Idiopathic Nephrotic SyndromePhase 2/3VAINNCT02896270
Valproic Acid for Idiopathic Nephrotic Syndrome
A Phase 2/3 study testing Valproic Acid in people with Idiopathic Nephrotic Syndrome and Focal Segmental Glomerulosclerosis, run by Universitair Ziekenhuis Brussel at 2 sites in Belgium.
Trial 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). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Idiopathic Nephrotic Syndrome, Focal Segmental Glomerulosclerosis, Minimal Change Disease
- What is being tested?
- Valproic Acid
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 15 participants
- Where
- 2 sitesBelgium
- Study dates
- Oct 2016to Dec 2019
- Phase
- Phase 2/3
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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 -
Who cannot join
- 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
Trial locations and contacts
2 sites in 1 country.
Belgium2 sites, 2 recruiting
- Recruiting
University Hospital Brussels
Brussels, 1090
Peter Janssens, MD, +32 2 477 6224, Peter.Janssens@uzbrussel.be
Nathalie Marmitte, Coordinator, +32 2 477 6224, Nathalie.Marmitte@uzbrussel.be
- Recruiting
UVC Brugmann
Brussels
Tatiana Besse-Hammer, MD
About this study
Official title: A Prospective Interventional Pilot Study on the Use of Valproic Acid for Treatment of Idiopathic Nephrotic Syndrome
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.
Read the 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
- 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.
- Being tested
- Valproic Acid
- Keywords
- idiopathic podocytopathies, Neprology
- Medical subject headings
- Nephrosis, Lipoid, Glomerulosclerosis, Focal Segmental
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Valproic AcidDrug
Also called Depakine Chrono 500©, Sanofi
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.
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: Drug: Valproic Acid |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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
Measured: 6 months
Secondary outcomes (4)
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.
Measured: 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.
Measured: 6 - 12 months
Evaluate the evolution of renal function estimated by MDRD-GFR
Evolution of renal function estimated by CKD-EPI
Measured: 12 months
Evaluate the tolerability of VPA in the setting of idiopathic podocytopathies
Evaluation adverse events
Measured: 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Sep 2016 (estimated)
- Study startOct 2016
- Primary completionOct 2019 (estimated)
- Study completionDec 2019 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Idiopathic Nephrotic SyndromeStudies of the same condition, open and under way.
- More trials run by Universitair Ziekenhuis BrusselThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2/3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 15 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Universitair Ziekenhuis Brussel
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Peter Janssens, MD, Principal investigator, University Hospital Brussels, Belgium
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02896270
- Sponsor's protocol ID
- UZB_20160728
- Acronym
- VAIN
- Status last verified
- Jun 2017
- First submitted
- 6 Sep 2016
- Last updated
- 20 Jun 2017
- Results posted
- No
- Data monitoring committee
- Yes
- Individual data sharing
- Yes: The data of this pilot study will be submitted for publication as soon as the anticipated 15 subjects have completed the study.
Study at a glance
From the registry record, last updated by the sponsor on 20 Jun 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Prospective Interventional Pilot Study on the Use of Valproic Acid for Treatment of Idiopathic Nephrotic Syndrome
- ClinicalTrials.gov ID
- NCT02896270
- Acronym / sponsor ID
- VAIN / UZB_20160728
- Keywords
- idiopathic podocytopathies, Neprology
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 15 participants (estimated)
- Lead sponsor
- Universitair Ziekenhuis BrusselUniversity, hospital or other
- Study start
- Oct 2016 (actual)
- Primary completion
- Oct 2019 (estimated)
- Study completion
- Dec 2019 (estimated)
- First posted
- 12 Sep 2016 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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
- 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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| single arm | Experimental | Drug: Valproic AcidPatients 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. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (8 listed)
- Able to give informed consent
- Biopsy proven idiopathic FSGS or MCD
- Organ function:
Key exclusion criteria (6 listed)
- Contraindication for VPA
- Secondary etiologies for FSGS or MCD
- Multiple organ transplantation
- Currently participating in another clinical trial
- Pregnant or lactating women
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Valproic AcidAlso: Depakine Chrono 500©, SanofiThe 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. | Drug | single arm |
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
4 secondary outcomes
Determine the disease response by the proportion of subjects with partial remission
Time frame: 6 - 12 months
Determine the extent to which standard immunosuppression can be reduced
Time frame: 6 - 12 months
Evaluate the evolution of renal function estimated by MDRD-GFR
Time frame: 12 months
Evaluate the tolerability of VPA in the setting of idiopathic podocytopathies
Time frame: 12 months
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 20 Jun 2017.
- Individual participant dataSharing planned: Yes. 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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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Peter Janssens, MD principal investigator
University Hospital Brussels, Belgium
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2/3 study testing Valproic Acid in people with Idiopathic Nephrotic Syndrome and Focal Segmental Glomerulosclerosis, run by Universitair Ziekenhuis Brussel at 2 sites in Belgium.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 8 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 20 Jun 2017. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in Belgium. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Peter Janssens, MD (+32 2 477 6224, peter.janssens@uzbrussel.be), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
In remission group induction is the proportion of patients in complete remission (time frame: 6 months). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 20 Jun 2017.