Clinical Trial: Valproic Acid for Idiopathic Nephrotic Syndrome
Study Status: Not yet recruiting
Recruit Status: Not yet recruiting
Study Type: Interventional
Official Title: A Prospective Interventional Pilot Study on the Use of Valproic Acid for Treatment of Idiopathic Nephrotic Syndrome
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 Summary:
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
- 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.
- VPA is effective in remission maintenance allowing reduction and cessation of chronic immunosuppression without relapse in patients with frequently relapsing FSGS or MCD.
Sponsor: Universitair Ziekenhuis Brussel
Current Primary Outcome:
- In remission group induction is the proportion of patients in complete remission [ Time Frame: 6 months ]Complete remission is defined as a reduction of proteinuria to <300mg/g creatinine or < 0.3g/d and normal serum creatinine (or stable creatinine if baseline creatinine before disease onset is well documented) and serum albumin > 3.5g/dL.
- In remission maintenance group is the proportion of patients able to reduce maintenance [ Time Frame: 6 months ]The proportion of patients able to reduce maintenance immunosuppression to a monotherapy of 4 mg methylprednisolone or less while remaining in complete remission
Original Primary Outcome: Same as current
Current Secondary Outcome:
- Determine the disease response by the proportion of subjects with partial remission [ Time Frame: 6 - 12 months ]
Remission induction patients with partial remission defined as a reduction in proteinuria to 0.3-3.5g/d or 300-3500mg/g creatinine and a decrease of at least 50% from baseline proteinuria and stable serum creatinine (change in creatinine < 25%) 6 months after inclusion into the study for FSGS.
Remission maintenance patients remaining in remission for at least 6 months after reduction of maintenance immunosuppression to monotherapy with 4 mg of methylprednisolone or less.
- Determine the extent to which standard immunosuppression can be reduced [ Time Frame: 6 - 12 months ]The proportion of "remission induction patients" attaining full or partial remission with 4mg methylprednisolone or less 6 months and 12 months after inclusion; The proportion of "remission maintenance patients" remaining in remission for at least 6 months after reduction of maintenance immunosuppression to monotherapy with 4 mg of methylprednisolone or less.
- Evaluate the evolution of renal function estimated by MDRD-GFR [ Time Frame: 12 months ]Evolution of renal function estimated by CKD-EPI
- Evaluate the tolerability of VPA in the setting of idiopathic podocytopathies [ Time Frame: 12 months ]Evaluation adverse events
Original Secondary Outcome: Same as current
Information By: Universitair Ziekenhuis Brussel
Dates:
Date Received: September 6, 2016
Date Started: October 2016
Date Completion: December 2019
Last Updated: September 6, 2016
Last Verified: August 2016
