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LZRSE-Col7A1 Engineered Autologous Epidermal Sheets

Phase 1

Epidermolysis Bullosa Dystrophica | Monoclonal antibody | Rare Disease |Abeona Therapeutics Inc.|Last Updated: Aug 22, 2023

Success Probability

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Market & Valuation

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Trial Design

UNCONTROLLEDDMC
Total Trials1
Total Enrollment12

FDA Designations

ORPHAN_DRUG

Clinical trial landscape

LZRSE-Col7A1 Engineered Autologous Epidermal Sheets · 1 trial · 2 indications

Phase 1 1
NCT01263379Gene Transfer for Recessive Dystrophic Epidermolysis BullosaEpidermolysis Bullosa Dystrophica
COMPLETED12 Analytics
PHASE1COMPLETED
Gene Transfer for Recessive Dystrophic Epidermolysis Bullosa
Epidermolysis Bullosa Dystrophica
NCT0126337912
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Study Endpoints

Primary Endpoints

Number of Wounds by Healing Category Per Investigator Visual Assessment
3, 6, 12 and 24 months post grafting

The graft site was clinically evaluated by the investigator with a global score of: 1) 100% to 75% healed, 2) 74% to 50% healed, 3) 49% or less healed with 100% meaning completely healed.

Percentage Surface Area of Wound Healing
3, 6 and 12 months post grafting

Percentage of wound area will be obtained using the Canfield system. Changes in dimensions between visits as well as changes in dimensions from baseline will be recorded. Dimensions of untreated wounded skin will be used for comparison

Secondary Endpoints

Number Participants Positive for NC2 Epitope as a Measure of Duration of Type VII Collagen Production
3 months, 6 months, 12 months, 24 months post-grafting
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Study Design & Arms

AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
LEAES treatmentEXPERIMENTALLZRSE-Col7A1 Engineered Autologous Epidermal Sheets (LEAES)

Interventions

NameTypeDescription
LZRSE-Col7A1 Engineered Autologous Epidermal SheetsBIOLOGICALThis 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.
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Eligibility Criteria

Age Range13 Years to N/A
SexALL
Healthy VolunteersNo
Study Sites1

Inclusion Criteria: 1. Clinical diagnosis of recessive dystrophic epidermolysis bullosa (RDEB) 2. 13 years old or older and willing and able to give assent/consent 3. Confirmation of RDEB diagnosis by immunofluorescence (IF) and electron microscopy (EM) 4. NC1\[+\] and mAb LH24 antibody staining ne...

Countries:United States
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