NCT02830009

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.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
5

participants targeted

Target at below P25 for not_applicable

Timeline
Completed

Started May 2013

Typical duration for not_applicable

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

May 1, 2013

Completed
2 months until next milestone

Primary Completion

Last participant's last visit for primary outcome

July 1, 2013

Completed
2.4 years until next milestone

Study Completion

Last participant's last visit for all outcomes

December 1, 2015

Completed
7 months until next milestone

First Submitted

Initial submission to the registry

July 8, 2016

Completed
4 days until next milestone

First Posted

Study publicly available on registry

July 12, 2016

Completed
Last Updated

July 13, 2016

Status Verified

July 1, 2016

Enrollment Period

2 months

First QC Date

July 8, 2016

Last Update Submit

July 12, 2016

Conditions

Primary Hyperoxaluria Type 1Idiopathic Hypercalciuria

Outcome Measures

Primary Outcomes (1)

  • presence of protein markers in urine

    24 hours

Secondary Outcomes (1)

  • Presence of discriminative and robust protein markers in urine

    24 hours

Study Arms (4)

Primary Hyperoxaluria patient

OTHER
Other: Urines samplesOther: Blood sample

Primary Hyperoxaluria patient's siblings

OTHER
Other: Urines samplesOther: Blood sample

Idiopathic hypercalciuria patients

OTHER
Other: Urines samplesOther: Blood sample

Healthy volunteers

OTHER
Other: Urines samplesOther: Blood sample

Interventions

Urines samplesOTHER

24-hour urines will be collected

Healthy volunteersIdiopathic hypercalciuria patientsPrimary Hyperoxaluria patientPrimary Hyperoxaluria patient's siblings
Blood sampleOTHER
Healthy volunteersIdiopathic hypercalciuria patientsPrimary Hyperoxaluria patientPrimary Hyperoxaluria patient's siblings

Eligibility Criteria

Sexall
Healthy VolunteersYes
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

  • 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.

You may not qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Hospices Civils de Lyon

Lyon/bron, 69500, France

Location

MeSH Terms

Conditions

Primary hyperoxaluria type 1

Interventions

Blood Specimen Collection

Intervention Hierarchy (Ancestors)

Specimen HandlingClinical Laboratory TechniquesDiagnostic Techniques and ProceduresDiagnosisPuncturesSurgical Procedures, OperativeInvestigative Techniques

Study Design

Study Type
interventional
Phase
not applicable
Allocation
NON RANDOMIZED
Masking
NONE
Purpose
BASIC SCIENCE
Intervention Model
PARALLEL
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

July 8, 2016

First Posted

July 12, 2016

Study Start

May 1, 2013

Primary Completion

July 1, 2013

Study Completion

December 1, 2015

Last Updated

July 13, 2016

Record last verified: 2016-07

Data Sharing

IPD Sharing
Will not share

Locations

FR(1)