Back to resultsIDENTIFICATION OF A MULTI-ANALYTE PROFILE FOR PRIMARY HYPEROXALURIA AND COMPARISON WITH HEALTHY SIBLINGS AND IDIOPATHIC HYPERCALCIURIA (PH1)
Primary Purpose
Primary Hyperoxaluria Type 1, Idiopathic Hypercalciuria
Status
Completed
Phase
Not Applicable
Locations
France
Study Type
Interventional
Intervention
Urines samples
Blood sample
Sponsored by
About this trial
This is an interventional basic science trial for Primary Hyperoxaluria Type 1
Eligibility Criteria
Inclusion Criteria:
- 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.
Exclusion Criteria:
- 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
Sites / Locations
- Hospices Civils de Lyon
Arms of the Study
Arm 1
Arm 2
Arm 3
Arm 4
Arm Type
Other
Other
Other
Other
Arm Label
Primary Hyperoxaluria patient
Primary Hyperoxaluria patient's siblings
Idiopathic hypercalciuria patients
Healthy volunteers
Arm Description
Outcomes
Primary Outcome Measures
presence of protein markers in urine
Secondary Outcome Measures
Presence of discriminative and robust protein markers in urine
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT02830009
Brief Title
IDENTIFICATION OF A MULTI-ANALYTE PROFILE FOR PRIMARY HYPEROXALURIA AND COMPARISON WITH HEALTHY SIBLINGS AND IDIOPATHIC HYPERCALCIURIA
Acronym
PH1
Study Type
Interventional
2. Study Status
Record Verification Date
July 2016
Overall Recruitment Status
Completed
Study Start Date
May 2013 (undefined)
Primary Completion Date
July 2013 (Actual)
Study Completion Date
December 2015 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Hospices Civils de Lyon
4. Oversight
Data Monitoring Committee
No
5. Study Description
Brief 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.
The aim is to identify patterns of discriminating markers associated with primary hyperoxaluria type 1 (PH1) that will significantly improve clinical diagnosis and prognosis.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Primary Hyperoxaluria Type 1, Idiopathic Hypercalciuria
7. Study Design
Primary Purpose
Basic Science
Study Phase
Not Applicable
Interventional Study Model
Parallel Assignment
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
5 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Primary Hyperoxaluria patient
Arm Type
Other
Arm Title
Primary Hyperoxaluria patient's siblings
Arm Type
Other
Arm Title
Idiopathic hypercalciuria patients
Arm Type
Other
Arm Title
Healthy volunteers
Arm Type
Other
Intervention Type
Other
Intervention Name(s)
Urines samples
Intervention Description
24-hour urines will be collected
Intervention Type
Other
Intervention Name(s)
Blood sample
Primary Outcome Measure Information:
Title
presence of protein markers in urine
Time Frame
24 hours
Secondary Outcome Measure Information:
Title
Presence of discriminative and robust protein markers in urine
Time Frame
24 hours
10. Eligibility
Sex
All
Accepts Healthy Volunteers
Accepts Healthy Volunteers
Eligibility Criteria
Inclusion Criteria:
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.
Exclusion Criteria:
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
Facility Information:
Facility Name
Hospices Civils de Lyon
City
Lyon/bron
ZIP/Postal Code
69500
Country
France
12. IPD Sharing Statement
Plan to Share IPD
No
Learn more about this trial
IDENTIFICATION OF A MULTI-ANALYTE PROFILE FOR PRIMARY HYPEROXALURIA AND COMPARISON WITH HEALTHY SIBLINGS AND IDIOPATHIC HYPERCALCIURIA
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