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