NCT05697861

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.

Trial Health

80
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
72

participants targeted

Target at P50-P75 for all trials

Timeline
144mo left

Started Jul 2023

Longer than P75 for all trials

Geographic Reach
4 countries

4 active sites

Status
recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress19%
Jul 2023Mar 2038

First Submitted

Initial submission to the registry

January 13, 2023

Completed
13 days until next milestone

First Posted

Study publicly available on registry

January 26, 2023

Completed
6 months until next milestone

Study Start

First participant enrolled

July 10, 2023

Completed
14.7 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

March 1, 2038

Expected
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

March 1, 2038

Last Updated

August 26, 2025

Status Verified

May 1, 2025

Enrollment Period

14.7 years

First QC Date

January 13, 2023

Last Update Submit

August 20, 2025

Conditions

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

Outcome Measures

Primary Outcomes (1)

  • Incidence of Treatment-related SAEs and Protocol-specified AESIs

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

    up to 15 years

Secondary Outcomes (1)

  • PD biomarkers for ATTR including serum TTR and serum prealbumin

    up to 15 years

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)
Sampling MethodNon-Probability Sample
Study Population

Participating sites in an Intellia-sponsored clinical study of NTLA-2001.

You may qualify if:

  • 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.

You may not qualify if:

  • None

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (4)

Clinical Trial Site

Paris, France

RECRUITING

Clinical Trial Site

Auckland, New Zealand

RECRUITING

Clinical Trial Site

Umeå, Sweden

RECRUITING

Clinical Trial Site

London, United Kingdom

RECRUITING

Biospecimen

Retention: SAMPLES WITHOUT DNA

serum

MeSH Terms

Conditions

Amyloid Neuropathies, Familial

Condition Hierarchy (Ancestors)

Heredodegenerative Disorders, Nervous SystemNeurodegenerative DiseasesNervous System DiseasesAmyloid NeuropathiesPeripheral Nervous System DiseasesNeuromuscular DiseasesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesAmyloidosis, FamilialMetabolism, Inborn ErrorsMetabolic DiseasesNutritional and Metabolic DiseasesAmyloidosisProteostasis Deficiencies

Central Study Contacts

Trial Manager at Intellia

CONTACT

1-857-285-6200medicalinformation@intelliatx.com

Study Design

Study Type
observational
Observational Model
CASE ONLY
Time Perspective
PROSPECTIVE
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

January 13, 2023

First Posted

January 26, 2023

Study Start

July 10, 2023

Primary Completion (Estimated)

March 1, 2038

Study Completion (Estimated)

March 1, 2038

Last Updated

August 26, 2025

Record last verified: 2025-05

Data Sharing

IPD Sharing
Will not share

Locations

GB(1)FR(1)NZ(1)SE(1)