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 LocationsYou 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.
Participation in the MAGNITUDE study will range from about 18 months and up to about 4 years. Study participants can expect the following:
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.
Take the pre-screener to see if you may qualify!
Take the Pre-ScreenerThe 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.
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.
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.
Hereditary ATTR-CM
Wild-type ATTR-CM
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).
Here are some common questions and answers about study participation.
Clinical research studies help scientists and doctors explore whether a medicine is safe and whether it works. The information gathered in the study can help to better understand the disease as well as the effects of the study medication. Before a doctor can prescribe a new medicine, it must go through several phases of clinical research:
Phase 1: First study of the medicine in people mainly to test safety
Phase 2: Study of the medicine in people with the condition the medicine is for
Phase 3: Study confirming how well the medicine works
Phase 4: More research after the medicine is approved
The MAGNITUDE study is a Phase 3 study.
Clinical research studies rely on volunteers. Remember that taking part in the study is your choice. The rules and ethics that doctors must follow to practice medicine also apply to clinical research studies.
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Before enrolling in a clinical trial, you must sign an Informed Consent Form (ICF). The ICF contains information about the study, including study goals, how long the study will last, benefits and risks, and the tests and procedures you will receive as part of the study.
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Study participation usually involves visiting a clinic regularly, taking or receiving an investigational medicine, and having assessments to monitor your health. You can still see your regular doctor, but you should let them know that you are participating in a study. Subjects may be reimbursed for their time on study and for travel costs, if applicable.
Participation in clinical research studies is your choice, and you may stop at any time
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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.
If you have additional questions about participating in a clinical research study, contact 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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