Natural History of Patients With PH3 and a History of Stone Events (NCT04542590) | Clinical Trial Compass
CompletedNot Applicable
Natural History of Patients With PH3 and a History of Stone Events
United States, Canada, Germany7 participantsStarted 2021-09-09
Plain-language summary
This is a natural history study of adults, adolescents, and children (starting at birth) with genetically confirmed primary hyperoxaluria type 3 (PH3) who have a history of stone events during the last 3 years and/or the presence of pre existing stones detected by renal ultrasound at screening.
Who can participate
Age range
2 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Key inclusion criteria
* Genetically confirmed PH3
* For participants at least 2 years of age, history of stone events (defined as presence of calcifications in the urinary tract and/or kidney, their relative location, and the number and size of stones) during the last 3 years and/or presence of pre-existing stones detected by renal ultrasound at Screening
* Uox ≥ 0.7 mmol/24 hours (adjusted per 1.73 m2 BSA in participants \< 18 years of age) OR if not able to collect 24-hour urine, average spot Uox to creatinine ratio at Screening above the 95th percentile for age:
* \> 220 mmol/mol in participants \< 6 months
* \> 170 mmol/mol in participants from 6 months to \< 12 months
* \> 130 mmol/mol in participants 12 months to \< 2 years
* \> 100 mmol/mol in participants from 2 to \< 3 years and
* \> 80 mmol/mol in participants from 3 to 5 years
* eGFR at Screening ≥ 30 mL/min or for infants aged less than 12 months, serum creatinine below the 97th percentile of a healthy population
Key Exclusion Criteria:
* Prior or planned liver transplant within study period
* Currently receiving dialysis or anticipating dialysis during study period
* Unwillingness to comply with study procedures
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Collect stone formation data in PH3 patients
Timeframe: Assess participants' stone formation rates over the course of 2 years
2
Assess relationship between urine oxalate (Uox) levels and stone formations in PH3 patients
Timeframe: Measure participants' Uox levels over the course of 2 years
3
Collect data on the degree of nephrocalcinosis in PH3 patients
Timeframe: Assess the change in nephrocalcinosis grade over the course of 2 years
Trial details
NCT IDNCT04542590
SponsorDicerna Pharmaceuticals, Inc., a Novo Nordisk company