Primary Hyperoxaluria Type 1PH1NCT02830009
IDENTIFICATION OF A MULTI-ANALYTE PROFILE FOR PRIMARY HYPEROXALURIA AND COMPARISON WITH HEALTHY SIBLINGS AND IDIOPATHIC HYPERCALCIURIA
A study testing Urines samples and Blood sample in people with Primary Hyperoxaluria Type 1 and Idiopathic Hypercalciuria, run by Hospices Civils de Lyon at 1 site in France.
Trial summary
The aim of this study is to know the difference between protein profiles (multi-analyte profile) of PH1 patients, idiopathic hypercalciuria (IH) patients and PH1 patients 'siblings. Idiopathic hypercalciuria is a less severe kidney disease that PH1, which also leads to the formation of kidney stones. Read more about this study
- Who is it for?
- Any ageHealthy volunteers can take part
- Conditions the study is for
- Primary Hyperoxaluria Type 1, Idiopathic Hypercalciuria
- What is being tested?
- Urines samples and Blood sample
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 5 participants
- Where
- 1 siteFrance
- Study dates
- May 2013to Dec 2015
- Phase
- Interventional
- 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 Any age
- All sexes
- Healthy volunteers accepted
- Child, Adult, Older adult
Who may be able to join
- Diagnosed with primary hyperoxaluria, type 1 (PH1-Cohort A); OR
- Confirmed with AGXT mutation analysis (PH1-Cohort A)
- Diagnosed with idiopathic hypercalciuria (IHC- Cohort B);
- Potential subject diagnosed with PH1 or IH and has both data entered into the registry and has matched, archived random and 24-hour urine specimens obtained prior to any treatment intervention OR is consented and enrolled into the registry or this specific study during the program;
- Healthy siblings of PH1 patients known not to have PH or any another stone disease or chronic disease will be consented and enrolled into this study through the local sites where their sibling is being treated for PH1 (this study meets the criteria for expedited review through local or central IRBs);
- Healthy non-sibling controls known not to have PH or any another stone disease or chronic disease (Healthy Control-Cohort C);
- There is no upper or lower limit to the pediatric age range of enrolling infant, children and adolescent subjects, although it is understood that accurate and complete 24-hour urine collection in very young children and infants will be problematic and will be seriously considered in advance of individual patient or healthy controls enrollment;
- eGFR (Glomerular Filtration Rate) > 60 mL/min x 1.73 m2 with PH1 and IH patient cohorts matched by mean eGFR from their initial study (or registry) enrollment/ data collection.
Who cannot join
- Unwilling to provide written parent consent or adolescent assent to enroll into the International Registry or this study;
- Potential PH1, hypercalciuria, or siblings of PH1 patients with other chronic or acute illness or disease that could potentially confound proteomic results;
- Healthy intra-familial siblings unwilling to provide a blood sample for serum creatinine;
- Unwilling to provide urine specimens or permit data abstraction for the registry or this study.
- Not covered by, or having the right to, Social Security
Trial locations and contacts
1 site in 1 country.
About this study
The aim of this study is to know the difference between protein profiles (multi-analyte profile) of PH1 patients, idiopathic hypercalciuria (IH) patients and PH1 patients 'siblings. Idiopathic hypercalciuria is a less severe kidney disease that PH1, which also leads to the formation of kidney stones.
The aim is to identify patterns of discriminating markers associated with primary hyperoxaluria type 1 (PH1) that will significantly improve clinical diagnosis and prognosis.
- Being tested
- Urines samples, Blood sample
- Medical subject headings
- Primary hyperoxaluria type 1
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Urines samplesOther
24-hour urines will be collected
Blood sampleOther
| Study group | Role | What they receive |
|---|---|---|
| Primary Hyperoxaluria patient | Other | Not described Receives: Other: Urines samples; Other: Blood sample |
| Primary Hyperoxaluria patient's siblings | Other | Not described Receives: Other: Urines samples; Other: Blood sample |
| Idiopathic hypercalciuria patients | Other | Not described Receives: Other: Urines samples; Other: Blood sample |
| Healthy volunteers | Other | Not described Receives: Other: Urines samples; Other: Blood sample |
What is being measured
The primary outcome is the main question the study is designed to answer.
presence of protein markers in urine
Measured: 24 hours
Secondary outcomes (1)
Presence of discriminative and robust protein markers in urine
Measured: 24 hours
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Jul 2016 (estimated)
- Study startMay 2013
- Primary completionJul 2013
- Study completionDec 2015
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Primary Hyperoxaluria Type 1Studies of the same condition, open and under way.
- More trials run by Hospices Civils de LyonThe 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
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Basic science
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 5 participants (actual)
Sponsor and investigators
- Lead sponsor
- Hospices Civils de Lyon
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02830009
- Sponsor's protocol ID
- 2010-604
- Acronym
- PH1
- Status last verified
- Jul 2016
- First submitted
- 8 Jul 2016
- Last updated
- 13 Jul 2016
- Results posted
- No
- Data monitoring committee
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 13 Jul 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- IDENTIFICATION OF A MULTI-ANALYTE PROFILE FOR PRIMARY HYPEROXALURIA AND COMPARISON WITH HEALTHY SIBLINGS AND IDIOPATHIC HYPERCALCIURIA
- ClinicalTrials.gov ID
- NCT02830009
- Acronym / sponsor ID
- PH1 / 2010-604
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 5 participants (actual)
- Lead sponsor
- Hospices Civils de LyonUniversity, hospital or other
- Study start
- May 2013
- Primary completion
- Jul 2013 (actual)
- Study completion
- Dec 2015 (actual)
- First posted
- 12 Jul 2016 (estimated)
- 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 aim of this study is to know the difference between protein profiles (multi-analyte profile) of PH1 patients, idiopathic hypercalciuria (IH) patients and PH1 patients 'siblings. Idiopathic hypercalciuria is a less severe kidney disease that PH1, which also leads to the formation of kidney stones.
The aim is to identify patterns of discriminating markers associated with primary hyperoxaluria type 1 (PH1) that will significantly improve clinical diagnosis and prognosis.
Study design
- Primary purpose
- Basic science
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| Primary Hyperoxaluria patient | Other | Other: Urines samples, Other: Blood sample |
| Primary Hyperoxaluria patient's siblings | Other | Other: Urines samples, Other: Blood sample |
| Idiopathic hypercalciuria patients | Other | Other: Urines samples, Other: Blood sample |
| Healthy volunteers | Other | Other: Urines samples, Other: Blood sample |
Who may qualify
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (8 listed)
- Diagnosed with primary hyperoxaluria, type 1 (PH1-Cohort A); OR
- Confirmed with AGXT mutation analysis (PH1-Cohort A)
- Diagnosed with idiopathic hypercalciuria (IHC- Cohort B);
- Potential subject diagnosed with PH1 or IH and has both data entered into the registry and has matched, archived random and 24-hour urine specimens obtained prior to any treatment intervention OR is consented and enrolled into the registry or this specific study during the program;
- Healthy siblings of PH1 patients known not to have PH or any another stone disease or chronic disease will be consented and enrolled into this study through the local sites where their sibling is being treated for PH1 (this study meets the criteria for expedited review through local or central IRBs);
Key exclusion criteria (5 listed)
- Unwilling to provide written parent consent or adolescent assent to enroll into the International Registry or this study;
- Potential PH1, hypercalciuria, or siblings of PH1 patients with other chronic or acute illness or disease that could potentially confound proteomic results;
- Healthy intra-familial siblings unwilling to provide a blood sample for serum creatinine;
- Unwilling to provide urine specimens or permit data abstraction for the registry or this study.
- Not covered by, or having the right to, Social Security
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Urines samples24-hour urines will be collected | Other | Healthy volunteers, Idiopathic hypercalciuria patients, Primary Hyperoxaluria patient, Primary Hyperoxaluria patient's siblings |
| Blood sample | Other | Healthy volunteers, Idiopathic hypercalciuria patients, Primary Hyperoxaluria patient, Primary Hyperoxaluria patient's siblings |
Primary outcome
presence of protein markers in urine
Time frame: 24 hours
1 secondary outcome
Presence of discriminative and robust protein markers in urine
Time frame: 24 hours
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 13 Jul 2016.
- Individual participant dataSharing planned: No
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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.
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?
A study testing Urines samples and Blood sample in people with Primary Hyperoxaluria Type 1 and Idiopathic Hypercalciuria, run by Hospices Civils de Lyon at 1 site in France.
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 8 inclusion and 5 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 13 Jul 2016. 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 France. 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?
presence of protein markers in urine (time frame: 24 hours). 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 13 Jul 2016.