Hereditary Angioedema
Conditions
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.
Interventions
None listed
Sponsors
Intellia Therapeutics
Study design
Observational model
CASE_ONLY
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| 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
| Measure | Time frame | Description |
|---|---|---|
| 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 | — |
| 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 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 | — |
| Change from baseline in QoL parameters as measured by the MOXIE Angioedema-QoL instrument. | up to 5 years | — |
Countries
Australia, France, Germany, Netherlands, New Zealand, United Kingdom
Outcome results
None listed