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Kidney DiseaseApril 2021Summary reviewed June 2026

What the ILLUMINATE-A Trial Found — Lumasiran for Primary Hyperoxaluria Type 1

Researchers tested lumasiran, an RNA-based treatment, in people with primary hyperoxaluria type 1 (PH1), a rare genetic disease where the liver makes too much oxalate, leading to kidney stones and kidney failure. After 6 months, lumasiran reduced urinary oxalate by 65% compared to placebo, and 84% of patients reached normal or near-normal levels.

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.