Proteinuric DiseasesPhase 2/3PIRATENCT02343094
Phenylbutyrate in Proteinuric Nephropathies
A Phase 2/3 study testing PBA 7,5g/d and PBA 15g/d in people with Proteinuric Diseases, run by Assistance Publique - Hôpitaux de Paris at 1 site in France.
Trial summary
The purpose of this study is to determine whether sodium phenylbutyrate can reduce Lcn2 urinary expression in proteinuric patients. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Proteinuric Diseases
- What is being tested?
- PBA 7,5g/d and PBA 15g/d
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 26 participants
- Where
- 1 siteFrance
- Study dates
- Feb 2015to Jul 2016
- 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
- Chronic Kidney disease for ore than 3 months
- Proteinuria > 1g/d or 0,1g/mmmol creatinine
- eGFR >30ml/mn/1,73m2
- written informed consent
- affiliated with social security health insurance
- risk of non-adherence to protocol and visits
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- patients having a cardiac insufficiency of grade 3 or 4
- patient requiring of a strict salt-free diet
- patients under corticoids or immunosuppresseurs
- clinical intolerance in the treatment
- intolerance in the fructose, the syndrome of malabsorption glucose and galactose or a deficit in sucrase / isomaltase
- patients treated by Probenecide
Who cannot join
- Women with childbearing potential
- Recent (<3 months) modification of ACE inhibitors or ARB
- Acute renal failure
- eGFR <30ml/mn/1,73m2
- Nephrotic syndrome (albuminélia <30g/l)
- Infection with HIV, HCV, HBV
- Liver insufficiency
- No affiliated with social security health insurance
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Phenylbutyrate Effect on Lcn2 Urianry Expression in Proteinuric Patients
The purpose of this study is to determine whether sodium phenylbutyrate can reduce Lcn2 urinary expression in proteinuric patients.
Read the detailed description
Proteinuria is a major prognosis factor of chronic kidney disease (CKD) progression. Convergent evidences from clinical and experimental studies indicate that albuminuria and proteinuria are not simply a marker of CKD progression, but an active player in the evolution of the disease. Mechanistically, it has been shown that proteinuria induces endoplasmic reticulum stress in tubular cells, leading to induction of lipocalin 2/NGAL, a critical element of CKD progression. Moreover, proteinuric mice treated with phenylbutyrate are protected from CKD progression.
The aim of this study is to evaluate the efficacy of phenybutyrate, a molecular chaperone which inhibits ER stress, on the proteinuria-induced NGAL expression. Urinary NGAL/creatinine ratio will be evaluated in proteinuric patients before and under treatment with phenylbutyrate.
- Conditions
- Proteinuric Diseases
- Being tested
- PBA 7,5g/d, PBA 15g/d
- Keywords
- Proteinuria, Lipocalin 2, Neutrophil gelatinase associated lipocalin, ELISA
- Medical subject headings
- Proteinuria
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PBA 7,5g/dDrug
Treatment for 14 days with PBA
PBA 15g/dDrug
Treatment for 14 days with PBA
| Study group | Role | What they receive |
|---|---|---|
| PBA 7,5g/d | Experimental | Treatment for 14 days Receives: Drug: PBA 7,5g/d |
| PBA 15g/d | Experimental | Treatment for 14 days Receives: Drug: PBA 15g/d |
What is being measured
The primary outcome is the main question the study is designed to answer.
Urinary Lcn2/creatinine ratio
Reduction of 50 % of Lcn2 concentration, measured with ELISA
Measured: Day 7, Day 15, Day 21, Day 28
Secondary outcomes (4)
Urinary protein/creatinine ratio
Reduction of 30% of the proteinurie and the albuminuria
Measured: Day 7
Urinary protein/creatinine ratio
Reduction of 30% of the proteinurie and the albuminuria
Measured: Day 15
Urinary protein/creatinine ratio
Reduction of 30% of the proteinurie and the albuminuria
Measured: Day 21
Urinary protein/creatinine ratio
Reduction of 30% of the proteinurie and the albuminuria
Measured: Day 28
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Jan 2015 (estimated)
- Study startFeb 2015
- Primary completionDec 2015
- Study completionJul 2016
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Proteinuric DiseasesStudies of the same condition, open and under way.
- More trials run by Assistance Publique - Hôpitaux de ParisThe 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
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 26 participants (actual)
Sponsor and investigators
- Lead sponsor
- Assistance Publique - Hôpitaux de Paris
- Sponsor type
- University, hospital or other
- Collaborators
- URC-CIC Paris Descartes Necker Cochin
- Responsible party
- Sponsor
- Investigators
- Bertrand KNEBELMANN, Principal investigator, Service de Néphrologie Adulte, Hôpital Necker
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02343094
- Sponsor's protocol ID
- 2013-003924-35
- Acronym
- PIRATE
- Status last verified
- Oct 2025
- First submitted
- 15 Jan 2015
- Last updated
- 20 Nov 2025
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 20 Nov 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phenylbutyrate Effect on Lcn2 Urianry Expression in Proteinuric Patients
- ClinicalTrials.gov ID
- NCT02343094
- Acronym / sponsor ID
- PIRATE / 2013-003924-35
- Conditions
- Proteinuric Diseases
- Keywords
- Proteinuria, Lipocalin 2, Neutrophil gelatinase associated lipocalin, ELISA
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 26 participants (actual)
- Lead sponsor
- Assistance Publique - Hôpitaux de ParisUniversity, hospital or other
- Collaborators
- URC-CIC Paris Descartes Necker Cochin
- Study start
- Feb 2015
- Primary completion
- Dec 2015 (actual)
- Study completion
- Jul 2016 (actual)
- First posted
- 21 Jan 2015 (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 purpose of this study is to determine whether sodium phenylbutyrate can reduce Lcn2 urinary expression in proteinuric patients.
Detailed description
Proteinuria is a major prognosis factor of chronic kidney disease (CKD) progression. Convergent evidences from clinical and experimental studies indicate that albuminuria and proteinuria are not simply a marker of CKD progression, but an active player in the evolution of the disease. Mechanistically, it has been shown that proteinuria induces endoplasmic reticulum stress in tubular cells, leading to induction of lipocalin 2/NGAL, a critical element of CKD progression. Moreover, proteinuric mice treated with phenylbutyrate are protected from CKD progression.
The aim of this study is to evaluate the efficacy of phenybutyrate, a molecular chaperone which inhibits ER stress, on the proteinuria-induced NGAL expression. Urinary NGAL/creatinine ratio will be evaluated in proteinuric patients before and under treatment with phenylbutyrate.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| PBA 7,5g/d | Experimental | Drug: PBA 7,5g/dTreatment for 14 days |
| PBA 15g/d | Experimental | Drug: PBA 15g/dTreatment for 14 days |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (12 listed)
- Chronic Kidney disease for ore than 3 months
- Proteinuria > 1g/d or 0,1g/mmmol creatinine
- eGFR >30ml/mn/1,73m2
- written informed consent
- affiliated with social security health insurance
Key exclusion criteria (8 listed)
- Women with childbearing potential
- Recent (<3 months) modification of ACE inhibitors or ARB
- Acute renal failure
- eGFR <30ml/mn/1,73m2
- Nephrotic syndrome (albuminélia <30g/l)
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 |
|---|---|---|
| PBA 7,5g/dTreatment for 14 days with PBA | Drug | PBA 7,5g/d |
| PBA 15g/dTreatment for 14 days with PBA | Drug | PBA 15g/d |
Primary outcome
Urinary Lcn2/creatinine ratio
Time frame: Day 7, Day 15, Day 21, Day 28
Reduction of 50 % of Lcn2 concentration, measured with ELISA
4 secondary outcomes
Urinary protein/creatinine ratio
Time frame: Day 7
Urinary protein/creatinine ratio
Time frame: Day 15
Urinary protein/creatinine ratio
Time frame: Day 21
Urinary protein/creatinine ratio
Time frame: Day 28
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 20 Nov 2025.
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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.
Bertrand KNEBELMANN principal investigator
Service de Néphrologie Adulte, Hôpital Necker
1 site 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 PBA 7,5g/d and PBA 15g/d in people with Proteinuric Diseases, run by Assistance Publique - Hôpitaux de Paris at 1 site in France.
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 12 inclusion and 8 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: finished, as of the sponsor's last update on 20 Nov 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in France. 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?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Urinary Lcn2/creatinine ratio (time frame: Day 7, Day 15, Day 21, Day 28). 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 Nov 2025.