Trials Sponsors Conditions Drugs Pricing

Conditions

Epidermolysis Bullosa RDEB Recessive Dystrophic Epidermolysis Bullosa

Keywords

Chronic wounds

Outcome Measures

Primary Outcomes (5)

Safety Endpoint (number of treatment-related and treatment-emergent adverse events (AEs) and serious adverse events (SAEs)
The number of treatment-related and treatment-emergent adverse events (TEAEs), treatment-emergent wound adverse events (TEWAEs), and serious adverse events (SAEs), including systemic and wound-specific adverse events.
6 months
Safety Endpoint (number of patients and wounds that have an infection or any related adverse event.)
The number of patients and wounds that have an infection or any related adverse event (AE).
6 months
Safety Endpoint (number of patients and wounds that result in hospitalization (serious adverse event )
The number of patients and wounds that result in hospitalization (SAE).
6 months
Safety Endpoint (Incidence of squamous cell carcinoma)

Other Outcomes (8)

Proportion of RDEB wounds with ≥50%, ≥75%, and complete
6 months
Extent of pain reduction assessed by the Wong-Baker FACES scale
6 months
Longitudinal change in scores of Worst Itch Numeric Rating Scale
6 months
+5 more other outcomes

Study Arms (1)

EB-101 Surgical application of RDEB wounds

EXPERIMENTAL

New or Previously Treated RDEB Patients

Biological: EB-101 Surgical application of RDEB wounds

Interventions

EB-101 Surgical application of RDEB woundsBiological

EB-101 autologous RDEB keratinocytes isolated from skin biopsies and transduced with a recombinant retrovirus containing a full-length COL7A1 expression cassette for C7 Other Names: • LZRSE-Col7A1 Engineered Autologous Epidermal Sheets \[LEAES\]

Also known as: pz-cel

EB-101 Surgical application of RDEB wounds

Registry-reported names are shown in full. Names without a link have not yet been matched to a Syfrah profile.

Eligibility Criteria

Age12 Months+

Sexall

Healthy VolunteersNo

Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

Clinical diagnosis of DEB.
Age 12 months and older.

Sponsors & Collaborators

Abeona Therapeutics, Inc
Industrylead

Study Sites (2)

Stanford University

Redwood City, California, 94063, United States

University of Massachusetts Medical School

Worcester, Massachusetts, 01605, United States

MeSH Terms

Conditions

Epidermolysis BullosaEpidermolysis Bullosa Dystrophica

Condition Hierarchy (Ancestors)

Skin AbnormalitiesCongenital AbnormalitiesCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesSkin Diseases, GeneticGenetic Diseases, InbornSkin DiseasesSkin and Connective Tissue DiseasesSkin Diseases, VesiculobullousCollagen DiseasesConnective Tissue Diseases

Study Officials

Angela Iheanacho, MS
Abeona Therapeutics, Inc
STUDY DIRECTOR
Sarah Abdelwahab, MD
Abeona Therapeutics, Inc
STUDY DIRECTOR

Study Design

Study Type: interventional
Phase: phase 3
Allocation: NA
Masking: NONE
Purpose: TREATMENT
Intervention Model: SINGLE GROUP
Sponsor Type: INDUSTRY
Responsible Party: SPONSOR

Study Record Dates

First Submitted

January 23, 2023

First Posted

February 13, 2023

Study Start

April 2, 2023

Actual (reported)

Primary Completion

September 30, 2025

Estimated (date passed)

This estimate has passed. The registry has not confirmed an actual date for this milestone.

A Phase 3b Study for the Treatment of Dystrophic Epidermolysis Bullosa (DEB) in New and Previously EB-101 Treated Patients

Brief Summary

Registry Overview

Trial Relationships

Study Timeline

First Submitted

First Posted

Study Start

Primary Completion

Study Completion

Conditions

Keywords

Outcome Measures

Primary Outcomes (5)

Other Outcomes (8)

Study Arms (1)

EB-101 Surgical application of RDEB wounds

Interventions

Eligibility Criteria

You may qualify if:

Sponsors & Collaborators

Study Sites (2)

MeSH Terms

Conditions

Condition Hierarchy (Ancestors)

Study Officials

Study Design

Study Record Dates

You may not qualify if:

Data Sharing

Source record

Locations

Brief Summary

Registry Overview

Trial Relationships

Related Scientific Literature

Study Timeline

First Submitted

First Posted

Study Start

Primary Completion

Study Completion

Conditions

Keywords

Outcome Measures

Primary Outcomes (5)

Other Outcomes (8)

Study Arms (1)

EB-101 Surgical application of RDEB wounds

Interventions

Eligibility Criteria

You may qualify if:

Sponsors & Collaborators

Study Sites (2)

MeSH Terms

Conditions

Condition Hierarchy (Ancestors)

Study Officials

Study Design

Study Record Dates

You may not qualify if:

Data Sharing

Source record

Locations