Study of ALXN2220 Versus Placebo in Adults With ATTR-CM
Purpose
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.
Condition
- Transthyretin Amyloid Cardiomyopathy
Eligibility
- Eligible Ages
- Between 18 Years and 90 Years
- Eligible Genders
- All
- Accepts Healthy Volunteers
- No
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
- Phase
- Phase 3
- Study Type
- Interventional
- Allocation
- Randomized
- Intervention Model
- Parallel Assignment
- Primary Purpose
- Treatment
- Masking
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Arm Groups
| Arm | Description | Assigned Intervention |
|---|---|---|
|
Experimental 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 Comparator Placebo |
Participants will receive placebo via IV infusion q4w for at least 24 months up to a maximum of 48 months. |
|
Recruiting Locations
Research Site
Nashville, Tennessee 37232
Nashville, Tennessee 37232
More Details
- Status
- Recruiting
- Sponsor
- Alexion Pharmaceuticals, Inc.