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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
To evaluate the efficacy and safety of a single dose of NTLA-2001 compared to placebo in participants with ATTR-CM.
(Show More)
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
Recruiting
Enrollment Count
765
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
2025-03-06
Participation Criteria
Eligible Age
18 Years - 90 Years
Standard Ages
Adult, Older adult
Sexes Eligible for Study
ALL
Locations
No.of Trial Sites
92
Locations
Argentina,Singapore,Hungary,United States,Czechia,United Kingdom,Portugal,Spain,New Zealand,Canada,Austria,Netherlands,Sweden,Taiwan,Brazil,Denmark,Korea, Republic of,Italy,Israel,Australia,France,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
Clinical Publications
(Presentation) Activity of Follow-On Dosing for an Investigational In Vivo CRISPR-Based Lipid Nanoparticle Therapy in Transthyretin Amyloidosis - Peripheral Nerve Society Annual Meeting 2024
(Presentation) Nexiguran Ziclumeran (nex-z, Also Known as NTLA-2001), an Investigational In Vivo CRISPR-Based Therapy for Patients With Transthyretin Amyloidosis With Cardiomyopathy (ATTR-CM): Interim Report of the Phase 1 Study - AHA Scientific Sessions 2024
Lessons from the first-in-human in vivo CRISPR/Cas9 editing of the TTR gene by NTLA-2001 trial in patients with transthyretin amyloidosis with cardiomyopathy
CRISPR-Cas9 In Vivo Gene Editing for Transthyretin Amyloidosis
News and Press Releases
Intellia Therapeutics Announces Anticipated 2025 Milestones and Strategic Reorganization to Prioritize the Advancement of its Late-Stage Programs, NTLA-2002 and Nexiguran Ziclumeran (nex-z)
Preclinical Publications
(Presentation) Enabling the development of serum [TTR] as a biomarker for treatment of ATTR amyloidosis - 4th International ATTR Amyloidosis Meeting for Patients and Doctors 2023
A Single Administration of CRISPR/Cas9 Lipid Nanoparticles Achieves Robust and Persistent In Vivo Genome Editing
Protocol
Clinical Trial Protocol
Related NCTID
Phase 1: NCT04601051