MAGNITUDE: Phase 3 Study to evaluate NTLA-2001 in Participants with ATTR with Cardiomyopathy
Research type
Research Study
Full title
MAGNITUDE: A Phase 3, Multinational, Multicenter, Randomized, Double-blind, Placebo-controlled Study to Evaluate the Efficacy and Safety of NTLA-2001 in Participants with Transthyretin Amyloidosis with Cardiomyopathy (ATTR-CM)
IRAS ID
1007949
Contact name
Rubin Modi
Contact email
Sponsor organisation
Intellia Therapeutics, Inc
Eudract number
2023-507220-23
Research summary
Potential patients will be asked to participate in this research study because they have a condition known as Transthyretin (TTR) Amyloidosis with Cardiomyopathy or ATTR-CM, where the heart muscle does not pump blood through the body as it should, because of build-up of abnormal protein between the muscle fibres in the heart. The specific protein is called transthyretin amyloid (TTR).
In the absence of treatment, it is a progressive and fatal disease. There are no available therapies for its treatment in the UK.
Intellia Therapeutics, Inc. has developed an investigational medication called NTLA-2001. NTLA-2001 consists of a CRISPR/Cas9 gene editing system, which can “knockout” the TTR gene. This means NTLA-2001 permanently changes the DNA so the gene stops making the TTR protein. By using the CRISPR/Cas 9 technology, NTLA-2001 will disable the TTR gene, but this does not involve the insertion of any permanent genetic material into the body.
The purpose of this study is to learn about the effects of NTLA-2001 on ATTR-CM and see how safe and effective NTLA-2001 is for patients with ATTR-CM.
This is a placebo-controlled trial. For every 3 participants in this study, 2 participants will receive NTLA-2001, and 1 will receive placebo (fluids that contain no active ingredient). The NTLA-2001 and the placebo will be masked so neither the patient, nor study doctor will not know if the patient received NTLA-2001 or placebo.
Approximately 765 participants will take part in this study at approximately 120 study sites in 32 countries.
This is an event-driven study. This means that the study will be complete after a certain number of cardiovascular events (such as hospitalisation for abnormal heart rhythm) have occurred in the whole population of study participants, but every participant will be studied for a minimum of 18 months.REC name
North East - York Research Ethics Committee
REC reference
24/NE/0005
Date of REC Opinion
30 Jan 2024
REC opinion
Further Information Favourable Opinion