Clinical Trial: Trial to Evaluate Safety and Tolerability of ALN-TTR02 in Transthyretin (TTR) Amyloidosis

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

Official Title: A Phase 2, Open-Label, Multi-Dose, Dose Escalation Trial to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Intravenous Infusions of ALN-TTR02 in Patients With TTR

Brief Summary: The purpose of this study is to determine the safety, tolerability, pharmacokinetics and pharmacodynamics of multiple doses of ALN-TTR02 in patients with transthyretin (TTR) mediated amyloidosis (ATTR).

Detailed Summary:
Sponsor: Alnylam Pharmaceuticals

Current Primary Outcome: Safety and tolerability in ATTR patients [ Time Frame: Up to 56 Days ]

The proportion of subjects experiencing adverse events (AEs), serious adverse events (SAEs) and study drug discontinuation


Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Pharmacokinetics (PK) of ALN-TTR02 [ Time Frame: Up to 208 days ]
    Cmax, Area Under Curve, Tmax
  • Serum transthyretin (TTR) protein [ Time Frame: Up to 208 days ]
    Determination of % Lowering to pretreatment/Baseline Levels


Original Secondary Outcome: Same as current

Information By: Alnylam Pharmaceuticals

Dates:
Date Received: June 7, 2012
Date Started: May 2012
Date Completion:
Last Updated: April 22, 2014
Last Verified: April 2014