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

NCT06622668 | Withdrawn Phase 1 DMC

• 1 other identifier: ITL-3001-CL-101
• Study Type: interventional
• Target: N/A
• Locations: 1 country
• Sites: 1

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

Conditions

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

Keywords

AATD

Outcome Measures

Primary Outcomes (1)

• Safety and Tolerability

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

  From NTLA-3001 infusion up to week156 post infusion

Secondary Outcomes (3)

• 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.

Study Arms (1)

Arm 1

EXPERIMENTAL

Biological: Biological NTLA-3001

Interventions

Biological NTLA-3001 BIOLOGICAL

IV administration of AAV and CRISPR/Cas9 gene editing system

Arm 1

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

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

You may not qualify if:

• 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.

Sponsors & Collaborators

• Intellia Therapeutics: lead

Study Sites (1)

New Zealand Clinical Research

Aukland, 1010, New Zealand

Location

MeSH Terms

Conditions

Lung Diseases; alpha 1-Antitrypsin Deficiency

Condition Hierarchy (Ancestors)

Respiratory Tract Diseases; Liver Diseases; Digestive System Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Subcutaneous Emphysema; Emphysema; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 30, 2024
• First Posted: October 2, 2024
• Study Start: November 20, 2024
• Primary Completion: January 9, 2025
• Study Completion: January 9, 2025
• Last Updated: January 17, 2025

Record last verified: 2025-01

Data Sharing

• IPD Sharing: Will not share

Locations

NZ (1)