Phase 1 Two-Part (Open-label, Single Ascending Dose (Part 1) and Open-label, Single Dose Expansion (Part 2)) Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of NTLA-2001 in Patients With Hereditary Transthyretin Amyloidosis With Polyneuropathy (ATTRv-PN) and Patients With Transthyretin Amyloidosis-Related Cardiomyopathy (ATTR-CM)

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Record identity and dates

Source

ClinicalTrials.gov

Source record ID

NCT04601051

Title field or adapter rule

protocolSection.identificationModule.officialTitle

Ingested At

2026-04-25T22:28:31.668954+00:00

Published At

2026-01-30T00:00:00+00:00

Record Updated At

2026-01-30

Timestamp Basis

source retrieval time was not retained by the feed adapter

Date limitation

source retrieval time was not retained by the feed adapter

Fields recorded by this source

protocolSection.conditionsModule.conditions

["Transthyretin-Related (ATTR) Familial Amyloid Polyneuropathy", "Transthyretin-Related (ATTR) Familial Amyloid Cardiomyopathy", "Wild-Type Transthyretin Cardiac Amyloidosis"]

protocolSection.statusModule.lastUpdatePostDateStruct.date

"2026-01-30"

protocolSection.identificationModule.nctId

"NCT04601051"

protocolSection.identificationModule.officialTitle

"Phase 1 Two-Part (Open-label, Single Ascending Dose (Part 1) and Open-label, Single Dose Expansion (Part 2)) Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of NTLA-2001 in Patients With Hereditary Transthyretin Amyloidosis With Polyneuropathy (ATTRv-PN) and Patients With Transthyretin Amyloidosis-Related Cardiomyopathy (ATTR-CM)"

protocolSection.statusModule.overallStatus

"COMPLETED"

protocolSection.designModule.phases

["PHASE1"]

protocolSection.statusModule.resultsFirstPostDateStruct.date

""

Study condition terms

• Transthyretin-Related (ATTR) Familial Amyloid Polyneuropathy (protocolSection.conditionsModule.conditions)
• Transthyretin-Related (ATTR) Familial Amyloid Cardiomyopathy (protocolSection.conditionsModule.conditions)
• Wild-Type Transthyretin Cardiac Amyloidosis (protocolSection.conditionsModule.conditions)

Intervention details are not present in the imported fields for this record. No drug-condition relationship is inferred from co-listing.

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