Clinical Trial: The Study of an Investigational Drug, ALN-TTR02 (Patisiran), for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis in Patients Who Have Already Been Treated With ALN-TTR02 (Patisiran)

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

Official Title: A Phase 2, Multicenter, Open-Label, Extension Study to Evaluate the Long-Term Safety, Clinical Activity, and Pharmacokinetics of ALN-TTR02 in Patients With Familial Amyloidotic Polyneuropathy Who Have

Brief Summary: The purpose of this study is to evaluate the safety and tolerability of long-term dosing with ALN-TTR02 (patisiran) in patients with transthyretin (TTR) mediated amyloidosis (ATTR).

Detailed Summary:
Sponsor: Alnylam Pharmaceuticals

Current Primary Outcome: Safety and tolerability of long-term dosing with ALN-TTR02 (patisiran) in ATTR patients [ Time Frame: Up to 56 days post last dose ]

Original Primary Outcome: Safety and tolerability of long-term dosing with ALN-TTR02 in ATTR patients [ Time Frame: Up to 56 days post last dose ]

Current Secondary Outcome:

  • Assessment of changes from baseline in serum TTR lowering associated with long-term dosing of ALN-TTR02 (patisiran) [ Time Frame: Up to 56 days post last dose ]
  • Assessment of changes from baseline in neurologic impairment associated with long-term dosing of ALN-TTR02 (patisiran) [ Time Frame: Up to 56 days post last dose ]
  • Assessment of changes from baseline in quality of life and disability associated with long-term dosing of ALN-TTR02 (patisiran) [ Time Frame: Up to 56 days post last dose ]
  • Assessment of changes from baseline in motor function associated with long-term dosing of ALN-TTR02 (patisiran) [ Time Frame: Up to 56 days post last dose ]
  • Assessment of changes from baseline in nutritional status associated with long-term dosing of ALN-TTR02 (patisiran) [ Time Frame: Up to 56 days post last dose ]


Original Secondary Outcome:

  • Assessment of changes from baseline in serum TTR lowering associated with long-term dosing of ALN-TTR02 [ Time Frame: Up to 56 days post last dose ]
  • Assessment of changes from baseline in neurologic impairment associated with long-term dosing of ALN-TTR02 [ Time Frame: Up to 56 days post last dose ]
  • Assessment of changes from baseline in quality of life and disability associated with long-term dosing of ALN-TTR02 [ Time Frame: Up to 56 days post last dose ]
  • Assessment of changes from baseline in motor function associated with long-term dosing of ALN-TTR02 [ Time Frame: Up to 56 days post last dose ]
  • Assessment of changes from baseline in nutritional status associated with long-term dosing of ALN-TTR02 [ Time Frame: Up to 56 days post last dose ]


Information By: Alnylam Pharmaceuticals

Dates:
Date Received: October 9, 2013
Date Started: October 2013
Date Completion:
Last Updated: October 17, 2016
Last Verified: October 2016