A Phase 3 Study of NTLA-2001 in ATTRv-PN
Recruiting
Phase 3
Interventional Study
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
Pivotal Trial
This treatment is in the last trial phase before FDA approval.
Prior Safety Data
This treatment has already been tested in at least one earlier human trial.
At a Glance
- Age
- 18 – 85
- Sex
- Any
- Trial phase
- Phase 3
- Study type
- Interventional
- Purpose
- Treatment
- Participants needed
- 60 (estimated)
- Sponsor
- Intellia Therapeutics · Industry
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About This Trial
This study will be conducted to evaluate the efficacy and safety of a single dose of nexiguran ziclumeran (NTLA-2001) compared to placebo in participants with ATTRv-PN.
Trial Locations
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Eligibility Criteria
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…
Contacts