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

Intellia Therapeutics

Status and phase: Enrolling; Phase 3

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

Treatments: Drug: Normal Saline as Placebo; Biological: nexiguran ziclumeran

Study type: Interventional

Funder types: Industry

Identifiers

NCT06672237

ITL-2001-CL-311

Details and patient eligibility

About

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.

Full description

This is a multinational, multicenter, double-blind, placebo-controlled study in approximately 50 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.

Enrollment: 50 estimated patients

Sex: All

Ages: 18 to 85 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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50 participants in 2 patient groups, including a placebo group

nexiguran ziclumeran

Experimental group

Description:

nexiguran ziclumeran 55 mg by single IV infusion

Treatment:

Biological: nexiguran ziclumeran

Normal Saline

Placebo Comparator group

Description:

Placebo; Normal saline (0.9% NaCl) by single IV infusion

Treatment:

Drug: Normal Saline as Placebo