Transthyretin Amyloid Cardiomyopathy (ATTR CM)
Conditions
Brief summary
This study will test a medicine, NNC6019-0001, for people who have a heart disease due to TTR amyloidosis. It will look at how safe this medicine is in the long term and if it can reduce symptoms of a heart disease due to TTR amyloidosis, such as heart failure. It is an extension to a study called A research study to look at how a new medicine called NNC6019-0001 works and how safe it is for people who have a heart disease due to TTR amyloidosis. Only participants who have completed that study will be invited for this new study. Participants will get NNC6019-0001, regardless of whether they got placebo or NNC6019-0001 in the first study. The study will last for up to 157 weeks (36 months/3 years).
Interventions
DRUGNNC6019-0001
Intravenous NNC6019-0001 every 4 weeks added to the standard of care until Week 140.
Sponsors
Novo Nordisk A/S
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to 85 Years
Healthy volunteers
No
Inclusion criteria
* Completed study intervention in NN6019-4940 and attended the last study visit (week 64; visit 16), and no later than 12 weeks after visit 16. * Expected to be on stable doses of cardiovascular medical therapy 6 weeks prior to the enrolment visit.
Exclusion criteria
* A prior solid organ transplant. * Presence or history of malignant neoplasm (other than basal or squamous cell skin cancer, in-situ 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 (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. * Body weight greater than (\>) 120 kilograms (kg) (264.6 pounds \[lb\]) at screening.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of Treatment Emergent Adverse Events | From baseline (week 0) up to visit 39 (week 156) | Measured as events. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change in N-terminal-pro brain natriuretic peptide (NT-proBNP) | From baseline (week 0) to visit 28 (week 104) | Measured in percentage. |
| Change in Myocardial Extracellular Volume (ECV) | From baseline (week 0) to visit 28 (week 104) | Measured in percentage-points. |
| Change in 6-Minute Walk Test (6MWT) | From baseline (week 0) to visit 28 (week 104) | Measured in meters. |
| Change in Troponin I | From baseline (week 0) to visit 28 (week 104) | Measured in nanogram per milliliter (ng/mL). |
| Change in Global Longitudinal Strain (GLS) on Echocardiography | From baseline (week 0) to visit 28 (week 104) | Measured in percentage-points. |
| Change in Kansas City Cardiomyopathy Questionnaire (KCCQ) Clinical Summary Score (CSS) | From baseline (week 0) to visit 28 (week 104) | The KCCQ is a disease-specific health status instrument composed of 23 items that quantify the domains of physical limitation, symptoms, self-efficacy, social limitation, and health-related quality of life limitation from heart failure. The overall summary score and all domains have been independently demonstrated to be valid, reliable, and responsive to clinical change. CSS scores range from 0 to 100 and lower scores represent more severe symptoms and/or limitations and scores of 100 indicate no symptoms, no limitations, and excellent quality of life. Measured in score on a scale. |
Countries
Canada, Czechia, France, Germany, Italy, Japan, Netherlands, Portugal, Spain, United States
Outcome results
None listed