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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.
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
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| single arm | Experimental | 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. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Valproic Acid | 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. |
| Measure | Description | Time Frame |
|---|---|---|
| In remission group induction is the proportion of patients in complete remission | 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. | 6 months |
| In remission maintenance group is the proportion of patients able to reduce maintenance | The proportion of patients able to reduce maintenance immunosuppression to a monotherapy of 4 mg methylprednisolone or less while remaining in complete remission | 6 months |
| Measure | Description | Time Frame |
|---|---|---|
| Determine the disease response by the proportion of subjects with partial remission | 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. |
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Inclusion Criteria:
Able to give informed consent
Biopsy proven idiopathic FSGS or MCD
Organ function:
Exclusion Criteria:
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Peter Janssens, MD | Contact | +32 2 477 6224 | peter.janssens@uzbrussel.be | |
| Nathalie Marmitte, Coordinator | Contact | +32 2 477 6224 | nathalie.marmitte@uzbrussel.be |
| Name | Affiliation | Role |
|---|---|---|
| Peter Janssens, MD | University Hospital Brussels, Belgium | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University Hospital Brussels | Recruiting | Brussels | 1090 | Belgium |
The data of this pilot study will be submitted for publication as soon as the anticipated 15 subjects have completed the study.
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| ID | Term |
|---|---|
| D009402 | Nephrosis, Lipoid |
| D005923 | Glomerulosclerosis, Focal Segmental |
| ID | Term |
|---|---|
| D009401 | Nephrosis |
| D007674 | Kidney Diseases |
| D014570 | Urologic Diseases |
| D052776 | Female Urogenital Diseases |
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| ID | Term |
|---|---|
| D014635 | Valproic Acid |
| ID | Term |
|---|---|
| D010421 | Pentanoic Acids |
| D014631 | Valerates |
| D000144 | Acids, Acyclic |
| D002264 | Carboxylic Acids |
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|
| 6 - 12 months |
| Determine the extent to which standard immunosuppression can be reduced | 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. | 6 - 12 months |
| Evaluate the evolution of renal function estimated by MDRD-GFR | Evolution of renal function estimated by CKD-EPI | 12 months |
| Evaluate the tolerability of VPA in the setting of idiopathic podocytopathies | Evaluation adverse events | 12 months |
| UVC Brugmann | Recruiting | Brussels | Belgium |
|
| D005261 |
| Female Urogenital Diseases and Pregnancy Complications |
| D000091642 | Urogenital Diseases |
| D052801 | Male Urogenital Diseases |
| D005921 | Glomerulonephritis |
| D009393 | Nephritis |
| D009930 |
| Organic Chemicals |
| D005232 | Fatty Acids, Volatile |
| D005227 | Fatty Acids |
| D008055 | Lipids |