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

Phase 1

Recruiting

• Conditions: -Hereditary Transthyretin Amyloidosis with Polyneuropathy (ATTRv-PN)-Transthyretin Amyloidosis-Related Cardiomyopathy (ATTR-CM); MedDRA version: 20.0Level: PTClassification code: 10019889Term: Hereditary neuropathic amyloidosis Class: 100000004850; MedDRA version: 20.0Level: PTClassification code: 10007509Term: Cardiac amyloidosis Class: 100000004849; Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

• Registration Number: CTIS2023-507385-11-00
• Lead Sponsor: Intellia Therapeutics Inc.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2024-04-08
• Last Update Posted: 21 August 2024

Recruitment & Eligibility

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

Inclusion Criteria

1. A participant has completed or discontinued from an Intellia-sponsored clinical study in which a complete or partial dose of NTLA-2001 was received., 2. A participant has provided informed consent for the LTFU study., 3. A participant is willing to attend study visits, complete protocol-required follow-up schedule, and comply with the study requirements.

Exclusion Criteria

None.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: To evaluate the long-term safety of NTLA-2001 in previously treated participants;Secondary Objective: To evaluate long-term pharmacodynamic effects of NTLA-2001 in previously treated participants;Primary end point(s): For ATTR-CM and ATTRv-PN participants, incidence of: • Treatment-related SAEs • Protocol-specified CEIs

Secondary Outcome Measures

Name	Time	Method
Secondary end point(s):PD biomarkers for ATTR over time: • Serum TTR • Serum prealbumin