Clinical Trial: A Safety and Tolerability Study of an Investigational Drug, ALN-TTRSC02, in Healthy Subjects

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional

Official Title: A Phase 1, Randomized, Single-Blind, Placebo Controlled, Single-Ascending Dose, Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics Study of Subcutaneously Administered ALN-TTRSC02 in Healthy

Brief Summary: The purpose of this study is to determine the safety, tolerability, pharmacokinetics and pharmacodynamics of ALN-TTRSC02 in healthy subjects.

Detailed Summary:
Sponsor: Alnylam Pharmaceuticals

Current Primary Outcome: Safety of ALN-TTRSC02, evaluated by the proportion of subjects experiencing adverse events (AEs) [ Time Frame: Day 1 through to Day 118 ]

Original Primary Outcome: Same as current

Current Secondary Outcome:

• Profile of pharmacokinetics (PK) of ALN-TTRSC02 [ Time Frame: Day 1 through to Day 3 ]
  Cmax
• Profile of pharmacokinetics (PK) of ALN-TTRSC02 [ Time Frame: Day 1 through to Day 3 ]
  tmax
• Profile of pharmacokinetics (PK) of ALN-TTRSC02 [ Time Frame: Day 1 through to Day 3 ]
  AUC
• Profile of pharmacokinetics (PK) of ALN-TTRSC02 [ Time Frame: Day 1 through to Day 3 ]
  t½
• Profile of pharmacokinetics (PK) of ALN-TTRSC02 [ Time Frame: Day 1 through to Day 3 ]
  CL/F
• Profile of pharmacokinetics (PK) of ALN-TTRSC02 [ Time Frame: Day 1 through to Day 3 ]
  Vss/F
• Profile of pharmacokinetics (PK) of ALN-TTRSC02 [ Time Frame: Day 1 through to Day 3 ]
  Vz/F
• Profile of pharmacokinetics (PK) of ALN-TTRSC02 [ Time Frame: Day 1 through to Day 118 ]
  fe
• Profile of pharmacokinetics (PK) of ALN-TTRSC02 [ Time Frame: Day 1 through to Day 3 ]
  CLR
• Effect of ALN-TTRSC02 on serum TTR levels as measured by reduction from baseline in serum TTR [ Time Frame: Day 1 through to Day 118 ]
• Effect of ALN-TTRSC02 on serum Vitamin A levels as measured by reduction from baseline in serum Vitamin A [ Time Frame: Screening through to Day 118 ]

Original Secondary Outcome:

• Profile of pharmacokinetics (PK) of ALN-TTRSC02 [ Time Frame: Day 1 through to Day 3 ]
  Cmax
• Profile of pharmacokinetics (PK) of ALN-TTRSC02 [ Time Frame: Day 1 through to Day 3 ]
  tmax
• Profile of pharmacokinetics (PK) of ALN-TTRSC02 [ Time Frame: Day 1 through to Day 3 ]
  AUC
• Profile of pharmacokinetics (PK) of ALN-TTRSC02 [ Time Frame: Day 1 through to Day 3 ]
  t½
• Profile of pharmacokinetics (PK) of ALN-TTRSC02 [ Time Frame: Day 1 through to Day 3 ]
  CL/F
• Profile of pharmacokinetics (PK) of ALN-TTRSC02 [ Time Frame: Day 1 through to Day 3 ]
  Vss/F
• Profile of pharmacokinetics (PK) of ALN-TTRSC02 [ Time Frame: Day 1 through to Day 3 ]
  Vz/F
• Profile of pharmacokinetics (PK) of ALN-TTRSC02 [ Time Frame: Day 1 through to Day 3 ]
  fe
• Profile of pharmacokinetics (PK) of ALN-TTRSC02 [ Time Frame: Day 1 through to Day 3 ]
  CLR
• Effect of ALN-TTRSC02 on serum TTR levels as measured by reduction from baseline in serum TTR [ Time Frame: Day 1 through to Day 118 ]
• Effect of ALN-TTRSC02 on serum Vitamin A levels as measured by reduction from baseline in serum Vitamin A [ Time Frame: Screening through to Day 118 ]

Information By: Alnylam Pharmaceuticals

Dates:
Date Received: May 18, 2016
Date Started: May 2016
Date Completion: March 2018
Last Updated: February 10, 2017
Last Verified: February 2017