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Cardiac amyloidosis – Trials in Disease

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Ongoing Clinical Trials for Cardiac Amyloidosis

Currently, 19 clinical trials are investigating new treatments and diagnostic methods for cardiac amyloidosis, a rare condition where abnormal protein deposits accumulate in the heart tissue. These studies are taking place across multiple European countries and involve various therapeutic approaches, including gene therapy, small molecule drugs, and advanced imaging techniques. Trials are recruiting patients with different forms of the condition, including hereditary and wild-type transthyretin amyloidosis and AL amyloidosis.

Clinical trial locations

Long-Term Safety Study of NTLA-2001 for Patients with Hereditary Transthyretin Amyloidosis with Polyneuropathy or Transthyretin Amyloidosis-Related Cardiomyopathy

This study focuses on the long-term safety of NTLA-2001, a gene therapy treatment, in patients who previously received it for hereditary transthyretin amyloidosis affecting nerves or the heart. The main goal is to monitor participants over an extended period to identify any treatment-related side effects.

Inclusion criteria: Participants must have completed or stopped a previous study where they received NTLA-2001. They need to provide informed consent and be willing to attend follow-up visits according to the study schedule.

Exclusion criteria: Those who have not been previously treated or belong to vulnerable populations requiring special protection cannot participate.

Study focus: The trial evaluates long-term safety by monitoring serious adverse events and clinical markers, particularly serum transthyretin and prealbumin levels. Regular follow-up visits will continue until August 2038.

Investigational drug: NTLA-2001 is a gene-editing therapy administered by intravenous infusion. It targets the TTR gene responsible for producing abnormal proteins in hereditary transthyretin amyloidosis. The study monitors its long-term safety after initial treatment.

Study on Long-Term Safety of Eplontersen for Adults with Transthyretin-Mediated Amyloid Cardiomyopathy

This study examines the long-term safety of eplontersen in adults with transthyretin-mediated amyloid cardiomyopathy, where abnormal protein deposits affect heart function. The treatment is given by injection under the skin.

Inclusion criteria: Participants must have completed a previous eplontersen study or have satisfactory participation in a related study as determined by their doctor. They must be willing to take vitamin A supplements and can be of any gender within the specified age range.

Exclusion criteria: People with heart conditions other than ATTR-CM, those unable to follow study procedures, pregnant or breastfeeding individuals, and those with serious medical conditions that might interfere with the study cannot participate.

Study focus: The trial monitors long-term safety through regular health assessments, including blood tests, physical exams, and heart function tests. It also tracks changes in biomarkers like serum TTR levels and walking ability over time, continuing until August 2029.

Investigational drug: Eplontersen is an antisense oligonucleotide administered subcutaneously to reduce production of the transthyretin protein involved in the disease process.

Study on the Effects and Safety of ALXN2220 for Adults with Transthyretin Amyloid Cardiomyopathy

This trial evaluates the effectiveness and safety of ALXN2220 in patients re-treated for transthyretin amyloid cardiomyopathy. ALXN2220 is a monoclonal antibody designed to target amyloid proteins in the heart.

Inclusion criteria: Adults aged 18 or older with ATTR-CM who have received at least one dose of ALXN2220 in a previous study and tolerated it well may participate. They must provide informed consent and agree to effective contraception measures.

Exclusion criteria: Those without an ATTR-CM diagnosis or who belong to vulnerable populations cannot participate.

Study focus: The trial assesses how the body responds to re-treatment with ALXN2220 using cardiac imaging techniques like cMRI and scintigraphy. It monitors heart function changes, side effects, vital signs, and blood levels of the medication.

Investigational drug: ALXN2220 is a recombinant human anti-ATTR monoclonal antibody given as an infusion solution to help remove amyloid deposits from the heart.

Study on the Effects of Dobutamine on Heart Function in Patients with Wild-type Transthyretin Amyloid Cardiomyopathy

This Danish study investigates how dobutamine affects heart function in patients with wild-type transthyretin amyloid cardiomyopathy.

Inclusion criteria: Participants must have confirmed ATTRwt through genetic testing, imaging, or biopsy. They should be receiving loop diuretics, be classified as NYHA class II-IV, be at least 65 years old, and have a left ventricular ejection fraction below 50% or stroke volume index below 35 ml/m².

Exclusion criteria: Those without ATTRwt, outside the age range, or belonging to vulnerable populations cannot participate.

Study focus: The study assesses dobutamine’s impact on cardiac output and filling pressures using right heart catheterization and echocardiography. It aims to achieve a 10% increase in cardiac output or 20% increase in stroke volume index, concluding by December 2025.

Investigational drug: Dobutamine is a beta-adrenergic agonist administered intravenously to stimulate heart contractions and improve blood flow.

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

This Spanish study examines whether Entresto, containing valsartan and sacubitril, can improve heart pumping ability in patients with transthyretin cardiac amyloidosis and reduced ejection fraction.

Inclusion criteria: Patients must be 18 or older with diagnosed transthyretin amyloid cardiomyopathy (ATTRv or ATTRwt) confirmed by tissue testing or non-invasive methods. They should have heart failure with left ventricular ejection fraction of 40% or less and be in NYHA functional class I, II, or III.

Exclusion criteria: Those with other serious heart conditions unrelated to transthyretin cardiac amyloidosis, recent heart attacks, severe kidney or liver disease, pregnancy, participation in other trials, allergies to the study medication, uncontrolled blood pressure, recent drug or alcohol abuse, or recent strokes cannot participate.

Study focus: The trial monitors heart pumping ability through regular echocardiograms over 12 months, comparing those receiving Entresto versus placebo. It aims to see if there is a 5% or greater improvement in left ventricular ejection fraction.

Investigational drug: Sacubitril/valsartan is an angiotensin receptor-neprilysin inhibitor taken orally in tablet form to help relax blood vessels and reduce strain on the heart.

Study on the Impact of Dapagliflozin on Quality of Life and Exercise Capacity in Patients with Transthyretin Cardiac Amyloidosis

This Italian study investigates whether dapagliflozin can improve quality of life and exercise capacity in patients with ATTR cardiac amyloidosis.

Inclusion criteria: Participants must be between 18 and 90 years old with established ATTR cardiac amyloidosis diagnosis confirmed by specific tests. They need a history of heart failure and stable cardiovascular medications for at least two weeks before enrollment.

Exclusion criteria: Those without ATTR-CA diagnosis, outside the age range, unable to perform exercise tests, pregnant or breastfeeding, with other serious health conditions, or unable to provide informed consent cannot participate.

Study focus: The trial measures changes in six-minute walking distance and quality of life using questionnaires. It also monitors safety and potential adjustments in diuretic medications over the study period, concluding by June 2025.

Investigational drug: Dapagliflozin is an SGLT2 inhibitor taken orally in tablet form, primarily used to lower blood sugar but being studied for its potential heart benefits in ATTR-CA patients.

Study on the Use of Florbetaben (18F) PET Imaging for Diagnosing Cardiac AL Amyloidosis in Patients

This study evaluates a new imaging technique using florbetaben (18F) PET to diagnose cardiac AL amyloidosis, a condition where abnormal proteins accumulate in the heart.

Inclusion criteria: Participants must be at least 18 years old and considered for cardiac amyloidosis diagnosis. This includes those with systemic amyloidosis without proven heart involvement, plasma cell disorders, abnormal free light chain levels, or heart failure with preserved ejection fraction, plus signs of heart involvement like wall thickness over 12mm or elevated NT-proBNP levels.

Exclusion criteria: Those with other types of amyloidosis not specified in the study or outside the age range cannot participate.

Study focus: The trial assesses how accurately florbetaben (18F) PET imaging can identify cardiac AL amyloidosis compared to standard diagnostic methods. Visual assessment of heart images by medical professionals helps determine the presence of amyloid deposits.

Investigational drug: Florbetaben (18F) is a radiopharmaceutical imaging agent injected intravenously that binds to amyloid deposits, making them visible on PET scans.

Study on the Use of Flutemetamol (18F) for Diagnosing Cardiac Amyloidosis in Patients

This Italian study examines the use of flutemetamol (18F), known as Vizamyl, for detecting amyloid deposits in patients with different types of cardiac amyloidosis through PET imaging.

Inclusion criteria: Adults aged 18 or older with diagnosed cardiac amyloidosis, including ATTRwt, ATTRv, and AL types, may participate. Diagnosis should be based on cardiac examination, biomarker assays, baseline EKG, echocardiography, cardiac MRI, positive scintigraphy, biopsy results, or genetic testing.

Exclusion criteria: Those with non-amyloid-related heart conditions, unable to provide informed consent, pregnant or breastfeeding, or with recent participation in other trials cannot participate.

Study focus: The trial evaluates how well flutemetamol (18F) binds to cardiac amyloid deposits and whether it can effectively differentiate between genetic types of the condition. It concludes by July 2025.

Investigational drug: Vizamyl is a radiopharmaceutical tracer administered intravenously that binds to amyloid plaques, allowing visualization through PET imaging.

Study to Diagnose Cardiac Amyloidosis in Patients with Monoclonal Component Using Florbetaben (18F) PET-CT

This Italian study compares traditional invasive diagnostic methods with non-invasive PET-CT imaging using 18F-florbetaben for diagnosing AL amyloidosis in patients with monoclonal component.

Inclusion criteria: Adults over 18 who can provide informed consent with NT-proBNP values greater than 332 ng/L, left ventricular wall thickness greater than 12mm, specific MRI patterns, or BNP greater than 81 ng/L may participate. The clinical setting should be compatible with cardiac amyloidosis as judged by experienced clinicians.

Exclusion criteria: Those already diagnosed with cardiac amyloidosis, without a monoclonal component, or unable to undergo required PET/CT scans cannot participate.

Study focus: The trial monitors participants for side effects during and after imaging and tracks changes in amyloid deposit intensity over 12 months.

Investigational drug: 18F-florbetaben, administered through Neuraceq injection, is a radiopharmaceutical that binds to amyloid deposits for PET-CT visualization.

Study to Evaluate the Safety and Effectiveness of Vutrisiran in Patients with Transthyretin Amyloidosis and Heart Disease

This multi-country study assesses the long-term safety of vutrisiran in patients continuing treatment from previous trials or switching from patisiran for ATTR amyloidosis with cardiomyopathy.

Inclusion criteria: Participants must have ATTR amyloidosis with cardiomyopathy and either be in specific parent studies or have completed 24 months in the vutrisiran parent study. They must agree to take vitamin A supplements and provide informed consent.

Exclusion criteria: Those with different types of amyloidosis, outside the specified age range, unable to continue the study medication, unable to provide consent, or belonging to vulnerable populations cannot participate.

Study focus: The trial monitors safety through regular adverse event tracking and assesses changes in heart-related health scores, biomarkers, mortality, and cardiovascular events until June 2027.

Investigational drug: Vutrisiran is an RNA interference therapeutic administered subcutaneously that reduces production of transthyretin protein involved in amyloid deposits.

Summary

The 19 ongoing clinical trials for cardiac amyloidosis demonstrate significant international collaboration, with multiple studies taking place across European countries. Italy, Spain, and France show particularly high concentrations of trial activity, hosting 10 or more trials each. Several key therapeutic approaches are being investigated, including RNA interference therapies like vutrisiran and patisiran, antisense oligonucleotides such as eplontersen, monoclonal antibodies including ALXN2220, and protein stabilizers like tafamidis. Multiple trials focus on advanced diagnostic imaging techniques using radiopharmaceuticals for more accurate, non-invasive diagnosis of different amyloidosis subtypes. The trials predominantly recruit patients with transthyretin-mediated amyloidosis, both hereditary and wild-type forms, though some studies also include AL amyloidosis patients. Many studies emphasize long-term safety monitoring, reflecting the chronic nature of the condition and the need for extended treatment observation periods. Study durations range from 12 months to several years, with some safety extension trials continuing until 2028 or beyond.

Ongoing Clinical Trials on Cardiac amyloidosis

  • Study on the Effectiveness and Safety of Vutrisiran and Patisiran for Patients with Hereditary Transthyretin Amyloidosis

    Not recruiting

    1 1 1 1
    Investigated diseases:
    Investigated drugs:
    Belgium Bulgaria Cyprus Italy The Netherlands Portugal +1

  • Study on the Effectiveness and Safety of CAEL-101 with Plasma Cell Dyscrasia Treatment in Patients with Stage IIIa AL Amyloidosis

    Not recruiting

    1 1 1
    Investigated diseases:
    Investigated drugs:
    Austria Czechia France Germany Greece Italy +2

  • Study on the Use of Florbetaben (18F) PET Imaging for Diagnosing Cardiac AL Amyloidosis in Patients

    Not recruiting

    1 1 1 1
    Investigated diseases:
    Investigated drugs:
    Germany Spain

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