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

• Conditions: Hereditary Angioedema

• Registration Number: NCT06262399
• Lead Sponsor: Intellia Therapeutics

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.

Detailed Description

Not available

Study Timeline

• Status Verified: 2024-01
• Study First Submitted: 2024-01-12
• Study First Submitted QC: 2024-02-07
• Study First Posted: 2024-02-16
• Study Start: 2024-04-12
• Last Update Submitted: 2024-10-10
• Last Update Posted: 2024-10-14
• Primary Completion: 2040-04
• Study Completion: 2040-04

Recruitment & Eligibility

• Status: ENROLLING_BY_INVITATION
• Sex: All
• Target Recruitment: 100

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.

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Exclusion Criteria

None

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Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
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 Outcome Measures

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

Trial Locations

Locations (5)

Hôpital Claude Huriez; 🇫🇷 Lille, France

Centre National de Reference - Grenoble; 🇫🇷 Grenoble, France

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