ENVISION: A Study to Evaluate the Efficacy and Safety of Givosiran (ALN-AS1) in Patients With Acute Hepatic Porphyrias (AHP)
Acute Hepatic Porphyria, Acute Intermittent Porphyria, Porphyria, Acute Intermittent
About this trial
This is an interventional treatment trial for Acute Hepatic Porphyria focused on measuring Acute Intermittent Porphyria (AIP), Acute Hepatic Porphyria (AHP), Hereditary Coproporphyria (HCP), Variegate Porphyria (VP), ALA dehydratase deficient porphyria (ADP) (ALAD), RNAi therapeutic, Porphyria
Eligibility 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
Sites / Locations
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Arms of the Study
Arm 1
Arm 2
Experimental
Placebo Comparator
Givosiran/Givosiran
Placebo/Givosiran
Givosiran 2.5 mg/kg administered subcutaneously (SC), monthly (QM), for 6 months during the 6-Month Double-blind (DB) Period, followed by givosiran 2.5 mg/kg or 1.25 mg/kg SC, QM for 29 months during the Open-label Extension (OLE) Period.
Matching placebo (normal saline [0.9% NaCl]) was administered SC, QM, for 6 months during the 6-Month DB Period, followed by givosiran 2.5 mg/kg or 1.25 mg/kg SC, QM for 29 months during the OLE period.