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Clinical Trial Details — Status: Completed

Administrative data

NCT number NCT03529877
Other study ID # allo-APZ2-EB-II-01
Secondary ID
Status Completed
Phase Phase 1/Phase 2
First received
Last updated
Start date February 16, 2019
Est. completion date November 26, 2021

Study information

Verified date March 2022
Source RHEACELL GmbH & Co. KG
Contact n/a
Is FDA regulated No
Health authority
Study type Interventional

Clinical Trial 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).


Description:

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.


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Study Design


Related Conditions & MeSH terms


Intervention

Biological:
allo-APZ2-EB
intravenous infusion of allo-APZ2-EB

Locations

Country Name City State
Austria EB-Haus Austria; Salzburger Landeskliniken (SALK); Paracelsus Medizinische Privatuniversität Salzburg (PMU) Salzburg
France Hôpital Saint-Louis; Département de dermatologie Paris
Germany Department of Dermatology, Medical Center-University of Freiburg Freiburg
United Kingdom Great Ormond Street Hospital; Dermatology Department London
United Kingdom King's College London; St John's Institute of Dermatology; London
United States University of Minnesota, Masonic Cancer Center and Medical Center Minneapolis Minnesota

Sponsors (4)

Lead Sponsor Collaborator
RHEACELL GmbH & Co. KG FGK Clinical Research GmbH, Granzer Regulatory Consulting & Services, Ticeba GmbH

Countries where clinical trial is conducted

United States,  Austria,  France,  Germany,  United Kingdom, 

Outcome

Type Measure Description Time frame Safety issue
Primary 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 Week 12 post baseline, or last available post-baseline measurement if the Week 12 measurement is missing (last observation carried forward [LOCF])
Primary Assessment of adverse event (AE) occurrence All AEs occurring during the clinical trial will be registered, documented and evaluated. Up to 24 months
Secondary 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 between baseline and week 12 post baseline (without LOCF)
Secondary 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 Week 12 post baseline, or last available post-baseline measurement if the Week 12 measurement is missing (LOCF);
Secondary 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 between baseline and week 12 post baseline (without LOCF)
Secondary 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 between baseline and day 17 post baseline
Secondary 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 between baseline and day 17 post baseline
Secondary 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 between baseline and day 35 post baseline
Secondary 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 between baseline and day 35 post baseline
Secondary Inflammation (measured by panel of inflammation markers) A panel of inflammation markers will be measured and evaluated. between baseline and day 17, day 35 and week 12 post baseline
Secondary Pain assessment as per NRS Pain assessment as per numerical rating scale (NRS) will be evaluated. between baseline and day 17, day 35 and week 12 post baseline
Secondary Itch assessment as per NRS Itch assessment as per numerical rating scale (NRS) will be evaluated. between baseline and day 17, day 35 and week 12 post baseline
Secondary Differences in patient's quality of life in EB Assessment of quality of life data using an EB-specific quality of life questionnaire between baseline and day 17, day 35 and week 12 post baseline
Secondary Physical examination until Week 12; A full physical examination will be performed and abnormal physical examination results will be evaluated and reported as AEs. At Screening, baseline, day 17, day 35 and week 12
Secondary Vital signs: Body temperature until Week 12; Body temperature will be evaluated at Screening, baseline, day 17, day 35 and week 12 At Screening, baseline, day 17, day 35 and week 12
Secondary Vital signs: Blood pressure until Week 12; Blood pressure will be evaluated at Screening, baseline, day 17, day 35 and week 12 At Screening, baseline, day 17, day 35 and week 12
Secondary Vital signs: Heart rate until Week 12; Heart rate will be evaluated at Screening, baseline, day 17, day 35 and week 12 At Screening, baseline, day 17, day 35 and week 12
Secondary Overall survival at month 24 month 24 post baseline
See also
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Recruiting NCT05944250 - A Pilot Study to Evaluate a Temporary Skin Substitute (Spincare® Matrix) for Wound Healing in RDEB Patients N/A
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Terminated NCT02984085 - Clinical Trial to Assess Safety and Efficacy of Autologous Cultured Epidermal Grafts Containing Epidermal Stem Cells Genetically Modified in Patients With RDEB. Phase 1/Phase 2
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