Pediatric Clinical Trial Initiated for Primary Hyperoxaluria Type 1!

Alnylam Pharmaceuticals has been developing lumasiran as a potential treatment for primary hyperoxaluria type 1 (PH1). This investigation began with ILLUMINATE-A, a clinical trial whose results should be announced by the end of this year. ILLUMINATE-A enrolled 30 participants age 6 and older. The company has just announced the initiation of ILLUMINATE-B, a Phase 3 study, which will enroll 8 participants who range from infancy to age 5. This new trial is an exciting step forward in the development of lumasiran for the pediatric PH1 population.

Lumasiran has already received Breakthrough Therapy Designation and Orphan Drug Designation from the FDA as well as Priority Medicines Designation and Orphan Drug Designation from the EMA.


This new trial is a global trial which will be held at multiple trial sites. The goal of this study is to examine the safety and efficacy of the treatment for pediatric patients.

Lumasiran is a RNAi therapy that is subcutaneously administered. The dose of the treatment in this study will be individualized for each patient, according to their body weight. The primary endpoint of this trial is reduction of urinary oxalate at six months of treatment compared to baseline. Researchers will also investigate eGFR, tolerability, safety, as well as patient’s quality of life.

Results from this trial should be announced by mid-2020.

Phase 2 OLE Study

In addition to the news of this pediatric trial, Alnylam has announced positive results from their Phase 2 OLE study. Results included an evaluation of 18 patients who were taking a variety of doses of lumasiran on a variety of regimens.

Alnylam was highly encouraged by the reductions in urinary oxalate in these patients. The mean reduction was 72% among all patients. Also evaluated was the reduction in urinary 24-hour oxalate:creatinine ratio. The mean reduction was found to be 77% among all patients.

There were only two serious adverse events reported, and neither were related to the treatment. 12 patients did report less serious AEs. No patients needed to discontinue the therapy.

The results from this investigation were recently presented at the ISN 2019 Annual Meeting in Melbourne, Australia. You can view these full results here.

Overall, these clinical updates from Alnylam show continued support for the development of lumasiran for the PH1 patient population and the scientific community is excited about the further investigation of this therapy.

You can read more about these clinical trial updates here.

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