Trials / Withdrawn
WithdrawnNCT06622668
NTLA-3001 in Adults with Alpha-1 Antitrypsin Deficiency-Associated Lung Disease
Phase 1/2 Multicenter, Open-label Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of NTLA-3001 in Participants with Alpha-1 Antitrypsin Deficiency (AATD)-Associated Lung Disease
- Status
- Withdrawn
- Phase
- Phase 1 / Phase 2
- Study type
- Interventional
- Enrollment
- 0 (actual)
- Sponsor
- Intellia Therapeutics · Industry
- Sex
- All
- Age
- 18 Years – 75 Years
- Healthy volunteers
- Not accepted
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
Detailed description
This study consists of 2 parts: Phase 1 is an open-label, single-arm ascending dose study to characterize the safety and activity of NTLA3001 and identify the dose for evaluation in Phase 2. Phase 2 will follow as an open-label, dose expansion study to further characterize the safety and clinical activity of NTLA-3001 and provide an initial assessment of the effect of NTLA-3001 on clinical measures of pulmonary function.
Conditions
- Lung Disease
- Pulmonary Disease
- AATD
- Alpha-1 Antitrypsin Deficiency
- Alpha-1 Antitrypsin Deficiency-associated Lung Disease
Interventions
| Type | Name | Description |
|---|---|---|
| BIOLOGICAL | Biological NTLA-3001 | IV administration of AAV and CRISPR/Cas9 gene editing system |
Timeline
- Start date
- 2024-11-20
- Primary completion
- 2025-01-09
- Completion
- 2025-01-09
- First posted
- 2024-10-02
- Last updated
- 2025-01-17
Locations
1 site across 1 country: New Zealand
Source: ClinicalTrials.gov record NCT06622668. Inclusion in this directory is not an endorsement.