Status:
RECRUITING
A Phase 3 Study of NTLA-2001 in ATTRv-PN
Lead Sponsor:
Intellia Therapeutics
Collaborating Sponsors:
Regeneron Pharmaceuticals
Conditions:
Neuromuscular Disease
Neuromuscular Diseases (NMD)
Eligibility:
All Genders
18-85 years
Phase:
PHASE3
Brief Summary
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.
Detailed Description
This is a multinational, multicenter, double-blind, placebo-controlled study in approximately 60 participants, who will be randomized 1:1 to receive a single infusion of either nexiguran ziclumeran or...
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 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
Key Trial Info
Start Date :
November 22 2024
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
August 1 2028
Estimated Enrollment :
60 Patients enrolled
Trial Details
Trial ID
NCT06672237
Start Date
November 22 2024
End Date
August 1 2028
Last Update
April 16 2026
Active Locations (14)
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1
Hospital Británico de Buenos Aires
Buenos Aires, Argentina
2
Hospital Italiano de Buenos Aires (HIBA)
Buenos Aires, Argentina
3
Hospital El Cruce
San Juan Bautista, Argentina
4
Hospital das Clinicas da Universidade Estadual de Campinas (UNICAMP)
Campinas, Brazil