Long-Term Follow-Up (LTFU) of Subjects Dosed With NTLA-2001

Transthyretin-Related (ATTR) Familial Amyloid Polyneuropathy Clinical Trial

Official title:

Long-Term Follow-Up of Subjects Treated With NTLA-2001

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05697861
• Other study ID #: ITL-2001-CL-999
• Secondary ID: 2022-003405-30
• Status: Recruiting
• 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
• 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

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