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NCT01263379 · ClinicalTrials.gov registry record · Phase 1
Gene Transfer for Recessive Dystrophic Epidermolysis Bullosa
A Phase 1 study of Epidermolysis Bullosa and Epidermolysis Bullosa Dystrophica, sponsored by Abeona Therapeutics.
- Completed
- Registry status
- Phase 1
- Development phase
- 12
- Enrollment target
- 1
- Study location
NCT01263379: Completed Phase 1 study of Epidermolysis Bullosa and Epidermolysis Bullosa Dystrophica, sponsored by Abeona Therapeutics.
NCT01263379 is a Phase 1 study of Epidermolysis Bullosa and Epidermolysis Bullosa Dystrophica that has completed, run by Abeona Therapeutics. The registered enrollment target is 12 participants, below the 19-participant average among 10 other Epidermolysis Bullosa trials with a reported enrollment target (37% lower). The trial reports 1 study location across 1 state. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT01263379, a Phase 1 study of Epidermolysis Bullosa and Epidermolysis Bullosa Dystrophica, has completed, sponsored by Abeona Therapeutics.
- COMPLETED
- Registry status
- Phase 1
- Development phase
- 12 participants
- Enrollment target
- 1
- Study location
Study 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.
Primary Outcome
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.
Conditions Studied
Interventions
- BIOLOGICAL LZRSE-Col7A1 Engineered Autologous Epidermal Sheets
Study Locations (1)
California
- Stanford University, School of Medicine, Dept of Dermatology - Redwood City
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 12 participants |
| Start Date | 2010-10-05 |
| Est. Completion | 2022-03-09 |
| Phase | Phase 1 |
What the finished NCT01263379 record still lists
NCT01263379 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 12 participants, a relatively small participant target, below the 19-participant average among 10 other Epidermolysis Bullosa trials with a reported enrollment target (37% lower).
The record links to 2 conditions, with Epidermolysis Bullosa appearing as the primary indexed condition, and to 1 intervention - of which LZRSE-Col7A1 Engineered Autologous Epidermal Sheets is the first listed.
NCT01263379 reports a single indexed study location in California.
Frequently Asked Questions
What is clinical trial NCT01263379 about?
NCT01263379 is a clinical study titled "Gene Transfer for Recessive Dystrophic Epidermolysis Bullosa". 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.
What is the current status of trial NCT01263379?
This trial is currently completed. It is a Phase 1 study. The enrollment target is 12 participants. The study started on 2010-10-05. Estimated completion is 2022-03-09.
What conditions does trial NCT01263379 study?
This clinical trial studies the following conditions: Epidermolysis Bullosa, Epidermolysis Bullosa Dystrophica.
What interventions are being tested in trial NCT01263379?
The interventions under investigation include: LZRSE-Col7A1 Engineered Autologous Epidermal Sheets (BIOLOGICAL).
Who is sponsoring clinical trial NCT01263379?
This trial is sponsored by Abeona Therapeutics, which has 8 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT01263379 being conducted?
This trial has 1 study location across California. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
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Where NCT01263379's enrollment target sits among peer trials
12 6th of 10 higher than 4 of 10 other Epidermolysis Bullosa trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Epidermolysis Bullosa trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
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