Other Clinical Trial - Long-Term Follow-Up (LTFU) of NCT05697861

Updated 06/21/2024

Clinical Trial Details — Status: Recruiting

Administrative data
NCT number	NCT05697861
Other study ID #	ITL-2001-CL-999
Secondary ID	2022-003405-30
Status	Recruiting
Phase
First received
Last updated
Start date	July 10, 2023
Est. completion date	March 2038

Study information
Verified date	March 2024
Source	Intellia Therapeutics
Contact	Trial Manager at Intellia
Phone	1-833-888-0387
Email	clinicalscience[@]intelliatx.com
Is FDA regulated	No
Health authority
Study type	Observational

Clinical Trial 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.

Recruitment information / eligibility
Status	Recruiting
Enrollment	72
Est. completion date	March 2038
Est. primary completion date	March 2038
Accepts healthy volunteers	No
Gender	All
Age group	18 Years and older
Eligibility	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. Exclusion Criteria: None

Study Design

Related Conditions & MeSH terms

Amyloid Neuropathies
Amyloid Neuropathies, Familial
Amyloidosis
Cardiomyopathies
Polyneuropathies
Transthyretin-Related (ATTR) Familial Amyloid Cardiomyopathy
Transthyretin-Related (ATTR) Familial Amyloid Polyneuropathy

Locations
Country	Name	City	State
New Zealand	Clinical Trial Site	Auckland
Sweden	Clinical Trial Site	Umea
United Kingdom	Clinical Trial Site	London

Sponsors *(1)*
Lead Sponsor	Collaborator
Intellia Therapeutics

Countries where clinical trial is conducted

New Zealand, Sweden, United Kingdom,

Outcome
Type	Measure	Description	Time frame	Safety issue
Primary	Incidence of Treatment-related SAEs and Protocol-specified AESIs	Long-term safety assessment of any treatment-related SAEs and protocol-specified AESIs	up to 15 years
Secondary	PD biomarkers for ATTR including serum TTR and serum prealbumin	Long-term assessment of serum TTR and serum prealbumin	up to 15 years

See also
	Status	Clinical Trial	Phase
	Withdrawn	`NCT04882735` - Efficacy and Safety of Acoramidis (AG10) in Subjects With Transthyretin Amyloid Polyneurophathy (ATTRibute-PN)	Phase 3
	Withdrawn	`NCT04418024` - Efficacy and Safety of AG10 in Subjects With Transthyretin Amyloid Polyneurophathy	Phase 3
	Active, not recruiting	`NCT04601051` - Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of NTLA-2001 in Patients With Hereditary Transthyretin Amyloidosis With Polyneuropathy (ATTRv-PN) and Patients With Transthyretin Amyloidosis-Related Cardiomyopathy (ATTR-CM)	Phase 1
	Recruiting	`NCT03237494` - TRAMmoniTTR Study Genetic Screening of an At-risk Population for hATTR and Monitoring of TTR Positive Subjects
	Withdrawn	`NCT02713880` - Biomarker for Transthyretin-Related Familial Amyloidotic Polyneuropathy (BioTRAP)