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Gene Transfer for Recessive Dystrophic Epidermolysis Bullosa
NCT01263379 · View on ClinicalTrials.gov ↗
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.
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 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT01263379
The ClinicalTrials.gov registry entry for NCT01263379 describes a study currently listed as completed. It is categorized as Phase 1, which is the standard way researchers label where a study sits along the investigational pathway from early safety work through later efficacy and post-marketing evaluation. The registered enrollment target is 12 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Abeona Therapeutics, which has 4 total studies on file at ClinicalTrials.gov, and sponsors are the parties responsible for study design, oversight, and regulatory filings.
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. Interventions can include drugs, devices, procedures, behavioral programs, or observational arms, and each is tracked as a separate registry field so that downstream queries can filter accurately. When a trial lists multiple interventions, it usually reflects a multi-arm design or a comparison protocol rather than a single treatment being tested in isolation. The brief summary published in the registry is the clearest source of protocol intent and should be read before drawing conclusions from any sidebar tags.
Geographic footprint matters for practical reasons: NCT01263379 reports 1 study location spanning 1 distinct geographic area — top geographies include California. A larger site network tends to correlate with broader recruitment capacity, but it does not imply anything about study quality, and site-level enrollment status can diverge from the overall registry status shown above. Every data point on this page comes from the public ClinicalTrials.gov dataset and is reproduced here for reference only; it is not a medical recommendation, an endorsement of the sponsor, or an invitation to enroll. Verify current status, eligibility criteria, and contact details directly at ClinicalTrials.gov, and discuss any participation decision with your own healthcare provider.
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. These conditions were identified from the trial registry and reflect the primary focus areas of the research.
What interventions are being tested in trial NCT01263379?
The interventions under investigation include: LZRSE-Col7A1 Engineered Autologous Epidermal Sheets (BIOLOGICAL). Each intervention is being evaluated for safety and efficacy as part of this clinical study.
Who is sponsoring clinical trial NCT01263379?
This trial is sponsored by Abeona Therapeutics, which has 4 total clinical trials registered on ClinicalTrials.gov. The sponsor is responsible for the study's design, funding, and regulatory compliance.
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.
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