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

N/AEnrolling By InvitationNCT06262399

Intellia Therapeutics100 enrolled

Overview

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 Type

OBSERVATIONAL

Enrollment

100

Conditions

Hereditary Angioedema

Eligibility

Sex: ALL

Medical Language ↔ Plain English

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

Locations (7)

Campbelltown Hospital

Campbelltown, New South Wales, Australia

Centre National de Reference - Grenoble

Grenoble, France

Hôpital Claude Huriez

Lille, France

Charite-Universitätsmedizin Berlin

Berlin, Germany

Outcomes

Primary Outcomes

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

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

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

Time frame: up to 15 years

Change from baseline in healthcare utilization for HAE attacks

Time frame: up to 15 years

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

Time frame: up to 5 years

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

Time frame: up to 5 years

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

Time frame: up to 5 years

Data from ClinicalTrials.gov

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