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MAGNITUDE: a Phase 3 Study of NTLA-2001 in Participants with Transthyretin Amyloidosis with Cardiomyopathy (ATTR-CM)

PHASE3RECRUITING

To evaluate the efficacy and safety of a single dose of NTLA-2001 compared to placebo in participants with ATTR-CM.

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Study details:

This is a multinational, multicenter, double-blind, placebo-controlled study in approximately 765 participants, who will be randomized to receive a single infusion of either NTLA-2001 or placebo.

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Eligibility criteria

Researchers look for people who fit a certain description, called eligibility criteria. See if you qualify.

Inclusion criteria

Documented diagnosis of ATTR amyloidosis with cardiomyopathy

Medical history of heart failure (HF)

Symptoms of HF are optimally managed and clinically stable within 28 days prior to administration of study intervention

Screening NT-proBNP, a blood marker of HF severity, greater than or equal to 1000 pg/mL (or greater than or equal to 2000 pg/mL if participant has known atrial fibrillation)

Exclusion criteria

New York Heart Association (NYHA) Class IV HF

Polyneuropathy Disability score of IV (confined to wheelchair or bed)

Has hepatitis B, hepatitis C or human immunodeficiency virus (HIV) infection

History of active malignancy within 3 years prior to screening

RNA silencer therapy (patisiran, inotersen and/or eplontersen) within 12 months prior to dosing. Any prior vutrisiran use is not allowed

Initiation of tafamidis within 6 months prior to study dosing

Estimated glomerular filtration rate (eGFR) <30 mL/min/1.73m^2

Liver failure

Uncontrolled blood pressure

Unable or unwilling to take vitamin A supplementation for the duration of the study

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Eligibility

Age eligible for study : 18 and older

Healthy volunteers accepted : No

Gender eligible for study: All

Things to know

Study dates

Study start: 2023-12-13

Primary completion: 2027-12-01

Study completion finish: 2028-04-01

Study type

TREATMENT

Phase

PHASE3

Trial ID

NCT06128629

Intervention or treatment

BIOLOGICAL: NTLA-2001

DRUG: Placebo

Conditions

• Transthyretin Amyloidosis (ATTR) with Cardiomyopathy

Image related to Transthyretin Amyloidosis (ATTR) with Cardiomyopathy

Condition: Transthyretin Amyloidosis (ATTR) with Cardiomyopathy
BIOLOGICAL: NTLA-2001 and other drugs
Adelaide, Not Specified, Australia and more
Sponsor: Intellia Therapeutics

You may be eligible to participate in this trial based on your search. Check eligibility by answering some questions.

Are you running this trial? If you're a clinic or sponsor, you can claim this study. Claim or learn more.

Find a site

Closest Location:

Royal Adelaide Hospital

Research sites nearby

Select from list below to view details:

Royal Adelaide Hospital
Adelaide, Not Specified, Australia
The Canberra Hospital
Garran, Australian Capital Territory, Australia
Westmead Hospital
Westmead, New South Wales, Australia
Cairns Hospital
Cairns, Queensland, Australia

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Participant Group/Arm	Intervention/Treatment
EXPERIMENTAL: NTLA-2001 Single intravenous (IV) infusion of NTLA-2001	BIOLOGICAL: NTLA-2001 NTLA-2001 (55mg) by IV infusion
PLACEBO_COMPARATOR: Placebo Single IV infusion of normal saline	DRUG: Placebo Normal saline (0.9% NaCl) by IV infusion

What is the study measuring?

Primary outcome

Primary Outcome Measure	Primary Outcome Description	Primary Outcome Time Frame
Composite outcome of cardiovascular (CV) mortality and CV events	Not Specified	Maximum study duration is dependent on event rates and is estimated to be at least 18 months and up to 48 months

Secondary outcome

Secondary Outcome Measure	Secondary Outcome Description	Secondary Outcome Time Frame
Change in baseline to month 18 in serum TTR	Not Specified	Baseline, Month 18
Change from baseline to month 18 in KCCQ-OS score	Not Specified	Baseline, Month 18

Frequently Asked Questions

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You may be eligible to participate in this trial based on your search.Apply for study

Are you running this trial? If you're a clinic or sponsor, you can claim this study.Claim this trial

References

Clinical Trials Gov: MAGNITUDE: a Phase 3 Study of NTLA-2001 in Participants with Transthyretin Amyloidosis with Cardiomyopathy (ATTR-CM)