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

Intellia Therapeutics

Overview

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

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.

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Conditions

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

Interventions

Biological NTLA-3001BIOLOGICAL

IV administration of AAV and CRISPR/Cas9 gene editing system

Eligibility

Sex: ALLMin age: 18 YearsMax age: 75 Years

Medical Language ↔ Plain English

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.

Locations (1)

New Zealand Clinical Research

Aukland, New Zealand

Outcomes

Primary Outcomes

Safety and Tolerability

To evaluate the safety and tolerability of NTLA-3001 as determined by adverse events (AEs) and dose limiting toxicities (DLTs)

Time frame: From NTLA-3001 infusion up to week156 post infusion

Secondary Outcomes

Pharmacodynamics

To evaluate the PD effect of NTLA-3001

Time frame: From NTLA-3001 infusion up to week 156 post infusion

Immune Response

To evaluate the immune response to NTLA-3001

Time frame: From NTLA-3001 infusion up to week 156 post infusion

Vector Shedding

To evaluate vector shedding following administration of NTLA-3001

Time frame: From NTLA-3001 infusion up to week 156 post infusion, only during phase 2.

Data from ClinicalTrials.gov

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