ENVISION: A Study to Evaluate the Efficacy and Safety of Givosiran (ALN-AS1) in Patients With Acu… (NCT03338816) | Clinical Trial Compass
CompletedPhase 3
ENVISION: A Study to Evaluate the Efficacy and Safety of Givosiran (ALN-AS1) in Patients With Acute Hepatic Porphyrias (AHP)
United States94 participantsStarted 2017-11-16
Plain-language 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).
Who can participate
Age range12 Years
SexALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
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
What they're measuring
1
Annualized Rate of Porphyria Attacks in Participants With Acute Intermittent Porphyria (AIP)