Phenylbutyrate in Proteinuric Nephropathies (PIRATE)

November 17, 2025 updated by: Assistance Publique - Hôpitaux de Paris

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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

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 Type

Interventional

Enrollment (Actual)

26

Phase

  • Phase 2
  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • France
      • Paris, France, 75015
        • Hopital Necker Enfants Malades

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  • 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

Exclusion Criteria:

  • 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
  • inclusion in another protocol of biomedical research
  • risk of non-adherence to protocol and visits
  • 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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: PBA 7,5g/d
Treatment for 14 days
Drug: PBA 7,5g/d
Treatment for 14 days with PBA
Experimental: PBA 15g/d
Treatment for 14 days
Drug: PBA 15g/d
Treatment for 14 days with PBA

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Urinary Lcn2/creatinine ratio
Time Frame: Day 7, Day 15, Day 21, Day 28
Reduction of 50 % of Lcn2 concentration, measured with ELISA
Day 7, Day 15, Day 21, Day 28

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Urinary protein/creatinine ratio
Time Frame: Day 7
Reduction of 30% of the proteinurie and the albuminuria
Day 7
Urinary protein/creatinine ratio
Time Frame: Day 15
Reduction of 30% of the proteinurie and the albuminuria
Day 15
Urinary protein/creatinine ratio
Time Frame: Day 21
Reduction of 30% of the proteinurie and the albuminuria
Day 21
Urinary protein/creatinine ratio
Time Frame: Day 28
Reduction of 30% of the proteinurie and the albuminuria
Day 28

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Assistance Publique - Hôpitaux de Paris

Collaborators

URC-CIC Paris Descartes Necker Cochin

Investigators

  • Principal Investigator: Bertrand KNEBELMANN, Service de Néphrologie Adulte, Hôpital Necker

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

February 1, 2015

Primary Completion (Actual)

December 1, 2015

Study Completion (Actual)

July 1, 2016

Study Registration Dates

First Submitted

January 15, 2015

First Submitted That Met QC Criteria

January 15, 2015

First Posted (Estimated)

January 21, 2015

Study Record Updates

Last Update Posted (Actual)

November 20, 2025

Last Update Submitted That Met QC Criteria

November 17, 2025

Last Verified

October 1, 2025

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 2013-003924-35

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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