Clinical trial NCT02896270: Valproic Acid for Idiopathic Nephrotic Syndrome
Official title: A Prospective Interventional Pilot Study on the Use of Valproic Acid for Treatment of Idiopathic Nephrotic Syndrome
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: PHASE2
PHASE3
Conditions: Idiopathic Nephrotic Syndrome
Focal Segmental Glomerulosclerosis
Minimal Change Disease
Keywords: idiopathic podocytopathies
Neprology
Lead sponsor: Universitair Ziekenhuis Brussel
Sponsor class: OTHER
First posted: 2016-09-12
Enrollment: 15
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: The trial investigates the use of VPA (Valproic Acid) for the treatment of adult patients with biopsy proven idiopathic focal segmentel glomerulosclerosis (FSGS) or minimal change disease (MCD).

VPA used as an add-on to steroids might induce clinical remission in a first category of patients and potentially reduce the dose of maintenance immunosuppression required to maintain remission thereafter.

In a second category of patients VPA might allow the reduction or even cessation of immunosuppression while clinical remission is maintained.

Detailed description: Idiopathic MCD to treat diseases with a considerable associated morbidity and mortality. Current treatment options are limited, have limited efficacy and a considerable side effect profile. Recent findings in a murine model suggest that VPA treatment in an early phase of renal disease could halt or even prevent the development of proteinuria and the progression of kidney damage. VPA is a commonly used and easy available oral antiepileptic agent with a favorable side effect profile compared to the current standard of care agents for podocytopathies.

This trial investigates wether

1. VPA on top of or in substitution of standard of care agents is effective in remission induction in patients with FSGS or MCD with proteinuria resistant to first line therapy with corticosteroids.
2. VPA is effective in remission maintenance allowing reduction and cessation of chronic immunosuppression without relapse in patients with frequently relapsing FSGS or MCD.

Eligibility criteria: Inclusion Criteria:

* Able to give informed consent
* Biopsy proven idiopathic FSGS or MCD
* Organ function:

  * Bilirubin/AST/ALT\< 2 ULN
  * PLT\>100.000 10\*6/L
  * INR 1.5 except if on anti-vitamin K treatment
  * Lipase \<1.5 ULN
  * Creatinine clearance \>30ml/min -

Exclusion Criteria:

* Contraindication for VPA
* Secondary etiologies for FSGS or MCD
* Multiple organ transplantation
* Currently participating in another clinical trial
* Pregnant or lactating women
* Women unwilling to take efficient contraceptive measures for the duration of the study

Primary outcomes:
measure: In remission group induction is the proportion of patients in complete remission
timeFrame: 6 months
measure: In remission maintenance group is the proportion of patients able to reduce maintenance
timeFrame: 6 months
Secondary outcomes:
measure: Determine the disease response by the proportion of subjects with partial remission
timeFrame: 6 - 12 months
measure: Determine the extent to which standard immunosuppression can be reduced
timeFrame: 6 - 12 months
measure: Evaluate the evolution of renal function estimated by MDRD-GFR
timeFrame: 12 months
measure: Evaluate the tolerability of VPA in the setting of idiopathic podocytopathies
timeFrame: 12 months
Other outcomes:
Locations:
city: Brussels
country: Belgium
facility: University Hospital Brussels
city: Brussels
country: Belgium
facility: UVC Brugmann
Study officials:
affiliation: University Hospital Brussels, Belgium
name: Peter Janssens, MD
role: PRINCIPAL_INVESTIGATOR
References: