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
Immunohistochemical Algorithm for the Diagnosis and Classification of Hepatocellular Carcinomas With TERT, TP53 and CTNNB1 Mutation
The presence of certain mutations in HCC, such as TERT and TP53, have a suspected or proven prognostic role in resected patients, but lack a morphological or immunophenotypic counterpart, which would allow us to define which tumours are rationally candidate for molecular biology analysis. The identification of a histological and immunohistochemical algorithm to determine which HCCs to take to NGS for prognostic purposes will save time and economic costs in the laboratory by rationalising available resources. In the future, the application of the immunohistochemical and molecular algorithm on biopsy could allow better prognosis and predictivity even before surgery/therapy.
Photon Radiotherapy Plus Tremelimumab/Durvalumab for BCLC Stage B and C HCC
Tremelimumab plus durvalumab (the STRIDE regimen) is an approved first-line therapy for unresectable hepatocellular carcinoma (HCC); however, it demonstrates limited efficacy, with an objective response rate (ORR) of only 20.1%. Radiation therapy (RT) is highly effective in controlling localized solid tumors and has become an integral component of the treatment algorithm for unresectable HCC. Preclinical studies have shown that combining RT with PD-L1/PD-1 blockade promotes immunogenic cell death and enhances antigen presentation by dendritic cells, thereby boosting systemic T cell-mediated antitumor responses in mouse models. The addition of CTLA-4 inhibition further enhances antigen cross-priming following RT. Recent retrospective data also indicate that combining RT with immune-oncology agents is associated with improved overall survival and prolonged time to progression compared to RT or immunotherapy alone. However, the clinical benefit and immunologic impact of combining RT with tremelimumab and durvalumab have not yet been evaluated in prospective clinical trials for unresectable HCC. This phase II, single-arm clinical trial aims to assess the safety, efficacy, and immunologic effects of combining proton RT with tremelimumab and durvalumab in patients with unresectable HCC.