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Completed

NCT Number: NCT05661916

A Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of 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.

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Key information

Conditions

Transthyretin-Mediated Amyloidosis

Age range

18 year–65 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1

Primary location

Clinical Trial Site

London, SE1 1YR, United Kingdom

Who can participate

Healthy volunteers accepted: Yes

Only the study team can determine whether someone qualifies for participation.

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).

Treatment and study plan

ALN-TTRSC04

Drug

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

Placebo

Drug

Placebo will be administered by SC injection.

Primary outcomes

  1. Frequency of Adverse Events

    Time frame: Up to 20 months

Secondary outcomes

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

    Time frame: Up to 18 months

  2. Maximum Observed Plasma Concentration (Cmax) of ALN-TTRSC04

    Time frame: Day 1 up to Day 4

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

    Time frame: Day 1 up to Day 4

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

    Time frame: Day 1 up to Day 4

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

    Time frame: Up to Day 2

Sponsors and collaborators

Lead sponsor

Alnylam Pharmaceuticals

Industry

Registry information

Official study title

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

Important dates

Study start
2023
Primary completion
2025
Study completion
2025
First posted
Dec 22, 2022
Registry last updated
Feb 17, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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