RecruitingRare disease

Study on the Effects of Valsartan and Sacubitril for Patients with Transthyretin Cardiac Amyloidosis and Heart Failure with Reduced Ejection Fraction

Verified siteRegistered drugNo placebo

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What is this trial about?

A plain-language summary of the goals, design and what participants do

This clinical trial is focused on studying the effects of a medication called Entresto, which contains the active substances valsartan and sacubitril, on two heart conditions: transthyretin cardiac amyloidosis and heart failure with reduced ejection fraction. Transthyretin cardiac amyloidosis is a condition where abnormal proteins build up in the heart, affecting its function. Heart failure with reduced ejection fraction is a type of heart failure where the heart does not pump blood as well as it should.

The purpose of this study is to determine how treatment with Entresto affects the heart's ability to pump blood in patients with these conditions. Participants in the study will receive either Entresto or a placebo, and their heart function will be monitored over a period of 12 months. The main focus will be on changes in the heart's pumping ability, measured by a test called an echocardiogram, which uses sound waves to create images of the heart.

Throughout the study, participants will undergo regular check-ups and echocardiograms to assess any changes in their heart function. The goal is to see if there is an improvement in the heart's pumping ability after 12 months of treatment with Entresto compared to those who do not receive the medication. This research aims to provide valuable insights into the potential benefits of Entresto for patients with these specific heart conditions.

The research process

The trial runs in 5 steps – from screening to follow-up. Each step says what happens and what the team monitors.

Step 1
Initial assessment
Upon joining the study, an initial assessment will be conducted to confirm eligibility. This includes verifying the diagnosis of transthyretin cardiac amyloidosis and heart failure with reduced ejection fraction. The assessment will ensure that the left ventricular ejection fraction is 40% or less, as per the study requirements.
Step 2
Medication administration
Participants will begin taking Entresto, which contains the active substances valsartan and sacubitril. The medication is provided in the form of film-coated tablets, with a dosage of 24 mg/26 mg. The tablets are to be taken orally, as prescribed by the study team.
Step 3
Regular follow-up visits
Throughout the 12-month treatment period, regular follow-up visits will be scheduled. These visits are essential for monitoring health status and assessing the impact of the medication on heart function. Adjustments to the treatment plan may be made based on these assessments.
Step 4
Echocardiogram assessments
Echocardiograms will be performed at the start of the study and after 12 months of treatment. These tests will measure the left ventricular ejection fraction to evaluate any changes in heart function. An improvement is defined as an increase of 5% or more compared to the baseline measurement.
Step 5
Completion of treatment
At the end of the 12-month period, a final assessment will be conducted to determine the overall impact of the treatment. This includes a review of all collected data and a final echocardiogram to compare with the initial results.

Who can join the trial?

4 criteria

Patients must be 18 years of age or older, and can be of any gender.
Patients must have been diagnosed with transthyretin amyloid cardiomyopathy. This can be either the hereditary form (known as ATTRv) or the wild-type form (known as ATTRwt). The diagnosis can be confirmed by a tissue test showing TTR amyloid deposits or through non-invasive methods as per guidelines. For ATTRv, a genetic test showing the specific TTR mutation is needed.
Patients should have been initially evaluated or be under follow-up in specialized units for Cardiomyopathy, Heart Failure, or Amyloidosis at the centers participating in the study.
Patients must have heart failure with reduced ejection fraction. This means the heart's left ventricle pumps out less than or equal to 40% of the blood it contains with each beat. They should be in functional class I, II, or III according to the New York Heart Association (NYHA), which describes the severity of heart failure symptoms.

Who cannot join the trial?

10 criteria

Patients with any other serious heart conditions that are not related to **transthyretin cardiac amyloidosis** cannot participate.
Patients who have had a heart attack in the last 3 months are not eligible.
Patients with severe kidney disease are excluded.
Patients with severe liver disease cannot take part in the study.
Patients who are pregnant or breastfeeding are not allowed to participate.
Patients who are currently participating in another clinical trial are excluded.
Patients with a history of allergic reactions to the study medication, **Sacubitril/Valsartan**, cannot participate.
Patients with uncontrolled high blood pressure are not eligible.
Patients with a history of drug or alcohol abuse in the past year are excluded.
Patients who have had a stroke in the last 6 months cannot participate.

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Verified sites

All sites with verified contact details – recruitment status may not be available; ask directly

Verified
Bellvitge University Hospital
L'hospitalet De Llobregat, Spain
Verified
Hospital Clinic De Barcelona
Barcelona, Spain
Verified
Hospital Universitario De Salamanca
Salamanca, Spain

Trial locations

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Investigated drugs

Sacubitril/Valsartan is a medication used in this clinical trial to help improve heart function in patients with a specific type of heart disease called transthyretin amyloid cardiomyopathy, which is a condition where abnormal proteins build up in the heart. This medication is also used for patients who have heart failure with reduced ejection fraction, meaning their heart is not pumping blood as well as it should. Sacubitril/Valsartan works by relaxing blood vessels and reducing the strain on the heart, which can help improve the heart's ability to pump blood and may lead to better heart function over time.

What is already known about the treatment

Sacubitril/Valsartan – This medication is taken orally in the form of tablets and is used to treat heart conditions. It is currently being studied for its effects on transthyretin cardiac amyloidosis and heart failure with reduced ejection fraction. The main therapeutic use is to improve heart function by increasing the heart's ability to pump blood. At the molecular level, it works by blocking certain proteins that can cause blood vessels to tighten, which helps to lower blood pressure and reduce strain on the heart. It is classified as an angiotensin receptor-neprilysin inhibitor, which means it helps relax blood vessels and improve blood flow.

Investigated diseases

Transthyretin cardiac amyloidosis – Transthyretin cardiac amyloidosis is a condition where abnormal protein deposits, known as amyloid, accumulate in the heart tissue. This buildup can lead to the thickening and stiffening of the heart walls, affecting its ability to pump blood effectively. Over time, the heart's function may decline, leading to symptoms such as fatigue, shortness of breath, and swelling in the legs. The disease progresses as more amyloid deposits form, further impairing heart function. It is a type of amyloidosis specifically involving the transthyretin protein, which is produced in the liver. The condition is considered rare and can be hereditary or acquired.