Study of ALXN2220 Versus Placebo in Adults With ATTR-CM
Phase 3
Recruiting
- Conditions
- Transthyretin Amyloid Cardiomyopathy
- Interventions
- Drug: Placebo
- Registration Number
- NCT06183931
- Lead Sponsor
- Alexion Pharmaceuticals, Inc.
- Brief Summary
The primary objective of this study is to access 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 total occurrences of all-cause mortality (ACM) and cardiovascular (CV) clinical events.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 1000
Inclusion Criteria
- Diagnosis of ATTR-CM with either wild-type or variant TTR genotype
- End-diastolic interventricular septal wall thickness ≥ 12 mm on echocardiography measured at Screening
- NT-proBNP > 2000 pg/mL at Screening
- Treatment with a loop diuretic for at least 30 days prior to Screening
- History of heart failure NYHA Class II-IV at Screening
- Life expectancy of ≥ 6 months as per the Investigator's judgment
- Males and females of childbearing ability must use contraception
Exclusion Criteria
- Known leptomeningeal amyloidosis
- Known light chain (AL) or secondary amyloidosis (AA), or any other form of systemic amyloidosis
- 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
- Uncontrolled clinically significant cardiac arrhythmia, per Investigator's assessment
- LVEF < 30% on echocardiography
- Renal failure requiring dialysis or an eGFR < 20 mL/min/1.73 m2 at Screening
- Polyneuropathy with PND score IV
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description ALXN2220 ALXN2220 Participants will receive weight-based dose of ALXN2220 via intravenous (IV) infusion every 4 weeks (q4w) for at least 24 months up to a maximum of 48 months. Placebo Placebo Participants will receive placebo via IV infusion q4w for at least 24 months up to a maximum of 48 months.
- Primary Outcome Measures
Name Time Method Total Occurrence of ACM and CV clinical events during the Blinded Treatment Period Baseline up to the end study (up to Month 48)
- Secondary Outcome Measures
Name Time Method Change From Baseline in Kansas City Cardiomyopathy Questionnaire-Overall Score (KCCQ-OS) at Month 24 Baseline, Month 24 Time to CV-related Mortality Baseline up to the end of study (up to Month 48) Change from Baseline in Six-minute Walk Test (6MWT) at Month 24 Baseline, Month 24 Rate of CV Clinical Events Baseline up to the end of study (up to Month 48) Time to ACM Baseline up to the end of study (up to Month 48)
Trial Locations
- Locations (1)
Research Site
🇬🇧Manchester, United Kingdom