Contact SCGE
Your email
Message
Send
SCGE Consortium Home
About SCGE TCDC
Contact Us
License
Home
Gene Therapy Trial Browser
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
(Click here for study contact information)
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