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

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

Status

Enrolling by invitation

Phases

Unknown

Study type

Observational

Source

ClinicalTrials.gov

Registry ID

NCT05697861

Enrollment

Registered

2023-01-26

Start date

2023-07-10

Completion date

2038-03-01

Last updated

2026-08-03

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Transthyretin-Related (ATTR) Familial Amyloid Cardiomyopathy, Transthyretin-Related (ATTR) Familial Amyloid Polyneuropathy, Wild-Type Transthyretin Cardiac Amyloidosis

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.

Interventions

None listed

Study design

Observational model

CASE_ONLY

Time perspective

PROSPECTIVE

Eligibility

Sex/Gender

ALL

Age

18 Years to No maximum

Healthy volunteers

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

Design outcomes

Primary

Measure	Time frame	Description
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

Measure	Time frame	Description
PD biomarkers for ATTR including serum TTR and serum prealbumin	up to 15 years	Long-term assessment of serum TTR and serum prealbumin

Countries

France, New Zealand, Sweden, United Kingdom

Outcome results