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WITHDRAWNPhase 1/2NCT06622668

NTLA-3001 in Adults with Alpha-1 Antitrypsin Deficiency-Associated Lung Disease

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) -associat...

Lead sponsor: Intellia Therapeutics•1 locations•View source on ClinicalTrials.gov

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Sponsors

Lead Sponsor

Intellia Therapeutics

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.

Age

18 - 75 years

Sex

ALL

Healthy Volunteers

No

Inclusion Criteria

•Inclusion:
•1. Age 18 years to 75 years
•2. Diagnosis of AATD ZZ/ZNull genotypes
•3. FEV1 ≥35% and ≤65%
•4. No evidence of liver cirrhosis
•5. Adequate chemistry and hematology measures at screening
•6. Participants must agree not to participate in another interventional study for the duration of this trial.
•7. Participants must be capable of providing signed informed consent

Exclusion Criteria

•1. AATD genotypes outside of ZZ or ZNull
•2. Participants with total antibodies to adeno-associated virus (AAV) serotype above laboratory assay cut off
•3. Participants who have known negative reaction or hypersensitivity to any lipid nanoparticles (LNP) component.
•4. Any condition that, in the Investigator's opinion, could adversely affect the safety of the participant.
•5. Unwilling to comply with study procedures.

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Data from ClinicalTrials.govLast updated on ClinicalTrials.gov: January 17, 2025Terms

Trial snapshot

StatusWITHDRAWN

PhasePHASE1/PHASE2

Enrollment—

Start dateNov 2024

Last updatedJan 17, 2025

Condition

Lung Disease Pulmonary Disease AATD Alpha-1 Antitrypsin Deficiency Alpha-1 Antitrypsin Deficiency-associated Lung Disease

Locations shown

New Zealand Clinical Research

Aukland

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Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: January 17, 2025Data synced to Clareo: May 25, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.

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