MAGNITUDE: A Phase 3 Study of NTLA-2001 in Participants With Transthyretin Amyloidosis With Cardiomyopathy (ATTR-CM)
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ClinicalTrials.gov Identifier: NCT06128629 |
Recruitment Status :
Recruiting
First Posted : November 13, 2023
Last Update Posted : May 2, 2024
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Condition or disease | Intervention/treatment | Phase |
---|---|---|
Transthyretin Amyloidosis (ATTR) With Cardiomyopathy | Biological: NTLA-2001 Drug: Placebo | Phase 3 |
Study Type : | Interventional (Clinical Trial) |
Estimated Enrollment : | 765 participants |
Allocation: | Randomized |
Intervention Model: | Parallel Assignment |
Intervention Model Description: | Participants will be randomized in a 2:1 ratio to NTLA-2001 or placebo |
Masking: | Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor) |
Primary Purpose: | Treatment |
Official 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) |
Actual Study Start Date : | December 13, 2023 |
Estimated Primary Completion Date : | December 2027 |
Estimated Study Completion Date : | April 2028 |
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
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Drug: Placebo
Normal saline (0.9% NaCl) by IV infusion |
- Composite outcome of cardiovascular (CV) mortality and CV events [ Time Frame: Maximum study duration is dependent on event rates and is estimated to be at least 18 months and up to 48 months ]
- Change in baseline to month 18 in serum TTR [ Time Frame: Baseline, Month 18 ]
- Change from baseline to month 18 in KCCQ-OS score [ Time Frame: Baseline, Month 18 ]
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Ages Eligible for Study: | 18 Years to 90 Years (Adult, Older Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
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
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT06128629
Contact: Trial Manager at Intellia | 1-857-285-6200 ext 6 | medicalinformation@intelliatx.com |
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Christchurch, New Zealand | |
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Richmond Pharmacology | Recruiting |
London, United Kingdom |
Responsible Party: | Intellia Therapeutics |
ClinicalTrials.gov Identifier: | NCT06128629 |
Other Study ID Numbers: |
ITL-2001-CL-301 |
First Posted: | November 13, 2023 Key Record Dates |
Last Update Posted: | May 2, 2024 |
Last Verified: | May 2024 |
Individual Participant Data (IPD) Sharing Statement: | |
Plan to Share IPD: | No |
Studies a U.S. FDA-regulated Drug Product: | Yes |
Studies a U.S. FDA-regulated Device Product: | No |
TTR Amyloidosis Cardiomyopathy NTLA-2001 ATTR-CM Transthyretin ATTR TTR-mediated amyloidosis |
Amyloidosis, Hereditary Amyloidosis, Hereditary, Transthyretin-Related Amyloidosis Transthyretin amyloid cardiomyopathy TTR cardiomyopathy Wild-type TTR V122I Amyloidosis, Wild Type |
Amyloid Neuropathies, Familial Cardiomyopathies Amyloidosis Heart Diseases Cardiovascular Diseases Proteostasis Deficiencies Metabolic Diseases Heredodegenerative Disorders, Nervous System |
Neurodegenerative Diseases Nervous System Diseases Amyloid Neuropathies Peripheral Nervous System Diseases Neuromuscular Diseases Genetic Diseases, Inborn Amyloidosis, Familial Metabolism, Inborn Errors |