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

Recruiting

• Conditions: Transthyretin-Related (ATTR) Familial Amyloid Cardiomyopathy; Transthyretin-Related (ATTR) Familial Amyloid Polyneuropathy; Wild-Type Transthyretin Cardiac Amyloidosis

• Registration Number: NCT05697861
• Lead Sponsor: Intellia Therapeutics

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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2023-01-13
• Study First Submitted QC: 2023-01-24
• Study First Posted: 2023-01-26
• Study Start: 2023-07-10
• Status Verified: 2024-03
• Last Update Submitted: 2024-03-25
• Last Update Posted: 2024-03-26
• Primary Completion: 2038-03
• Study Completion: 2038-03

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 72

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.

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Exclusion Criteria

None

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Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Incidence of Treatment-related SAEs and Protocol-specified AESIs	up to 15 years	Long-term safety assessment of any treatment-related SAEs and protocol-specified AESIs

Secondary Outcome Measures

Name	Time	Method
PD biomarkers for ATTR including serum TTR and serum prealbumin	up to 15 years	Long-term assessment of serum TTR and serum prealbumin

Trial Locations

Locations (1)

Clinical Trial Site; 🇬🇧 London, United Kingdom