Long-Term Follow-Up (LTFU) of Subjects Treated With NTLA 2002

November 25, 2025 updated by: Intellia Therapeutics

Long-Term Follow-Up of Subjects Treated With NTLA 2002

This is a follow-up study of subjects who received NTLA-2002 in a previous clinical trial as an observational evaluation of the long-term effects of the investigational therapy.

Study Overview

Status

Enrolling by invitation

Conditions

Study Type

Observational

Enrollment (Estimated)

100

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
    • New South Wales
      • Campbelltown, New South Wales, Australia, 2560
        • Campbelltown Hospital
  • France
      • Grenoble, France
        • Centre National de Reference - Grenoble
      • Lille, France, 59037
        • Hôpital Claude Huriez
  • Germany
      • Berlin, Germany, 12203
        • Charite-Universitatsmedizin Berlin
  • Netherlands
      • Amsterdam, Netherlands
        • University of Amsterdam Academic Medical Center
  • New Zealand
      • Auckland, New Zealand
        • New Zealand Clinical Research
  • United Kingdom
      • Cambridge, United Kingdom
        • Cambridge University Hospitals NHS Foundation Trust, Addenbrooke's Hospital

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

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Sampling Method

Non-Probability Sample

Study Population

Participating sites in an Intellia-sponsored clinical study of NTLA-2002.

Description

Inclusion Criteria:

  1. A subject has completed or discontinued from an Intellia-sponsored or -supported treatment protocol in which a complete or partial dose of NTLA-2002 was received.
  2. A subject has provided informed consent for the LTFU study.
  3. A subject is willing to attend study visits, complete protocol-required follow-up schedule, and comply with the study requirements.

Exclusion Criteria:

None

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

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Incidence of treatment-related Adverse Events (AEs); incidence of treatment-related Serious Adverse Events (SAEs); incidence of treatment-related Adverse Events of Special Interest (AESIs) defined per protocol
Time Frame: up to 15 years
up to 15 years

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
To evaluate the long-term efficacy of NTLA-2002 in previously treated subjects
Time Frame: up to 15 years
Rate of Hereditary Angioedema (HAE) attacks overall and rate of HAE attacks requiring acute therapy. Total plasma kallikrein protein level.
up to 15 years
Change from baseline in consumption of on-demand HAE medications for reported HAE attacks
Time Frame: up to 15 years
up to 15 years
Change from baseline in healthcare utilization for HAE attacks
Time Frame: up to 15 years
up to 15 years
Change from baseline in QoL parameters as measured by the MOXIE Angioedema-QoL instrument.
Time Frame: up to 5 years
up to 5 years
Change from baseline in QoL parameters as measured by the EQ-5D-5L instrument.
Time Frame: up to 5 years
up to 5 years
Change from baseline in QoL parameters as measured by the WPAI:GH instrument.
Time Frame: up to 5 years
up to 5 years

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)

April 12, 2024

Primary Completion (Estimated)

April 1, 2040

Study Completion (Estimated)

April 1, 2040

Study Registration Dates

First Submitted

January 12, 2024

First Submitted That Met QC Criteria

February 7, 2024

First Posted (Actual)

February 16, 2024

Study Record Updates

Last Update Posted (Actual)

November 28, 2025

Last Update Submitted That Met QC Criteria

November 25, 2025

Last Verified

April 1, 2025

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • ITL-2002-CL-999
  • 2022-003778-22 (EudraCT Number)

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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