Enrolling by invitation NCT05697861
Long-Term Follow-Up (LTFU) of Subjects Dosed With NTLA-2001
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- This is an observational study: the protocol does not assign a study treatment.
- Who it may be relevant to
- Registry conditions: Transthyretin-Related (ATTR) Familial Amyloid Polyneuropathy, Transthyretin-Related (ATTR) Familial Amyloid Cardiomyopathy, Wild-Type Transthyretin Cardiac Amyloidosis. Basic parameters: from 18 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- France, New Zealand, Sweden, United Kingdom
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Official title
Long-Term Follow-Up of Subjects Treated With NTLA-2001
Overview
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.
Primary outcome measures
- Incidence of Treatment-related SAEs and Protocol-specified AESIs [Time frame: up to 15 years]
Secondary outcome measures (1)
- PD biomarkers for ATTR including serum TTR and serum prealbumin [Time frame: up to 15 years]
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
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Observational model
- Case-only
Study locations
France · 1 center
- Clinical Trial Site — Paris
New Zealand · 1 center
- Clinical Trial Site — Auckland
Sweden · 1 center
- Clinical Trial Site — Umeå
United Kingdom · 1 center
- Clinical Trial Site — London
Identifiers
NCT: NCT05697861 · ITL-2001-CL-999 · 2022-003405-30