A Phase 1, Randomized, Double-Blind, Placebo Controlled, Single-Ascending Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics (PK), and Pharmacodynamics (PD) of Subcutaneously Administered ALN-TTRSC04 in Healthy Subjects

Alnylam Pharmaceuticals2 sites in 1 country96 target enrollmentJanuary 16, 2023

ConditionsTransthyretin-Mediated Amyloidosis

InterventionsALN-TTRSC04 Placebo

Overview

Phase: Phase 1
Intervention: ALN-TTRSC04
Conditions: Transthyretin-Mediated Amyloidosis
Sponsor: Alnylam Pharmaceuticals
Enrollment: 96
Locations: 2
Primary Endpoint: Frequency of Adverse Events
Status: Completed
Last Updated: 2 months ago

Overview Investigators Eligibility Criteria Arms & Interventions Outcomes Sites Similar Trials

Overview

Brief Summary

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

Registry

clinicaltrials.gov

Start Date

January 16, 2023

End Date

September 30, 2025

Last Updated

2 months ago

Study Type

Interventional

Study Design

Parallel

Sex

All

Investigators

Sponsor

Alnylam Pharmaceuticals

Responsible Party

Sponsor

Eligibility Criteria

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).

Arms & Interventions

ALN-TTRSC04

Participants will be administered a single dose of ALN-TTRSC04.

Intervention: ALN-TTRSC04

Placebo

Participants will be administered a single dose of placebo.

Intervention: Placebo

Outcomes

Primary Outcomes

Frequency of Adverse Events

Time Frame: Up to 20 months

Secondary Outcomes

Area Under the Concentration-time Curve (AUC) of ALN-TTRSC04(Day 1 up to Day 4)
Fraction of ALN-TTRSC04 Excreted in the Urine (fe)(Up to Day 2)
Maximum Observed Plasma Concentration (Cmax) of ALN-TTRSC04(Day 1 up to Day 4)
Time to Maximum Observed Plasma Concentration (Tmax) of ALN-TTRSC04(Day 1 up to Day 4)
Change from Baseline in Serum Transthyretin (TTR) Levels Over Time(Up to 18 months)