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

• Status: Completed
• Trial end date: 2017-09-06
• Target enrollment: 0
• Participant gender: All

Summary

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.

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

Details

• Lead Sponsor: Alnylam Pharmaceuticals

Criteria

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