Trial purpose
Cancer treatment
Tumor type
Haematological
Age
18 - 90
Trial acronym
Cardio-TTRansform
Clinical summary
Summary
This is a randomised, double blind trial evaluating the efficacy of eplontersen compared to placebo for 120 weeks in people with Transthyretin-Mediated Amyloid Cardiomyotherapy (ATTR-CM) receiving available standard of care (SoC). Participants will be randomly assigned to either the Experimental Arm or Placebo Comparator Arm. Participants in the Experimental Arm will receive eplontersen, administered via an injection, once every 4 weeks. Participants in the Placebo Comparator group will receive a placebo, administered via injection, once every 4 weeks. All participants will also receive daily supplemental doses of the recommended daily allowance of vitamin A.
Conditions
This trial is treating people with Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR CM)
Eligibility
Inclusion
- 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 millimeters (mm) on Screening echocardiogram
- New York Heart Association (NYHA) class I-III
Exclusion
- 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 alterations in immunoglobulin free light chain (FLC) ratio unless fat, bone marrow, or heart biopsy confirming the absence of light chain and the presence of TTR protein by mass spectrometry or immunoelectron microscopy. For participants with chronic kidney disease (CKD) and without presence of monoclonal protein in blood and urine, the acceptable FLC ratio is 0.26-2.25. Results different from that may be discussed with local hematologist, Investigator and Medical Monitor if the risks associated with the biopsy outweigh the benefits
- 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 ribonucleic acid (RNA) therapeutic (including small interfering ribonucleic acid [siRNA]; does not apply to COVID-19 mitochondrial [mRNA] vaccinations)
- Current treatment with diflunisal, doxycycline, with or without ursodeoxycholic acid, and/or non-dihydropyridine calcium-channel blocker (e.g., verapamil, diltiazem). Participants receiving any of these agents must respect a wash-out period of 14 days before randomization.
Exclusion
- You have certain types of non-cancer medical conditions.
- You have had certain treatments, surgical procedures or drugs.
Clinical trials have complex eligibility criteria, and other criteria may apply for this trial. Ask your doctor about whether this trial could be right for you.
More information
Trial Identifiers
Information on this page is partially produced from ClinicalTrials.gov *. View further details about this trial on the registry via the links below:
Trial sponsor
Ionis Pharmaceuticals, Inc.
Scientific 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)
Get Support
You might find it helpful to speak to someone who has 'been there before'. Our Peer Connect program can provide one-on-one phone support from someone who understands what you're going through and has clinical trials experience.
Know more about Peer Connect
If you need cancer information and practical support for yourself, a carer, family or friend, contact Cancer Council to speak with an experienced health professional on 13 11 20.
Get support
Learn more about clinical trials through this collection of resources in languages other than English.
View information