A Research Study to Look at How a New Medicine Called NNC6019-0001 Works and How Safe it is for People Who Have Heart Disease Due to Transthyretin (TTR) Amyloidosis


Trial ID: NCT05442047


This study is testing a potential new medicine, NNC6019-0001, for people who have a heart disease due to TTR amyloidosis.The study will look at if this medicine can reduce the symptoms of a heart disease due to TTR amyloidosis, such as heart failure. Participants will either get NNC6019-0001 (apotential new medicine) or placebo (a medicine which has no effect on the body). Which treatment participants get is decided by chance. The chance of getting NNC6019-0001 is two times higher than getting placebo. NNC6019-0001 is not yet approved in any country or region in the world. It is a new medicine that doctors cannot prescribe yet. Participants will get an infusion of the study medicine 13 times, once every 4 weeks. The study will last for about 64 weeks after the first dose of medicine. Participants cannot participate in this study if they have a heart disease other than a heart disease due to TTR amyloidosis.

Official Title

Efficacy and Safety of NNC6019-0001 at Two Dose Levels in Participants With Transthyretin Amyloid Cardiomyopathy (ATTR CM)

Stanford Investigator(s)

Kevin M. Alexander, MD, FACC, FHFSA
Kevin M. Alexander, MD, FACC, FHFSA

Assistant Professor of Medicine (Cardiovascular Medicine) at the Stanford University Medical Center


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

   - 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

   - Body weight >120 kilogram (kg) (264.6 pounds [lb]) at screening.

   - History of contrast allergy or adverse reactions to gadolinium-containing agents.


drug: NNC6019-0001

drug: Placebo (NNC6019-0001)


Contact Information

Stanford University
School of Medicine
300 Pasteur Drive
Stanford, CA 94305