Long-Term Follow-Up (LTFU) of Subjects Dosed With NTLA-2001 | NCT05697861

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

NCT05697861

Sponsor

Intellia Therapeutics

Status

Recruiting

Last Update Posted

Aug 26, 2025Actual

Enrollment

72Estimated

Phase

Not provided

Conditions

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

Interventions

Not provided

Countries

France
New Zealand
Sweden
United Kingdom

Protocol Section

Identification Module Status Module Sponsor/Collaborators Module Oversight Module Description Module Conditions Module Design Module Arms/Interventions Module Outcomes Module Eligibility Module Contacts/Locations Module References Module IPD Sharing Statement Module

Results Section

Participant Flow Module Baseline Characteristics Module Outcome Measures Module Adverse Events Module More Info Module

Annotation Section

Annotation Module

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module Condition Browse Module Intervention Browse Module

Protocol Section

Identification Module

NCT ID

Results Section

No data available

No data is available for this block.

Annotation Section

No data available

No data is available for this block.

Document Section

No data available

No data is available for this block.

Derived Section

Miscellaneous Info Module

Version Holder

ID	Type	Description	Link
2022-003405-30	EudraCT Number

Status Module

Record Verification Date

May 2025

Overall Recruitment Status or Expanded Access Status

Recruiting

Last Known Status

Not provided

Delayed Posting

Not provided

Why Stopped

Not provided

Expanded Access Info

Start Date

Jul 10, 2023Actual

Primary Completion Date

Mar 2038Estimated

Completion Date

Mar 2038Estimated

First Submitted Date

Jan 13, 2023

First Submission Date that Met QC Criteria

Jan 24, 2023

First Posted Date

Jan 26, 2023Actual

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

Aug 20, 2025

Last Update Posted Date

Aug 26, 2025Actual

Oversight Module

Has Data Monitoring Committee (DMC)

Is FDA Regulated Drug

Not provided

Is FDA Regulated Device

Not provided

Is Unapproved Device

Not provided

Pediatric Postmarket Surveillance of a Device Product

Not provided

Product Exported from US

FDAAA801 Violation

Not provided

Description Module

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.

Detailed Description

Not provided

Design Module

Study Type

Observational

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Target Follow-Up Duration

Not provided

Phases

Not provided

Interventional Study Design

Allocation

Not provided

Intervention Model

Biospecimen

Retention

Enrollment

72Estimated

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
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

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

Other Outcomes

Not provided

Eligibility Module

Eligibility Criteria

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

Accepts Healthy Volunteers

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

18 Years

Maximum Age

Not provided

Standard Ages

AdultOlder Adult

Study Population

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

Sampling Method

Non-Probability Sample

Contacts/Locations Module

Central Contacts

Name	Role	Phone	Extension	Email
Trial Manager at Intellia	Contact	1-857-285-6200	Ext. 6	medicalinformation@intelliatx.com

Overall Officials

Not provided

Locations

Facility	Status	City	State	ZIP	Country	Contacts
Clinical Trial Site	Recruiting	Paris			France
Clinical Trial Site

IPD Sharing Statement Module

Plan to Share IPD

Description

Not provided

Types

Not provided

Time Frame

Not provided

Access Criteria

Not provided

URL

Not provided

Condition Browse Module

MeSH Terms

ID	Term
D028227	Amyloid Neuropathies, Familial

Ancestor Terms

ID	Term
D020271	Heredodegenerative Disorders, Nervous System
D019636	Neurodegenerative Diseases
D009422	Nervous System Diseases
D017772	Amyloid Neuropathies

Browse Leaves

Not provided

Browse Branches

Not provided