Ongoing, recruitingTherapeutic confirmatory (Phase III)Interventional

A Phase 3, Randomized, Double-blind, Placebo-controlled Multicenter Study to Evaluate the Efficacy and Safety of Patisiran in Patients with Transthyretin Amyloidosis with Cardiomyopathy (ATTR Amyloidosis with Cardiomyopathy)

This research study is looking into a new treatment called Onpattro for people who have a heart condition known as ATTR amyloidosis with cardiomyopathy. This condition affects your heart's ability to pump blood properly. The study aims to see how well Onpattro works and if it's safe. Participants will be given either the study medicine or a dummy treatment (placebo) without knowing which one they receive. Researchers will check changes in walking ability and other heart health measures over 12 months. This type of study helps doctors understand if new medicines can effectively treat serious conditions and improve patients' lives.

At a glance

Status

Ongoing, recruiting

Phase

Therapeutic confirmatory (Phase III)

Sponsor

Alnylam Pharmaceuticals Inc.

Enrolment target

Start

25 Nov 2024

Transthyretin Amyloidosis with Cardiomyopathy (ATTR Amyloidosis with Cardiomyopathy)

What is this study about?

This study is about a heart condition called ATTR amyloidosis with cardiomyopathy. In this condition, abnormal proteins build up in your heart, making it stiff and harder for it to pump blood around your body. This can lead to symptoms like shortness of breath and tiredness.

The main goal of this study is to see if a medication called Onpattro can help people with this heart condition. Researchers want to find out if Onpattro can improve your ability to walk and generally make you feel better and healthier compared to people who don't receive the active medicine. They will also be carefully checking for any side effects.

This is a "Phase 3" study, which means the medicine has already been tested in smaller groups and is now being looked at in a larger group of people to confirm its benefits and safety. By comparing Onpattro to a 'dummy' treatment (a placebo), doctors can get a clear picture of how effective the new medicine truly is. This information is crucial for deciding if Onpattro could become a new standard treatment for ATTR amyloidosis.

Key takeaways

This study investigates Onpattro for a serious heart condition.
It aims to improve walking ability and heart health.
Participants receive either Onpattro or a dummy treatment.
The study lasts about 12 months with ongoing follow-up.
Regular health checks and walking tests are part of participation.
It's a Phase 3 study, looking to confirm benefits and safety.

Who may be eligible?

This study is looking for adults aged 18 years and older. Both men and women can take part.

To be considered for the study, you must have been diagnosed with Transthyretin Amyloidosis with Cardiomyopathy. There will be other specific medical checks to make sure the study is right for you and safe for you to take part in.

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.

Am I 18 years old or older?
Have I been diagnosed with Transthyretin Amyloidosis with Cardiomyopathy?
Am I able to attend regular hospital visits for assessments?
Am I comfortable completing walking tests and health questionnaires?

Answer every question to see your result.

What does participation involve?

If you decide to take part, you will be randomly assigned to receive either the study medicine, Onpattro, or a dummy treatment (placebo). Neither you nor your doctor will know which one you are receiving – this is standard practice in these types of studies and helps us get accurate results. Both Onpattro and the dummy treatment will be given through a drip (infusion) into your vein.

You will have regular hospital visits for medical checks, blood tests, and to assess your heart health and how well you can walk. These assessments typically involve a "6-minute walk test" where you walk as far as you can in six minutes. You will also fill out questionnaires about your quality of life and symptoms.

These visits will happen over about 12 months. After this initial period, there will be ongoing follow-up to monitor your health. The medical team will explain the exact schedule of visits and what each will involve if you are interested.

Potential risks and benefits

Taking part in a clinical trial may offer potential benefits, such as access to a new treatment before it's widely available and more frequent medical monitoring. However, there are also potential risks, including side effects from the study medicine or the dummy treatment, and the possibility that the medicine may not help your condition. All procedures carry some level of risk. You have the right to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (8)

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

—
Unverified
Denmark
—
Unverified
Belgium
—
Unverified
Italy
—
Unverified
Sweden
—
Unverified
Czechia
—
Unverified
Netherlands
—
Unverified
France
—
Unverified
Portugal

Common questions

What is ATTR amyloidosis with cardiomyopathy?

It's a heart condition where abnormal proteins build up in your heart, making it less effective at pumping blood.

What is Onpattro?

Onpattro is the new experimental medicine being tested in this study for ATTR amyloidosis with cardiomyopathy.

Will I know if I'm getting the real medicine or a dummy treatment?

No, neither you nor your doctor will know. This is a standard way to ensure reliable study results.

How long will I be in the study?

The main part of the study lasts about 12 months, with follow-up beyond that.

Can I leave the study at any time?

Yes, you can choose to leave the study at any point, and your regular medical care will not be affected.