Hyperoxaluria, PrimaryLUMA-KIDSNCT07874074
Follow-up of Pediatric Patients Affected by Primary Hyperoxaluria Type 1 (PH1) Treated With Lumasiran
An observational study of Hyperoxaluria, Primary, run by Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico at 1 site in Italy.
Trial summary
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… Read more about this study
- Who is it for?
- Ages 0 to 18 years
- Conditions the study is for
- Hyperoxaluria, Primary
- How many people
- 30 participants
- Where
- 1 siteItaly
- Study dates
- 6 Jul 2022to 15 Jun 2028
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 0 to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Study population: Pediatric patients with type 1 hyperoxaluria undergoing Lumasiran treatment at the participating centers.
Who may be able to join
- Clinical diagnosis of type 1 hyperoxaluria with genetic confirmation prior to the initiation of treatment.
- Initiation of Lumasiran treatment during the study period, OR
- Initiation of Lumasiran treatment within the year immediately preceding the start of the study, provided that all data regarding the therapy already administered and the subsequent clinical course are available.
Who cannot join
- Patients with type 1 hyperoxaluria who have undergone prior liver or kidney transplantation
- Patients with type 1 hyperoxaluria undergoing dialysis treatment
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
Italy1 site
Fondazione IRCCS Cà Granda Ospedale Maggiore Policlinico
Milan, Lombardy, 20122
About this study
Official title: FOLLOW UP DI PAZIENTI PEDIATRICI AFFETTI DA IPEROSSALURIA DI TIPO 1 IN TRATTAMENTO CON LUMASIRAN
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.
- Conditions
- Hyperoxaluria, Primary
- Medical subject headings
- Hyperoxaluria, Primary
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| Pediatric patients affected by PH1 in treatment with Lumasiran | 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. |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: 1-5 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Oct 2026
- Study start6 Jul 2022
- Primary completion31 Jul 2027 (estimated)
- Study completion15 Jun 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hyperoxaluria, PrimaryStudies of the same condition, open and under way.
- More trials run by Fondazione IRCCS Ca' Granda, Ospedale Maggiore PoliclinicoThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 30 participants (estimated)
Sponsor and investigators
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Giovanni Montini, Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07874074
- Sponsor's protocol ID
- 06.07.2022_0033688_U
- Acronym
- LUMA-KIDS
- Status last verified
- Oct 2026
- First submitted
- 8 Oct 2026
- Last updated
- 14 Oct 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- 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.
Study at a glance
From the registry record, last updated by the sponsor on 14 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- FOLLOW UP DI PAZIENTI PEDIATRICI AFFETTI DA IPEROSSALURIA DI TIPO 1 IN TRATTAMENTO CON LUMASIRAN
- ClinicalTrials.gov ID
- NCT07874074
- Acronym / sponsor ID
- LUMA-KIDS / 06.07.2022_0033688_U
- Conditions
- Hyperoxaluria, Primary
- Study type
- Observational
- Recruitment status
- Enrolling by invitation
- Enrolment
- 30 participants (estimated)
- Lead sponsor
- Fondazione IRCCS Ca' Granda, Ospedale Maggiore PoliclinicoUniversity, hospital or other
- Study start
- 6 Jul 2022 (actual)
- Primary completion
- 31 Jul 2027 (estimated)
- Study completion
- 15 Jun 2028 (estimated)
- First posted
- 14 Oct 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Pediatric patients affected by PH1 in treatment with Lumasiran | – | –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. |
Who may qualify
- Ages
- 0 to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
- Study population
- Pediatric patients with type 1 hyperoxaluria undergoing Lumasiran treatment at the participating centers.
Key inclusion criteria (3 listed)
- Clinical diagnosis of type 1 hyperoxaluria with genetic confirmation prior to the initiation of treatment.
- Initiation of Lumasiran treatment during the study period, OR
- Initiation of Lumasiran treatment within the year immediately preceding the start of the study, provided that all data regarding the therapy already administered and the subsequent clinical course are available.
Key exclusion criteria (2 listed)
- Patients with type 1 hyperoxaluria who have undergone prior liver or kidney transplantation
- Patients with type 1 hyperoxaluria undergoing dialysis treatment
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Reduction in urinary oxalate excretion and plasma oxalate levels
Time frame: 1-5 years
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
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 14 Oct 2026.
- Individual participant dataSharing planned: 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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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Hyperoxaluria, Primary, run by Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico at 1 site in Italy.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: enrolling by invitation, as of the sponsor's last update on 14 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Italy. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Reduction in urinary oxalate excretion and plasma oxalate levels (time frame: 1-5 years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 14 Oct 2026.