Valproic Acid for Idiopathic Nephrotic Syndrome

NCT ID: NCT02896270

Last Updated: 2017-06-20

Basic Information

• Recruitment Status: UNKNOWN
• Clinical Phase: PHASE2/PHASE3
• Total Enrollment: 15 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2016-10-31
• Study Completion Date: 2019-12-31

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.

Conditions

Idiopathic Nephrotic Syndrome; Focal Segmental Glomerulosclerosis; Minimal Change Disease

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

single arm

Patients will start study treatment on Day1 and will be treated with a dose of 250mg twice daily of the valproic acid slow release formulation (Depakine Chrono© - Sanofi Pharma Belgium).

Control of valproic acid serum levels after 4 to 7 days. The dose will be progressively increased targeting valproic acid serum levels in the target range for use of the drug as an anti-epileptic (50-100µg/ml).

During the study, visits will be performed every month and at the end of treatment. The duration of the study is 12 months. Continuation of valproic acid after completion of the study will be at the investigators discretion.

Group Type: EXPERIMENTAL

Valproic Acid

Intervention Type: DRUG

The concomitant immunosuppressive regimen is to be reduced at the discretion of the investigators. It is suggested to lower immunosuppressive therapy only in valproic acid target trough levels have been attained.

Interventions

Valproic Acid

The concomitant immunosuppressive regimen is to be reduced at the discretion of the investigators. It is suggested to lower immunosuppressive therapy only in valproic acid target trough levels have been attained.

Intervention Type: DRUG

Other Intervention Names

Depakine Chrono 500©, Sanofi

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

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Universitair Ziekenhuis Brussel

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Peter Janssens, MD

Role: PRINCIPAL_INVESTIGATOR

University Hospital Brussels, Belgium

Locations

University Hospital Brussels

Brussels, , Belgium

Site Status: RECRUITING

UVC Brugmann

Brussels, , Belgium

Site Status: RECRUITING

Countries

Belgium

Central Contacts

Peter Janssens, MD

Role: CONTACT

peter.janssens@uzbrussel.be

+32 2 477 6224

Nathalie Marmitte, Coordinator

Role: CONTACT

nathalie.marmitte@uzbrussel.be

+32 2 477 6224

Facility Contacts

Peter Janssens, MD

Role: primary

Peter.Janssens@uzbrussel.be

+32 2 477 6224

Nathalie Marmitte, Coordinator

Role: backup

Nathalie.Marmitte@uzbrussel.be

+32 2 477 6224

Tatiana Besse-Hammer, MD

Role: primary

Other Identifiers

UZB_20160728

Identifier Type: -

Identifier Source: org_study_id