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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Phase
Phase 1
Status
Completed
Study type
Interventional
Design
Non randomized · None · Treatment
Enrollment
72actual
Sites
4
Countries
France, New Zealand, Sweden +1
Every value on this page is a field the sponsor filed with ClinicalTrials.gov, reproduced. Dates are their own estimates, revised as a study runs, unless the registry marks them actual. sources →
Source: ClinicalTrials.gov, retrieved 2026-08-19
Dates
each axis at the precision it was filed, with the registry's own basis| Date | Filed | Basis | What it is |
|---|---|---|---|
| Start | 2020-11-05 | actual | When the study began enrolling |
| Primary completion | 2025-09-12 | actual | The date the last participant is measured for the primary outcome — the readout window |
| Study completion | 2025-09-12 | actual | The whole study's end, after follow-up |
| First posted | 2020-10-23 | actual | When this record first appeared on the registry |
| Results posted | — | When the sponsor posted results to the registry | |
| Record updated | 2026-01-30 | actual | The sponsor's own last edit to this record — every projection above is as current as this date |
What it studies
Conditions
- Transthyretin-Related (ATTR) Familial Amyloid Polyneuropathy
- Transthyretin-Related (ATTR) Familial Amyloid Cardiomyopathy
- Wild-Type Transthyretin Cardiac Amyloidosis
Intervention
- Biological: NTLA-2001
Primary outcomes
what the primary-completion date above is the date OFNumber of Participants with Treatment-Emergent Adverse Events
measured up to Day 730
Number of Participants with Clinically Significant Clinical Laboratory Test Findings
measured up to Day 730
Number of Participants with Clinically Significant Safety Measurements
measured up to Day 730
Percent Change from Baseline in Serum TTR (enzyme-linked immunosorbent assay [ELISA])
measured up to Day 730
Percent Change from Baseline in Serum Prealbumin
measured up to Day 730
Mean Area Under the Plasma Concentration-Time Curve from Time Zero to the Time of the Last Measurable Concentration (AUClast) for DMG-PEG2k, LP000001, Cas9 mRNA, and sgRNA
measured up to Day 730
Mean Area Under the Plasma Concentration-Time Curve from Time Zero to Infinity (AUCinf) for DMG-PEG2k, LP000001, Cas9 mRNA, and sgRNA
measured up to Day 730
Mean Maximum Concentration (Cmax) for DMG-PEG2k, LP000001, Cas9 mRNA, and sgRNA
measured up to Day 730
Mean Time of the Maximum Concentration (Tmax) for DMG-PEG2k, LP000001, Cas9 mRNA, and sgRNA
measured up to Day 730
Mean Terminal Half-Life (t½) for DMG-PEG2k, LP000001, Cas9 mRNA, and sgRNA
measured up to Day 730
Mean Apparent Clearance (CL) for DMG-PEG2k, LP000001, Cas9 mRNA, and sgRNA
measured up to Day 730
Mean Volume of Distribution (Vd) for DMG-PEG2k, LP000001, Cas9 mRNA, and sgRNA
measured up to Day 730
Change from Baseline in Anti-Drug Antibody to NTLA-2001 and Anti-Cas9 Protein Antibody to Transgene Product Levels
measured up to Day 730
Publications
- PMID 41002250 — linked by the registry
- PMID 39555828 — linked by the registry
- PMID 34215024 — linked by the registry
- PMID 34175043 — linked by the registry
Linked on the registry record. A “linked by the registry” entry is NLM's own automated PubMed match, not a claim by the sponsor that the paper reports this study.
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