NCT05661916

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.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
96

participants targeted

Target at P75+ for phase_1

Timeline
Completed

Started Jan 2023

Typical duration for phase_1

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

December 15, 2022

Completed
7 days until next milestone

First Posted

Study publicly available on registry

December 22, 2022

Completed
25 days until next milestone

Study Start

First participant enrolled

January 16, 2023

Completed
2.7 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

September 30, 2025

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

September 30, 2025

Completed
Last Updated

February 17, 2026

Status Verified

February 1, 2026

Enrollment Period

2.7 years

First QC Date

December 15, 2022

Last Update Submit

February 13, 2026

Conditions

Transthyretin-Mediated Amyloidosis

Keywords

ATTR amyloidosissiRNA

Outcome Measures

Primary Outcomes (1)

  • Frequency of Adverse Events

    Up to 20 months

Secondary Outcomes (5)

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

    Up to 18 months

  • 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

  • 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

Study Arms (2)

ALN-TTRSC04

EXPERIMENTAL

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

Drug: ALN-TTRSC04

Placebo

PLACEBO COMPARATOR

Participants will be administered a single dose of placebo.

Drug: Placebo

Interventions

ALN-TTRSC04DRUG

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

ALN-TTRSC04
PlaceboDRUG

Placebo will be administered by SC injection.

Placebo

Eligibility Criteria

Age18 Years - 65 Years
Sexall
Healthy VolunteersYes
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • Body mass index (BMI) ≥18.0 kg/m\^2 and ≤30 kg/m\^2 assessed at Screening.

You may not qualify if:

  • 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).

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Clinical Trial Site

London, SE1 1YR, United Kingdom

Location

Related Publications (1)

  • Badri P, Kolachana K, Duong A, Lasko M, Nandi T, Mehrotra N, Robbie GJ. Platform Assessment of Concentration-QTc Relationship Across GalNAc-siRNA Molecules. Clin Pharmacokinet. 2026 Feb;65(2):293-311. doi: 10.1007/s40262-025-01606-0. Epub 2025 Dec 12.

    PMID: 41388232DERIVED

Study Officials

  • Medical Director

    Alnylam Pharmaceuticals Inc

    STUDY DIRECTOR

Study Design

Study Type
interventional
Phase
phase 1
Allocation
RANDOMIZED
Masking
TRIPLE
Who Masked
PARTICIPANT, CARE PROVIDER, INVESTIGATOR
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

December 15, 2022

First Posted

December 22, 2022

Study Start

January 16, 2023

Primary Completion

September 30, 2025

Study Completion

September 30, 2025

Last Updated

February 17, 2026

Record last verified: 2026-02

Data Sharing

IPD Sharing
Will not share

Locations

GB(1)