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

February 13, 2026 updated by: Alnylam Pharmaceuticals

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

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

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

96

Phase

  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

14 years to 61 years (Adult, Older Adult)

Accepts Healthy Volunteers

Yes

Description

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).

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Triple

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: ALN-TTRSC04
Participants will be administered a single dose of ALN-TTRSC04.
Drug: ALN-TTRSC04
ALN-TTRSC04 will be administered by subcutaneous (SC) injection.
Placebo Comparator: Placebo
Participants will be administered a single dose of placebo.
Drug: Placebo
Placebo will be administered by SC injection.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Frequency of Adverse Events
Time Frame: Up to 20 months
Up to 20 months

Secondary Outcome Measures

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

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Alnylam Pharmaceuticals

Investigators

  • Study Director: Medical Director, Alnylam Pharmaceuticals Inc

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

January 16, 2023

Primary Completion (Actual)

September 30, 2025

Study Completion (Actual)

September 30, 2025

Study Registration Dates

First Submitted

December 15, 2022

First Submitted That Met QC Criteria

December 15, 2022

First Posted (Actual)

December 22, 2022

Study Record Updates

Last Update Posted (Actual)

February 17, 2026

Last Update Submitted That Met QC Criteria

February 13, 2026

Last Verified

February 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • ALN-TTRSC04-001

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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