A Phase 3 Study of NTLA-2001 in ATTRv-PN

PHASE3RecruitingINTERVENTIONAL
Enrollment

50

Participants

Timeline

Start Date

November 22, 2024

Primary Completion Date

July 31, 2027

Study Completion Date

August 31, 2028

Conditions
Neuromuscular DiseaseNeuromuscular Diseases (NMD)Neurodegenerative DiseaseNeurodegenerative Disease, HereditaryNeurodegenerative DiseasesNeuromuscular DiseasesNerve DisordersNervous System DiseaseNervous System DiseasesGenetic Disease, InbornAmyloidosis, FamilialAmyloidosis, HereditaryAmyloidosisPolyneuropathiesAmyloid NeuropathiesAmyloid Neuropathies, FamilialPeripheral Nervous System DiseasePeripheral Nervous System DiseasesMetabolism, Inborn ErrorsMetabolic Diseases
Interventions
BIOLOGICAL

nexiguran ziclumeran

nexiguran ziclumeran 55 mg by single IV infusion

DRUG

Normal Saline as Placebo

Normal saline (0.9% NaCl) by single IV infusion

Trial Locations (16)

Unknown

RECRUITING

Hospital Británico de Buenos Aires, Buenos Aires

RECRUITING

Hospital Italiano de Buenos Aires (HIBA), Buenos Aires

RECRUITING

Hospital El Cruce, San Juan Bautista

RECRUITING

Westmead Hospital, Westmead

RECRUITING

Hospital das Clinicas da Universidade Estadual de Campinas (UNICAMP), Campinas

RECRUITING

HCFMRP - Hospital das Clinicas da Faculdade de Medicina de Ribeirao Preto, Ribeirão Preto

RECRUITING

Instituto de Educacao, Pesquisa e Gestao em Saude, Rio de Janeiro

RECRUITING

PSEG Centro de Pesquisa Clinica S.A., São Paulo

RECRUITING

National Institute of Medical Sciences and Nutrition - Salvador Zubiran (INCMNSZ), Tlalpan

RECRUITING

New Zealand Clinical Research, Auckland

RECRUITING

National University Hospital, Singapore

RECRUITING

Singapore General Hospital, Singapore

ACTIVE_NOT_RECRUITING

National Taiwan University Hospital, Taipei

ACTIVE_NOT_RECRUITING

Taipei Veterans General Hospital, Taipei

ACTIVE_NOT_RECRUITING

Chang Gung Medical Foundation - Linkou Branch, Taoyuan District

RECRUITING

Siriraj Hospital, Bangkok

Sponsors
All Listed Sponsors
collaborator

Regeneron Pharmaceuticals

INDUSTRY

lead

Intellia Therapeutics

INDUSTRY

NCT06672237 - A Phase 3 Study of NTLA-2001 in ATTRv-PN | Biotech Hunter | Biotech Hunter