Primary Hyperoxaluria Type 3PHYOX-OBXNCT04542590
Natural History of Patients With PH3 and a History of Stone Events
An observational study of Primary Hyperoxaluria Type 3, run by Dicerna Pharmaceuticals, Inc., a Novo Nordisk company at 6 sites in 5 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 2 years and older
- Conditions the study is for
- Primary Hyperoxaluria Type 3
- How many people
- 7 participants
- Where
- 6 sitesin 5 countries
- Study dates
- 9 Sep 2021to 23 Jul 2024
- How long will I take part?
- 2 Years
- Phase
- Observational
- Are there results?
- None posted in the registry
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 2 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: This is a natural history study in adults, adolescents, and children (starting from birth) with genetically confirmed PH3 who have a history of stone events during the last 3 years and/or the presence of preexisting stones detected by renal ultrasounds at Screening, and an eGFR > 30 mL/min/1.73 m2 or serum creatinine below the 97th percentile for patients younger than 12 months old.
Who may be able to join
- 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
Show all 9Show fewer
- > 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
Who cannot join
- Prior or planned liver transplant within study period
- Currently receiving dialysis or anticipating dialysis during study period
- Unwillingness to comply with study procedures
Trial locations and contacts
6 sites in 5 countries.
6 sites in 5 countries
United States2 sites
Clinical Trial Site
Boston, Massachusetts, 02115
Clinical Trial Site
New York, New York, 10016
About this study
Official title: A Natural History Study of Patients With Genetically Confirmed Primary Hyperoxaluria Type 3 and, as Applicable Per Age, a History of Stone Events
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.
Read the detailed description
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.
Potential participants are screened over an up-to-7 week period according to the eligibility criteria and will then be followed every 6 months over an up-to-2-year period to determine the annualized new stone formation rate (number of new stones being formed per year) and the change in the degree of nephrocalcinosis (if applicable).
New stone formation is defined as occurrence of any of the following:
- Spontaneous stone passage in the absence of pre-existing stones
- Stone passage occurring without change in the number of pre-existing stones detected by renal ultrasound
- Appearance of new stones on renal ultrasound or 100% or more growth of a pre-existing stone (estimated area)
- Surgical removal of newly formed stones Note: Spontaneous passage or surgical removal of pre-existing stones does not meet the criteria for new stone formation.
This is a non-interventional study that will last up to 2 years.
- Conditions
- Primary Hyperoxaluria Type 3
- Keywords
- primary hyperoxaluria, PH3, observational, kidney stones, nephrocalcinosis
- Medical subject headings
- Hyperoxaluria, Primary, Kidney Calculi, Nephrocalcinosis
What is being measured
The primary outcome is the main question the study is designed to answer.
Collect stone formation data in PH3 patients
Collect data on the rate of new stone formation in PH3 patients of at least 2 years of age
Measured: Assess participants' stone formation rates over the course of 2 years
Assess relationship between urine oxalate (Uox) levels and stone formations in PH3 patients
Explore the potential relationship between Uox levels and new stone formation in patients (≥ 2 years of age) with genetically confirmed PH3 and relatively intact renal function
Measured: Measure participants' Uox levels over the course of 2 years
Collect data on the degree of nephrocalcinosis in PH3 patients
Collect data on the degree of nephrocalcinosis in PH3 patients of at least 2 years of age
Measured: Assess the change in nephrocalcinosis grade over the course of 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Sep 2020
- Study start9 Sep 2021
- Primary completion23 Jul 2024
- Study completion23 Jul 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Primary Hyperoxaluria Type 3Studies of the same condition, open and under way.
- 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
- 7 participants (actual)
- Target duration
- 2 Years
Sponsor and investigators
- Lead sponsor
- Dicerna Pharmaceuticals, Inc., a Novo Nordisk company
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Verity Rawson, Study director, Dicerna, A Novo Nordisk Company
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04542590
- Sponsor's protocol ID
- DCR-PHXC-502
- Acronym
- PHYOX-OBX
- Status last verified
- Nov 2024
- First submitted
- 20 Aug 2020
- Last updated
- 2 Dec 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 2 Dec 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Natural History Study of Patients With Genetically Confirmed Primary Hyperoxaluria Type 3 and, as Applicable Per Age, a History of Stone Events
- ClinicalTrials.gov ID
- NCT04542590
- Acronym / sponsor ID
- PHYOX-OBX / DCR-PHXC-502
- Conditions
- Primary Hyperoxaluria Type 3
- Keywords
- primary hyperoxaluria, PH3, observational, kidney stones, nephrocalcinosis
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 7 participants (actual)
- Lead sponsor
- Dicerna Pharmaceuticals, Inc., a Novo Nordisk companyIndustry (pharma, biotech, devices)
- Study start
- 9 Sep 2021 (actual)
- Primary completion
- 23 Jul 2024 (actual)
- Study completion
- 23 Jul 2024 (actual)
- First posted
- 9 Sep 2020 (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.
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.
Detailed description
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.
Potential participants are screened over an up-to-7 week period according to the eligibility criteria and will then be followed every 6 months over an up-to-2-year period to determine the annualized new stone formation rate (number of new stones being formed per year) and the change in the degree of nephrocalcinosis (if applicable).
New stone formation is defined as occurrence of any of the following:
- Spontaneous stone passage in the absence of pre-existing stones
- Stone passage occurring without change in the number of pre-existing stones detected by renal ultrasound
- Appearance of new stones on renal ultrasound or 100% or more growth of a pre-existing stone (estimated area)
- Surgical removal of newly formed stones Note: Spontaneous passage or surgical removal of pre-existing stones does not meet the criteria for new stone formation.
This is a non-interventional study that will last up to 2 years.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Target follow-up
- 2 Years
Who may qualify
- Ages
- 2 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- This is a natural history study in adults, adolescents, and children (starting from birth) with genetically confirmed PH3 who have a history of stone events during the last 3 years and/or the presence of preexisting stones detected by renal ultrasounds at Screening, and an eGFR > 30 mL/min/1.73 m2 or serum creatinine below the 97th percentile for patients younger than 12 months old.
Key inclusion criteria (9 listed)
- 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:
- 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 (3 listed)
- Prior or planned liver transplant within study period
- Currently receiving dialysis or anticipating dialysis during study period
- Unwillingness to comply with study procedures
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
Collect stone formation data in PH3 patients
Time frame: Assess participants' stone formation rates over the course of 2 years
Collect data on the rate of new stone formation in PH3 patients of at least 2 years of age
Assess relationship between urine oxalate (Uox) levels and stone formations in PH3 patients
Time frame: Measure participants' Uox levels over the course of 2 years
Explore the potential relationship between Uox levels and new stone formation in patients (≥ 2 years of age) with genetically confirmed PH3 and relatively intact renal function
Collect data on the degree of nephrocalcinosis in PH3 patients
Time frame: Assess the change in nephrocalcinosis grade over the course of 2 years
Collect data on the degree of nephrocalcinosis in PH3 patients of at least 2 years of age
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 2 Dec 2024.
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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. This study is not recruiting at the moment.
Verity Rawson study director
Dicerna, A Novo Nordisk Company
6 sites in 5 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Primary Hyperoxaluria Type 3, run by Dicerna Pharmaceuticals, Inc., a Novo Nordisk company at 6 sites in 5 countries.
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 9 inclusion and 3 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: finished, as of the sponsor's last update on 2 Dec 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 6 sites in United States, Canada, Germany, Poland, United Kingdom. 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?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Collect stone formation data in PH3 patients (time frame: Assess participants' stone formation rates over the course of 2 years). The study lists 3 primary outcomes in all. 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 2 Dec 2024.