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Updated May 2026 · ClinicalTrials.gov

RECRUITINGPhase 3INTERVENTIONAL

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

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)

A Phase 3 Study of NTLA-2001 in ATTRv-PN (NCT06672237) is a Phase 3 interventional studying Neuromuscular Disease and Neuromuscular Diseases (NMD), sponsored by Intellia Therapeutics. RECRUITING as of the most recent ClinicalTrials.gov update. Talk to your doctor before contacting the trial site.

Important: This information is not medical advice. Talk to your doctor about whether a clinical trial is right for you.

About This Trial

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.

What Stage of Research Is This?

Phase 3 trials confirm efficacy and safety in large patient groups (often 300–3,000+) and form the evidence base for an FDA approval submission. For Neuromuscular Disease, Phase 3 studies typically randomize participants between the investigational treatment and either a placebo or current standard of care. A successful Phase 3 result is the threshold most treatments need to clear before regulatory approval.

This trial is currently recruiting participants. The sponsor has registered the study with ClinicalTrials.gov as actively enrolling, which means new applicants who meet the eligibility criteria can be considered for screening. Trial status can change between updates — confirm current recruiting status with the study contact before traveling for a screening visit.

Target enrollment of 60 participants puts this in the typical range for a Phase 2-style efficacy study or a moderate Phase 3 trial in a focused Neuromuscular Disease subpopulation. At this scale, the study has enough statistical power to detect a clear treatment effect but is not the largest cohort in the field.

Who May Be Eligible (Plain English)

Who May Qualify: - Diagnosis of ATTRv-PN - Karnofsky Performance Status (KPS) ≥ 60 Who Should NOT Join This Trial: - 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 weakened immune system 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 Always talk to your doctor about whether this trial is right for you.

These are translations of the protocol\'s inclusion and exclusion criteria, simplified for patients and caregivers. The original clinical text appears below. Eligibility is ultimately confirmed by the trial site\'s screening process — this summary is a starting point for a conversation with your doctor, not a final determination.

Original Eligibility Criteria

View original clinical language
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

Treatments Being Tested

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

Locations (14)

Trial sites listed on ClinicalTrials.gov for this study. Site activation status can vary — confirm with the specific site before traveling for a screening visit.

Hospital Británico de Buenos Aires
Buenos Aires, Argentina
Hospital Italiano de Buenos Aires (HIBA)
Buenos Aires, Argentina
Hospital El Cruce
San Juan Bautista, Argentina
Hospital das Clinicas da Universidade Estadual de Campinas (UNICAMP)
Campinas, Brazil
HCFMRP - Hospital das Clinicas da Faculdade de Medicina de Ribeirao Preto
Ribeirão Preto, Brazil
Instituto de Educacao, Pesquisa e Gestao em Saude
Rio de Janeiro, Brazil
PSEG Centro de Pesquisa Clinica S.A.
São Paulo, Brazil
National Institute of Medical Sciences and Nutrition - Salvador Zubiran (INCMNSZ)
Tlalpan, Mexico
National University Hospital
Singapore, Singapore
Singapore General Hospital
Singapore, Singapore
National Taiwan University Hospital
Taipei, Taiwan
Taipei Veterans General Hospital
Taipei, Taiwan
Chang Gung Medical Foundation - Linkou Branch
Taoyuan, Taiwan
Siriraj Hospital
Bangkok, Thailand

How to Talk to Your Doctor About This Trial

Bring the printable summary of this trial — including the NCT ID (NCT06672237), the sponsor (Intellia Therapeutics), and the key eligibility criteria — to your next appointment. Your doctor can review the inclusion and exclusion criteria against your medical history, lab values, and current treatments to assess whether you are likely to qualify. They can also help you weigh whether trial participation makes sense alongside your existing care plan.

Useful questions to walk through together: What does the trial protocol require beyond standard care? How long is the active treatment phase, and how long is follow-up? Are there study visits at sites I can reach? Who pays for the trial-specific procedures, and who pays for standard-of-care portions? See our 25 questions to ask about clinical trials guide for a more complete checklist.

Authoritative Sources

The official record for this trial lives on ClinicalTrials.gov — the federal registry maintained by the National Library of Medicine at NIH. For background on how this trial fits into the FDA approval pathway, see the FDA drug approval process. For oncology-specific guidance for patients considering trials, the National Cancer Institute publishes patient-oriented overviews. International trial registries are aggregated by the WHO ICTRP.

Frequently Asked Questions

What is the NCT06672237 clinical trial studying?

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. The full protocol is registered on ClinicalTrials.gov and includes the primary outcome measures, eligibility criteria, and study endpoints.

Who can participate in NCT06672237?

Eligibility for this trial depends on the specific inclusion and exclusion criteria set by the sponsor. The plain-English summary above translates the most important criteria into accessible language; the official clinical text is preserved in the collapsible section underneath. Whether you fit any specific trial is a medical decision your doctor needs to confirm — bring the trial information to your treating physician for a full review against your medical history.

How do I contact the trial site for NCT06672237?

Contact information registered with ClinicalTrials.gov is shown in the sidebar of this page. Before reaching out, confirm with your treating physician that this trial is appropriate for your situation. The trial site will then walk you through the screening process to determine final eligibility.

Is participating in a clinical trial safe?

Clinical trials in the United States are regulated by the FDA and overseen by Institutional Review Boards (IRBs) that review the protocol for safety. Risk varies by trial — Phase 1 studies test new treatments in humans for the first time, while Phase 3 trials use treatments that have already passed earlier safety screening. The informed consent document for any specific trial details the known risks and what to expect. Discuss those risks with your physician before deciding whether to participate.

Where can I verify the data on this page?

Every detail on this page comes directly from the ClinicalTrials.gov API. Click "View on ClinicalTrials.gov" in the sidebar to see the official, unmodified record. The federal record is always authoritative; this page is a structured presentation with a plain-English eligibility translation. For background on how clinical trials are regulated, see the FDA drug approval process documentation.

How This Page Is Built

Every field on this page is pulled directly from the ClinicalTrials.gov API v2 — no estimates, no proxies. The plain-English eligibility translation is generated from the original protocol text and reviewed for fidelity to the underlying clinical criteria. The original clinical text remains visible in the collapsible section above so users and clinicians can verify the translation. Read the full methodology for the data pipeline and known limitations.

Source: ClinicalTrials.gov API v2 record for NCT06672237. Maintained by the National Library of Medicine at NIH. Public domain. Cite as: "TrialFinderData. NCT06672237. Data: ClinicalTrials.gov."

Medical disclaimer: This page is informational, not medical advice. Talk to your doctor about whether a clinical trial is right for you.

Last updated 2026-05-08 · Data from ClinicalTrials.gov.