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

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

Status
Completed
Phases
Phase 1
Study type
Interventional
Source
ClinicalTrials.gov
Registry ID
NCT05661916
Enrollment
96
Registered
2022-12-22
Start date
2023-01-16
Completion date
2025-09-30
Last updated
2026-02-17

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Transthyretin-Mediated Amyloidosis

Keywords

ATTR amyloidosis, siRNA

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.

Interventions

DRUGALN-TTRSC04

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

DRUGPlacebo

Placebo will be administered by SC injection.

Sponsors

Alnylam Pharmaceuticals
Lead SponsorINDUSTRY

Study design

Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
TRIPLE (Subject, Caregiver, Investigator)

Eligibility

Sex/Gender
ALL
Age
18 Years to 65 Years
Healthy volunteers
Yes

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).

Design outcomes

Primary

MeasureTime frame
Frequency of Adverse EventsUp to 20 months

Secondary

MeasureTime frame
Change from Baseline in Serum Transthyretin (TTR) Levels Over TimeUp to 18 months
Maximum Observed Plasma Concentration (Cmax) of ALN-TTRSC04Day 1 up to Day 4
Time to Maximum Observed Plasma Concentration (Tmax) of ALN-TTRSC04Day 1 up to Day 4
Area Under the Concentration-time Curve (AUC) of ALN-TTRSC04Day 1 up to Day 4
Fraction of ALN-TTRSC04 Excreted in the Urine (fe)Up to Day 2

Countries

United Kingdom

Contacts

STUDY_DIRECTORMedical Director

Alnylam Pharmaceuticals Inc

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 18, 2026