Allogeneic ABCB5-positive Dermal Mesenchymal Stromal Cell... | NCT05838092

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

NCT05838092

Sponsor

RHEACELL GmbH & Co. KG

Status

Active, not recruiting

Last Update Posted

Dec 10, 2025Actual

Enrollment

9Actual

Phase

Phase 3

Conditions

Epidermolysis Bullosa

Interventions

allo-APZ2-OTS
Placebo

Countries

United States

Protocol Section

Identification Module Status Module Sponsor/Collaborators Module Oversight Module Description Module Conditions Module Design Module Arms/Interventions Module Outcomes Module Eligibility Module Contacts/Locations Module References Module IPD Sharing Statement Module

Results Section

Participant Flow Module Baseline Characteristics Module Outcome Measures Module Adverse Events Module More Info Module

Annotation Section

Annotation Module

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module Condition Browse Module Intervention Browse Module

Protocol Section

Identification Module

NCT ID

Results Section

No data available

No data is available for this block.

Annotation Section

No data available

No data is available for this block.

Document Section

No data available

No data is available for this block.

Derived Section

Miscellaneous Info Module

Version Holder

Status Module

Record Verification Date

Dec 2025

Overall Recruitment Status or Expanded Access Status

Active, not recruiting

Last Known Status

Not provided

Delayed Posting

Not provided

Why Stopped

Not provided

Expanded Access Info

Start Date

Oct 18, 2023Actual

Primary Completion Date

Aug 29, 2025Actual

Completion Date

Jul 2026Estimated

First Submitted Date

Apr 20, 2023

First Submission Date that Met QC Criteria

Apr 20, 2023

First Posted Date

May 1, 2023Actual

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

Dec 3, 2025

Last Update Posted Date

Dec 10, 2025Actual

Description Module

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. An additional baseline-controlled open-label arm will be included to investigate the safety and efficacy of allo-APZ2-OTS administered intravenously to subjects with JEB and to RDEB subjects < 1 year.

Detailed Description

Not provided

Design Module

Study Type

Interventional

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Not provided

Target Follow-Up Duration

Not provided

Phases

Phase 3

Interventional Study Design

Allocation

Biospecimen

No data available

No data is available for this block.

Enrollment

9Actual

Label	Type	Description	Intervention Names
Verum	Experimental		Drug: allo-APZ2-OTS
Placebo	Placebo Comparator		Drug: Placebo

Name	Type	Description	Arm Group Labels	Other Names
allo-APZ2-OTS	Drug	Allogeneic dermal ABCB5-positive Mesenchymal Stromal Cells (ABCB5+ MSCs)	Verum

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
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	6 Months

Secondary Outcomes

Measure	Description	Time Frame
Time to complete target wound closure from baseline		6 Months

Other Outcomes

Not provided

Eligibility Module

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.

Accepts Healthy Volunteers

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

6 Months

Maximum Age

Not provided

Standard Ages

ChildAdultOlder Adult

Study Population

Not provided

Sampling Method

Not provided

Facility	Status	City	State	ZIP	Country	Contacts
Children's Hospital Colorado		Aurora	Colorado	80045	United States
Masonic Cancer Center and Medical Center Minneapolis

ID	Term
D012868	Skin Abnormalities
D000013	Congenital Abnormalities
D009358	Congenital, Hereditary, and Neonatal Diseases and Abnormalities
D012873	Skin Diseases, Genetic