allo-APZ2-EB-III
Research type
Research Study
Full title
A double-blind, randomized, placebo-controlled, interventional, multicenter, phase III clinical trial to investigate the safety and efficacy of ABCB5-positive mesenchymal stromal cells (ABCB5+ MSCs) on epidermolysis bullosa (EB)
IRAS ID
1006269
Contact name
Cristina Daniele
Contact email
Sponsor organisation
RHEACELL GmbH & Co. KG
Eudract number
2021-006076-16
Clinicaltrials.gov Identifier
Research summary
This is a double-blind, randomized, placebo-controlled, interventional, multicenter, phase III clinical trial to investigate the safety and efficacy of allo-APZ2-OTS in participants with RDEB (≥ 1 year). The safety and efficacy of allo-APZ2-OTS will be assessed for JEB participants and for RDEB participants < 1 year in an additional open-label study arm. The active substance of the investigational product (IP) allo-APZ2-OTS consists of allogeneic ABCB5+ MSCs isolated as a high-potent subpopulation of expanded human skin-derived MSC cultures. The IP will be given intravenously to participants with RDEB and JEB at the dose of 2 × 10e6 ABCB5+ cells/kg in application solution. Participants will be screened at Visit 1 (Day -10 to -7). RDEB participants ≥ 1 year will be randomised to treatment with either allo-APZ2-OTS or placebo. Randomization will be stratified by target wound size (5 to < 26 cm squared, 26 to 50 cm squared). RDEB participants < 1 year and JEB participants will only be treated with allo-APZ2-OTS. Participants < 1 year (irrespective of the EB subtype) will be enrolled in a staggered fashion, according to which the next participant < 1 year can only be enrolled when the first application of the prior participant< 1 year showed no issues in safety and tolerability (assessed at Day 17 after first administration of the IP by the Data Monitoring Committee (DMC)). During the 6-month double-blind placebo-controlled period, RDEB participants ≥ 1 year will undergo treatment with either allo-APZ2-OTS or placebo initially 3 times within 5 weeks (Day 0, 17 ±3 days and 35 ±3 days) and then every 2 months at Month 3 (±3 days) and Month 5 (±3 days). The primary endpoint will be determined at the end of the double-blind placebo-controlled period at Month 6 (Visits 7 and 8).
REC name
South West - Central Bristol Research Ethics Committee
REC reference
22/SW/0133
Date of REC Opinion
24 Nov 2022
REC opinion
Further Information Favourable Opinion