Summary

Eligibility
for people ages 18-90 (full criteria)
Location
at La Jolla, California and other locations
Dates
study started
estimated completion

Description

Summary

To evaluate the efficacy of eplontersen compared to placebo for 120 weeks in patients with ATTR-CM receiving available standard of care (SoC). For more information, please visit https://www.cardio-ttransform.com.

Official Title

A Phase 3 Global, Double-Blind, Randomized, Placebo-Controlled Study to Evaluate the Efficacy and Safety of ION-682884 in Patients With Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR CM)

Details

This is a multicenter, double-blind study in approximately 750 participants, who will be randomized to receive subcutaneous (SC) injections of either eplontersen or placebo once every 4 weeks. Participants will also receive daily supplemental doses of the recommended daily allowance of vitamin A.

Keywords

Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR CM) TTR Amyloidosis Cardiomyopathy Cardiomyopathies Eplontersen

Eligibility

You can join if…

Open to people ages 18-90

  • Females must be non-pregnant and non-lactating, and either surgically sterile or post-menopausal or abstinent. If engaged in sexual relations of child-bearing potential, agree to use 1 highly effective contraceptive method
  • Males must be surgically sterile or, abstinent or, if engaged in sexual relations with a woman of child-bearing potential, the participant or the participant's non-pregnant female partner must be using a highly effective contraceptive method
  • Amyloid deposits in cardiac or non-cardiac tissue confirmed by Congo Red (or equivalent) staining OR technetium scintigraphy (99mTc -3,3-diphosphono-1,2- propanodicarboxylic acid [DPD-Tc], 99m Tc-pyrophosphate [PYP-Tc], or 99m Tc-hydroxymethylene-diphosphonate [HMDP-Tc]) with Grade 2 or 3 cardiac uptake in the absence of abnormal light chains ratio, centrally confirmed
  • End-diastolic interventricular septum thickness of > 12 mm on Screening echocardiogram
  • New York Heart Association (NYHA) class I-III

You CAN'T join if...

  • Acute coronary syndrome, unstable angina, stroke, transient ischemic attack (TIA), coronary revascularization, cardiac device implantation, cardiac valve repair, or major surgery within 3 months of Screening
  • Cardiomyopathy not primarily caused by ATTR-CM, for example, cardiomyopathy due to hypertension, valvular heart disease, or ischemic heart disease
  • Monoclonal gammopathy of undetermined significance (MGUS) and/or immunoglobulin free light chain ratio < 0.26 and > 1.65, unless fat, bone marrow, or heart biopsy confirming the absence of light chain by mass spectrometry or immunoelectron microscopy
  • Prior liver or heart transplant, and/or Left Ventricular Assist Device (LVAD) or anticipated liver transplant or LVAD within 1 year after randomization
  • Current or previous treatment with Tegsedi™ (inotersen) or Onpattro™ (patisiran) or other oligonucleotide or RNA therapeutic (including siRNA)
  • Current treatment with diflunisal, doxycycline, and/or calcium-channel blocker. Participants receiving any of these agents must respect a wash-out period of 14 days before randomization.

Locations

  • Altman Clinical and Translational Research Institute Center for Clinical Research in progress, not accepting new patients
    La Jolla California 92037 United States
  • Cedars-Sinai Medical Center accepting new patients
    Los Angeles California 90048 United States

Details

Status
accepting new patients at some sites,
but this study is not currently recruiting here
Start Date
Completion Date
(estimated)
Sponsor
Ionis Pharmaceuticals, Inc.
Links
CARDIO-TTRansform Website
ID
NCT04136171
Phase
Phase 3 Cardiomyopathy Research Study
Study Type
Interventional
Participants
Expecting 750 study participants
Last Updated