ENVISION: A Study to Evaluate the Efficacy and Safety of Givosiran (ALN-AS1) in Patients With Acute Hepatic Porphyrias (AHP)

• Status: Completed
• Trial end date: 2021-05-31
• Target enrollment: 0
• Participant gender: All

Summary

The purpose of this study is to evaluate the effect of subcutaneous givosiran (ALN-AS1), compared to placebo, on the rate of porphyria attacks in patients with Acute Hepatic Porphyrias (AHP).

• Phase: Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Alnylam Pharmaceuticals

Criteria

Inclusion Criteria:

• ≥ 12 years of age

• Diagnosed with Acute Hepatic Porphyria (Acute Intermittent Porphyria, Hereditary Corproporhyria, Variegate Porphyria, aminolevulinic acid (ALA) dehydratase deficient porphyria)

• Elevated urinary or plasma porphobilinogen (PBG) or ALA values within the past year,

• Have active disease, with at least 2 documented porphyria attacks within the last 6 months

• Willing to discontinue or not initiate the use of prophylactic hemin throughout the study.

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

Exclusion Criteria:

• Clinically significant abnormal laboratory results

• Anticipated liver transplantation

• History of multiple drug allergies or intolerance to subcutaneous injections

• Active HIV, hepatitis C virus, or hepatitis B virus infection(s)

• History of recurrent pancreatitis