NEURO-TTRansform is a clinical trial for people living with hereditary transthyretin-mediated amyloid polyneuropathy (hATTR-PN)

About the Study

The purpose of the NEURO-TTRansform study is to determine if an investigational antisense drug is safe and effective at improving the quality of life for people living with hATTR-PN. There is no placebo group in this study. All study participants will be divided in two different treatment groups.

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 AKCEA-TTR-LRx once every 4 weeks (monthly).

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The second group will receive a subcutaneous injection of inotersen (a drug approved in the US, Canada, and most European countries for the treatment of hATTR-PN) once a week. After 8 months, participants in the inotersen group will stop receiving inotersen, and will be crossed over to the group receiving AKCEA-TTR-LRx.

Participants in both groups will also receive daily supplemental doses of the recommended daily allowance of vitamin A.

Participation in this study will last about 26 months, which includes a total treatment period of 65 weeks followed by a post-treatment evaluation period.


Hereditary transthyretin-mediated amyloid polyneuropathy (hATTR-PN)

Eligibility Criteria

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

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Have been diagnosed with polyneuropathy symptoms due to hereditary transthyretin amyloidosis, including genetic testing to confirm the genetic mutation in the TTR gene

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Are 18 to 82 years old

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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)*

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Are still able to walk (with or without an aid)


Are willing to take a vitamin A supplement

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

Study Type



Phase 3 Identifier

NCT04136184 Take me there arrow

Frequently Asked Questions

Contact for more information.

What is AKCEA-TTR-LRx?
AKCEA-TTR-LRx is an investigational antisense drug being developed for the potential treatment of ATTR-CM. AKCEA-TTR-LRx 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, AKCEA-TTR-LRx has reduced the level of the TTR protein in the blood of animals and healthy volunteers.

How often is AKCEA-TTR-LRx dosed?
AKCEA-TTR-LRx 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 (AKCEA-TTR-LRx) in the NEURO-TTRansform trial?
No, you will be randomly assigned to receive either AKCEA-TTR-LRx or TEGSEDI (inotersen). TEGSEDI (inotersen) is a drug approved in the US, most European countries, and Canada for the treatment of hATTR-PN; inotersen is also given subcutaneously, but more frequently and at a higher dose than AKCEA-TTR-LRx.

This is an open-label study, which means that you will know whether you are taking AKCEA-TTR-LRx or TEGSEDI (inotersen). If you are randomly assigned to receive TEGSEDI (inotersen), you will be switched to AKCEA-TTR-LRx after 8 months of treatment with TEGSEDI (inotersen).

What does participation in the NEURO-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 the doctors conducting the study confirm that you are eligible for the trial, the study will involve monthly visits during the first 4 months, then quarterly visits thereafter. More details will be provided to you by the physician.

Am I allowed to take VYNDAQEL® (tafamidis)* during the trial?
No, you are not eligible for the study if you are taking VYNDAQEL (tafamidis). If you and your doctor consider stopping VYNDAQEL (tafamidis) treatment, you can enter NEURO-TTRansform 14 days after you took your last dose of VYNDAQEL (tafamidis).

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

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

How much will it cost to participate in NEURO-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).

*VYNDAQEL (tafamidis) is a registered trademark of Pfizer Inc.