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

January 16, 2025 updated by: Intellia Therapeutics

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

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

Study Overview

Status

Withdrawn

Conditions

Intervention / Treatment

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.

Study Type

Interventional

Phase

  • Phase 2
  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • New Zealand
      • Aukland, New Zealand, 1010
        • New Zealand Clinical Research

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Arm 1
Biological: Biological NTLA-3001
IV administration of AAV and CRISPR/Cas9 gene editing system

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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)
From NTLA-3001 infusion up to week156 post infusion

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Pharmacodynamics
Time Frame: From NTLA-3001 infusion up to week 156 post infusion
To evaluate the PD effect of NTLA-3001
From NTLA-3001 infusion up to week 156 post infusion
Immune Response
Time Frame: From NTLA-3001 infusion up to week 156 post infusion
To evaluate the immune response to NTLA-3001
From NTLA-3001 infusion up to week 156 post infusion
Vector Shedding
Time Frame: From NTLA-3001 infusion up to week 156 post infusion, only during phase 2.
To evaluate vector shedding following administration of NTLA-3001
From NTLA-3001 infusion up to week 156 post infusion, only during phase 2.

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Intellia Therapeutics

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

November 20, 2024

Primary Completion (Estimated)

January 9, 2025

Study Completion (Actual)

January 9, 2025

Study Registration Dates

First Submitted

September 30, 2024

First Submitted That Met QC Criteria

September 30, 2024

First Posted (Actual)

October 2, 2024

Study Record Updates

Last Update Posted (Actual)

March 25, 2025

Last Update Submitted That Met QC Criteria

January 16, 2025

Last Verified

January 1, 2025

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • ITL-3001-CL-101

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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