A Phase 3 Study of NTLA-2001 in ATTRv-PN
Recruiting now · Phase 3 · Has a placebo group
Conditions studied: 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
In brief
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.
Key facts
- Study ID
- NCT06672237
- Run by
- Intellia Therapeutics
- People needed
- 60
- Starts
- 2024-11-22
- Expected to finish
- 2028-08-01
- Last updated by the study team
- 2026-04-16
Who can join
Age: 18 and older, up to 85. Sex: any. Healthy volunteers: not accepted.
You may qualify if…
- Diagnosis of ATTRv-PN
- Karnofsky Performance Status (KPS) ≥ 60
You may not qualify if…
- 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 or IV heart failure
- Liver failure
- Hepatitis B, hepatitis C or human immunodeficiency virus (HIV) infection
- Prior receipt of a TTR silencer (Small interfering RNA (siRNA) or Antisense oligonucleotides (ASOs))
- Estimated Glomerular Filtration Rate < 30 mL/min/1.73 m2
- Unable or unwilling to take vitamin A supplementation for the duration of the study
- History of liver disease
Where it is running
- Siriraj Hospital — Bangkok, Thailand (enrolling)
- Hospital Italiano de Buenos Aires (HIBA) — Buenos Aires, Argentina (enrolling)
- Hospital El Cruce — San Juan Bautista, Argentina (enrolling)
- Hospital das Clinicas da Universidade Estadual de Campinas (UNICAMP) — Campinas, Brazil (enrolling)
- HCFMRP - Hospital das Clinicas da Faculdade de Medicina de Ribeirao Preto — Ribeirão Preto, Brazil (enrolling)
- Instituto de Educacao, Pesquisa e Gestao em Saude — Rio de Janeiro, Brazil (enrolling)
- PSEG Centro de Pesquisa Clinica S.A. — São Paulo, Brazil (enrolling)
- National Institute of Medical Sciences and Nutrition - Salvador Zubiran (INCMNSZ) — Tlalpan, Mexico (enrolling)
- National University Hospital — Singapore, Singapore (enrolling)
- Singapore General Hospital — Singapore, Singapore (enrolling)
- Hospital Británico de Buenos Aires — Buenos Aires, Argentina (enrolling)
- Taipei Veterans General Hospital — Taipei, Taiwan
- Chang Gung Medical Foundation - Linkou Branch — Taoyuan, Taiwan
- National Taiwan University Hospital — Taipei, Taiwan
Full record on ClinicalTrials.gov
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