Study of ALXN2220 versus Placebo in Adults with ATTR-CM

JRCT ID: jRCT2031230517

Registered date:18/12/2023

Basic Information

• Recruitment status: Recruiting
• Health condition(s) or Problem(s) studied: Transthyretin Amyloid Cardiomyopathy
• Date of first enrollment: 04/01/2024
• Target sample size: 1000
• Countries of recruitment: Argentina,Japan,Australia,Japan,Brazil,Japan,Canada,Japan,China,Japan,France,Japan,Germany,Japan,Italy,Japan,Poland,Japan,South Korea,Japan,Spain,Japan,Taiwan,Japan,Turkey,Japan,United Kingdom,Japan,United States of America,Japan,Austria,Japan,Belgium,Japan,Czech Republic,Japan,Denmark,Japan,Greece,Japan,Hungary,Japan,Ireland,Japan,Israel,Japan,Netherlands,Japan,New Zealand,Japan,Norway,Japan,Switzerland,Japan
• Study type: Interventional

Intervention(s)

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 of either 2400mg, 3200mg or 4800mg of ALXN2220 once q4w via IV injection.
Experimental: Placebo
Starting at Day 1 of the Randomized Evaluation Period participants will receive placebo for a total from 24 to 48 months. Participants will receive placebo once q4w via IV injection.

Outcome(s)

• Primary Outcome: 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

Key inclusion & exclusion criteria

• Age minimum: Not applicable
• Age maximum: Not applicable

Include 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

Exclude 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

Related Information

• Primary Sponsor: Hirose Hiroshi