ATTR CM: Efficacy and Safety of NNC6019-0001 in Transthyretin Amyloid Cardiomyopathy (ATTR CM)

What is the Purpose of this Study?

The purpose of this study is to determine whether an investigational drug called NNC6019-0001 can be used to treat people with transthyretin (TTR) amyloid cardiomyopathy, a heart disease due to TTR amyloidosis. Participants will undergo tests to check their heart (electrocardiograms, echocardiograms and magnetic resonance imaging), in addition to submitting blood samples and performing walking tests. Patients will also complete questionnaires about quality of life and health status.

People with a heart disease due to TTR amyloidosis have changes in a specific protein circulating in the blood, called transthyretin. This changed protein can be detrimental, as it may increase the risk of heart failure or other complications. In previous studies of NNC6019-0001, a reduction in transthyretin was observed. In this study, participants will be randomly assigned to receive NNC6019-0001 or placebo. (They will have a 2:1 chance of getting the study drug.)

The information collected may also help researchers better understand heart disease due to TTR amyloidosis or related diseases; how the study drug works in the body; and how to improve the treatment of people with a heart disease due to TTR amyloidosis or related diseases.

Eligibility

Male or female.
Age greater than or equal to (>=) 18 to less than (<) 85 years at the time of signing informed consent.
Have an established diagnosis of Transthyretin amyloid cardiomyopathy (ATTR CM) with either wild-type transhyretin (TTR) or hereditary transthyretin (TTR) genotype as per local standards.
Expected to be on stable doses of cardiovascular medical therapy 6 weeks prior to the randomisation visit.

Inclusion Criteria:

Male or female.
Age greater than or equal to (>=) 18 to less than (<) 85 years at the time of signing informed consent.
Have an established diagnosis of Transthyretin amyloid cardiomyopathy (ATTR CM) with either wild-type transhyretin (TTR) or hereditary transthyretin (TTR) genotype as per local standards.
Expected to be on stable doses of cardiovascular medical therapy 6 weeks prior to the randomisation visit.
Known end-diastolic interventricular septal wall thickness greater than or equal to (>=) 12 millimeters (mm).
Presently classified as New York Heart Association (NYHA) Class II-III.
N-terminal-pro brain natriuretic peptide (NT-proBNP) concentration greater than or equal to (>=) 650 picograms per milliliter (pg/mL) in sinus cardiac rhythm and greater than (>) 1000 pg/mL in atrial fibrillation at screening.
Completed greater than or equal to (>=) 150 meters to less than or equal to (<=) 450 meters on the 6-minute walk test (MWT) at screening.
Estimated glomerular filtration rate (eGFR) greater than or equal to (>=) 25 milliliter per minute per 1.73 meter square (mL/min/1.73 m^2) at screening.

Exclusion Criteria:

Cardiomyopathy not primarily caused by transthyretin amyloid cardiomyopathy transthyretin amyloid cardiomyopathy (ATTR CM), for example, cardiomyopathy due to hypertension, valvular heart disease, or ischemic heart disease.
A prior solid organ transplant.
Planned solid organ transplant during the study.
Presence or history of malignant neoplasm (other than basal or squamous cell skin cancer, insitu carcinomas of the cervix, or in-situ/high grade prostatic intraepithelial neoplasia (PIN) or low-grade prostate cancer) within 5 years before screening.
Current treatment with calcium channel blockers with conduction system effects (example [e.g.], verapamil, diltiazem). The use of dihydropyridine calcium channel blockers is allowed. The use of digoxin will only be allowed if required for management of atrial fibrillation with rapid ventricular response.
Acute coronary syndrome, unstable angina, stroke, transient ischemic attack (TIA), coronary revascularization, cardiac valve repair, or major surgery within 3 months of screening.
Body weight >120 kilogram (kg) (264.6 pounds [lb]) at screening.
History of contrast allergy or adverse reactions to gadolinium-containing agents.

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Where can I participate?

Beverly

More about this Clinical Trial

What is the full name of this clinical trial?

Efficacy and safety of NNC6019-0001 at two dose levels in participants with transthyretin amyloid cardiomyopathy

Study Details

Disease Type/Condition

ATTR-CM, Cardiac Amyloidosis

Principal Investigator

Cole, Robert

Age Group

Adult

Phase

N/A

IRB Number

STUDY00002102

ClinicalTrials.gov ID

NCT05442047

Key Eligibility

ClinicalTrials.gov

How do I learn more about this study?

clinicaltrials@cshs.org