Transthyretin amyloid cardiomyopathy (wild-type and variant)
Conditions
Interventions
- Drug: NNC6019-0001
NNC6019-0001 will be administered IV.
- Drug: Placebo (NNC6019-0001)
Placebo matched to NNC6019-0001 will be administered IV.
Sponsors
Brian Malling
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: - Male or female. - Age 18 years or above at the time of signing the informed consent. - Have an established diagnosis of ATTR-CM (wild-type ATTR [ATTRwt] or variant ATTR [ATTRv]), with cardiac amyloid infiltration, increased left ventricular (LV) wall thickness, and HF. Note: Target ATTRv recruitment is approximately 15 percent of the study population. 1. Cardiac amyloid infiltration demonstrated by: - Cardiac biopsy positive for TTR amyloid, OR - Grade 2 or 3 cardiac uptake at pyrophosphate (PYP)/diphosphono-1,2-propanodicarboxylic acid (DPD)/ hydroxymethylene diphosphonate (HMDP) scintigraphy with single-photon emission computed tomography (SPECT/CT) combined with an extracardiac biopsy positive for TTR amyloid, OR - Grade 2 or 3 cardiac uptake at PYP/DPD/HMDP scintigraphy with SPECT/CT combined with normal serum free light chain ratio, and negative serum and urine protein electrophoresis with immunofixation (SPIE & UPIE). Notes: - Non-invasive diagnostic pathway will be confirmed by a centralised expert review. - Bone tracer scintigraphy will be conducted using 99m-technetium (Tc)-labelled pyrophosphate (99mTc-PYP), 99mTc-labelled 3,3-diphosphono-1,2-propanodicarboxylic acid (99mTc-DPD), or 99mTc-labeled hydroxymethylene diphosphonate (99mTc-HMDP).
Exclusion criteria
Exclusion criteria: - Acute coronary syndrome, unstable angina, stroke, transient ischemic attack, coronary revascularisation, cardiac device implantation, cardiac valve repair, or major surgery within 60 days of screening. - Prior solid organ transplant or planned solid organ transplant during the study. - Left ventricular ejection fraction (LVEF) less than 30 percent as assessed by centralised review of echocardiography. - Presence or history of malignant neoplasm (other than basal or squamous cell skin cancer, in situ carcinomas of the cervix, carcinoma in situ/high-grade prostatic intraepithelial neoplasia [PIN], low-risk prostate cancer, or on stable therapy for prostate cancer) within 3 years before screening. - End-stage renal disease (estimated glomerular filtration rate [eGFR] less than 15 mL/min/1.73 m^2 at screening, or chronic/intermittent haemodialysis or peritoneal dialysis).
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Number of occurrences of composite endpoint of cardiovascular (CV) deaths and recurrent CV events (CV hospitalisations and urgent heart failure [HF] visits) [Time Frame: From baseline (week 0) to end of study (EOS) (up to approximately 4 years)] - Measured as count of events. | — |
Countries
Australia, Belgium, Brazil, Canada, China, Czechia, Denmark, France, Germany, Italy, Japan, Netherlands, South Korea, Spain, United Kingdom, United States
Contacts
Public ContactClinical trial contact
ICON Clinical Research GK
Outcome results
None listed