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

NCT06262399 | Enrolling By Invitation

• 2 other identifiers: ITL-2002-CL-9992022-003778-22
• Study Type: observational
• Target: 100
• Locations: 6 countries
• Sites: 7

Brief 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.

Conditions

Hereditary Angioedema

Outcome Measures

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

  up to 15 years

Secondary Outcomes (6)

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

  up to 15 years

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

  up to 15 years

• Change from baseline in healthcare utilization for HAE attacks

  up to 15 years

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

  up to 5 years

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

  up to 5 years

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

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

You may qualify if:

• 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.

You may not qualify if:

• None

Sponsors & Collaborators

• Intellia Therapeutics: lead

Study Sites (7)

Campbelltown Hospital

Campbelltown, New South Wales, 2560, Australia

Location

Centre National de Reference - Grenoble

Grenoble, France

Location

Hôpital Claude Huriez

Lille, 59037, France

Location

Charite-Universitätsmedizin Berlin

Berlin, 12203, Germany

Location

University of Amsterdam Academic Medical Center

Amsterdam, Netherlands

Location

New Zealand Clinical Research

Auckland, New Zealand

Location

Cambridge University Hospitals NHS Foundation Trust, Addenbrooke's Hospital

Cambridge, United Kingdom

Location

Biospecimen

Retention: SAMPLES WITHOUT DNA

Serum

MeSH Terms

Conditions

Angioedemas, Hereditary

Condition Hierarchy (Ancestors)

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

Study Design

• Study Type: observational
• Observational Model: CASE ONLY
• Time Perspective: PROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 12, 2024
• First Posted: February 16, 2024
• Study Start: April 12, 2024
• Primary Completion (Estimated): April 1, 2040
• Study Completion (Estimated): April 1, 2040
• Last Updated: November 28, 2025

Record last verified: 2025-04

Data Sharing

• IPD Sharing: Will not share

Locations

AU (1); NL (1); NZ (1); FR (2); DE (1); GB (1)