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Clinical Trial Report

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
Dose 2
Dose 3
Dose 4
Dose 5

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

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
Recent Updates Plan to dose the first patient in the pivotal Phase 3 MAGNITUDE-2 trial for ATTRv-PN in 1Q25.

Resources/Links