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

Intellia Therapeutics

Status and phase: Withdrawn; Phase 2; Phase 1

Conditions: AATD; Pulmonary Disease; Alpha-1 Antitrypsin Deficiency-associated Lung Disease; Lung Disease; Alpha-1 Antitrypsin Deficiency

Treatments: Biological: Biological NTLA-3001

Study type: Interventional

Funder types: Industry

Identifiers

NCT06622668

ITL-3001-CL-101

Details and patient eligibility

About

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

Full 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.

Sex: All

Ages: 18 to 75 years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion 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.

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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0 participants in 1 patient group

Arm 1

Experimental group

Treatment:

Biological: Biological NTLA-3001