NTLA-2002 in Adults With Hereditary Angioedema (HAE) (NTLA-2002)

March 23, 2026 updated by: Intellia Therapeutics

Phase 1/2 Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of NTLA-2002 in Adults With Hereditary Angioedema (HAE)

This study will be conducted to evaluate the safety, tolerability, activity, pharmacokinetics, and pharmacodynamics of NTLA-2002 in adults with Hereditary Angioedema (HAE).

Study Overview

Status

Active, not recruiting

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

37

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

  • Australia
      • Campbelltown, Australia
        • Clinical Trial Site
  • France
      • Grenoble, France
        • Clinical Trial Site
      • Lille, France
        • Clinical Trial Site
      • Paris, France
        • Clinical Trial Site
  • Germany
      • Berlin, Germany
        • Clinical Trial Site
      • Frankfurt, Germany
        • Clinical Trial Site
  • Netherlands
      • Amsterdam, Netherlands
        • Clinical Trial Site
  • New Zealand
      • Auckland, New Zealand
        • Clinical Trial Site
  • United Kingdom
      • Cambridge, United Kingdom
        • Clinical Trial Site

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

18 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  1. Age >18 years
  2. Diagnosis of HAE Types I or II
  3. Ability to provide evidence of HAE attacks to meet the screening requirement
  4. Subjects must have access to, and the ability to use, ≥ 1 acute medication(s) to treat angioedema attacks.
  5. Adequate chemistry and hematology measures at screening
  6. Subjects must agree not to participate in another interventional study for the duration of this trial.
  7. Subjects must be capable of providing signed informed consent

Exclusion Criteria:

  1. Concurrent diagnosis of any other type of recurrent angioedema
  2. Subjects who have known negative reaction or hypersensitivity to any lipid nanoparticles (LNP) component.
  3. Any condition that, in the Investigator's opinion, could adversely affect the safety of the subject.
  4. 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: Randomized
  • Interventional Model: Sequential Assignment
  • Masking: Double

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Phase 1 Study Arm
Participants assigned to 1 of 3 dose-escalation cohorts will receive a single dose of NTLA-2002 on Day 1 and will then be followed for 104 weeks. Primary observation period is 16 weeks.
Biological: Biological NTLA-2002
CRISPR/Cas9 gene editing system delivered by LNP for IV administration
Experimental: Phase 2 Experimental Study Arm
Participants randomized to NTLA-2002 (2 dose levels), will receive a single dose of NTLA-2002 on Day 1 and will then be followed for 104 weeks. Primary observation period is 16 weeks.
Biological: Biological NTLA-2002
CRISPR/Cas9 gene editing system delivered by LNP for IV administration
Placebo Comparator: Phase 2 Placebo Comparator Study Arm
Participants randomized to placebo will receive IV normal saline on Day 1 and will then be followed for up to 104 weeks. Primary observation period is 16 weeks.
Other: Normal Saline IV Administration
The administration of IV normal saline
Experimental: Placebo Crossover and Follow-On Dosing Substudy Arm
Participants assigned to this Substudy Arm (participants who previously received either 25mg or placebo only) will have the opportunity to receive a single dose of NTLA-2002 (50mg) and will then be followed for 52 weeks.
Biological: Biological NTLA-2002
CRISPR/Cas9 gene editing system delivered by LNP for IV administration

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Safety and tolerability of NTLA-2002 as determined by adverse events (AEs) and dose limiting toxicities (DLTs)
Time Frame: From NTLA-2002 infusion up to week 104 post-infusion
(Phase 1 only)
From NTLA-2002 infusion up to week 104 post-infusion
Number of HAE attacks per month (Weeks 1-16)
Time Frame: From study drug infusion up to week 16 post-infusion
(Phase 2 only)
From study drug infusion up to week 16 post-infusion

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Change from baseline in total plasma kallikrein protein level
Time Frame: From NTLA-2002 infusion up to week 104 post-infusion
(Phase 1 & 2)
From NTLA-2002 infusion up to week 104 post-infusion
Plasma and urine concentrations for DMG-PEG2k, LP000001, Cas9 mRNA, and sgRNA
Time Frame: From NTLA-2002 infusion up to week 104 post-infusion
(Phase 1 & 2)
From NTLA-2002 infusion up to week 104 post-infusion
Safety and tolerability of NTLA-2002 as determined by AEs
Time Frame: From study drug infusion up to week 104 post-infusion
(Phase 2 only)
From study drug infusion up to week 104 post-infusion
Number of HAE attacks per month (Weeks 5-16)
Time Frame: From week 6 post-infusion up to week 16 post-infusion
(Phase 2 only)
From week 6 post-infusion up to week 16 post-infusion
Number of HAE attacks per month requiring acute therapy (Weeks 1-16, Weeks 5-16)
Time Frame: From study drug infusion up to week 16 post-infusion
(Phase 2 only)
From study drug infusion up to week 16 post-infusion

Collaborators and Investigators

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

Sponsor

Intellia Therapeutics

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

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)

December 10, 2021

Primary Completion (Actual)

April 4, 2024

Study Completion (Estimated)

July 1, 2026

Study Registration Dates

First Submitted

November 3, 2021

First Submitted That Met QC Criteria

November 12, 2021

First Posted (Actual)

November 15, 2021

Study Record Updates

Last Update Posted (Actual)

March 24, 2026

Last Update Submitted That Met QC Criteria

March 23, 2026

Last Verified

March 1, 2026

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • ITL-2002-CL-001

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

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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