Clinical Trials List
Protocol Numberallo-APZ2-EB-III
NCT Number(ClinicalTrials.gov Identfier)NCT05838092
Active
2023-07-01 - 2026-09-30
Phase III
Recruiting1
ICD-10Q81.0
Epidermolysis bullosa simplex
ICD-10Q81.1
Epidermolysis bullosa letalis
ICD-10Q81.2
Epidermolysis bullosa dystrophica
ICD-10Q81.8
Other epidermolysis bullosa
ICD-10Q81.9
Epidermolysis bullosa, unspecified
ICD-10Q82.8
Other specified congenital malformations of skin
ICD-10Q82.9
Congenital malformation of skin, unspecified
ICD-9757.39
Other specified anomalies of skin
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)
-
Trial Applicant
Syneos Health
-
Sponsor
-
Trial scale
Multi-Regional Multi-Center
-
Update
2026/03/01
Investigators and Locations
Principal Investigator
Chao-Kai Hsu
Co-Principal Investigator
- Wei-Ting Tu Division of Dermatology
- Chao-Chun Yang Division of Dermatology
The Actual Total Number of Participants Enrolled
0 Recruiting
Condition/Disease
Epidermolysis Bullosa
Objectives
The objective of this clinical trial is to investigate the safety and efficacy of intravenous allo-APZ2-OTS compared to placebo in patients with subclinical dystrophic epidermolysis bullosa (RDEB). An additional baseline-controlled open-label group will be included to investigate the safety and efficacy of intravenous allo-APZ2-OTS in patients with jEB and RDEB <1 year of age.
Test Drug
Intravenous infusion
Active Ingredient
ABCB5+ MSC
Dosage Form
246
Dosage
10.5 × 10^6 cells/mL
Endpoints
The proportion of RDEB subjects aged ≥1 year whose target wounds were completely healed by month 6 (complete healing by month 6a and confirmed by month 6b, 2 weeks later).
Inclution Criteria
Inclusion Criteria:
Male or female subjects from 6 months of age with a diagnosis of RDEB confirmed by genetic testing or by a skin biopsy with immunofluorescence mapping
Subject is eligible to participate in this clinical trial based on general health condition;
Subject with a target wound meeting the following criteria: 5-50 cm2, ≥ 21 days and < 9 months, no signs of acute infection;
Patient/legal representative understands the nature of the procedure and provide written informed consent/assent prior to any clinical trial procedure;
Women of childbearing potential must have a negative urine pregnancy test at Visit 1. Women of childbearing potential, male participants, and their partner must be willing to use highly effective contraceptive methods during the course of the entire clinical trial.
Male or female subjects from 6 months of age with a diagnosis of RDEB confirmed by genetic testing or by a skin biopsy with immunofluorescence mapping
Subject is eligible to participate in this clinical trial based on general health condition;
Subject with a target wound meeting the following criteria: 5-50 cm2, ≥ 21 days and < 9 months, no signs of acute infection;
Patient/legal representative understands the nature of the procedure and provide written informed consent/assent prior to any clinical trial procedure;
Women of childbearing potential must have a negative urine pregnancy test at Visit 1. Women of childbearing potential, male participants, and their partner must be willing to use highly effective contraceptive methods during the course of the entire clinical trial.
Exclusion Criteria
Exclusion Criteria:
Any current tumor diseases, including squamous cell carcinoma and basal cell carcinoma;
Any known allergies to components of the IP or premedication;
Patient/legal representative anticipated to be unwilling or unable to comply with the requirements of the protocol;
Pregnant or lactating women;
Current or previous (within 30 days of screening) treatment with another IP, or participation and/or under follow-up in another interventional clinical trial;
Previous participation in this clinical trial (except for screening failures);
Clinically significant or unstable concurrent disease or other clinical contraindications like an uncontrolled or poorly controlled mental health condition of the subject and/or his/her legal representative that could impact on patient's safety or interfere with study compliance such as inability to attend scheduled study visits; Confidential
Employees of the sponsor, or employees or relatives of the investigator.
Any current tumor diseases, including squamous cell carcinoma and basal cell carcinoma;
Any known allergies to components of the IP or premedication;
Patient/legal representative anticipated to be unwilling or unable to comply with the requirements of the protocol;
Pregnant or lactating women;
Current or previous (within 30 days of screening) treatment with another IP, or participation and/or under follow-up in another interventional clinical trial;
Previous participation in this clinical trial (except for screening failures);
Clinically significant or unstable concurrent disease or other clinical contraindications like an uncontrolled or poorly controlled mental health condition of the subject and/or his/her legal representative that could impact on patient's safety or interfere with study compliance such as inability to attend scheduled study visits; Confidential
Employees of the sponsor, or employees or relatives of the investigator.
The Estimated Number of Participants
-
Taiwan
7 participants
-
Global
79 participants
