What the trial was testing
The ENVISION enrolled 94 patients with liver disease. The study was sponsored by Alnylam Pharmaceuticals and tracked outcomes across the full group of patients who matched the trial's eligibility profile.
It was a large trial designed to confirm whether the treatment works well enough for wider use. Trials at this stage are designed to produce evidence regulators and physicians can act on — not just observations to follow up later.
What the results showed
Patients taking givosiran had 74% fewer porphyria attacks than those on placebo.
The New England journal of medicine · 2020 · NCT03338816
These findings — that people taking givosiran had three-quarters fewer porphyria attacks than those on placebo — were published in the The New England journal of medicine and represent the headline result of the study.
Researchers tracked outcomes across 94 patients enrolled in the trial. The result was consistent enough across the group that the team felt confident reporting it.
What this means for patients
For patients with liver disease, this result changes the calculus on what to ask their care team about. Whether it changes day-to-day care depends on factors like disease subtype, prior treatments, and where the patient is in their care journey.
What you can do now
Givosiran is FDA-approved for acute hepatic porphyria and is given as a monthly injection under the skin. It significantly reduces attacks but can cause liver enzyme changes and kidney function changes that need monitoring. Talk to your doctor about whether givosiran is right for you, especially if you're having frequent attacks.
Eligibility for the treatments mentioned above depends on specific test results and clinical history. Bring this summary, the trial name, and your most recent labs or pathology report to your next visit.
Open liver disease trials
Study of GST-HG131&141 Tablets Compared With Placebo in Patients With Chronic Hepatitis B
A randomized, double-blind, placebo-controlled Phase IIa clinical study to evaluate the safety and efficacy of GST-HG131/GST-HG141 tablets in patients with chronic hepatitis B
Development of a FibroScan Liver Examination Using a Single Probe
This is an exploratory, international, prospective, interventional, multicenter clinical investigation that will take place in 1 Hong Kong site and 3 French sites and 309 adults patients will be included. The study objective is to assess the LSM reproducibility between the FibroScan examination performed with the Single Probe (SP) and the FibroScan examination performed with the reference probes (M and XL).