 (measured by percentage change of a patient's iscorEB)
iscorEB: instrument for scoring clinical outcome of research for epidermolysis bullosa; measured in percentage change to baseline score
Time frame: between baseline and day 35 post baseline
Inflammation (measured by panel of inflammation markers)
A panel of inflammation markers will be measured and evaluated.
Time frame: between baseline and day 17, day 35 and week 12 post baseline
Pain assessment as per NRS
Pain assessment as per numerical rating scale (NRS) will be evaluated.
Time frame: between baseline and day 17, day 35 and week 12 post baseline
Itch assessment as per NRS
Itch assessment as per numerical rating scale (NRS) will be evaluated.
Time frame: between baseline and day 17, day 35 and week 12 post baseline
Differences in patient's quality of life in EB
Assessment of quality of life data using an EB-specific quality of life questionnaire
Time frame: between baseline and day 17, day 35 and week 12 post baseline
Physical examination until Week 12;
A full physical examination will be performed and abnormal physical examination results will be evaluated and reported as AEs.
Time frame: At Screening, baseline, day 17, day 35 and week 12
Vital signs: Body temperature until Week 12;
Body temperature will be evaluated at Screening, baseline, day 17, day 35 and week 12
Time frame: At Screening, baseline, day 17, day 35 and week 12
Vital signs: Blood pressure until Week 12;
Blood pressure will be evaluated at Screening, baseline, day 17, day 35 and week 12
Time frame: At Screening, baseline, day 17, day 35 and week 12
Vital signs: Heart rate until Week 12;
Heart rate will be evaluated at Screening, baseline, day 17, day 35 and week 12
Time frame: At Screening, baseline, day 17, day 35 and week 12
Overall survival at month 24
Time frame: month 24 post baseline