CARDIO-TTRansform is a clinical trial for people living with transthyretin-mediated amyloid cardiomyopathy (ATTR-CM)

About the Study

The purpose of the CARDIO-TTRansform study is to determine if eplontersen, an investigational antisense drug, is safe and effective at slowing the progression of cardiomyopathy and improving the quality of life for people living with hereditary or wild-type ATTR-CM.

Study participants will be randomly divided into two groups:

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The first group will receive subcutaneous injections (under the skin, like an insulin injection) of eplontersen once every 4 weeks.

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The second group will receive a subcutaneous injection of a placebo every 4 weeks.

All Participants in both groups will also receive daily supplemental doses of the recommended daily allowance of vitamin A and remain on current standard of care.

Participation in this study will last about 36 months, which includes a total treatment period of 120 weeks followed by 20 weeks of post-treatment evaluation.


Transthyretin-mediated amyloid cardiomyopathy (wild-type and hereditary ATTR-CM)

Eligibility Criteria

You may be eligible to participate in the CARDIO-TTRansform study if you:

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Have been diagnosed with mild or moderate cardiomyopathy due to transthyretin amyloidosis (hereditary or wild-type)

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Are 18 to 90 years old

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Are willing to take a vitamin A supplement

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Have NOT had a liver or heart transplant

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Have NOT been previously treated with TEGSEDI™ (inotersen) or ONPATTRO® (patisiran)*

*TEGSEDI is a trademark of Akcea Therapeutics, Inc.
*ONPATTRO is a registered trademark of Alnylam Pharmaceuticals, Inc.

Study Type



Phase 3 Identifier

NCT04136171 Take me there arrow

Frequently Asked Questions

Contact [email protected] for more information.

What is eplontersen?
Eplontersen is an investigational antisense drug being developed for the potential treatment of ATTR-CM. Eplontersen is a ligand-conjugated antisense (LICA) drug designed to reduce the production of transthyretin, or TTR protein, to treat all types of TTR amyloidosis (ATTR). In previous studies, eplontersen has reduced the level of the TTR protein in the blood of animals and healthy volunteers.

How often is eplontersen dosed?
Eplontersen is given once every 4 weeks (monthly) as a subcutaneous injection; (under the skin), similar to an insulin injection.

Will everyone receive the study medication (eplontersen in the CARDIO-TTRansform trial?
No, you will be randomly assigned to receive either eplontersen or placebo on current standard of care. Current standard of care means that you have approximately a 50% chance of being assigned to eplontersen and approximately a 50% chance of being assigned to placebo on top of standard of care. This is a double-blind study, which means that neither you nor the study staff will know whether you are taking eplontersen or placebo.

You will continue to receive concomitant approved and available treatments for your condition—such as VYNDAMAX® (tafamidis)* or VYNDAQEL® (tafamidis meglumine)—per your physician’s discretion.

What does participation in the CARDIO-TTRansform trial involve?
Before you begin the study, you will have 1-2 visits in-clinic during the screening period to determine your eligibility for the trial. After screening the physician conducting the study will confirm that you are eligible for the trial.

The study will involve quarterly visits for the duration of the trial. More details will be provided to you by the physician.

Am I allowed to take VYNDAMAX® (tafamidis)* or VYNDAQEL® (tafamidis meglumine) during the trial?
Yes, if available in your country and prescribed by your physician. VYNDAMAX® (tafamidis) and VYNDAQEL® (tafamidis meglumine) are currently approved for the treatment of transthyretin amyloidosis cardiomyopathy in the US and is expected to be approved in other regions soon.

If I am taking or have taken ONPATTRO® (patisiran) or TEGSEDI™ (inotersen), am I allowed to participate?
No, you are not eligible for the study if you are taking or have taken ONPATTRO (patisiran) or TEGSEDI (inotersen) in the past.

If I am on diflunisal or doxycycline, am I allowed to participate?
Current treatment with diflunisal or doxycycline is not allowed during the study. If you are receiving any of these agents, you must stop taking the medication 14 days before starting your participation in the study.

Where will the CARDIO-TTRansform trial be taking place?
This is a global study and will include several countries potentially within Europe, Asia, North America, Asia-Pacific, and South America.

When is the study expected to start?
This study is currently enrolling.

How much will it cost to participate in CARDIO-TTRansform?
There is no cost associated with participation in this study. Travel expenses (such as transportation and hotel, if required) will be provided. You may also be reimbursed for certain travel expenses that are related to your study visits (such as meals, parking, and taxi fares).

*VYNDAMAX (tafamidis) and VYNDAQEL (tafamidis meglumine) are registered trademarks of Pfizer Inc.