ClinicalTrials.gov record NCT05697861

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

Record copied from ClinicalTrials.gov: Oct 10, 2026AI summaries generated: Sep 17, 2026

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

StatusEnrolling by invitationThe study enrols only people the study team invites, for example patients already at a particular clinic. It is not open to the public.
Study typeObservationalA study in which researchers do not assign participants to a treatment or other intervention. They observe or measure what happens, which may include study visits, tests, questionnaires or a review of medical records.
Age18 years old and older
SexAll
LocationFrance, New Zealand, Sweden, United Kingdom
AI summaryWhat this study is about, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

This is a prospective observational long-term follow-up of people who previously received NTLA-2001 in an Intellia-sponsored study. It tracks treatment-related serious adverse events and protocol-specified adverse events of special interest, and measures biomarkers such as serum TTR and prealbumin. Follow-up can continue for up to 15 years.

What it is studying. The study is testing the long-term safety profile and biological effects of prior NTLA-2001 dosing by monitoring treatment-related serious adverse events and specific biomarkers (serum TTR and serum prealbumin) over time.

What the researchers hope to learn. To describe long-term safety (incidence of treatment-related serious adverse events and protocol-specified adverse events of special interest) and to track pharmacodynamic biomarkers for ATTR, including serum transthyretin (TTR) and serum prealbumin, after NTLA-2001 dosing.

What this study is about

The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.

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.

Eligibility

The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.

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

Study description and what participation involves

The registry record has no detailed description beyond the summary above.

AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

This is an observational long‑term follow‑up: participants will have safety monitoring and pharmacodynamic (PD) biomarker measurements (serum TTR and serum prealbumin) with data collected for up to 15 years. The registry lists monitoring for treatment‑related serious adverse events and protocol‑specified adverse events plus the listed biomarkers. The record does not provide details on visit frequency, other specific tests or procedures, or participant services, and it does not list specific invasive procedures.

How the study is structured. A prospective, case-only observational follow-up: people who were previously dosed with NTLA-2001 are followed over time (up to 15 years) with periodic safety assessments and blood biomarker measurements. No new experimental treatments are assigned as part of this study (observational follow-up of prior dosing).

Questions you could ask

These questions are suggestions for a conversation with the study team or your own clinician, not advice.

  • This is an observational study, so the study itself gives no treatment. What would taking part involve for me, and would it change my usual care?
  • The registry says participants are enrolled by invitation. Who decides who is invited, and how?
  • The listed sites are in France, New Zealand, Sweden, United Kingdom. Is any of them reachable for me, and does the study help with travel?
  • Which of the eligibility criteria would need to be checked with tests before I could join?

More on wild-type ATTR amyloidosis

This trial is also listed on this site under ATTRv amyloidosis.

Clinical trials121 trials listed for wild-type ATTR amyloidosisEvery trial the registry lists for wild-type ATTR amyloidosis, with recruitment status, phase and sites, updated from ClinicalTrials.gov.See all trials →Research updatesWhat changed in wild-type ATTR amyloidosis researchLatest monthly update: September 2026. Includes: Frailty and support needs. New research summarized each month in plain language, with links to every source paper.Read the latest update →

Important dates

Study start
Jul 10, 2023
Primary completion
March 2038 (estimated)
Study completion
March 2038 (estimated)
Target enrollment
72 participants (estimated)
Last updated on registry
Aug 3, 2026
Data verified
Oct 10, 2026

Sponsor and research team

The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.

Lead sponsor
Intellia Therapeutics

Participating sites (4)

  • Clinical Trial Site

    Paris · France

  • Clinical Trial Site

    Auckland · New Zealand

  • Clinical Trial Site

    Umeå · Sweden

  • Clinical Trial Site

    London · United Kingdom

Terms used on this page · explanations by MedicalUpdates.ai

follow-up
The period after treatment or enrolment during which participants are observed or contacted, for example to record side effects or outcomes.
clinical trial
A study in which researchers assign participants to one or more interventions, such as a drug, procedure, device, behavioural programme or other approach, to measure their effects.
investigational
A treatment that is being tested and has not been approved for this use. It may be approved for a different condition. Being studied does not mean it works or is available outside the study.
informed consent
The process of explaining a study, including its risks and what participation involves, so that a person can decide freely whether to take part. Consent is usually documented in writing, and a participant can choose to leave the study later.
Inclusion Criteria
The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
Exclusion Criteria
The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.

The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this site; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.

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