Long-term follow-up of patients treated with NTLA-2001

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

Observational Intellia Therapeutics · NCT05697861

This study looks at how NTLA-2001 has affected people with certain types of amyloidosis over the long term after they were treated in a previous trial.

Quick facts

Study typeObservational
Enrollment72 (estimated)
Ages18 Years and up
SexAll
SponsorIntellia Therapeutics Industry-sponsored
Locations4 sites (Paris and 3 other locations)
Trial IDNCT05697861 on ClinicalTrials.gov

What this trial studies

This observational study aims to evaluate the long-term effects of NTLA-2001 in patients who have previously participated in an Intellia-sponsored clinical trial. It focuses on individuals diagnosed with transthyretin-related familial amyloid polyneuropathy, familial amyloid cardiomyopathy, or wild-type transthyretin cardiac amyloidosis. Participants will be monitored to assess the ongoing impact of the investigational therapy over time. The study does not involve new interventions but rather collects data on the outcomes of those previously treated.

Who should consider this trial

Good fit: Ideal candidates are individuals who have completed or discontinued from a previous clinical trial involving NTLA-2001.

Not a fit: Patients who have not received NTLA-2001 in prior studies will not benefit from this follow-up study.

Why it matters

Potential benefit: If successful, this study could provide valuable insights into the long-term safety and efficacy of NTLA-2001 for patients with amyloid-related conditions.

How similar studies have performed: While this study is observational and follows previous trials, similar long-term follow-up studies have shown success in providing critical data on investigational therapies.

Eligibility criteria

Show full inclusion / exclusion 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 this trial is running

Paris and 3 other locations

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
Conditions Transthyretin-Related Familial Amyloid PolyneuropathyTransthyretin-Related Familial Amyloid CardiomyopathyWild-Type Transthyretin Cardiac Amyloidosis
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.