RecruitingOBSERVATIONAL

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

This study is a long-term check-up for individuals who previously took part in a clinical trial involving a new medicine called NTLA-2001. This medicine was given to people with specific conditions affecting their nerves and heart, caused by a protein called transthyretin. These conditions include Familial Amyloid Polyneuropathy (nerve problems), Familial Amyloid Cardiomyopathy (heart problems that run in families), and Wild-Type Transthyretin Cardiac Amyloidosis (heart problems not inherited). The main goal is to carefully watch these participants over time to understand the lasting effects of the NTLA-2001 treatment. They are not giving out new treatment in this study, just observing those who already received it.

At a glance

Status

Recruiting

Sponsor

Intellia Therapeutics

Enrolment target

Start

10 Jul 2023

Estimated completion

01 Mar 2038

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

What is this study about?

This study, called a 'Long-Term Follow-Up,' is all about checking in with people who have already been part of a research study for a new medicine called NTLA-2001. This medicine was developed to help individuals with specific conditions that affect their nerves and heart. These conditions are linked to a protein called transthyretin, which can build up in the body and cause problems.

There are a few types of these conditions: one affects the nerves (Familial Amyloid Polyneuropathy), another affects the heart and runs in families (Familial Amyloid Cardiomyopathy), and a third affects the heart but isn't inherited (Wild-Type Transthyretin Cardiac Amyloidosis).

The purpose of this current study is to simply observe and learn what happens to these participants over a longer period. The researchers want to understand how the NTLA-2001 treatment, which participants received in a previous study, continues to affect their health in the long run. They are not giving out new doses of the medicine in this follow-up study; they are just keeping an eye on how everyone is doing.

Key takeaways

This study is a follow-up for people who previously received NTLA-2001.
It helps researchers understand the long-term effects of the treatment.
No new medication is given in this specific study.
Participation involves observation and health checks.
It's for people with certain heart and nerve conditions.

Who may be eligible?

To be part of this follow-up study, you must have already taken part in an earlier study where you received some amount of the NTLA-2001 medicine. It doesn't matter if you finished that previous study or had to stop early.

Also, you need to agree to join this specific follow-up study. Before you decide, the study team will explain everything clearly, and you'll need to give your permission in writing.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

Did you take part in an earlier study where you received NTLA-2001?
Are you willing to give your written permission to join this follow-up study?
Are you 18 years old or older?

Answer every question to see your result.

What does participation involve?

If you join this study, you won't be given any new medication. Instead, it's about observing your health over time. This will likely involve regular check-ups, which might include talking to a study doctor or nurse, having your vital signs checked, and potentially having some blood tests or other examinations. The specific timing and number of visits would be explained in full detail by the study team. The total duration of your participation would also be clear before you agree to join.

Potential risks and benefits

The main benefit of taking part is contributing to a better understanding of how the NTLA-2001 treatment works in the long run, which could help future patients. Since no new treatment is given, the direct risks are generally low, mostly related to the inconvenience of follow-up visits and tests. However, there's always a small risk with any medical procedure, like blood draws. You are free to withdraw from the study at any time without giving a reason.

Locations (4)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

Clinical Trial Site
Verified postcode
Paris, France· Recruiting
Clinical Trial Site
Verified postcode
Auckland, New Zealand· Recruiting
Clinical Trial Site
Verified postcode
Umeå, Sweden· Recruiting
Clinical Trial Site
Verified postcode
London, United Kingdom· Recruiting

Common questions

What is NTLA-2001?

NTLA-2001 is a new medicine that was given to people in earlier research studies for certain heart and nerve conditions.

Am I being given new medicine in this study?

No, you will not receive any new NTLA-2001 medicine in this follow-up study. We are just checking on people who received it previously.

Why is this study happening?

This study wants to learn about the long-term effects of the NTLA-2001 treatment that participants received in older studies.

Do I have to do anything special to join?

You just need to have been part of an earlier NTLA-2001 study and agree to participate in this follow-up part.

What does 'Transthyretin-Related Amyloidosis' mean?

It's a group of conditions where a protein called transthyretin builds up in the body, causing problems mainly in the nerves or heart. This study is for people who had different types of this condition.

How to find out more

Trial Manager at Intellia

medicalinformation@intelliatx.com 1-857-285-6200 Official trial record

Always speak to your GP or specialist before deciding to take part in a study.