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A Phase 3 Study of NTLA-2001 in ATTRv-PN

NCT06672237 · Status: RECRUITING · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 60

Last updated 2026-04-16

No results posted yet for this study

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.

Conditions

  • Genetic Disease, Inborn
  • Amyloidosis, Familial
  • Amyloidosis, Hereditary
  • Amyloidosis
  • Polyneuropathies
  • Amyloid Neuropathies
  • Nervous System Diseases
  • Neuromuscular Disease
  • Neuromuscular Diseases (NMD)
  • Neurodegenerative Disease
  • Neurodegenerative Disease, Hereditary
  • Neurodegenerative Diseases
  • Neuromuscular Diseases
  • Nerve Disorders
  • Nervous System Disease
  • Amyloid Neuropathies, Familial
  • Peripheral Nervous System Disease
  • Peripheral Nervous System Diseases
  • Metabolism, Inborn Errors
  • Metabolic Diseases

Interventions

BIOLOGICAL

nexiguran ziclumeran

nexiguran ziclumeran 55 mg by single IV infusion

DRUG

Normal Saline as Placebo

Normal saline (0.9% NaCl) by single IV infusion

Sponsors & Collaborators

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
PARALLEL

Eligibility

Min Age
18 Years
Max Age
85 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2024-11-22
Primary Completion
2027-07-31
Completion
2028-08-31
FDA Drug
Yes

Countries

  • Argentina
  • Brazil
  • Mexico
  • Singapore
  • Taiwan
  • Thailand

Study Locations

More Related Trials

Entities

Drugs
Diseases
Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT06672237 on ClinicalTrials.gov