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

ENVISION: A phase 3 randomized, double-blind, placebo-controlled multicenter study with an open-label extension to evaluate the efficacy and safety of givosiran in patients with acute hepatic porphyrias

Status

Active, not recruiting

Phases

Phase 3

Study type

Interventional

Source

JPRN

Registry ID

JPRN-jRCT2080223832

Enrollment

Registered

2018-03-05

Start date

2018-05-30

Completion date

Unknown

Last updated

2026-06-29

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Acute hepatic porphyrias (AHP)

Interventions

investigational material(s) Generic name etc : Givosiran Sodium INN of investigational material : Givosiran Therapeutic category code : 399 Agents affecting metabolism, n.e.c. Dosage and Administratio

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: 1.>=12 years of age 2.Diagnosed with Acute Hepatic Porphyria (Acute Intermittent Porphyria, Hereditary Corproporhyria, Variegate Porphyria, ALA dehydratase deficient porphyria) 3.Elevated urinary or plasma PBG or ALA values within the past year 4.Have active disease, with at least 2 documented porphyria attacks within the last 6 months 5.Willing to discontinue or not initiate the use of prophylactic hemin throughout the study 6.Women of child bearing potential must have a negative serum pregnancy test, not be nursing, and use acceptable contraception

Exclusion criteria

Exclusion criteria: 1.Clinically significant abnormal laboratory results 2.Anticipated liver transplantation 3.History of multiple drug allergies or intolerance to subcutaneous injections 4.Active HIV, hepatitis C virus, or hepatitis B virus infection(s) 5.History of recurrent pancreatitis

Design outcomes

Primary

Measure	Time frame
efficacy The annualized rate of porphyria attacks in patients with AIP	—

Secondary

Measure	Time frame
safety efficacy -The pharmacodynamic (PD) effect of Givosiran on urine levels of delta-aminolevulinic acid (ALA) in patients with AIP -The pharmacodynamic (PD) effect of Givosiran on urine levels of Porphobilinogen (PBG) in patients with AIP -Annualized rate of hemin administrations in AIP patients -Annualized rate of porphyria attacks in patients with AHP -Pain as measured by the Brief Pain Inventory-Short Form numeric rating scale, a 0 to 10 point scale with 10 rated as worst pain -Nausea as measured by the numeric rating scale, a 0 to 10 point scale with 10 rated as worst nausea -Fatigue as measured by the Brief Fatigue Inventory-Short Form numeric rating scale, a 0 to 10 point scale with 10 rated as worst fatigue -Change from baseline in the Physical Component Summary of the 12-Item Short Form Survey (SF-12)	—

Measure

Time frame

safety efficacy -The pharmacodynamic (PD) effect of Givosiran on urine levels of delta-aminolevulinic acid (ALA) in patients with AIP -The pharmacodynamic (PD) effect of Givosiran on urine levels of Porphobilinogen (PBG) in patients with AIP -Annualized rate of hemin administrations in AIP patients -Annualized rate of porphyria attacks in patients with AHP -Pain as measured by the Brief Pain Inventory-Short Form numeric rating scale, a 0 to 10 point scale with 10 rated as worst pain -Nausea as measured by the numeric rating scale, a 0 to 10 point scale with 10 rated as worst nausea -Fatigue as measured by the Brief Fatigue Inventory-Short Form numeric rating scale, a 0 to 10 point scale with 10 rated as worst fatigue -Change from baseline in the Physical Component Summary of the 12-Item Short Form Survey (SF-12)

—

Countries

Asia except Japan, Europe, Japan, North America, Oceania, South America

Outcome results

None listed

Source: JPRN (via WHO ICTRP) · Data processed: Jul 3, 2026