A Study of Patients with Primary Hyperoxaluria Type 3 with a History of Stone Events
Tab Title Description
Describes the nature of a clinical study. Types include:
- Observational study — observes people and measures outcomes without affecting results.
- Interventional study (clinical trial) — studies new tests, treatments, drugs, surgical procedures or devices.
- Medical records research — uses historical information collected from medical records of large groups of people to study how diseases progress and which treatments and surgeries work best.
- Rochester, Minnesota: 20-010654
NCT ID: NCT04542590
Sponsor Protocol Number: DCR-PHXC-502
About this study
The objective of this study is to collect data on stone formation and the degree of nephrocalcinosis in patients (≥ 2 years of age) with genetically confirmed PH3 and relatively intact renal function and to explore the potential relationship between Uox and new stone formation.
This is a natural history study of adults, adolescents, and children (≥ 2 years of age) 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.
The relationship between the level of Uox and the incidence of kidney stones and/or nephrocalcinosis in patients with PH3 has not been established. The goal of this study is to record 24-hour Uox levels and the incidence of new stone formation and/or the degree of nephrocalcinosis in patients with PH3 over time.
Participant eligibility includes age, gender, type and stage of disease, and previous treatments or health concerns. Guidelines differ from study to study, and identify who can or cannot participate. There is no guarantee that every individual who qualifies and wants to participate in a trial will be enrolled. Contact the study team to discuss study eligibility and potential participation.
- Male and female participants starting at birth.
- Diagnosis of PH3 confirmed by genotyping (historically available genotype information is acceptable for study eligibility).
- For participants at least 2 years of age, history of stone events during the last 3 years and/or presence of preexisting stones detected by renal ultrasound at Screening.
- 24-hour Uox ≥ 0.7 mmol (adjusted per 1.73 m^2 BSA in participants < 18 years of age) on each of the 2 collections during Screening; OR If not able to collect 24-hour urine, average spot Uox to creatinine ratio at Screening above the 95th percentile for age (Matos et al, 1990):
- > 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.
- If able to collect 24-hour urine, less than 20% variation between the two 24-hour urinary creatinine measurements in the Screening period. Individuals who do not achieve < 20% variation between the 2 Screening values may undergo a second round of urine collection. An extra 14 calendar days may be added to the Screening window for participants to complete a second round of urine collection. Should potential participants again fail to achieve < 20% variation, they will be excluded from participation.
- Estimated GFR at Screening ≥ 30 mL/min/1.73 m^2, calculated using the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) equation in participants aged ≥ 18 years (Levey & Stevens, 2010), or the multivariate equation by Schwartz in participants aged 12 months to 17 years (Schwartz et al., 2012); OR For infants aged less than 12 months, serum creatinine below the 97th percentile of a healthy population (Boer et al., 2010).
- Body weight ≥ 5.7 kg.
- Participant (and/or participant’s parent or legal guardian if participant is a minor [defined as patient < 18 years of age, or younger than the age of majority, according to local regulations]) is capable of giving signed informed consent, which includes compliance with the requirements and restrictions listed in the ICF and in this protocol:
- Adolescents (12 to < 18 years of age, or older than 12 years but younger than the age of majority, according to local regulations) must be able to provide written assent for participation;
- For children younger than 12 years of age, assent will be based on local regulations.
- Prior hepatic transplantation; or planned transplantation within the study period.
- Currently receiving dialysis or anticipating requirement for dialysis during the study period.
- Inability or unwillingness to comply with the specified study procedures.
Eligibility last updated 9/14/21. Questions regarding updates should be directed to the study team contact.
Participating Mayo Clinic locations
Study statuses change often. Please contact the study team for the most up-to-date information regarding possible participation.
|Mayo Clinic Location
Mayo Clinic principal investigator
John Lieske, M.D.
Closed for enrollment
Publications are currently not available