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Allogeneic ABCB5-positive Dermal Mesenchymal Stromal Cells for Treatment of Epidermolysis Bullosa (Phase III)

Primary Purpose

Epidermolysis Bullosa

Status
Not yet recruiting
Phase
Phase 3
Locations
United States
Study Type
Interventional
Intervention
allo-APZ2-OTS
Placebo
Sponsored by
RHEACELL GmbH & Co. KG
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Epidermolysis Bullosa

Eligibility Criteria

6 Months - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

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. 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.

Sites / Locations

  • Masonic Cancer Center and Medical Center Minneapolis

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Placebo Comparator

Arm Label

Verum

Placebo

Arm Description

Outcomes

Primary Outcome Measures

Proportion of subjects with complete target wound closure
Proportion of subjects with complete healing of target wound, determined by the investigator, as compared to baseline

Secondary Outcome Measures

Time to complete target wound closure from baseline

Full Information

First Posted
April 20, 2023
Last Updated
April 20, 2023
Sponsor
RHEACELL GmbH & Co. KG
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1. Study Identification

Unique Protocol Identification Number
NCT05838092
Brief Title
Allogeneic ABCB5-positive Dermal Mesenchymal Stromal Cells for Treatment of Epidermolysis Bullosa (Phase III)
Official 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)
Study Type
Interventional

2. Study Status

Record Verification Date
April 2023
Overall Recruitment Status
Not yet recruiting
Study Start Date
June 1, 2023 (Anticipated)
Primary Completion Date
December 1, 2024 (Anticipated)
Study Completion Date
December 1, 2025 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
RHEACELL GmbH & Co. KG

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes

5. Study Description

Brief Summary
The aim of this clinical trial is to investigate the safety and efficacy of allo-APZ2-OTS administered intravenously to subjects with recessive dystrophic epidermolysis bullosa (RDEB) compared to placebo.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Epidermolysis Bullosa

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
74 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
Verum
Arm Type
Experimental
Arm Title
Placebo
Arm Type
Placebo Comparator
Intervention Type
Drug
Intervention Name(s)
allo-APZ2-OTS
Intervention Description
Allogeneic dermal ABCB5-positive Mesenchymal Stromal Cells (ABCB5+ MSCs)
Intervention Type
Drug
Intervention Name(s)
Placebo
Intervention Description
Placebo
Primary Outcome Measure Information:
Title
Proportion of subjects with complete target wound closure
Description
Proportion of subjects with complete healing of target wound, determined by the investigator, as compared to baseline
Time Frame
12 months
Secondary Outcome Measure Information:
Title
Time to complete target wound closure from baseline
Time Frame
12 months

10. Eligibility

Sex
All
Minimum Age & Unit of Time
6 Months
Accepts Healthy Volunteers
No
Eligibility 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. 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.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Cristina Daniele
Phone
+49 6221 71833-66
Email
cristina.daniele@rheacell.com
Facility Information:
Facility Name
Masonic Cancer Center and Medical Center Minneapolis
City
Minneapolis
State/Province
Minnesota
ZIP/Postal Code
55455
Country
United States

12. IPD Sharing Statement

Plan to Share IPD
Undecided

Learn more about this trial

Allogeneic ABCB5-positive Dermal Mesenchymal Stromal Cells for Treatment of Epidermolysis Bullosa (Phase III)

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