NTLA-3001 in Adults with Alpha-1 Antitrypsin Deficiency-Associated Lung Disease
NCT06622668 · Status: WITHDRAWN · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL
Last updated 2025-01-17
Summary
This study will be conducted to evaluate the safety, tolerability, clinical activity, pharmacokinetics, and pharmacodynamics of NTLA-3001 in adults with alpha-1 antitrypsin deficiency (AATD) -associated lung disease
Conditions
- Lung Disease
- Pulmonary Disease
- AATD
- Alpha-1 Antitrypsin Deficiency
- Alpha-1 Antitrypsin Deficiency-associated Lung Disease
Interventions
- BIOLOGICAL
-
Biological NTLA-3001
IV administration of AAV and CRISPR/Cas9 gene editing system
Sponsors & Collaborators
- lead INDUSTRY
Study Design
- Allocation
- NA
- Purpose
- TREATMENT
- Masking
- NONE
- Model
- SINGLE_GROUP
Eligibility
- Min Age
- 18 Years
- Max Age
- 75 Years
- Sex
- ALL
- Healthy Volunteers
- No
Timeline & Regulatory
- Start
- 2024-11-20
- Primary Completion
- 2025-01-09
- Completion
- 2025-01-09
Countries
- New Zealand
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