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

A Phase 1, Single-ascending Dose, Multiple-ascending Dose, and Multi-dose Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics Study of Subcutaneously Administered ALN AS1 in Patients With Acute Intermittent Porphyria (AIP)

The purpose of this study is to evaluate the safety and tolerability of givosiran (ALN-AS1) in AIP patients as well as to characterize pharmacokinetics (PK) and pharmacodynamics (PD) of ALN-AS1 in AIP patients.

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 intolerance to subcutaneous injection