allo-APZ2-EB-II-01 - Phase I/IIA CT of Allo-APZ2-EB
Research type
Research Study
Full title
An interventional, multicenter, single arm, phase I/IIa clinical trial to investigate the efficacy and safety of allo-APZ2-EB on epidermolysis bullosa (EB)
IRAS ID
245586
Contact name
Kathrin Dieter
Contact email
Sponsor organisation
RHEACELL GmbH & Co. KG
Eudract number
2018-001009-98
Clinicaltrials.gov Identifier
Duration of Study in the UK
2 years, 0 months, 0 days
Research summary
Summary of Research
This clinical trial is a scientific research study on patients who have epidermolysis bullosa (EB). EB is a serious hereditary skin disease that does not currently have a cure. People with the condition find that their skin blisters and tears with the slightest mechanical stress. Blisters, wounds and scars are not only limited to the skin, but they can also occur on mucous tissue in the mouth, eyes, food pipe, stomach and intestines.For this clinical trial, so-called stem cells will be obtained from the skin of healthy donors and then fed and grown in a laboratory under special conditions so that a solution can then be administered to the participating patients via a vein. This should reduce blister formation, and trigger and speed up wound healing.
The medicine containing these special stem cells is called allo-APZ2-EB, and it is being investigated in this clinical trial. This is a clinical trial without a control group; this means that all patients will be treated with the drug allo-APZ2-EB, that is being investigated in this clinical trial. In total, the study drug will be given three times, at intervals of approximately 2 weeks.
In the UK this study will be conducted at two sites, with patients visiting the sites five times within one year.
Allo-APZ2-EB belongs to a group of drugs known as advanced therapy medicinal products. It is a medicinal product that is under clinical investigation. That means it is not yet approved by the authorities for the treatment. To date, it has never previously been used on humans.
The objective of the clinical trial is to evaluate whether the use of allo-APZ2-EB can improve the disease in such a way that the EB symptoms are reduced, and wounds and blisters partially - or in the best-case scenario even completely - heal.Summary of Results
Treatment with the study drug resulted in an improvement of the epidermolysis bullosa disease activity and scarring index (EBDASI) activity score and a slight improvement in the EBDASI overall score at Week 12 • The clinical outcome as assessed by the instrument for scoring clinical outcome of research for epidermolysis bullosa (iscorEB)clinician subscore showed a general improvement which was most pronounced at Week 12 • Treatment with the study drug reduced itch (-37.5% at Day 35) and to a slighter extent also pain (-25.0% at Day 35) as assessed by the patient on an numeric rating scale (NRS); • Treatment with study drug facilitated skin wound closure at week 12. In addition, the treatment with study drug prolongated natural wound recurrence. The wounds that did not fully close during the 12-week treatment and efficacy period significantly decreased in size. The development of new wounds was significantly decelerated by the treatment with study drug.
• In general, the study drug was well tolerated with no evidence of safety concerns. Two serious and drug related-related events of Hypersensitivity occurred. Twelve and 24 months after the first allo-APZ2-EB application, the overall survival was 100%.REC name
North East - York Research Ethics Committee
REC reference
18/NE/0240
Date of REC Opinion
18 Sep 2018
REC opinion
Further Information Favourable Opinion