An observational study in Hyperoxaluria, Primary, sponsored by Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico. Enrolling by invitation at 1 site in Italy. Open to participants aged 0 Years to 18 Years. Per ClinicalTrials.gov, last updated 2026-10-14.
Sponsored by Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico · Observational
The primary objective of the study is to confirm, in pediatric patients with type 1 hyperoxaluria treated with Lumasiran, the reduction in urinary oxalate excretion and plasma oxalate levels-already described at six months-on a larger number of patients and with an extended follow-up (1-5 years), and to evaluate in the same patients the ultrasound-detected reduction in the number of kidney stones over time from the initiation of treatment.
47 studies on the registry are indexed under Hyperoxaluria, Primary; 13 are open to participants now.
This study's planned enrollment of 30 is below the median of 365 across 15 observational studies indexed under Hyperoxaluria, Primary.
Browse Hyperoxaluria, Primary studies →Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico is the lead sponsor of 232 studies on the registry; 83 are open to participants now.
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Pediatric patients with type 1 hyperoxaluria undergoing Lumasiran treatment at the participating centers.
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Exclusion Criteria:
Considering both previously diagnosed patients who initiated treatment in the year prior to the study launch and the new diagnoses reasonably expected during the five-year study period, it is anticipated that approximately 30 patients in total will be enrolled across the various Centers affected by this rare disease. This would therefore represent the largest cohort of patients treated with Lumasiran for this pathology to date (in the Illuminate-A trial, which has had the largest sample size so far, only 26 patients were assigned to the treatment group). Our study is necessarily an open-label, single-arm clinical trial (as were Illuminate-B and Illuminate-C). In fact, since the Illuminate-A study provided absolutely convincing evidence of the drug's efficacy in these patients, including a control group would not be ethically justified.
Reduction in urinary oxalate excretion and plasma oxalate levels
The primary objective of the study is to confirm, in pediatric patients affected by type 1 hyperoxaluria and treated with Lumasiran, the reduction in urinary oxalate excretion and plasma oxalate levels-already described at six months-within a larger patient population and over an extended follow-up period (1-5 years), as well as to evaluate the ultrasound-detected reduction in the number of kidney stones in these same patients over time from the initiation of treatment
Time frame: 1-5 years
Plan to share: No — The LUMA-KIDS study does not plan to share Individual Participant Data (IPD). This decision prioritizes the protection of participant confidentiality and aligns with the study's ethical guidelines. As such, no IPD collected throughout the trial will be made available publicly or for secondary research purposes.
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From the registry record's own update history. This site started tracking changes on Sep 25, 2026; for anything earlier, see the record history on ClinicalTrials.gov ↗
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Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico →