Transthyretin Amyloid Cardiomyopathy
Conditions
Interventions
Experimental: ALXN2220
Starting at Day 1 of the Randomized Evaluation Period participants will receive ALXN2220 for a total from 24 to 48 months. Participants will receive bodyweight dependent doses o
Sponsors
Furuta Keiko
Eligibility
Inclusion criteria
Inclusion criteria: 1. Male or female over 18 years or more to 90 years or under of age at time of randomization 2. Centrally confirmed diagnosis of ATTR-CM with either wild-type or variant TTR genotype 3. End-diastolic interventricular septal wall thickness 11 mm or more for women or 12 mm or more for men on echocardiography measured at Screening 4. NT-proBNP > 2000 pg/mL measured by a central laboratory at Screening 5. Treatment with a loop diuretic for at least 30 days prior to Screening 6. History of heart failure as documented by one of the following events within 1 year prior to Screening: a. heart failure hospitalization b. urgent heart failure visit c. episode of volume overload documented by NT-proBNP > 2000 pg/mL (or equivalent BNP) 7. NYHA Class II-IV at Screening 8. Life expectancy of 6 months or more as per the Investigator's judgment
Exclusion criteria
Exclusion criteria: 1. Known leptomeningeal amyloidosis 2. Known light chain (AL) or secondary amyloidosis (AA), or any other form of systemic amyloidosis 3. History of multiple myeloma 4. Cardiomyopathy not primarily caused by ATTR-CM, for example, cardiomyopathy primarily due to hypertension, valvular heart disease, or ischemic heart disease per Investigator's assessment 5. Acute coronary syndrome, unstable angina, stroke, transient ischemic attack, coronary revascularization, cardiac device implantation, cardiac valve repair, or major surgery within 3 months of Screening 6. Uncontrolled hypertension (average resting systolic BP > 160 mmHg or diastolic BP > 100 mmHg at Screening) 7. Average resting systolic BP < 90 mm Hg or symptomatic orthostatic hypotension at Screening per Investigator's assessment 8. Uncontrolled clinically significant cardiac arrhythmia, per Investigator's assessment 9. LVEF < 30% on echocardiography 10. Hemoglobin < 8 g/dL for women or < 9 g/dL for men measured by central laboratory at Screening 11. Platelet count < 100000/mm3 or other disorder associated with clinically significant thrombocytopenia measured by central laboratory at Screening 12. Participants with renal failure requiring dialysis or who have an eGFR by CKD-Epi formula < 20 mL/min/1.73 m2 measured by a central laboratory at Screening 13. Body weight < 40 kg at Screening
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| To assess the efficacy of ALXN2220 in the treatment of adult participants with ATTR-CM by evaluating the difference between the ALXN2220 and placebo groups as assessed by the composite endpoint of ACM and total CV clinical events | — |
Countries
Argentina, Australia, Austria, Belgium, Brazil, Canada, China, Czech Republic, Denmark, France, Germany, Greece, Hungary, Ireland, Israel, Italy, Japan, Netherlands, New Zealand, Norway, Poland, South Korea, Spain, Switzerland, Taiwan, Turkey, United Kingdom, United States of America
Contacts
Public ContactKeiko Furuta
Alexion Pharma GK
Outcome results
None listed