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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT06672237




Registration number
NCT06672237
Ethics application status
Date submitted
1/11/2024
Date registered
4/11/2024

Titles & IDs
Public title
A Phase 3 Study of NTLA-2001 in ATTRv-PN
Scientific 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)
Secondary ID [1] 0 0
ITL-2001-CL-311
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Neuromuscular Disease 0 0
Neuromuscular Diseases (NMD) 0 0
Neurodegenerative Disease 0 0
Neurodegenerative Disease, Hereditary 0 0
Neurodegenerative Diseases 0 0
Neuromuscular Diseases 0 0
Nerve Disorders 0 0
Nervous System Disease 0 0
Nervous System Diseases 0 0
Genetic Disease, Inborn 0 0
Amyloidosis, Familial 0 0
Amyloidosis, Hereditary 0 0
Amyloidosis 0 0
Polyneuropathies 0 0
Amyloid Neuropathies 0 0
Amyloid Neuropathies, Familial 0 0
Peripheral Nervous System Disease 0 0
Peripheral Nervous System Diseases 0 0
Metabolism, Inborn Errors 0 0
Metabolic Diseases 0 0
Condition category
Condition code
Neurological 0 0 0 0
Other neurological disorders
Neurological 0 0 0 0
Neurodegenerative diseases
Musculoskeletal 0 0 0 0
Other muscular and skeletal disorders
Metabolic and Endocrine 0 0 0 0
Metabolic disorders
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Metabolic and Endocrine 0 0 0 0
Other metabolic disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Other - nexiguran ziclumeran
Treatment: Drugs - Normal Saline as Placebo

Experimental: nexiguran ziclumeran - nexiguran ziclumeran 55 mg by single IV infusion

Placebo comparator: Normal Saline - Placebo; Normal saline (0.9% NaCl) by single IV infusion


Treatment: Other: nexiguran ziclumeran
nexiguran ziclumeran 55 mg by single IV infusion

Treatment: Drugs: Normal Saline as Placebo
Normal saline (0.9% NaCl) by single IV infusion
Intervention code [1] 0 0
Treatment: Other
Intervention code [2] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Modified Neuropathy Impairment Score +7 (mNIS+7)
Timepoint [1] 0 0
18 Months
Primary outcome [2] 0 0
Serum TTR
Timepoint [2] 0 0
29 Days
Secondary outcome [1] 0 0
Norfolk Quality of Life-Diabetic Neuropathy (Norfolk QoL-DN) Questionnaire
Timepoint [1] 0 0
18 Months
Secondary outcome [2] 0 0
Modified Body Mass Index (mBMI)
Timepoint [2] 0 0
18 Months
Secondary outcome [3] 0 0
Serum TTR
Timepoint [3] 0 0
18 Months

Eligibility
Key inclusion criteria
* Diagnosis of ATTRv-PN
* Karnofsky Performance Status (KPS) = 60
Minimum age
18 Years
Maximum age
85 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key 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

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
22/11/2024
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/08/2028
Actual
Sample size
Target
50
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
Recruitment outside Australia
Country [1] 0 0
New Zealand
State/province [1] 0 0
Auckland

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Intellia Therapeutics
Address
Country
Other collaborator category [1] 0 0
Commercial sector/industry
Name [1] 0 0
Regeneron Pharmaceuticals
Address [1] 0 0
Country [1] 0 0

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Trial Manager at Intellia
Address 0 0
Country 0 0
Phone 0 0
1-857-285-6200
Fax 0 0
Email 0 0
medicalinformation@intelliatx.com
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
No/undecided IPD sharing reason/comment


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results not provided in https://clinicaltrials.gov/study/NCT06672237