NCT03529877

Allogeneic ABCB5-positive Stem Cells for Treatment of Epidermolysis Bullosa

An Interventional, Multicenter, Single Arm, Phase I/IIa Clinical Trial to Investigate the Efficacy and Safety of Allo-APZ2-EB on Epidermolysis Bullosa (EB)

• Status: Completed
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 16 (actual)
• Sponsor: RHEACELL GmbH & Co. KG · Industry
• Sex: All
• Age: 0 Years – 55 Years
• Healthy volunteers: Not accepted

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).

Detailed 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.

Conditions

• Recessive Dystrophic Epidermolysis Bullosa

Interventions

Type	Name	Description
BIOLOGICAL	allo-APZ2-EB	intravenous infusion of allo-APZ2-EB

Timeline

• Start date: 2019-02-16
• Primary completion: 2021-11-26
• Completion: 2021-11-26
• First posted: 2018-05-18
• Last updated: 2022-10-07

Locations

6 sites across 5 countries: United States, Austria, France, Germany, United Kingdom

Regulatory

• FDA-regulated drug study