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Gene Therapy

LZRSE-Col7A1 Engineered Autologous Epidermal Sheets for Epidermolysis Bullosa

Phase 1 & 2
Waitlist Available
Led By Jean Tang, MD, PhD
Research Sponsored by Abeona Therapeutics, Inc
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Constipation, can enroll up to Grade 2 (inclusive)
Dysphagia, can enroll up to Grade 4 (inclusive)
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 3, 6, 12 and 24 months post grafting
Awards & highlights

Study Summary

This trial will use the patient's own skin cells that have been genetically engineered to express a protein called type VII collagen. The corrected cells will be transplanted back to the patient.

Eligible Conditions
  • Epidermolysis Bullosa

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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You can have constipation up to a certain level (Grade 2 or lower) to participate in the study.
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You have difficulty swallowing, but it is no worse than a Grade 4 level.
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You can join the trial if you have keratitis that is no worse than Grade 4.
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You have bone pain, but it is not worse than Grade 2.
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The investigators and the doctor in charge may make some exceptions to the rules based on their judgement.
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You have been diagnosed with a rare skin disorder called recessive dystrophic epidermolysis bullosa (RDEB).

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~3, 6, 12 and 24 months post grafting
This trial's timeline: 3 weeks for screening, Varies for treatment, and 3, 6, 12 and 24 months post grafting for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Number of Wounds by Healing Category Per Investigator Visual Assessment
Percentage Surface Area of Wound Healing
Secondary outcome measures
Number Participants Positive for NC2 Epitope as a Measure of Duration of Type VII Collagen Production
Other outcome measures
Number of Participants With Presence of Anchoring Fibrils (AF)

Side effects data

From 2022 Phase 1 & 2 trial • 12 Patients • NCT01263379
86%
Wound infection
43%
Pruritus
29%
Neoplasms benign, malignant and unspecified, squamous cell carcinoma
29%
Pain
14%
Wound complication
14%
Postoperative hemorrhage
100%
80%
60%
40%
20%
0%
Study treatment Arm
LEAES Treatment

Trial Design

1Treatment groups
Experimental Treatment
Group I: LEAES treatmentExperimental Treatment1 Intervention
LZRSE-Col7A1 Engineered Autologous Epidermal Sheets (LEAES)
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
LZRSE-Col7A1 Engineered Autologous Epidermal Sheets
2010
Completed Phase 2
~20

Find a Location

Who is running the clinical trial?

National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)NIH
483 Previous Clinical Trials
1,086,934 Total Patients Enrolled
1 Trials studying Epidermolysis Bullosa
Abeona Therapeutics, IncLead Sponsor
11 Previous Clinical Trials
1,089 Total Patients Enrolled
2 Trials studying Epidermolysis Bullosa
23 Patients Enrolled for Epidermolysis Bullosa
Stanford UniversityOTHER
2,374 Previous Clinical Trials
17,332,915 Total Patients Enrolled
9 Trials studying Epidermolysis Bullosa
136 Patients Enrolled for Epidermolysis Bullosa

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Is this research currently seeking participants?

"Accurate. Clinicaltrials.gov has reported that this clinical trial, which first went public on October 5th 2010 and was modified lastly on October 23rd 2020, is not currently recruiting patients. However, 28 other studies are actively enrolling participants at the moment."

Answered by AI
~1 spots leftby Mar 2025