What the trial was testing
The ILLUMINATE-A enrolled 39 patients with kidney 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
Lumasiran reduced urinary oxalate by 65%, with 84% of patients reaching normal or near-normal levels after 6 months.
The New England journal of medicine · 2021 · NCT03681184
These findings — that reached normal or near-normal urinary oxalate levels after 6 months of treatment — were published in the The New England journal of medicine and represent the headline result of the study.
Researchers tracked outcomes across 39 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 kidney 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
Lumasiran is FDA-approved for primary hyperoxaluria type 1. It's given as an injection under the skin and works by reducing the liver's production of oxalate, helping prevent kidney damage. If you have PH1, ask your doctor if lumasiran is right for you.
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 kidney disease trials
Prevention of Nephrotoxin-Induced Acute Kidney Injury Using Cilastatin
The goal of this clinical trial is to repurpose cilastatin for preventing acute kidney injury (AKI) in hospitalized patients receiving nephrotoxic medications. The trial will evaluate the efficacy of the re-purposed drug. The main questions it aims to answer are: \- whether cilastatin will prevent nephrotoxic AKI in hospitalized patients. Researchers will compare the drug cilastatin to a placebo (a look-alike substance that contains no drug) to see if drug cilastatin works to prevent AKI in hospitalized patients receiving nephrotoxic medications. Participants will: * Receive drug Cilastatin or a placebo intravenously every 6 hours for up to 24 hours after last exposure to nephrotoxic medication * Have blood test for kidney function every day they are on treatment. * Have a follow-up blood test at 90 days after randomization * Have a telephone survey at 90 days after randomization
Nephrotic Syndrome Study Network
Minimal change disease (MCD), focal segmental glomerulosclerosis (FSGS), and Membranous nephropathy (MN), generate an enormous individual and societal financial burden, accounting for approximately 12% of prevalent end stage renal disease (ESRD) cases (2005) at an annual cost in the US of more than $3 billion. However, the clinical classification of these diseases is widely believed to be inadequate by the scientific community. Given the poor understanding of MCD/FSGS and MN biology, it is not surprising that the available therapies are imperfect. The therapies lack a clear biological basis, and as many families have experienced, they are often not beneficial, and in fact may be significantly toxic. Given these observations, it is essential that research be conducted that address these serious obstacles to effectively caring for patients. In response to a request for applications by the National Institutes of Health, Office of Rare Diseases (NIH, ORD) for the creation of Rare Disease Clinical Research Consortia, a number of affiliated universities joined together with The NephCure Foundation the NIDDK, the ORDR, and the University of Michigan in collaboration towards the establishment of a Nephrotic Syndrome (NS) Rare Diseases Clinical Research Consortium. Through this consortium the investigators hope to understand the fundamental biology of these rare diseases and aim to bank long-term observational data and corresponding biological specimens for researchers to access and further enrich.