Clinical Trial: A Study to Evaluate Long-term Safety and Clinical Activity of ALN-AS1 in Patient With Acute Intermittent Porphyria (AIP)

Study Status: Enrolling by invitation
Recruit Status: Enrolling by invitation
Study Type: Interventional

Official Title: A Multicenter, Open-label Extension Study to Evaluate the Long-term Safety and Clinical Activity of Subcutaneously Administered ALN-AS1 in Patients With Acute Intermittent Porphy

Brief Summary: The purpose of this study is to determine the long-term safety, tolerability and pharmacokinetics of ALN-AS1 in AIP patients

Detailed Summary:
Sponsor: Alnylam Pharmaceuticals

Current Primary Outcome: The safety of ALN-AS1 evaluated by the proportion of patients experiencing adverse events [ Time Frame: Through Month 42 ]

Original Primary Outcome: Same as current

Current Secondary Outcome:

  • The pharmacodynamic (PD) effect of ALN-AS1 on urine levels of delta-aminolevulinic acid (ALA) [ Time Frame: Through Month 42 ]
  • The pharmacodynamic (PD) effect of ALN-AS1 on urine levels of Porphobilinogen (PBG) [ Time Frame: Through Month 42 ]
  • The frequency of porphyria attacks as measured by number of attacks [ Time Frame: Through Month 42 ]
  • Frequency of hematin administrations [ Time Frame: Through Month 42 ]


Original Secondary Outcome: Same as current

Information By: Alnylam Pharmaceuticals

Dates:
Date Received: October 28, 2016
Date Started: October 2016
Date Completion: August 2020
Last Updated: January 18, 2017
Last Verified: January 2017