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

Phase 1
Recruiting
Conditions
Transthyretin-Mediated Amyloidosis
Interventions
Drug: Placebo
Registration Number
NCT05661916
Lead Sponsor
Alnylam Pharmaceuticals
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.

Detailed Description

Not available

Recruitment & Eligibility

Status
RECRUITING
Sex
All
Target Recruitment
180
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 & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
PlaceboPlaceboParticipants will be administered a single dose of placebo.
ALN-TTRSC04ALN-TTRSC04Participants will be administered a single dose of ALN-TTRSC04.
Primary Outcome Measures
NameTimeMethod
Frequency of Adverse EventsUp to 20 months
Secondary Outcome Measures
NameTimeMethod
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
Time to Maximum Observed Plasma Concentration (Tmax) of ALN-TTRSC04Day 1 up to Day 4
Maximum Observed Plasma Concentration (Cmax) of ALN-TTRSC04Day 1 up to Day 4
Change from Baseline in Serum Transthyretin (TTR) Levels Over TimeUp to 18 months

Trial Locations

Locations (1)

Clinical Trial Site

🇬🇧

London, United Kingdom

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