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

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.

Recruiting

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Key information

Conditions

Transthyretin-Related (ATTR) Familial Amyloid Polyneuropathy Amyloid Neuropathies Amyloid Neuropathies, Familial Amyloidosis Amyloidosis, Familial Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Heredodegenerative Disorders, Nervous System Metabolic Diseases Metabolism, Inborn Errors Nervous System Diseases Neurodegenerative Diseases Neuromuscular Diseases Nutritional and Metabolic Diseases Peripheral Nervous System Diseases Proteostasis Deficiencies Transthyretin-Related (ATTR) Familial Amyloid Cardiomyopathy Wild-Type Transthyretin Cardiac Amyloidosis

Age range

18 year and older

Sex eligibility

All sexes

Study type

Observational

Primary location

Clinical Trial Site, Paris, France

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

A subject has completed or discontinued from an Intellia-sponsored clinical study in which a complete or partial dose of NTLA-2001 was received.
A subject has provided informed consent for the LTFU study.

Exclusion criteria

None

Treatment and study plan

Primary outcomes

Incidence of Treatment-related SAEs and Protocol-specified AESIs

Time frame: up to 15 years

Long-term safety assessment of any treatment-related SAEs and protocol-specified AESIs

Secondary outcomes

PD biomarkers for ATTR including serum TTR and serum prealbumin

Time frame: up to 15 years

Long-term assessment of serum TTR and serum prealbumin

Study contacts

Contact information is provided by the study sponsor or research team.

Trial Manager at Intellia

CONTACT

[email protected]

1-857-285-6200 ext. Ext. 6

Sponsors and collaborators

Lead sponsor

Intellia Therapeutics

Industry

Registry information

Official study title

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

Important dates

Study start: 2023
Primary completion: 2038
Study completion: 2038
First posted: Jan 26, 2023
Registry last updated: Aug 26, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Published trials that share one or more normalized conditions with this study.

Completed