A Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ALN-TTRSC04 in Healthy Subjects

Phase 1CompletedNCT05661916

Alnylam Pharmaceuticals96 enrolled

Overview

The purpose of this study is to evaluate the safety, tolerability, pharmacodynamics (PD) and pharmacokinetics (PK) of single ascending doses of ALN-TTRSC04.

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

TRIPLE

Enrollment

Conditions

Transthyretin-Mediated Amyloidosis

Interventions

ALN-TTRSC04DRUG

ALN-TTRSC04 will be administered by subcutaneous (SC) injection.

PlaceboDRUG

Placebo will be administered by SC injection.

Eligibility

Sex: ALLMin age: 18 YearsMax age: 65 YearsHealthy volunteers:

Medical Language ↔ Plain English

Inclusion Criteria: * Body mass index (BMI) ≥18.0 kg/m\^2 and ≤30 kg/m\^2 assessed at Screening. Exclusion Criteria: * Has an estimated glomerular filtration rate (eGFR) \<90 mL/min/1.73m\^2 at screening. * Has alanine aminotransferase (ALT), aspartate aminotransferase (AST) or direct bilirubin above the upper limit of normal (ULN).

Locations (1)

Clinical Trial Site

London, United Kingdom

Outcomes

Primary Outcomes

Frequency of Adverse Events

Time frame: Up to 20 months

Secondary Outcomes

Change from Baseline in Serum Transthyretin (TTR) Levels Over Time

Time frame: Up to 18 months

Maximum Observed Plasma Concentration (Cmax) of ALN-TTRSC04

Time frame: Day 1 up to Day 4

Time to Maximum Observed Plasma Concentration (Tmax) of ALN-TTRSC04

Time frame: Day 1 up to Day 4

Area Under the Concentration-time Curve (AUC) of ALN-TTRSC04

Time frame: Day 1 up to Day 4

Fraction of ALN-TTRSC04 Excreted in the Urine (fe)

Time frame: Up to Day 2

Data from ClinicalTrials.gov

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