Transthyretin-Related (ATTR) Familial Amyloid PolyneuropathyNCT05697861
Long-Term Follow-Up (LTFU) of Subjects Dosed With NTLA-2001
An observational study of Transthyretin-Related (ATTR) Familial Amyloid Polyneuropathy and Transthyretin-Related (ATTR) Familial Amyloid Cardiomyopathy, run by Intellia Therapeutics at 4 sites in 4 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Transthyretin-Related (ATTR) Familial Amyloid Polyneuropathy, Transthyretin-Related (ATTR) Familial Amyloid Cardiomyopathy, Wild-Type Transthyretin Cardiac Amyloidosis
- How many people
- 72 participants
- Where
- 4 sitesin 4 countries
- Study dates
- 10 Jul 2023to Mar 2038
- Phase
- Observational
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: Participating sites in an Intellia-sponsored clinical study of NTLA-2001.
Who may be able to join
- 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.
Who cannot join
- None
Trial locations and contacts
4 sites in 4 countries. Contact the study team or a site near you to ask about taking part.
4 sites in 4 countries
About this study
Official title: Long-Term Follow-Up of Subjects Treated With NTLA-2001
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.
- Conditions
- Transthyretin-Related (ATTR) Familial Amyloid Polyneuropathy, Transthyretin-Related (ATTR) Familial Amyloid Cardiomyopathy, Wild-Type Transthyretin Cardiac Amyloidosis
- Medical subject headings
- Amyloid Neuropathies, Familial
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of Treatment-related SAEs and Protocol-specified AESIs
Long-term safety assessment of any treatment-related SAEs and protocol-specified AESIs
Measured: up to 15 years
Secondary outcomes (1)
PD biomarkers for ATTR including serum TTR and serum prealbumin
Long-term assessment of serum TTR and serum prealbumin
Measured: up to 15 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 Jan 2023
- Study start10 Jul 2023
- Primary completionMar 2038 (estimated)
- Study completionMar 2038 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Transthyretin-Related (ATTR) Familial Amyloid PolyneuropathyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Case only
- Time perspective
- Prospective
- Enrolment
- 72 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Intellia Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05697861
- Sponsor's protocol ID
- ITL-2001-CL-999
- Other IDs
- 2022-003405-30
- Status last verified
- Jul 2026
- First submitted
- 13 Jan 2023
- Last updated
- 3 Aug 2026
- Results posted
- No
- Data monitoring committee
- No
- Individual data sharing
- No