| NCT ID | Title | Phase | Status | Enrollment | Velocity | Design | Start | Completion | Last Updated | Sites | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT03338816 | ENVISION: A Study to Evaluate the Efficacy and Safety of Givosiran (ALN-AS1) in Patients With Acute Hepatic Porphyrias (AHP) | PHASE3 | COMPLETED | 94 | — | — | Nov 16, 2017 | May 31, 2021 | Apr 22, 2024 | 36 | United States, Australia +16 |
Porphyria attacks were defined as meeting all of the following criteria: an acute episode of neurovisceral pain in the abdomen, back, chest, extremities and/or limbs, no other medically determined cause, and required treatment with intravenous (IV) dextrose or hemin, carbohydrates, or analgesics, or other medications such as antiemetics at a dose or frequency beyond the participant's usual daily porphyria management. The annualized rate of porphyria attacks is a composite endpoint which included porphyria attacks requiring hospitalization, urgent healthcare visit, or IV hemin administration at home.
| Arm | Type | Description |
|---|---|---|
| Givosiran/Givosiran | EXPERIMENTAL | 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. |
| Placebo/Givosiran | PLACEBO_COMPARATOR | 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. |
| Name | Type | Description |
|---|---|---|
| Givosiran | DRUG | Givosiran by SC |
| Placebo | DRUG | Matching placebo (normal saline \[0.9% NaCl\]) by SC |
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...