Clinical trial NCT03592030: Diagnostic and Prognostic Value of Miss-1 Study in Children and Adult With Nephrotic Syndrome MISSNEPHROTIQUE
Official title: Diagnostic and Prognostic Value of Miss-1 Study in Children and Adult With Nephrotic Syndrome MISSNEPHROTIQUE
Study type: OBSERVATIONAL
Overall status: UNKNOWN
Conditions: Nephrotic Syndrome
Keywords: Idiopathic nephrotic syndrome
Minimal change diseases
Primitive FSGS
Lead sponsor: Assistance Publique - Hôpitaux de Paris
Sponsor class: OTHER
First posted: 2018-07-19
Enrollment: 150
Sex: ALL
Minimum age (years): 12
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: All nephrotic patients from the participating centers

Brief summary: The nephrotic syndrome is a rare disease defined by a proteinuria \>3g/24h and a hypoalbuminemia \< 30g/L. Genetic and immune are the main causes. The acquired idiopathic nephrotic syndrome presents histologically minimal glomerular lesions, sometimes associated with segmental and focal hyalinosis. The idiopathic nephrotic syndrome (INS) represents 85% of children's glomerular nephropathy and 25-30% of adult's.

Relapses are frequents, and can be pejorative up to 10% and lead to end-stage kidney failure.

Another immune cause is the extramembranous glomerulonephritis mediated by molecular targets specific autoantibodies expressed at the podocytes surface.

Other immune causes include lupus nephropathy, ANCA vascularitis, Goodpasture disease, Berger disease.

Easy diagnosis between these causes can be made with the renal biopsy.

Miss-1, a new protein activated during a inflammatory event, could be an actor in nephrotic syndromes by modifying the podocyte's adhesion on the glomerular basal membrane. This would modulate the structure and function of the slit diaphragm, as well as junctions between the podocyte and the glomerular basal membrane, regulating podocytes' apoptosis.

Detailed description: This project is meant to propose and validate specific and non-invasive diagnostic and prognostic tests for the acquired idiopathic nephrotic syndrome.

These tests rely on the measure of Miss-1 expression in circulating blood cells on flow cytometry and its plasmatic concentration.

To date, no equivalent tests exist to diagnose idiopathic nephrotic syndrome (INS) from other causes.

These simple tests would allow a quick diagnosis of acquired INS by avoiding an invasive renal biopsy. It would also help anticipate the relapses of the disease and guide the treatment modalities as do nowadays the PLA2R antibodies in idiopathic membranous nephropathy.

We will propose the tests to every consent patient, hospitalized in the participating centers (Néphrologie pédiatrique of Robert Debré hospital, Néphrologie adulte of Tenon hospital) with a nephrotic syndrome.

Eligibility criteria: Inclusion Criteria:

* All new hospitalized patient
* Presenting a nephrotic syndrome according to its definition
* For which an anatomopathological diagnostic and its evolution can be or will be carried
* Children of any age can be included if they present a nephrotic syndrome

Exclusion Criteria:

- Patients already treated with glucocorticoids and/or immunosuppressor

Primary outcomes:
measure: Miss1 expression in circulating blood cells on flow cytometry at the time of diagnostic of nephrotic syndrome.
timeFrame: From Day 0 to 1 month
Secondary outcomes:
measure: Miss1 plasmatic concentration at the time of the diagnostic of nephrotic syndrome
timeFrame: From Day 0 to 1 month
measure: Miss1 expression in circulating blood cells on flow cytometry after remission
timeFrame: After remission, up to 1 month
measure: Miss1 plasmatic concentration after remission
timeFrame: After remission, up to 1 month
Other outcomes:
Locations:
city: Paris
country: France
facility: Service de Néphrologie et Dialyses Paris, Hôpital Tenon
Study officials:
affiliation: Assistance Publique - Hôpitaux de Paris
name: Jean-Jacques Boffa, Professor
role: PRINCIPAL_INVESTIGATOR
References:
citation: Shalhoub RJ. Pathogenesis of lipoid nephrosis: a disorder of T-cell function. Lancet. 1974 Sep 7;2(7880):556-60. doi: 10.1016/s0140-6736(74)91880-7.
citation: Eddy AA, Symons JM. Nephrotic syndrome in childhood. Lancet. 2003 Aug 23;362(9384):629-39. doi: 10.1016/S0140-6736(03)14184-0.
citation: van den Berg JG, Weening JJ. Role of the immune system in the pathogenesis of idiopathic nephrotic syndrome. Clin Sci (Lond). 2004 Aug;107(2):125-36. doi: 10.1042/CS20040095.