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Gene Transfer for Recessive Dystrophic Epidermolysis Bullosa

NCT01263379 · Status: COMPLETED · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 12

Last updated 2023-08-22

Study results available
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Summary

This trial will create a skin graft, which the investigators call "LEAES," using the patient's own skin cells that have been genetically engineered in the lab to express a missing protein called type VII collagen. The corrected cells will be transplanted back to the patient.

Conditions

  • Epidermolysis Bullosa Dystrophica
  • Epidermolysis Bullosa

Interventions

BIOLOGICAL

LZRSE-Col7A1 Engineered Autologous Epidermal Sheets

This trial will create a graft, which we call "LEAES", of the patient's own skin that has been genetically engineered in our lab to express this missing protein.

Sponsors & Collaborators

  • National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)

    collaborator NIH

  • Stanford University

    collaborator OTHER

  • Abeona Therapeutics, Inc

    lead INDUSTRY

Principal Investigators

  • Jean Tang, MD, PhD · Stanford University

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
13 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2010-10-05
Primary Completion
2022-03-09
Completion
2022-03-09
FDA Drug
Yes

Countries

  • United States

Study Locations

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Entities

Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT01263379 on ClinicalTrials.gov