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NCT06996756 · ClinicalTrials.gov registry record · Phase 1
Gene Therapy for Alpha 1- Antitrypsin Deficiency
A Phase 1 study of Alpha-1 Antitrypsin Deficiency, sponsored by Weill Medical College of Cornell University.
- Recruiting
- Registry status
- Phase 1
- Development phase
- 16
- Enrollment target
- 1
- Study location
NCT06996756: Recruiting Phase 1 study of Alpha-1 Antitrypsin Deficiency, sponsored by Weill Medical College of Cornell University.
NCT06996756 is a Phase 1 study of Alpha-1 Antitrypsin Deficiency that is actively recruiting participants, run by Weill Medical College of Cornell University. The registered enrollment target is 16 participants, below the 2,341-participant average among 24 other Alpha-1 Antitrypsin Deficiency trials with a reported enrollment target (99% lower). The trial reports 1 study location across 1 state. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT06996756, a Phase 1 study of Alpha-1 Antitrypsin Deficiency, is actively recruiting participants, sponsored by Weill Medical College of Cornell University.
- RECRUITING
- Registry status
- Phase 1
- Development phase
- 16 participants
- Enrollment target
- 1
- Study location
Study Summary
This is a study of gene therapy to treat alpha 1-antitrypsin (AAT) deficiency. This study aims to treat AAT deficiency with a single administration of AAV8hAAT(AVL), a gene therapy that codes for an oxidation resistant form of the AAT protein, which if safe and if efficacious, will protect the lung on a persistent basis. We hope to learn the safety/toxicity and initial evidence of efficacy of intravenous delivery of this gene therapy to alpha 1-antitrypsin deficient individuals.
Primary Outcome
Serious adverse events will only be included if assessed as related to the gene therapy.
Conditions Studied
Interventions
- BIOLOGICAL AAV8hAAT(AVL)
Study Locations (1)
New York
- WCMC Department of Genetic Medicine - New York
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 16 participants |
| Start Date | 2025-02-26 |
| Est. Completion | 2032-08-01 |
| Phase | Phase 1 |
What NCT06996756 shows while recruiting
NCT06996756 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 16 participants, a relatively small participant target, below the 2,341-participant average among 24 other Alpha-1 Antitrypsin Deficiency trials with a reported enrollment target (99% lower).
The record links to 1 condition, with Alpha-1 Antitrypsin Deficiency appearing as the primary indexed condition, and to 1 intervention - of which AAV8hAAT(AVL) is the first listed.
NCT06996756 reports a single indexed study location in New York.
Frequently Asked Questions
What is clinical trial NCT06996756 about?
NCT06996756 is a clinical study titled "Gene Therapy for Alpha 1- Antitrypsin Deficiency". This is a study of gene therapy to treat alpha 1-antitrypsin (AAT) deficiency. This study aims to treat AAT deficiency with a single administration of AAV8hAAT(AVL), a gene therapy that codes for an oxidation resistant form of the AAT protein, which if safe and if efficacious, will protect the lung ...
What is the current status of trial NCT06996756?
This trial is currently recruiting. It is a Phase 1 study. The enrollment target is 16 participants. The study started on 2025-02-26. Estimated completion is 2032-08-01.
What conditions does trial NCT06996756 study?
This clinical trial studies the following conditions: Alpha-1 Antitrypsin Deficiency.
What interventions are being tested in trial NCT06996756?
The interventions under investigation include: AAV8hAAT(AVL) (BIOLOGICAL).
Who is sponsoring clinical trial NCT06996756?
This trial is sponsored by Weill Medical College of Cornell University, which has 701 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT06996756 being conducted?
This trial has 1 study location across New York. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
Similar trials for Alpha-1 Antitrypsin Deficiency
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Where NCT06996756's enrollment target sits among peer trials
16 20th of 24 higher than 5 of 24 other Alpha-1 Antitrypsin Deficiency trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Alpha-1 Antitrypsin Deficiency 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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