Refer a Patient

Do You Have a Patient with Transthyretin Amyloid Cardiomyopathy (ATTR-CM) Who May Be Interested in the DepleTTR-CM Study?

About the DepleTTR-CM Study

The purpose of the DepleTTR-CM Study is to evaluate the safety and efficacy of an amyloid depleter investigational medication (ALXN2220) compared to placebo in adult participants with advanced ATTR-CM.

Study Objectives

The primary objectives of this study will be:

To assess the efficacy of ALXN2220 in the treatment of adult participants with ATTR-CM by evaluating the difference between the ALXN2220 and placebo as assessed by the combined endpoint of all-cause mortality (ACM) and the cumulative frequency of cardiovascular (CV) clinical events

To assess the effects of ALXN2220 on health-based quality of life (QoL) as measured by the change from baseline in Kansas City Cardiomyopathy Questionnaire-Overall Score (KCCQ-OS)

Study Design

The DepleTTR-CM Study is a phase 3, randomized, double-blind, placebo-controlled study to evaluate the safety and efficacy of ALXN2220. Participants will be randomized in a 2:1 ratio to receive either the investigational medication or placebo, which will be administered once every 4 weeks via intravenous (IV) infusion.

Participation will last approximately 2.5–4.5 years and consists of a screening period (35 days), a treatment period (2–4 years), and a safety follow-up period (4 months). Participants who complete the treatment period may be eligible to enroll in an open-label extension study, during which all participants will receive the investigational medication.

Key Inclusion Criteria

  • Male or female ≥ 18 to ≤ 90 years of age at time of randomization
  • Centrally confirmed diagnosis of ATTR-CM with either wild-type TTR or a variant TTR genotype based on:
    • Evidence of cardiac amyloidosis by echocardiography or cMRI and 1 of the following:
      • Endomyocardial or extra-cardiac biopsy with confirmatory TTR amyloid typing by either mass spectrometry, immunoelectron microscopy, or immunohistochemistry, or
      • Grade 2 or 3 cardiac uptake on planar 99mTc scintigraphy (99mTc DPD, 99mTc PYP, or 99mTc HMDP) in the absence of monoclonal gammopathy, or
      • Grade 2 or 3 cardiac uptake on 99mTc scintigraphy (99mTc DPD, 99mTc PYP, or 99mTc HMDP) AND confirmatory TTR amyloid typing by either immunohistochemistry or mass spectrometry in non-cardiac tissue in the presence of monoclonal gammopathy
  • Willing to be genetically tested for mutations in the TTR gene during screening, if genetic testing was not previously performed or if genetic results are not available
  • End-diastolic interventricular septal wall thickness ≥ 11 mm for women or ≥ 12 mm for men on echocardiography measured at screening
  • NT-proBNP > 2,000 pg/mL measured by a central laboratory at screening

Key Exclusion Criteria

  • LVEF less than 30% on echocardiography measured at a central laboratory
  • Uncontrolled clinically significant cardiac arrhythmia, per investigator’s assessment
  • Known leptomeningeal amyloidosis
  • Known light chain (AL) or secondary amyloidosis (AA), or any other form of systemic amyloidosis
  • History of multiple myeloma
  • 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
  • Acute coronary syndrome, unstable angina, stroke, transient ischemic attack, coronary revascularization, cardiac device implantation, cardiac valve repair, or major surgery within 3 months prior of screening
  • Uncontrolled hypertension (average resting systolic [BP] > 160 mmHG or diastolic BP > 100 mmHG at screening)
  • Prior treatment with an ATTR amyloid depleter (i.e., monoclonal anti-ATTR antibody), approved or in clinical development

Additional eligibility criteria apply.

DepleTTR-CM Study Research Sites

If you have a patient who may be interested in participating and you think may qualify, refer them to this study website to take the prescreening questionnaire to determine eligibility.