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NCT Number: NCT06672237

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

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.

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Key information

Conditions

Neuromuscular Disease Amyloid Neuropathies Amyloid Neuropathies, Familial Amyloidosis Amyloidosis, Familial Amyloidosis, Hereditary Amyloidosis, Hereditary, Transthyretin-Related Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Disease, Inborn Genetic Diseases, Inborn Heredodegenerative Disorders, Nervous System Metabolic Diseases Metabolism, Inborn Errors Nerve Disorders Nervous System Disease Nervous System Diseases Neurodegenerative Disease Neurodegenerative Disease, Hereditary Neurodegenerative Diseases Neuromuscular Diseases Neuromuscular Diseases (NMD) Nutritional and Metabolic Diseases Peripheral Nervous System Disease Peripheral Nervous System Diseases Polyneuropathies Proteostasis Deficiencies

Age range

18 year–85 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

Hospital Británico de Buenos Aires, Buenos Aires, Argentina

Loading trial locations.

About this study

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 placebo. To ensure all participants will have the potential to receive nexiguran ziclumeran, participants will have the option to cross over to the opposite study arm at Month 12 or Month 18, depending on study criteria.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

nexiguran ziclumeran

Biological

nexiguran ziclumeran 55 mg by single IV infusion

Other names: NTLA-2001

Normal Saline as Placebo

Drug

Normal saline (0.9% NaCl) by single IV infusion

Primary outcomes

  1. Modified Neuropathy Impairment Score +7 (mNIS+7)

    Time frame: 18 Months

  2. Serum TTR

    Time frame: 29 Days

Secondary outcomes

  1. Norfolk Quality of Life-Diabetic Neuropathy (Norfolk QoL-DN) Questionnaire

    Time frame: 18 Months

  2. Modified Body Mass Index (mBMI)

    Time frame: 18 Months

  3. Serum TTR

    Time frame: 18 Months

Study contacts

Contact information is provided by the study sponsor or research team.

Trial Manager at Intellia

CONTACT

[email protected]

1-857-285-6200 ext. 6

Sponsors and collaborators

Lead sponsor

Intellia Therapeutics

Industry

Collaborators

  • Regeneron Pharmaceuticals

Registry information

Official study title

MAGNITUDE-2: A Phase 3, Multinational, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of NTLA-2001 in Participants With Hereditary Transthyretin Amyloidosis With Polyneuropathy (ATTRv-PN)

Important dates

Study start
2024
Primary completion
2027
Study completion
2028
First posted
Nov 4, 2024
Registry last updated
Apr 16, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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