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Enrolling by Invitation

NCT Number: NCT06262399

Long-Term Follow-Up (LTFU) 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.

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Key information

Conditions

Hereditary Angioedema Angioedema Angioedemas, Hereditary Cardiovascular Diseases Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Hereditary Complement Deficiency Diseases Hypersensitivity Hypersensitivity, Immediate Immune System Diseases Immunologic Deficiency Syndromes Primary Immunodeficiency Diseases Skin Diseases Skin Diseases, Vascular Skin and Connective Tissue Diseases Urticaria Vascular Diseases

Sex eligibility

All sexes

Study type

Observational

Primary location

Campbelltown Hospital, Campbelltown, New South Wales, Australia

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • 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.
  • A subject has provided informed consent for the LTFU study.
  • A subject is willing to attend study visits, complete protocol-required follow-up schedule, and comply with the study requirements.

Exclusion criteria

None

Treatment and study plan

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

    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.

  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

Sponsors and collaborators

Lead sponsor

Intellia Therapeutics

Industry

Registry information

Official study title

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

Important dates

Study start
2024
Primary completion
2040
Study completion
2040
First posted
Feb 16, 2024
Registry last updated
Nov 28, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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