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Clinical trial

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)

NCT04601051 is registered in ClinicalTrials.gov with Intellia Therapeutics as lead sponsor.

NCT NCT04601051
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Registry record

As filed in ClinicalTrials.gov.

Title
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)
Lead sponsor
Intellia Therapeutics
Phase
Phase 1
Overall status
Completed
Start date
November 5, 2020
Completion date
September 12, 2025
Enrollment
72 (actual)
Sites
4

Conditions

As the registry lists them; a condition with its own page is linked.

transthyretin-related (attr) familial amyloid cardiomyopathy, transthyretin-related (attr) familial amyloid polyneuropathy, wild-type transthyretin cardiac amyloidosis