Trial Review

Did you know?

The ANZCTR now automatically displays published trial results and simplifies the addition of trial documents such as unpublished protocols and statistical analysis plans.

These enhancements will offer a more comprehensive view of trials, regardless of whether their results are positive, negative, or inconclusive.

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been endorsed by the ANZCTR. Before participating in a study, talk to your health care provider and refer to this information for consumers
Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT05697861




Registration number
NCT05697861
Ethics application status
Date submitted
13/01/2023
Date registered
26/01/2023
Date last updated
26/03/2024

Titles & IDs
Public title
Long-Term Follow-Up (LTFU) of Subjects Dosed With NTLA-2001
Scientific title
Long-Term Follow-Up of Subjects Treated With NTLA-2001
Secondary ID [1] 0 0
2022-003405-30
Secondary ID [2] 0 0
ITL-2001-CL-999
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Transthyretin-Related (ATTR) Familial Amyloid Polyneuropathy 0 0
Transthyretin-Related (ATTR) Familial Amyloid Cardiomyopathy 0 0
Wild-Type Transthyretin Cardiac Amyloidosis 0 0
Condition category
Condition code
Neurological 0 0 0 0
Other neurological disorders
Neurological 0 0 0 0
Neurodegenerative diseases
Metabolic and Endocrine 0 0 0 0
Metabolic disorders
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Metabolic and Endocrine 0 0 0 0
Other metabolic 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 SAEs and Protocol-specified AESIs
Timepoint [1] 0 0
up to 15 years
Secondary outcome [1] 0 0
PD biomarkers for ATTR including serum TTR and serum prealbumin
Timepoint [1] 0 0
up to 15 years

Eligibility
Key inclusion criteria
1. A subject has completed or discontinued from an Intellia-sponsored clinical study in which a complete or partial dose of NTLA-2001 was received.
2. A subject has provided informed consent for the LTFU study.
Minimum age
18 Years
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
10/07/2023
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/03/2038
Actual
Sample size
Target
72
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
Recruitment outside Australia
Country [1] 0 0
New Zealand
State/province [1] 0 0
Auckland
Country [2] 0 0
Sweden
State/province [2] 0 0
Umea
Country [3] 0 0
United Kingdom
State/province [3] 0 0
London

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-2001 in a previous clinical trial as an observational evaluation of the long-term effects of the investigational therapy.
Trial website
https://clinicaltrials.gov/study/NCT05697861
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
Trial Manager at Intellia
Address 0 0
Country 0 0
Phone 0 0
1-833-888-0387
Fax 0 0
Email 0 0
[email protected]
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/study/NCT05697861