Active not recruitingOBSERVATIONAL

Myocardial Effects in Patients With ATTRv With Polyneuropathy Treated With Patisiran or Vutrisiran

This research is for people in the UK living with a complex, inherited condition called ATTRv amyloidosis, which affects both their nerves and heart. While medicines like Patisiran and Vutrisiran are known to help nerve damage, doctors want to learn more about their effects on the heart. This study will carefully watch how the hearts of patients taking these medicines change over two years. They will use routine check-ups like heart scans and walking tests to gather information. The main goal is to get a clearer picture of how these treatments impact the heart's function and structure in people with ATTRv amyloidosis who also have nerve issues.

At a glance

Status

Active not recruiting

Sponsor

Rennes University Hospital

Enrolment target

Start

12 Apr 2024

Estimated completion

01 Dec 2027

Transthyretin Amyloidosis Amyloidosis, Hereditary

What is this study about?

This study is about a condition called ATTRv amyloidosis. It's a genetic disease where abnormal proteins build up in different parts of the body, causing problems. In many people with ATTRv amyloidosis, this build-up affects the nerves, leading to symptoms like numbness or weakness (this is called polyneuropathy). It can also affect the heart, making it harder for the heart to pump blood around the body.

Two medicines, Patisiran and Vutrisiran, have shown promising results in helping to slow down or stop the nerve damage caused by ATTRv amyloidosis. However, doctors don't fully understand how these medicines affect the heart in people who have both nerve and heart problems due to their ATTRv amyloidosis. This study aims to fill that gap in our knowledge.

The main goal is to carefully observe and measure how the hearts of people taking Patisiran or Vutrisiran change over a two-year period. By doing this, the researchers hope to get a better understanding of whether these treatments can protect the heart or improve its function and structure in people with ATTRv amyloidosis.

Key takeaways

This study focuses on how Patisiran or Vutrisiran affect the heart in ATTRv amyloidosis patients.
It uses information from your regular medical check-ups over two years.
No new medicines or extra procedures are involved beyond routine care.
The goal is to improve understanding of these treatments for future patients.
You must already be on Patisiran or Vutrisiran and have certain types of nerve and heart involvement.
Your participation helps doctors learn more about this complex condition.

Who may be eligible?

To join this study, you need to be an adult (18 years or older) and have a specific type of inherited amyloidosis called ATTRv. You must also have some nerve problems (polyneuropathy) that are either mild (stage 1) or moderate (stage 2). Another important point is that you should not have been treated for ATTRv amyloidosis before starting Patisiran or Vutrisiran.

You also need to be already taking Patisiran or Vutrisiran, prescribed by a hospital neurologist, and planning to continue this treatment for at least two years. Your heart condition, if you have one, should be classified as NYHA stage 1 or 2, which means your symptoms are generally mild to moderate with everyday activities.

You cannot participate if you are currently taking another medicine called Tafamidis alongside Patisiran or Vutrisiran. Also, if you have conditions that prevent you from having certain scans, like severe claustrophobia for an MRI or metal implants that aren't safe near MRI scanners, you won't be able to join. Women who could become pregnant would also need to discuss this with the study team.

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.

Are you 18 years old or older?
Do you have inherited ATTRv amyloidosis with nerve problems (polyneuropathy stage 1 or 2)?
Are you currently taking Patisiran or Vutrisiran for your ATTRv amyloidosis, prescribed by a neurologist?
Have you not taken other ATTRv treatments (like Tafamidis) at the same time as Patisiran or Vutrisiran?
Do you generally have mild to moderate heart symptoms if you have heart disease (NYHA stage 1 or 2)?
Are you able to have heart scans like MRI without any concerns (e.g., no severe claustrophobia or incompatible metal implants)?

Answer every question to see your result.

What does participation involve?

If you join this study, you won't be given new medicine; instead, the researchers will collect information from your routine hospital appointments over two years while you are on Patisiran or Vutrisiran. These appointments will include standard examinations your doctor would normally perform, such as physical checks, blood tests, and heart tests like an ECG (a recording of your heart's electrical activity), echocardiograms (ultrasound scans of your heart), cardiac MRI scans (detailed images of your heart), and special heart scans called scintigraphy.

In addition to these routine tests, specifically for the study, you'll be asked to complete some questionnaires about your quality of life and any nerve control issues you might have. You'll also do a walking test to see how far you can walk in six minutes. Data will be collected when you start treatment, and then again after one year and two years. The total duration of your participation in the study would be two years.

Potential risks and benefits

There are no new medicines or procedures in this study, so it should not introduce new risks beyond those already associated with your routine hospital care and treatment with Patisiran or Vutrisiran. You will undergo standard medical examinations, which have their own known, small risks. The potential benefits for you personally might include a more detailed look at your heart health over time, but the main benefit of this study is to help doctors better understand how Patisiran and Vutrisiran affect the heart in people with ATTRv amyloidosis. This knowledge can improve future care for others with the same condition. You are free to withdraw from the study at any time without affecting your medical treatment.

Locations (5)

CHU Bordeaux Haut-Levêque
Verified postcode
Bordeaux, France
APHP Henri Mondor
Verified postcode
Créteil, France
CHU Grenoble Alpes
Verified postcode
Grenoble, France
CHU Nancy Institut Louis Mathieu
Verified postcode
Nancy, France
CHU Rangueil Toulouse
Verified postcode
Toulouse, France

Common questions

What is ATTRv amyloidosis?

It's an inherited condition where faulty proteins build up in your body, damaging organs like your nerves and heart.

Are Patisiran and Vutrisiran new medicines?

They are modern treatments used to manage ATTRv amyloidosis, particularly the nerve problems it causes.

Will I receive different treatment if I join this study?

No, you will continue with your usual treatment plan. This study just collects extra information during your routine appointments.

What does a 'six-minute walk test' involve?

It's a simple test where you walk as far as you can in six minutes, usually down a hospital corridor, to measure your walking ability.

What if I change my mind about participating?

You are free to leave the study at any time, and it will not affect your medical care in any way.