RecruitingRare disease

Long‑Term Study of Acoramidis on Heart Function and Amyloid Burden in Patients with Transthyretin Amyloid Cardiomyopathy (ATTR‑CM)

Verified siteRegistered drugNo placebo

Clinical Trials Concierge

Prefer not to search? Our Concierge searches the trials for you.

What is this trial about?

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

The study focuses on Transthyretin Amyloidosis, a rare condition in which an abnormal protein builds up in the heart muscle, making the heart stiff and reducing its ability to pump blood effectively. Participants receive an oral medication called acoramidis, supplied as film‑coated tablets, which is intended to target the protein deposits and support heart health.

The purpose of the study is to determine whether long‑term treatment with acoramidis can improve the way the heart works and is safe to use. Heart function and structure are evaluated using imaging techniques such as Cardiac Magnetic Resonance and echocardiography, which create detailed pictures of the heart and help assess the amount of amyloid protein present.

During the trial, participants take the study medication each day for several years and attend regular clinic visits where heart scans and simple blood tests are performed. One key measurement is the LVEF, a number that shows how much blood the heart pumps out with each beat; improvements or stability in this value are used to gauge benefit. Any side effects or health changes are closely monitored throughout the study period.

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
Baseline assessment
After you join the study you will have a baseline visit where doctors will perform cardiac magnetic resonance imaging and echocardiography to evaluate your heart function and structure.
Blood samples will be taken to check your overall health and to establish a starting point for later comparisons.
Step 2
Start of medication
You will begin taking BEYONTTRA tablets, each containing 356 mg of acoramidis.
The prescribed dose is 1424 mg per day, which is taken orally as four tablets every day.
The medication will be taken continuously for up to 36 months unless a doctor decides otherwise.
Step 3
Monthly safety monitoring
Each month you will have a brief visit or phone contact to report any side effects, new symptoms, or health changes.
The study team will record any adverse events and assess whether the medication is well tolerated.
Step 4
Quarterly cardiac evaluations
At month 12, month 24, and month 36 you will return for detailed cardiac imaging, including magnetic resonance and echocardiography, to measure changes in heart function.
These visits also include routine lab tests and a review of your medication adherence.
Step 5
Final assessment and study completion
At month 36 a comprehensive evaluation will be performed to determine the overall effect of the treatment on heart function and structure.
After the final assessment the study medication will be discontinued, and you will receive information about any further medical follow‑up that may be recommended.

Who can join the trial?

13 criteria

Be between 18 and 80 years old when you sign the study consent form.
Be willing and able to sign an informed consent form, which means you understand the study and agree to take part.
Have a confirmed diagnosis of ATTR‑CM (a heart condition caused by abnormal transthyretin protein deposits), proven by the doctor before screening.
Have had TTR genetic testing done before screening, with the results documented.
Your heart failure must have been clinically stable for at least 6 weeks before the screening visit, meaning no recent worsening of symptoms.
Your blood test for NT‑proBNP (a marker that shows how much stress is on the heart) must be between 300 and 6,000 pg/mL.
Your kidney function measured by eGFR (estimated glomerular filtration rate) must be 40 mL/min/1.73 m² or higher.
If you are a woman who could become pregnant (WOCBP) or a man whose partner could become pregnant, you must agree to use a highly effective method of birth control during the study and for 30 days after the last dose. Men must also avoid sperm donation for at least 30 days after the last dose.
You must have baseline heart imaging that is acceptable: a CMR (cardiac MRI) and a TTE with strain (heart ultrasound that measures how the heart muscle stretches).
Your left‑ventricular wall thickness (the thickness of the heart muscle) must be at least 12 mm as measured by the ultrasound.
Your LVEF (left‑ventricular ejection fraction, the percentage of blood the heart pumps out each beat) must be greater than 35 %.
You must have a history of heart failure, shown by either a previous hospital stay for heart failure or ongoing treatment with a diuretic medication.
You must be classified as NYHA Class I or II (a scale that describes how much heart failure symptoms limit everyday activities).

Who cannot join the trial?

22 criteria

Having a pacemaker or implantable cardioverter‑defibrillator now or planned.
Taking heart medicines called calcium channel blockers that affect the heart’s electrical system (such as verapamil or diltiazem). Regular dihydropyridine calcium blockers are allowed, and digitalis only if needed for fast heart rhythm due to atrial fibrillation.
Having HIV infection or active hepatitis B or hepatitis C (people who have been cured are okay).
Having a non‑reversible heart muscle disease in the past 6 months, or an untreated or uncontrolled irregular heartbeat.
Being on kidney dialysis or having had a kidney transplant.
Having liver test results that are more than twice the normal upper limit for ALT, AST, or total bilirubin (or three times if you have Gilbert’s syndrome).
Having cancer within the last 5 years or currently having cancer, except for certain skin cancers (basal or squamous cell carcinoma) or early‑stage cervical cancer (carcinoma in situ) that has been treated.
Being allergic to acoramidis or any of its inactive ingredients (excipients).
Being pregnant or breastfeeding (or planning to breastfeed when the study drug is given). Pregnancy tests must be negative.
Having any other medical condition or lab result that the doctor thinks could make participation unsafe or affect the study results.
Participating in another experimental drug study within 30 days before screening or while the other drug is still in the body (five half‑lives).
Being unable to have a cardiac MRI (CMR) because of severe claustrophobia, body size limits, or other medical reasons.
Having a history of substance abuse, alcoholism, or a psychiatric condition that might prevent following the study plan.
Having had major surgery within the past 3 months or planning major surgery in the next year.
Expected to live less than 2 years.
Having had a heart attack, acute coronary syndrome, heart‑blood‑vessel surgery, stroke, or mini‑stroke (transient ischemic attack) within 90 days before screening.
Having unstable blood pressure or circulation (hemodynamic instability).
Likely to need a heart transplant within 12 months.
Having AL amyloidosis (a different type of amyloid disease) at screening.
Being in NYHA Class III or IV (moderate to severe heart failure symptoms) at screening.
Being in NAC Stage 3 (low kidney function and high heart‑stress marker) at screening.
Having taken acoramidis before or currently taking it.

Clinical Trials Concierge

Prefer not to search? Our Concierge searches the trials for you.

Tell us about your condition – we search every trial in Europe and connect you with the right site.

We usually reply within a few days

Verified sites

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

Bellvitge University Hospital
Verified
L'hospitalet De Llobregat, Spain
Hospital Universitario De Salamanca
Verified
Salamanca, Spain
Region Midtjylland
Responsive
Aarhus, Denmark

Trial locations

Where you can join this trial

Countries are shaded by recruitment status. Click a recruiting country to ask about joining there.

RecruitingNot yet recruiting

Not finding your country?

Not sure what to do next?

Joining a clinical trial can seem overwhelming. We guide you step by step, so you know exactly what to expect and how we support you through the process.

See the full process and FAQ

Investigated drugs

Acoramidis is an oral tablet taken by mouth that is being studied to see if it can help people with a heart condition called transthyretin amyloid cardiomyopathy. In this trial, the medicine is given for a long time to find out whether it can improve how well the heart pumps blood, reduce the amount of amyloid protein that builds up in the heart, and make the heart’s structure healthier. Researchers are also watching closely for any side effects to make sure the medicine is safe and well‑tolerated.

Acoramidis

What is already known about the treatment

BEYONTTRA 356 mg film-coated tablets - Acoramidis is taken by mouth as a film‑coated tablet. It is currently an investigational medicine being studied in clinical trials for transthyretin amyloid cardiomyopathy, a rare heart condition. The drug works by attaching to the transthyretin protein and keeping it stable, which helps stop the formation of amyloid deposits that can damage the heart. It belongs to the class of transthyretin stabilizers.

Investigated diseases

Transthyretin Amyloidosis - Transthyretin amyloidosis is a rare condition in which a protein called transthyretin folds incorrectly and forms amyloid deposits in body tissues. When these deposits accumulate in the heart, they cause the walls of the heart chambers to become stiff and thickened. This stiffness reduces the heart’s ability to fill and pump blood efficiently. Over time, the progressive buildup can lead to a gradual decline in cardiac function. The disease may occur spontaneously or be inherited, but the heart involvement follows a similar pattern of slow worsening.