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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
Indication Transthyretin Amyloidosis with Cardiomyopathy/Polyneuropathy
Compound Name Nexiguran ziclumeran (NTLA-2001)
Sponsor Intellia Therapeutics
Funder Type Industry
Status
Recruiting
Enrollment Count 765

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 2024-11-04

Participation Criteria

Eligible Age 18 Years - 90 Years
Standard Ages Adult, Older adult
Eligible Sex ALL

Locations

No.of Trial Sites 60
Locations Argentina,Singapore,United States,United Kingdom,Spain,New Zealand,Canada,Sweden,Taiwan,Denmark,Italy,Australia,France,Germany

Regulatory Information

Has US IND True
Recent Updates Initiate Phase III study for ATTRv-PN by year end

Resources/Links

Patents
  • US20230257747A1
  • WO2017173054A1
  • US20240076636A1
  • US20230287400A1
  • US20210087568A1
  • WO2020219876A1
  • US20220009878A1
Clinical Publications
News and Press Releases
Preclinical Publications
Protocol