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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT06262399




Registration number
NCT06262399
Ethics application status
Date submitted
12/01/2024
Date registered
16/02/2024
Date last updated
15/10/2024

Titles & IDs
Public title
Long-Term Follow-Up (LTFU) of Subjects Treated with NTLA 2002
Scientific title
Long-Term Follow-Up of Subjects Treated with NTLA 2002
Secondary ID [1] 0 0
2022-003778-22
Secondary ID [2] 0 0
ITL-2002-CL-999
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Hereditary Angioedema 0 0
Condition category
Condition code
Cardiovascular 0 0 0 0
Diseases of the vasculature and circulation including the lymphatic system
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Blood 0 0 0 0
Other blood disorders

Intervention/exposure
Study type
Observational
Patient registry
Target follow-up duration
Target follow-up type
Description of intervention(s) / exposure
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
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
Timepoint [1] 0 0
up to 15 years
Secondary outcome [1] 0 0
To evaluate the long-term efficacy of NTLA-2002 in previously treated subjects
Timepoint [1] 0 0
up to 15 years
Secondary outcome [2] 0 0
Change from baseline in consumption of on-demand HAE medications for reported HAE attacks
Timepoint [2] 0 0
up to 15 years
Secondary outcome [3] 0 0
Change from baseline in healthcare utilization for HAE attacks
Timepoint [3] 0 0
up to 15 years
Secondary outcome [4] 0 0
Change from baseline in QoL parameters as measured by the MOXIE Angioedema-QoL instrument.
Timepoint [4] 0 0
up to 5 years
Secondary outcome [5] 0 0
Change from baseline in QoL parameters as measured by the EQ-5D-5L instrument.
Timepoint [5] 0 0
up to 5 years
Secondary outcome [6] 0 0
Change from baseline in QoL parameters as measured by the WPAI:GH instrument.
Timepoint [6] 0 0
up to 5 years

Eligibility
Key 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.
Minimum age
No limit
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
None

Study design
Purpose
Duration
Selection
Timing
Prospective
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
12/04/2024
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/04/2040
Actual
Sample size
Target
100
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
Recruitment outside Australia
Country [1] 0 0
France
State/province [1] 0 0
Grenoble
Country [2] 0 0
France
State/province [2] 0 0
Lille
Country [3] 0 0
Netherlands
State/province [3] 0 0
Amsterdam
Country [4] 0 0
New Zealand
State/province [4] 0 0
Auckland
Country [5] 0 0
United Kingdom
State/province [5] 0 0
Cambridge

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Intellia Therapeutics
Address
Country

Ethics approval
Ethics application status

Summary
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.
Trial website
https://clinicaltrials.gov/study/NCT06262399
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/study/NCT06262399