ClinicalTrials.gov record NCT06672237

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

Record copied from ClinicalTrials.gov: Oct 10, 2026AI summaries generated: Sep 17, 2026

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)

StatusRecruitingThe study is currently enrolling participants. Only the study team can say whether a particular person can join.
Study typeInterventionalA study in which researchers assign participants to one or more interventions, such as a drug, procedure, device, behavioural programme or other approach, to measure their effects.
PhasePhase 3A later-stage study, usually with more participants, designed to give stronger evidence about how well a treatment works and how safe it is. It often compares the treatment with standard care or a placebo, and results may support an application for approval.
Age18 years old to 85 years old
SexAll
LocationArgentina, Brazil, Mexico, Singapore, Taiwan, Thailand
AI summaryWhat this study is about, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

This randomized, double-blind Phase 3 study compares a single IV infusion of NTLA-2001 with placebo in about 60 people with hereditary transthyretin amyloid polyneuropathy (ATTRv‑PN). Participants are randomized 1:1 and the study measures neuropathy scores and blood markers over 18 months. There is an option to cross over to the other arm at Month 12 or Month 18 under study rules.

What it is studying. The study is testing the efficacy and safety of a single infusion of nexiguran ziclumeran (NTLA-2001) compared with placebo in people with ATTRv‑PN. Key outcomes include change in neuropathy impairment (mNIS+7), serum transthyretin (TTR) levels, quality of life, and nutritional status over follow-up.

What the researchers hope to learn. To evaluate whether a single IV dose of NTLA-2001 affects neuropathy measures (mNIS+7), serum TTR levels, quality of life (Norfolk QoL‑DN), and modified BMI, and to collect safety data.

What this study is about

The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.

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.

Interventions being studied

Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.

Drug
  • Normal Saline as Placebo
Biological
  • nexiguran ziclumeran

Eligibility

The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.

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

Study description and what participation involves

The study's detailed description, as published on ClinicalTrials.gov.

Detailed description from the registry

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.

AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

Participants are randomly assigned (1:1) to receive a single IV infusion of nexiguran ziclumeran (NTLA‑2001, 55 mg) or a saline placebo, with study staff and participants blinded to assignment. You will have follow-up visits and assessments over 18 months, including neuropathy testing (mNIS+7), quality-of-life questionnaires (Norfolk QoL‑DN), weight/BMI checks (mBMI), and blood tests for serum TTR. Some participants may have the option to cross over to the opposite study arm at Month 12 or Month 18 if they meet study criteria. Participation requires willingness to take vitamin A supplements and to complete medical assessments and blood draws as scheduled.

How the study is structured. Randomized, parallel-group, quadruple‑blind (participants, caregivers, investigators, outcomes assessors) Phase 3 trial. About 60 participants are assigned 1:1 to one single IV infusion of NTLA-2001 (55 mg) or placebo (normal saline). Participants are followed for outcomes up to at least 18 months, with an option to cross over to the opposite arm at Month 12 or Month 18 if study criteria are met.

Questions you could ask

These questions are suggestions for a conversation with the study team or your own clinician, not advice.

  • Some participants receive a placebo. How is that decided, and what happens for placebo-group participants when the study ends?
  • The listed sites are in Argentina, Brazil, Mexico, Singapore, Taiwan, Thailand. Is any of them reachable for me, and does the study help with travel?
  • Which of the eligibility criteria would need to be checked with tests before I could join?

More on ATTRv amyloidosis

This trial is also listed on this site under wild-type ATTR amyloidosis.

Clinical trials106 trials listed for ATTRv amyloidosisEvery trial the registry lists for ATTRv amyloidosis, with recruitment status, phase and sites, updated from ClinicalTrials.gov.See all trials →Research updatesWhat changed in ATTRv amyloidosis researchLatest monthly update: September 2026. Includes: Gene-silencing drugs. New research summarized each month in plain language, with links to every source paper.Read the latest update →

Important dates

Study start
Nov 22, 2024
Primary completion
July 2027 (estimated)
Study completion
August 2028 (estimated)
Target enrollment
60 participants (estimated)
Last updated on registry
Apr 16, 2026
Data verified
Oct 10, 2026

Sponsor and research team

The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.

Lead sponsor
Intellia Therapeutics
Collaborators
  • Regeneron Pharmaceuticals

Central study contact

If you're interested in participating, the central contact can answer general questions about the study — including eligibility requirements and how to find a recruiting site near you. Actual eligibility is determined by the site's medical team.

Participating sites (14)

  • Hospital Británico de Buenos AiresRecruiting

    Buenos Aires · Argentina

  • Hospital Italiano de Buenos Aires (HIBA)Recruiting

    Buenos Aires · Argentina

  • Hospital El CruceRecruiting

    San Juan Bautista · Argentina

  • Hospital das Clinicas da Universidade Estadual de Campinas (UNICAMP)Recruiting

    Campinas · Brazil

  • HCFMRP - Hospital das Clinicas da Faculdade de Medicina de Ribeirao PretoRecruiting

    Ribeirão Preto · Brazil

  • Instituto de Educacao, Pesquisa e Gestao em SaudeRecruiting

    Rio de Janeiro · Brazil

  • PSEG Centro de Pesquisa Clinica S.A.Recruiting

    São Paulo · Brazil

  • National Institute of Medical Sciences and Nutrition - Salvador Zubiran (INCMNSZ)Recruiting

    Tlalpan · Mexico

  • National University HospitalRecruiting

    Singapore · Singapore

  • Singapore General HospitalRecruiting

    Singapore · Singapore

Terms used on this page · explanations by MedicalUpdates.ai

placebo
A treatment that looks like the study treatment but contains no active ingredient. Some participants receive it instead of the study treatment, usually without knowing which they got.
Karnofsky Performance Status
A scale from 100 (no complaints) down to 0 that describes how well a person can carry out daily activities. Studies often require a minimum score.
Estimated Glomerular Filtration Rate
A blood-test estimate of how well the kidneys are filtering. Lower numbers mean reduced kidney function; many studies set a minimum value.
Inclusion Criteria
The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
Exclusion Criteria
The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.
double-blind
Two types of people involved in the study do not know which intervention a participant is receiving. The ClinicalTrials.gov record specifies who is masked, for example participants, care providers, investigators or outcome assessors.
placebo-controlled
A study design in which one group receives a placebo so that the study treatment's effects can be compared with no active treatment.
randomized
Participants are assigned to the study groups by chance, like a coin toss, rather than chosen by the doctor or the participant. This is done so the groups are comparable.
1:1
How many participants go to each group. In a 2:1 ratio, two are assigned to the first group for every one assigned to the second.
study arm
One of the groups in a study. Each arm receives a particular intervention, comparator or no intervention, so the groups can be compared.

The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this site; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.

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