CARDIO-TTRansform: A Study to Evaluate the Efficacy and Safety of Eplontersen (Formerly Known as ION-682884, IONIS-TTR-LRx and AKCEA-TTR-LRx) in Participants With Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR CM)
N
Noel Dasgupta
Primary Investigator
Overview
To evaluate the efficacy of eplontersen compared to placebo in participants with ATTR-CMving available standard of care (SoC). For more information, please visit
https://www.cardio-ttransform.com.
Description
This is a multicenter, double-blind study in approximately 1400 participants, who will bedomized to receive subcutaneous (SC) injections of either eplontersen or placebo oncevery 4 weeks. Participants will also receive daily supplemental doses of the recommended
daily allowance of vitamin A.
Eligibility
You may be eligible for this study if you meet the following criteria:
-
Conditions:
Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR CM)
-
Age: Between 18 Years - 90 Years
-
Gender: All
Inclusion Criteria:
- Females must be non-pregnant and non-lactating, and either surgically sterile orusal or abstinent. If engaged in sexual relations of child-bearinggree to use 1 highly effective contraceptive method
- Males must be surgically sterile or, abstinent or, if engaged in sexual relations withwoman of child-bearing potential, the participant or the participant's non-pregnantust be using a highly effective contraceptive method
- Amyloid deposits in cardiac or non-cardiac tissue confirmed by Congo Red (orquivalent) staining OR technetium scintigraphy (99mTc -3,3-diphosphono-1,2-dicarboxylic acid [DPD-Tc], 99m Tc-pyrophosphate [PYP-Tc], or 99mTc-hydroxymethylene-diphosphonate [HMDP-Tc]) with Grade 2 or 3 cardiac uptake in thebsence of abnormal light chains ratio, centrally confirmed
- End-diastolic interventricular septum thickness of > 12 millimeters (mm) on Screeninghocardiogram
- New York Heart Association (NYHA) class I-III
Exclusion Criteria:
- Acute coronary syndrome, unstable angina, stroke, transient ischemic attack (TIA),y revascularization, cardiac device implantation, cardiac valve repair, orjor surgery within 3 months of Screening
- Cardiomyopathy not primarily caused by ATTR-CM, for example, cardiomyopathy due tohypertension, valvular heart disease, or ischemic heart disease
- Monoclonal gammopathy of undetermined significance (MGUS) and/or alterations inunoglobulin free light chain (FLC) ratio unless fat, bone marrow, or heart biopsyg the absence of light chain and the presence of TTR protein by massy or immunoelectron microscopy. For participants with chronic kidneydisease (CKD) and without presence of monoclonal protein in blood and urine, theble FLC ratio is 0.26-2.25. Results different from that may be discussed withhematologist, Investigator and Medical Monitor if the risks associated with thebiopsy outweigh the benefits
- Prior liver or heart transplant, and/or Left Ventricular Assist Device (LVAD) ord liver transplant or LVAD within 1 year after randomization
- Current or previous treatment with Tegsedi™ (inotersen) or Onpattro™ (patisiran) orher oligonucleotide or ribonucleic acid (RNA) therapeutic (including smallg ribonucleic acid [siRNA]; does not apply to COVID-19 mitochondrial [mRNA]vaccinations)
- Current treatment with diflunisal, doxycycline, with or without ursodeoxycholic acid,d/or non-dihydropyridine calcium-channel blocker (e.g., verapamil, diltiazem).Participants receiving any of these agents must respect a wash-out period of 14 daysbefore randomization.
Updated on
27 Apr 2024.
Study ID: ION-682884-CS2
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