Are you living with heart failure due to transthyretin amyloidosis with cardiomyopathy (ATTR-CM)?

If so, consider joining the MAGNITUDE study, a clinical trial testing a research medicine to see if advanced therapy can help people with ATTR with cardiomyopathy (ATTR-CM), by stopping the TTR gene from making the abnormal TTR protein that causes the disease.

Learn more and decide if the study is right for you.

See if You May Qualify! 

Are you living with heart failure due to transthyretin amyloidosis with cardiomyopathy (ATTR-CM)?

If so, consider joining the MAGNITUDE study, a clinical trial testing a research medicine to see if advanced therapy can help people with ATTR with cardiomyopathy (ATTR-CM), by stopping the TTR gene from making the abnormal TTR protein that causes the disease. Learn more and decide if the study is right for you.

See if You May Qualify!

What is the MAGNITUDE study?

The MAGNITUDE study is a clinical research study for people with ATTR and cardiomyopathy (ATTR-CM). Researchers want to know if the research medicine called NTLA-2001 is safe and what effect it has on ATTR-CM.

You may be compensated for your time and inconvenience related to study participation for the visits you complete. Certain costs related to study participation, such as travel and meals, will also be available for study participants for reimbursement. Please ask your study team for more information about it.

Study Clinic Locations

Who can join the MAGNITUDE study?

You may be able to join the MAGNITUDE study if you meet these criteria:

Between the ages of 18 and 90

Diagnosed with Heart Failure due to ATTR

On medication for heart failure and have experienced heart failure in the past year

Women are eligible if they can no longer have children

Other criteria apply. Your doctor can help you find out if you qualify.

What will happen during the study?

Participation in the MAGNITUDE study will range from about 18 months and up to about 4 years. Study participants can expect the following:

Screening and Baseline Period (up to 2 months)
  • Read the Informed Consent Form (ICF) and sign it if you agree to participate
  • Visit the study site a few times for study assessments such as a blood test, physical exam, and walking test to find out whether you qualify for the study
  • Have genetic testing to find out whether you have a TTR gene mutation if this is not already part of your medical record
Dosing Period
(1 day)
  • Be assigned at random to receive NTLA-2001 or a placebo (no active ingredients)—you are 2 times more likely to receive NTLA-2001 than to receive a placebo
  • Receive pre-medications commonly given to reduce the chance of a reaction to the infusion
  • Receive a one-time infusion of NTLA-2001 or placebo over about 4 hours
  • Have study assessments before, during, and after the infusion to check your safety and the effects of NTLA-2001. Assessments may include, but are not limited to, physical exams, heart tests, and blood tests
  • Start taking Vitamin A supplements, which you will continue to take regularly
Post-dosing Period
(18 months or more)
  • Return to the study site regularly for study assessments, such as physical exams, blood tests, heart tests, walking tests, and questionnaires. Attend study visits less frequently over time with visits approximately every 6 months after the first year.
  • Be asked to join a separate long-term follow-up study in which your health information is collected to help researchers learn about the long-term effects of NTLA-2001

Participation in a clinical study is voluntary. You can ask any questions you have and may leave the study at any time, for any reason.

Interested in the MAGNITUDE study?

Take the pre-screener to see if you may qualify!

Take the Pre-Screener

Study Medication

What is the study medication ?

The study medication is called NTLA-2001, and it is an investigational medicine. Investigational means NTLA-2001 has not been approved by any health or regulatory authority and can only be used in clinical research studies.

NTLA-2001 is given as a 1-time intravenous (IV) infusion into the arm or hand.

NTLA-2001 is a gene editing system known as CRISPR/Cas9. It is designed to find and “knockout” (disable) the TTR gene. This means it changes a person’s DNA, so the gene stops making the TTR protein. Click here to learn more about CRISPR and NTLA-2001.

Will I receive the study medication?

Participants in the MAGNITUDE study will be assigned at random to receive NTLA-2001 or a placebo, which has no active ingredients. You are 2 times more likely to receive NTLA-2001 than to receive a placebo. The study will be double-blind, which means neither you nor the study doctor will know if you are getting NTLA-2001 or a placebo.

About Transthyretin Amyloidosis with Cardiomyopathy (ATTR-CM)

What is ATTR with cardiomyopathy (ATTR-CM)?

ATTR-CM is a rare disease caused by the buildup of a protein that does not form correctly. The protein is called transthyretin (TTR), and the buildup happens when abnormal TTR protein gets stuck in different parts of the body. The buildup is known as amyloidosis. When TTR amyloidosis is in the heart, it is called ATTR with cardiomyopathy, or ATTR-CM.

There are 2 types of ATTR-CM:

Hereditary ATTR-CM

  • passed down from a parent
  • less common
  • symptoms usually seen in patients over the age of 60

Wild-type ATTR-CM

  • can happen with aging
  • more common
  • symptoms usually seen in patients over the age of 75

In both types, abnormal TTR protein builds up in the heart. This can happen because of a mutation (change) in a person’s genes (hereditary), or it can happen because of abnormal folding of the protein after it is made (wild-type).

About Clinical Research

Here are some common questions and answers about study participation.

If you have additional questions about participating in a clinical research study, contact a study clinic near you.

Study Clinic Locations

Find a study clinic near you!

Use the filters, map, and listing to find the study clinic closest to you. If you do not see a site listed near you, additional sites may be listed on clinicaltrials.gov. Contact the study clinic to learn more.

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