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

• Status: Active, not recruiting
• Trial end date: 2021-11-04
• Target enrollment: 0
• Participant gender: All

Summary

The purpose of this study is to determine the long-term safety, tolerability and pharmacokinetics of givosiran (ALN-AS1) in AIP patients

• Phase: Phase 1/Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Alnylam Pharmaceuticals

Criteria

Inclusion Criteria:

• Completed participation in Part C of study ALN-AS1-001

• Not on a scheduled regimen of hemin

• Women of child bearing potential must have a negative serum pregnancy test, not be nursing, and use acceptable contraception

• Willing and able to comply with the study requirements and to provide written informed consent

Exclusion Criteria:

• Clinically significant abnormal laboratory results

• Received an investigational agent (other than ALN-AS1) within 90 days before the first dose of study drug or are in follow-up of another clinical study

• History of multiple drug allergies or intolerance to subcutaneous injection