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

View on ClinicalTrials.gov ↗

At a glance

What the study gets you: Health checks and monitoring — no treatment given
Type of study: Observational (no treatment given)
Time in hospital: In-person visits at study sites — visit count not specified by the sponsor
Drug or intervention: Not specified by the sponsor
How long the study runs: Study runs about 176 months (dates as stated)
About the drug or intervention: Not specified by the sponsor
Patient visit burden: Not specified by the sponsor

Type of study: Observing health over time
Ages: 18 Years and over
Who: All
Number of participants: 72
Started: 2023-07-10
Last checked: 2026-07

Plain English Summary

What is this study?

• Testing a new treatment for transthyretin-related (attr) familial amyloid polyneuropathy
• Clinical study - 72 participants
• 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

Who can take part?

• Ages 18 Years and over
• Diagnosed with transthyretin-related (attr) familial amyloid polyneuropathy

Where?

• London - Clinical Trial Site

This is a simplified summary. Always discuss with your doctor before making any decisions.

About This Trial

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.

Transthyretin-Related (ATTR) Familial Amyloid PolyneuropathyTransthyretin-Related (ATTR) Familial Amyloid CardiomyopathyWild-Type Transthyretin Cardiac Amyloidosis

How this trial compares with your answers

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What we know so far

Condition· Matched your search

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Still need:

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Preliminary match based on your answers. Full eligibility requires on-site assessment including medical history, physical exam, and lab tests. This does not guarantee enrolment.

Eligibility at a Glance

Key info

Age: 18 Years and over
Who can join: All genders

What the study is looking for

✓A subject has completed or discontinued from an Intellia-sponsored clinical study in which a complete or partial...
✓A subject has provided agreement to take part for the LTFU study.

See the full criteria

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

Where Is This Study? (1 UK site)

Clinical Trial Site

London, United Kingdom

Data sourced from ClinicalTrials.gov · Last verified: 2026-07