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A Phase 3 Study to Evaluate the Efficacy and Safety of Nucresiran in Patients with Hereditary Transthyretin Amyloidosis with Polyneuropathy

Basic Information

Recruitment status	Recruiting
Health condition(s) or Problem(s) studied	Hereditary Transthyretin-Mediated Amyloidosis with Polyneuropathy (hATTR-PN)
Date of first enrollment	26/03/2026
Target sample size	5
Countries of recruitment	Austria,Japan,Belgium,Japan,Cyprus,Japan,The Czech Republic,Japan,Denmark,Japan,France,Japan,Germany,Japan,Greece,Japan,Ireland,Japan,Italy,Japan,The Netherlands,Japan,Portugal,Japan,Spain,Japan,Sweden,Japan,Argentina,Japan,Australia,Japan,Brazil,Japan,Canada,Japan,Malaysia,Japan,Mexico,Japan,South Korea,Japan,Switzerland,Japan,Taiwan,Japan,Turkey,Japan,The United Kingdom,Japan,The United States,Japan
Study type	Interventional
Intervention(s)	Investigational Drug Clinical Trial Component Code: ALN-TTRSC04 Non-Proprietary Name: Nucresiran (INN) Pharmacological Classification Code: 219 Other Cardiovascular Drugs Dosage and Administration: 300 mg subcutaneously once every 6 months. Comparator Drug Clinical Trial Component Code: Vutrisiran Sodium (JAN) Dosage and Administration: 25 mg subcutaneously once every 3 months.

Outcome(s)

Primary Outcome	Change from baseline in the Modified Neuropathy Impairment Score +7 (mNIS+7) compared to the external placebo group from the APOLLO study at Month 9
Secondary Outcome

Key inclusion & exclusion criteria

Age minimum	>= 18age old
Age maximum	<= 85age old
Gender	Both
Include criteria	- Has documented diagnosis of hATTR-PN - Has a diagnosis of hATTR amyloidosis with polyneuropathy with a documented TTR gene variant - Has a neuropathy impairment score (NIS) of 5 to 130 (inclusive) - Has a Karnofsky Performance Status (KPS) of >=60%
Exclude criteria	- Has had a liver transplant or is likely, in the opinion of the Investigator, to undergo liver transplantation during the Treatment Period of the study - Has known other (non-hATTR) forms of amyloidosis or clinical evidence of leptomeningeal amyloidosis - Has a New York Heart Association (NYHA) heart failure classification >2 - Has alanine aminotransferase (ALT) or aspartate aminotransferase (AST) >2.5 upper limit of normal (ULN) - Has total bilirubin >1.5 ULN - Has estimated glomerular filtration rate (eGFR) <=30 mL/min/1.73m^2 - Has other known causes of sensorimotor or autonomic neuropathy