A Phase 3 Study of NTLA-2001 in ATTRv-PN
← catalyst calendarNCT06672237 · readout ≤ 346 d
Sponsored by Intellia Therapeutics (industry) · NTLA — their whole pipeline →. With Regeneron Pharmaceuticals.
Phase
Phase 3
Status
Recruiting
Study type
Interventional
Design
Randomized · Quadruple · Treatment
Enrollment
60estimated
Sites
14
Countries
Argentina, Brazil, Mexico +3
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 | 2024-11-22 | actual | When the study began enrolling |
| Primary completion | Jul 2027 | estimated | The date the last participant is measured for the primary outcome — the readout window |
| Study completion | Aug 2028 | estimated | The whole study's end, after follow-up |
| First posted | 2024-11-04 | actual | When this record first appeared on the registry |
| Results posted | — | When the sponsor posted results to the registry | |
| Record updated | 2026-04-16 | actual | The sponsor's own last edit to this record — every projection above is as current as this date |
What it studies
Conditions
- Neuromuscular Disease
- Neuromuscular Diseases (NMD)
- Neurodegenerative Disease
- Neurodegenerative Disease, Hereditary
- Neurodegenerative Diseases
- Neuromuscular Diseases
- Nerve Disorders
- Nervous System Disease
- Nervous System Diseases
- Genetic Disease, Inborn
- Amyloidosis, Familial
- Amyloidosis, Hereditary
- Amyloidosis
- Polyneuropathies
- Amyloid Neuropathies
- Amyloid Neuropathies, Familial
- Peripheral Nervous System Disease
- Peripheral Nervous System Diseases
- Metabolism, Inborn Errors
- Metabolic Diseases
Interventions
- Biological: nexiguran ziclumeran — also filed as NTLA-2001
- Drug: Normal Saline as Placebo
Primary outcomes
what the primary-completion date above is the date OFModified Neuropathy Impairment Score +7 (mNIS+7)
measured 18 Months
Serum TTR
measured 29 Days
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