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nexiguran ziclumeran

Phase 3

Neuromuscular Disease | Monoclonal antibody | Rare Disease |Intellia Therapeutics, Inc.|Last Updated: Apr 16, 2026

Success Probability
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Trial Design
RandomizedDouble-BlindPLACEBO_CONTROLLEDDMC
Total Trials1
Total Enrollment60
FDA Designations
RMATORPHAN_DRUG
Clinical trial landscape

nexiguran ziclumeran · 1 trial · 20 indications

Phase 3 1
NCT06672237A Phase 3 Study of NTLA-2001 in ATTRv-PNNeuromuscular Disease
RECRUITING60 Analytics
PHASE3RECRUITING
A Phase 3 Study of NTLA-2001 in ATTRv-PN
Neuromuscular DiseaseUnlock trial analytics
Study Endpoints
Primary Endpoints
Modified Neuropathy Impairment Score +7 (mNIS+7)
18 Months
Serum TTR
29 Days
Secondary Endpoints
Norfolk Quality of Life-Diabetic Neuropathy (Norfolk QoL-DN) Questionnaire
18 Months
Modified Body Mass Index (mBMI)
18 Months
Serum TTR
18 Months
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Study Design & Arms
AllocationRANDOMIZED
MaskingQUADRUPLE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
nexiguran ziclumeranEXPERIMENTALnexiguran ziclumeran 55 mg by single IV infusion
Normal SalinePLACEBO_COMPARATORPlacebo; Normal saline (0.9% NaCl) by single IV infusion
Interventions
NameTypeDescription
nexiguran ziclumeranBIOLOGICALnexiguran ziclumeran 55 mg by single IV infusion
Normal Saline as PlaceboDRUGNormal saline (0.9% NaCl) by single IV infusion
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Eligibility Criteria
Age Range18 Years to 85 Years
SexALL
Healthy VolunteersNo
Study Sites14

Inclusion Criteria: * Diagnosis of ATTRv-PN * Karnofsky Performance Status (KPS) ≥ 60 Exclusion Criteria: * Other causes of amyloidosis (amyloidosis caused by non-TTR protein) * Other known causes of sensorimotor or autonomic neuropathy * Diabetes mellitus * New York Heart Association Class III o...

Countries:ArgentinaBrazilMexicoSingaporeTaiwanThailand
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Recent Changes (Last 90 Days)
LOWMay 26, 2026NCT06672237Status: ACTIVE_NOT_RECRUITING → RECRUITING
LOWMay 24, 2026NCT06672237studyFirstPostDate: changed