A Phase 1 Study of Givosiran (ALN-AS1) in Patients With Acute Intermittent Porphyria (AIP)

Parts A and B Inclusion Criteria: * Diagnosis of AIP * Urine PBG at Screening indicating patient is a high excreter * No clinically significant health concerns * Women of child bearing potential must have a negative pregnancy test, not be nursing, and use effective contraception * Willing to provide written informed consent and willing to comply with study requirements. Exclusion Criteria: * Porphyria attack within 6 months of screening * Started a new prescription medication within 3 months of screening * Clinically significant abnormal laboratory results * Received an investigational agent 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 Part C Inclusion Criteria: * Diagnosis of AIP * Patient experienced a porphyria attack or was taking medication to prevent attacks recently * No clinically significant health concerns * Women of child bearing potential must have a negative pregnancy test, not be nursing, and use effective contraception * Willing to provide written informed consent and willing to comply with study requirements. Exclusion Criteria: * Stared a new prescription medication within 3 months of screening * Clinically significant abnormal laboratory results * Received an investigational agent 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 intole…