A Phase 3 Study of NTLA-2001 in ATTRv-PN | NCT06672237

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

NCT06672237

Sponsor

Intellia Therapeutics

Status

Recruiting

Last Update Posted

Apr 16, 2026Actual

Enrollment

60Estimated

Phase

Phase 3

Conditions

Neuromuscular Disease
Neuromuscular Diseases (NMD)
Neurodegenerative Disease
Neurodegenerative Disease, Hereditary
Neurodegenerative Diseases
Neuromuscular Diseases
Nerve Disorders
Nervous System Disease
Nervous System Diseases
Genetic Disease, Inborn
Amyloidosis, Familial
Amyloidosis, Hereditary
Amyloidosis
Polyneuropathies
Amyloid Neuropathies
Amyloid Neuropathies, Familial
Peripheral Nervous System Disease
Peripheral Nervous System Diseases
Metabolism, Inborn Errors
Metabolic Diseases

Interventions

nexiguran ziclumeran
Normal Saline as Placebo

Countries

Argentina
Brazil
Mexico
Singapore
Taiwan
Thailand

Protocol Section

Identification Module Status Module Sponsor/Collaborators Module Oversight Module Description Module Conditions Module Design Module Arms/Interventions Module Outcomes Module Eligibility Module Contacts/Locations Module References Module IPD Sharing Statement Module

Results Section

Participant Flow Module Baseline Characteristics Module Outcome Measures Module Adverse Events Module More Info Module

Annotation Section

Annotation Module

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module Condition Browse Module Intervention Browse Module

Protocol Section

Identification Module

NCT ID

Status Module

Record Verification Date

Apr 2026

Overall Recruitment Status or Expanded Access Status

Recruiting

Last Known Status

Not provided

Delayed Posting

Not provided

Why Stopped

Not provided

Expanded Access Info

Start Date

Nov 22, 2024Actual

Primary Completion Date

Jul 2027Estimated

Completion Date

Aug 2028Estimated

First Submitted Date

Nov 1, 2024

First Submission Date that Met QC Criteria

Nov 1, 2024

First Posted Date

Nov 4, 2024Actual

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

Apr 13, 2026

Last Update Posted Date

Apr 16, 2026Actual

Name	Class
Regeneron Pharmaceuticals	INDUSTRY

Oversight Module

Has Data Monitoring Committee (DMC)

Yes

Is FDA Regulated Drug

Yes

Is FDA Regulated Device

Is Unapproved Device

Not provided

Pediatric Postmarket Surveillance of a Device Product

Not provided

Product Exported from US

Yes

FDAAA801 Violation

Not provided

Description Module

Brief 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.

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 Module

Conditions

Neuromuscular Disease
Neuromuscular Diseases (NMD)
Neurodegenerative Disease
Neurodegenerative Disease, Hereditary
Neurodegenerative Diseases
Neuromuscular Diseases
Nerve Disorders
Nervous System Disease
Nervous System Diseases
Genetic Disease, Inborn
Amyloidosis, Familial
Amyloidosis, Hereditary
Amyloidosis
Polyneuropathies
Amyloid Neuropathies
Amyloid Neuropathies, Familial
Peripheral Nervous System Disease
Peripheral Nervous System Diseases
Metabolism, Inborn Errors
Metabolic Diseases

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

Design Module

Study Type

Interventional

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Not provided

Target Follow-Up Duration

Not provided

Phases

Phase 3

Interventional Study Design

Allocation

Biospecimen

No data available

No data is available for this block.

Enrollment

60Estimated

Arms/Interventions Module

Arm Groups

Label	Type	Description	Intervention Names
nexiguran ziclumeran	Experimental	nexiguran ziclumeran 55 mg by single IV infusion	Biological: nexiguran ziclumeran
Normal Saline	Placebo Comparator	Placebo; Normal saline (0.9% NaCl) by single IV infusion	Drug: Normal Saline as Placebo

Interventions

Name	Type	Description	Arm Group Labels	Other Names
nexiguran ziclumeran	Biological	nexiguran ziclumeran 55 mg by single IV infusion	nexiguran ziclumeran

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
Modified Neuropathy Impairment Score +7 (mNIS+7)		18 Months
Serum TTR		29 Days

Secondary Outcomes

Measure	Description	Time Frame
Norfolk Quality of Life-Diabetic Neuropathy (Norfolk QoL-DN) Questionnaire		18 Months
Modified Body Mass Index (mBMI)		18 Months
Serum TTR

Other Outcomes

Not provided

Eligibility Module

Eligibility Criteria

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

Accepts Healthy Volunteers

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

18 Years

Maximum Age

85 Years

Standard Ages

AdultOlder Adult

Study Population

Not provided

Sampling Method

Not provided

Contacts/Locations Module

Central Contacts

Name	Role	Phone	Extension	Email
Trial Manager at Intellia	Contact	1-857-285-6200	6	medicalinformation@intelliatx.com

Overall Officials

Not provided

Locations

Facility	Status	City	State	ZIP	Country	Contacts
Hospital Británico de Buenos Aires	Recruiting	Buenos Aires			Argentina
Hospital Italiano de Buenos Aires (HIBA)

IPD Sharing Statement Module

Plan to Share IPD

Description

Not provided

Types

Not provided

Time Frame

Not provided

Access Criteria

Not provided

URL

Not provided

ID	Term
D009468	Neuromuscular Diseases
D019636	Neurodegenerative Diseases
D020271	Heredodegenerative Disorders, Nervous System
D009422	Nervous System Diseases
D030342	Genetic Diseases, Inborn
D028226	Amyloidosis, Familial
D000686	Amyloidosis
D011115	Polyneuropathies
D017772	Amyloid Neuropathies
D028227	Amyloid Neuropathies, Familial
D010523	Peripheral Nervous System Diseases
D008661	Metabolism, Inborn Errors
D008659	Metabolic Diseases
C567782	Amyloidosis, Hereditary, Transthyretin-Related

ID	Term
D009358	Congenital, Hereditary, and Neonatal Diseases and Abnormalities
D009750	Nutritional and Metabolic Diseases
D057165	Proteostasis Deficiencies

ID	Term
D000077324	Crystalloid Solutions
D007552	Isotonic Solutions
D012996	Solutions
D004364	Pharmaceutical Preparations

ID	Term
C000718870	NTLA-2001
D000077330	Saline Solution