Enrolling by invitation NCT06262399
Long-Term Follow-Up (LTFU) of Subjects Treated With NTLA 2002
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- This is an observational study: the protocol does not assign a study treatment.
- Who it may be relevant to
- Registry conditions: Hereditary Angioedema. Basic parameters: No limits · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- Australia, France, Germany, Netherlands, New Zealand +1
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Official title
Long-Term Follow-Up of Subjects Treated With NTLA 2002
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.
Primary outcome measures
- 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 outcome measures (6)
- To evaluate the long-term efficacy of NTLA-2002 in previously treated subjects [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]
Eligibility criteria
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
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Observational model
- Case-only
Study locations
France · 2 centers
- Centre National de Reference - Grenoble — Grenoble
- Hôpital Claude Huriez — Lille
Australia · 1 center
- Campbelltown Hospital — Campbelltown
Germany · 1 center
- Charite-Universitätsmedizin Berlin — Berlin
Netherlands · 1 center
- University of Amsterdam Academic Medical Center — Amsterdam
New Zealand · 1 center
- New Zealand Clinical Research — Auckland
United Kingdom · 1 center
- Cambridge University Hospitals NHS Foundation Trust, Addenbrooke's Hospital — Cambridge
Identifiers
NCT: NCT06262399 · ITL-2002-CL-999 · 2022-003778-22