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

study id #: NCT05661916

condition: Transthyretin-Mediated Amyloidosis

status: Recruiting

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

intervention: Drug: ALN-TTRSC04 Drug: Placebo

results: https://clinicaltrials.gov/study/NCT05661916

last updated: December 12, 2024

start date: 2023-01-16

estimated completion: 2025-09-01

phase of development: Phase 1

size / enrollment: 180

primary outcomes:

• Frequency of Adverse Events - Up to 20 months
  

secondary outcomes:

• 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
  

inclusion criteria:

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).

sponsor: Alnylam Pharmaceuticals

contacts: Alnylam Pharmaceuticals, 1-877-ALNYLAM, [email protected]

investigators: Study Director:Medical Director,Alnylam Pharmaceuticals Inc

trial center locations:

• United Kingdom
  Alnylam Clinical Trial Information Line