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Gene Therapy Trial Report

Summary

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


NCTID NCT06128629 (View at clinicaltrials.gov)
Description
Development Status Active
Indication Transthyretin Amyloidosis (ATTR) with Cardiomyopathy
Disease Ontology Term DOID:0050638
Compound Name NTLA-2001
Compound Alias Nexiguran ziclumeran
Sponsor Intellia Therapeutics
Funder Type Industry
Recruitment Status
Active not recruiting
Enrollment Count 1200 (ESTIMATED)
Results Posted Not Available

Therapy Information


Target Gene/Variant TTR
Therapy Type Gene editing
Therapy Route In-vivo
Mechanism of Action Gene inactivation
Route of Administration Intravenous
Drug Product Type MRNA, LNP
Target Tissue/Cell Hepatocyte
Delivery System Lipid encapsulation
Vector Type LNP
Editor Type Cas9 mRNA
Dose 1 55mg

Study Record Dates


Current Stage Phase3
Submit Date 2023-11-08
Completion Date 2028-04
Last Update 2025-12-02

Participation Criteria


Eligible Age 18 Years - 90 Years
Standard Ages Adult, Older adult
Sexes Eligible for Study ALL
Eligibility Criteria
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 600 pg/mL and less than 10,000 pg/mL 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 or acoramidis within 56 days 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
View Inclusion and Exclusion Criteria at ClinicalTrials.gov

Locations


No.of Trial Sites 126
Locations Singapore,United States,Czechia,Portugal,Austria,Netherlands,Sweden,Brazil,France,Argentina,Hungary,Japan,United Kingdom,Spain,New Zealand,Canada,Belgium,Norway,Taiwan,Denmark,Italy,Mexico,Israel,Australia,Germany

Regulatory Information


Has US IND True
FDA Designations Orphan Drug Designation, Regenerative Medicine Advanced Therapy
Recent Updates Clinical holds from October 2025 were lifted on both Phase 3 clinical trials (announced 3/2/26)

Resources/Links