Neuromuscular DiseasePhase 3NCT06672237
A Phase 3 Study of NTLA-2001 in ATTRv-PN
A Phase 3 study testing nexiguran ziclumeran in people with Neuromuscular Disease and Neuromuscular Diseases (NMD), run by Intellia Therapeutics at 14 sites in 6 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 85 years
- Conditions the study is for
- Neuromuscular Disease, Neuromuscular Diseases (NMD), Neurodegenerative Disease
- What is being tested?
- nexiguran ziclumeran
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 60 participants
- Where
- 14 sitesin 6 countries
- Study dates
- 22 Nov 2024to Aug 2028
- Phase
- Phase 3
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 85 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Diagnosis of ATTRv-PN
- Karnofsky Performance Status (KPS) ≥ 60
Who cannot join
- 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
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- 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
Trial locations and contacts
14 sites in 6 countries. Contact the study team or a site near you to ask about taking part.
14 sites in 6 countries
Brazil4 sites, 4 recruiting
- Recruiting
Hospital das Clinicas da Universidade Estadual de Campinas (UNICAMP)
Campinas
- Recruiting
HCFMRP - Hospital das Clinicas da Faculdade de Medicina de Ribeirao Preto
Ribeirão Preto
- Recruiting
Instituto de Educacao, Pesquisa e Gestao em Saude
Rio de Janeiro
- Recruiting
PSEG Centro de Pesquisa Clinica S.A.
São Paulo
Argentina3 sites, 3 recruiting
- Recruiting
Hospital Británico de Buenos Aires
Buenos Aires
- Recruiting
Hospital Italiano de Buenos Aires (HIBA)
Buenos Aires
- Recruiting
Hospital El Cruce
San Juan Bautista
Taiwan3 sites
- Active, not recruiting
National Taiwan University Hospital
Taipei
- Active, not recruiting
Taipei Veterans General Hospital
Taipei
- Active, not recruiting
Chang Gung Medical Foundation - Linkou Branch
Taoyuan
Singapore2 sites, 2 recruiting
- Recruiting
National University Hospital
Singapore
- Recruiting
Singapore General Hospital
Singapore
Mexico1 site, 1 recruiting
- Recruiting
National Institute of Medical Sciences and Nutrition - Salvador Zubiran (INCMNSZ)
Tlalpan
About this study
Official 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)
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.
Read the 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 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.
- Conditions
- Neuromuscular Disease, Neuromuscular Diseases (NMD), Neurodegenerative Disease, Neurodegenerative Disease, Hereditary, Neurodegenerative Diseases, Neuromuscular Diseases
- Being tested
- nexiguran ziclumeran, Normal Saline as Placebo
- Keywords
- TTR, Amyloidosis, Polyneuropathy, NTLA-2001, ATTR, ATTR-PN, ATTRv-PN, Transthyretin, TTR-mediated amyloidosis, Amyloidosis, hereditary, Amyloidosis, hereditary, transthyretin-related amyloidosis, Transthretin amyloid polyneuropathy, TTR PN, TTR polyneuropathy, nexiguran ziclumeran, nex-z, CRISPR
- Medical subject headings
- Neuromuscular Diseases, Neurodegenerative Diseases, Heredodegenerative Disorders, Nervous System, Nervous System Diseases, Genetic Diseases, Inborn, Amyloidosis, Familial, Amyloidosis, Polyneuropathies
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
nexiguran ziclumeranBiological
Also called NTLA-2001
nexiguran ziclumeran 55 mg by single IV infusion
Normal Saline as PlaceboDrug
Normal saline (0.9% NaCl) by single IV infusion
| Study group | Role | What they receive |
|---|---|---|
| nexiguran ziclumeran | Experimental | nexiguran ziclumeran 55 mg by single IV infusion Receives: Biological: nexiguran ziclumeran |
| Normal Saline | Placebo comparator | Placebo; Normal saline (0.9% NaCl) by single IV infusion Receives: Drug: Normal Saline as Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Modified Neuropathy Impairment Score +7 (mNIS+7)
Measured: 18 Months
Serum TTR
Measured: 29 Days
Secondary outcomes (3)
Norfolk Quality of Life-Diabetic Neuropathy (Norfolk QoL-DN) Questionnaire
Measured: 18 Months
Modified Body Mass Index (mBMI)
Measured: 18 Months
Serum TTR
Measured: 18 Months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Nov 2024
- Study start22 Nov 2024
- Primary completionJul 2027 (estimated)
- Study completionAug 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Neuromuscular DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- Participants will be randomized in a 1:1 ratio to nexiguran ziclumeran or placebo with opportunity for crossover.
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 60 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Intellia Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Regeneron Pharmaceuticals
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06672237
- Sponsor's protocol ID
- ITL-2001-CL-311
- Status last verified
- Apr 2026
- First submitted
- 1 Nov 2024
- Last updated
- 16 Apr 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No