CClinicalTrials.gg
Enrolling by invitationNCT06262399Updated Nov 28, 2025

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

An observational study in Hereditary Angioedema, sponsored by Intellia Therapeutics. Enrolling by invitation at 7 sites in 6 countries. Per ClinicalTrials.gov, last updated 2025-11-28.

Sponsored by Intellia Therapeutics · Observational

Study type
Observational
Model
Case-only
Time perspective
Prospective
Enrollment
100
Sex
All
01

Study summary

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.

02

Conditions studied

  • Hereditary Angioedema
03

In context

Angioedemas, Hereditary

171 studies on the registry are indexed under Angioedemas, Hereditary; 25 are open to participants now.

This study's planned enrollment of 100 is close to the median of 100 across 43 observational studies indexed under Angioedemas, Hereditary.

Browse Angioedemas, Hereditary studies →

Lead sponsor

Intellia Therapeutics is the lead sponsor of 9 studies on the registry; 4 are open to participants now.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
Child (0–17), Adult (18–64), Older adult (65+)
Sexes eligible
All
Accepts healthy volunteers
No
Sampling method
Non-probability sample

Study population

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

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

Exclusion Criteria:

None

05

Study design

Observational model
Case-only
Time perspective
Prospective
Enrollment
100 participants (estimated)
Patient registry
No
Biospecimen retention
Samples without dna
06

What researchers measure

Primary outcomes

  1. 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

Secondary outcomes

  1. To evaluate the long-term efficacy of NTLA-2002 in previously treated subjects

    Rate of Hereditary Angioedema (HAE) attacks overall and rate of HAE attacks requiring acute therapy. Total plasma kallikrein protein level.

    Time frame: up to 15 years

  2. Change from baseline in consumption of on-demand HAE medications for reported HAE attacks

    Time frame: up to 15 years

  3. Change from baseline in healthcare utilization for HAE attacks

    Time frame: up to 15 years

  4. Change from baseline in QoL parameters as measured by the MOXIE Angioedema-QoL instrument.

    Time frame: up to 5 years

  5. Change from baseline in QoL parameters as measured by the EQ-5D-5L instrument.

    Time frame: up to 5 years

  6. Change from baseline in QoL parameters as measured by the WPAI:GH instrument.

    Time frame: up to 5 years

07

Study locations

7 sites
  • Campbelltown Hospital
    Campbelltown, New South Wales 2560, Australia
  • Centre National de Reference - Grenoble
    Grenoble, France
  • Hôpital Claude Huriez
    Lille, 59037, France
  • Charite-Universitätsmedizin Berlin
    Berlin, 12203, Germany
  • University of Amsterdam Academic Medical Center
    Amsterdam, Netherlands
  • New Zealand Clinical Research
    Auckland, New Zealand
  • Cambridge University Hospitals NHS Foundation Trust, Addenbrooke's Hospital
    Cambridge, United Kingdom
08

References and documents

Individual participant data

Plan to share: No

No publications or documents are linked to this record.

09

Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on Nov 28, 2025, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
10

Registry details

Key details

Study ID
NCT06262399
Lead sponsor
Intellia Therapeutics
Responsible party
Sponsor
First posted
Feb 16, 2024
Start date
Apr 12, 2024
Primary completion
Apr 2040 (estimated)
Completion
Apr 2040 (estimated)
Last update
Nov 28, 2025

Oversight

Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

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No contact was published for this record. The registry link below has the sponsor’s details.

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