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
Overview
- Phase
- Phase 1
- Intervention
- Biological NTLA-3001
- Conditions
- Lung Disease
- Sponsor
- Intellia Therapeutics
- Enrollment
- 30
- Locations
- 1
- Primary Endpoint
- Safety and Tolerability
- Status
- Withdrawn
- Last Updated
- last year
Overview
Brief 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.
Investigators
Eligibility Criteria
Inclusion Criteria
- Not provided
Exclusion Criteria
- •AATD genotypes outside of ZZ or ZNull
- •Participants with total antibodies to adeno-associated virus (AAV) serotype above laboratory assay cut off
- •Participants who have known negative reaction or hypersensitivity to any lipid nanoparticles (LNP) component.
- •Any condition that, in the Investigator's opinion, could adversely affect the safety of the participant.
- •Unwilling to comply with study procedures.
Arms & Interventions
Arm 1
Intervention: Biological NTLA-3001
Outcomes
Primary Outcomes
Safety and Tolerability
Time Frame: From NTLA-3001 infusion up to week156 post infusion
To evaluate the safety and tolerability of NTLA-3001 as determined by adverse events (AEs) and dose limiting toxicities (DLTs)
Secondary Outcomes
- Pharmacodynamics(From NTLA-3001 infusion up to week 156 post infusion)
- Immune Response(From NTLA-3001 infusion up to week 156 post infusion)
- Vector Shedding(From NTLA-3001 infusion up to week 156 post infusion, only during phase 2.)