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Polycystic Kidney, Autosomal DominantPhase 2NCT01233869

Bosutinib For Autosomal Dominant Polycystic Kidney Disease

A Phase 2 study testing Bosutinib in people with Polycystic Kidney, Autosomal Dominant, run by Pfizer at 67 sites in 17 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
Search all trials

Trial summary

This purpose of this study is to determine if bosutinib reduces the rate of kidney enlargement in subjects with autosomal dominant polycystic kidney disease (ADPKD) entering the study with a total kidney volume greater than or equal to 750 cc and eGFR greater than or equal to 60 mL/min/1.73m2. Read more about this study

Who is it for?
Ages 18 to 50 years
Conditions the study is for
Polycystic Kidney, Autosomal Dominant
What is being tested?
Bosutinib
Type of treatment
Pill or oral medicine
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
172 participants
Where
67 sitesin 17 countries
Study dates
Dec 2010to Aug 2014
Phase
Phase 2
Are there results?
Yes, posted in the registrySummary in the doctors' view

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 to 50 years
  • All sexes
  • No healthy volunteers
  • Adult

Who may be able to join

  • Males and females, 18 to 50 years old at the time of consent.
  • Documented diagnosis of ADPKD (PKD-1 or PKD-2 genotypes allowed).
  • Total kidney volume ≥ 750 cc, as measured by centrally evaluated MRI.

Who cannot join

  • eGFR < 60 mL/min/1.73m2.
  • Uncontrolled hypertension (defined as systolic blood pressure ≥140 or diastolic blood pressure ≥90 mm Hg).
  • Any previous exposure to the bosutinib test article or receipt of other polycystic kidney disease (PKD) therapies.

Trial locations and contacts

67 sites in 17 countries.

67 sites in 17 countries

United States20 sites

  • Renal Associates of Baton Rouge

    Baton Rouge, Louisiana, 70808

  • Tufts Medical Center

    Boston, Massachusetts, 02111

  • Boise Kidney & Hypertension Institute, PLLC

    Caldwell, Idaho, 83605

  • University of Virginia Health System - Nephrology

    Charlottesville, Virginia, 22908

  • University of Virginia Health System

    Charlottesville, Virginia, 22908

  • Doylestown Hospital MRI

    Doylestown, Pennsylvania, 18901

  • Doylestown Hospital

    Doylestown, Pennsylvania, 18901

  • Nephrology/Hypertension Specialists

    Doylestown, Pennsylvania, 18901

  • Boise Kidney & Hypertension Institute, PLLC

    Meridian, Idaho, 83642

  • New York University - HHC CTSI Clinical Research Center

    New York, New York, 10016

  • Southwest Kidney Institute, PLC

    Phoenix, Arizona, 85004

  • Capital Nephrology Clinical Research

    Sacramento, California, 95825

  • Renal Associates, PA

    San Antonio, Texas, 78215

  • San Antonio Kidney Disease Center Physicians Group, P.L.L.C.

    San Antonio, Texas, 78229

  • The Polyclinic

    Seattle, Washington, 98104

  • Renal Remission and Hypertension Clinic

    Silverdale, Washington, 98383

  • Washington University School of Medicine

    St Louis, Missouri, 63110

  • Washington University

    St Louis, Missouri, 63110

  • Southwest Clinical Research Institute, LLC

    Tempe, Arizona, 85284

  • Southwest Kidney Institute, PLC

    Tempe, Arizona, 85284

Other trials in United States

Poland11 sites

  • Zaklad Diagnostyki Chorob Serca, II Katedra Kardiologii

    Gdansk, 80-210

  • Klinika Nefrologii, Transplantologii i Chorob Wewnetrznych

    Gdansk, 80-952

  • Specjalistyczny Szpital Zachodni im. Jana Pawla II w Grodzisku Mazowieckim

    Grodzisk Mazowiecki, 05-825

  • Krakowskie Centrum Medyczne NZOZ

    Krakow, 31-501

  • Klinika Nefrologii, Hipertensjologii i Chorob Wewnetrznych Katedry Chorob Wewnetrznych UWM

    Olsztyn, 10-561

  • Pracownia Echokardiografii, Oddzial Kardiologii

    Olsztyn, 10-561

  • Centrum Medyczne Aesculap

    Radom, 26-600

  • Klinika Kardiologii

    Szczecin, 70-111

  • Klinika Nefrologii, Transplantologii i Chorob Wewnetrznych

    Szczecin, 70-111

  • Szpital Powiatowy w Wolominie

    Wołomin, 05-200

  • SPZOZ Akademicki Szpital Kliniczny im. J. Mikulicza - Radeckiego

    Wroclaw, 50-556

Other trials in Poland

Czechia6 sites

  • Klinika gerontologicka a metabolicka

    Hradec Králové, 500 05

  • Krajska nemocnice Liberec

    Liberec, 460 63

  • Nemocnice Nove Mesto na Morave

    Nové Město na Moravě, 592 31

  • Fakultni poliklinika VFN

    Prague, 128 00

  • Vseobecna fakultni nemocnice v Praze

    Prague, 128 08

  • Pharmaceutical Research Associates CZ, s.r.o.

    Prague, 170 00

Other trials in Czechia

Romania4 sites

  • Institutul Clinic Fundeni, Centrul de Medicina Interna-Nefrologie

    Bucharest, 022328

  • Spitalul Clinic Dr. C. I. Parhon Iasi

    Iași, Jud. Iasi, 700503

  • Spitalul Clinic Municipal Dr. Gavril Curteanu Oradea

    Oradea, Jud. Bihor, 410469

  • SPITALUL CLINIC JUDETEAN DE URGENTA TIMISOARA ,Clinica de Nefrologie

    Timișoara, 300736

Other trials in Romania

Hungary3 sites

  • PRA Magyarorszag Kft. Klinikai Farmakologiai Vizsgalohely

    Budapest, 1077

  • Fovarosi Onkormanyzat Szent Imre Korhaz BSZMI Klinikai Farmakologiai Reszlege

    Budapest, 1115

  • Szegedi Tudomanyegyetem, AOK, Szent-Gyorgyi Albert Klinikai Kozpont I.sz.Belgyogyaszati Klinika

    Szeged, 6720

Other trials in Hungary

South Korea3 sites

  • Seoul National University Hospital, Department of Internal Medicine

    Seoul, 110-744

  • Samsung Medical Center/Division of Nephrology

    Seoul, 135-710

  • Eulji General Hospital

    Seoul, 139-872

Other trials in South Korea

Sweden3 sites

  • Sahlgrenska Universitetssjukhuset, Njurmedicin

    Gothenburg, 413 45

  • Karolinska Universitetssjukhuset Huddinge

    Stockholm, 141 86

  • Karolinska Universitetssjukhuset Solna

    Stockholm, 171 76

Other trials in Sweden

United Kingdom3 sites

  • BHF Glasgow Cardiovascular Research Centre, University of Glasgow

    Glasgow, G12 8TA

  • Renal and Urology Directorate, Leicester General Hospital

    Leicester, LE5 4PW

  • Morriston Hospital

    Swansea, Wales, SA6 6NL

Other trials in United Kingdom

Canada2 sites

  • Hopital du Sacre-Coeur de Montreal

    Montreal, Quebec, H4J 1C5

  • Toronto General Hospital

    Toronto, Ontario, M5G 2N2

Other trials in Canada

Italy2 sites

  • Istituti Ospitalieri di Cremona

    Cremona, 26100

  • A.O. Universitaria Ospedali Riuniti di Foggia

    Foggia, 71100

Other trials in Italy

Slovakia2 sites

  • SUMMIT CLINICAL RESEARCH, s.r.o., Oddelenie internej mediciny a klinickej farmakologie

    Bratislava, 831 01

  • Univerzitna nemocnica Bratislava

    Limbova 5, Bratislava Region, 83305

Other trials in Slovakia

Spain2 sites

  • Hospital Clinic I Provincial de Barcelona

    Barcelona, 08036

  • Hospital Universitari de Bellvitge

    L'Hospitalet de Llobregat, Barcelona, 08907

Other trials in Spain

Turkey (Türkiye)2 sites

  • Istanbul University, Istanbul Tip Fakultesi

    Istanbul, Capa, 34390

  • Dokuz Eylul Universitesi Hastanesi Ic Hastaliklari Anabilim Dali

    Izmir, Inciralti/ Narlidere, 35340

Other trials in Turkey (Türkiye)

Australia1 site

  • Monash Medical Centre

    Clayton, Victoria, 3168

Other trials in Australia

Lithuania1 site

  • Vilnius University Hospital Santariskiu Clinic, Public Institution, Centre of Nephrology

    Vilnius, 08661

Other trials in Lithuania

Moldova1 site

  • Spitalul Clinic Republican

    Chisinau, 2025

Other trials in Moldova

Switzerland1 site

  • Universitaetsspital Zuerich

    Zurich, 8091

Other trials in Switzerland

About this study

Official title: A Phase 2, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study Of The Safety, Clinical Activity And Pharmacokinetics Of Bosutinib (PF-05208763) Versus Placebo In Subjects With Autosomal Dominant Polycystic Kidney Disease (ADPKD)

This purpose of this study is to determine if bosutinib reduces the rate of kidney enlargement in subjects with autosomal dominant polycystic kidney disease (ADPKD) entering the study with a total kidney volume greater than or equal to 750 cc and eGFR greater than or equal to 60 mL/min/1.73m2.

Being tested
Bosutinib, Placebo
Keywords
Bosutinib, Autosomal Dominant Polycystic Kidney Disease
Medical subject headings
Polycystic Kidney, Autosomal Dominant

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • BosutinibDrug

    Once daily oral dose of 200 mg of bosutinib

  • BosutinibDrug

    Once daily oral dose of 400 mg of bosutinib transitioned to 200 mg/day

  • PlaceboDrug

    Once daily oral dose of placebo

Study groups in NCT01233869
Study groupRoleWhat they receive
Cohort AExperimentalNot described
Receives: Drug: Bosutinib
Cohort BExperimentalNot described
Receives: Drug: Bosutinib
Cohort CPlacebo comparatorNot described
Receives: Drug: Placebo

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Change From Baseline (CFB) in Total Kidney Volume (TKV) at Month 25

    TKV was measured by centrally evaluated Magnetic Resonance Imaging (MRI).

    Measured: Baseline and Month 25 (end of Initial Treatment Period Visit [ITPV])

Secondary outcomes (17)

  1. Change From Baseline in Estimated Glomerular Filtration Rate (eGFR) at Months 12, 24, 25 and Early Termination

    eGFR was centrally evaluated. Glomerular filtration rate (GFR) is an index of kidney function that describes the flow of filtered fluid through the kidney. The Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) equation was used to calculate eGFR. Month 25 is the end of the ITPV.

    Measured: Baseline, Month 12, Month 24, Month 25 (end of ITPV), and early termination

  2. Time to First Occurrence or Worsening of Hypertension

    The time to first occurrence or worsening of hypertension was observed (defined as the need for increased dose of or need for additional anti-hypertensive medication). The numbers presented correspond to the very first occurrence or worsening of hypertension in that treatment group.

    Measured: Baseline up to Month 25 (end of ITPV)

  3. Time to First Occurrence or Worsening of Back and/or Flank Pain

    The time to first occurrence or worsening of back and/or flank pain was observed (defined as initial onset of polycystic kidney disease [PKD]-related chronic back and/or flank pain; initiation of pain medication treatment for PKD-related chronic back and/or flank pain; addition of a pain medicine for treatment of PKD-related chronic back and/or flank pain; increase in dose of pain medication for treatment of PKD-related chronic back and/or flank pain). The numbers presented correspond to the very first occurrence or worsening of back and/or flank pain in that treatment group.

    Measured: Baseline up to Month 25 (end of ITPV)

  4. Time to First Occurrence of Gross Hematuria

    Gross hematuria is the presence of blood in the urine (defined as pink, red, or cola-colored urine due to the presence of red blood cells). The numbers presented correspond to the very first occurrence of gross hematuria in that treatment group.

    Measured: Baseline up to Month 25 (end of ITPV)

  5. Time to First Occurrence of Proteinuria

    Proteinuria is the presence of an excess of serum proteins in the urine, which may be an early sign of kidney disease. The numbers presented correspond to the very first occurrence of proteinuria in that treatment group.

    Measured: Baseline up to Month 25 (end of ITPV)

  6. Time to First Occurrence of End-Stage Renal Disease (ESRD) Requiring Dialysis >=56 Days

    ESRD is when the kidneys permanently fail to work at a level needed for daily life. No participants developed ESRD during the treatment period, therefore the analysis of the onset of ESRD requiring ≥56 days of dialysis was not performed.

    Measured: Baseline up to Month 25 (end of ITPV)

  7. Number of Participants With High Blood Urea Nitrogen (BUN) Levels

    A BUN test can reveal how well the kidneys are working by measuring the amount of urea nitrogen in the blood. A high BUN level (>1.3 times the upper limit of normal) may suggest that the kidneys are not working properly. Month 25 is the end of the ITPV.

    Measured: Day 15, Months 6, 12, 18, 24, and 25 (end of ITPV)

  8. Number of Participants With High Serum Creatinine (SCr) Levels

    A SCr test can reveal how well the kidneys are working by measuring the amount of urea nitrogen in the blood. A high SCr level (>1.3 times the upper limit of normal) may suggest that the kidneys are not working properly. Month 25 is the end of the ITPV.

    Measured: Day 15, Months 6, 12, 18, 24, and 25 (end of ITPV)

  9. Maximum Observed Plasma Concentration (Cmax) of Bosutinib

    Measured: Day 1 (pre-dose and 1, 3, 5 and 24 hours post-dose), Day 15 (pre-dose and 1, 2, 3, 4, 6, 8 and 24 hours post-dose)

  10. Time to Reach Maximum Observed Plasma Concentration (Tmax) of Bosutinib

    Measured: Day 1 (pre-dose and 1, 3, 5 and 24 hours post-dose), Day 15 (pre-dose and 1, 2, 3, 4, 6, 8 and 24 hours post-dose)

  11. Area Under the Concentration-Time Profile From Time 0 to the Dosing Interval (AUCtau) of Bosutinib

    Area under the concentration-time profile from time 0 to time tau, the dosing interval, where tau=24 hours.

    Measured: Day 1 (pre-dose and 1, 3, 5 and 24 hours post-dose), Day 15 (pre-dose and 1, 2, 3, 4, 6, 8 and 24 hours post-dose)

  12. Lowest Concentration Observed During the Dosing Interval (Cmin) of Bosutinib

    Measured: Day 15 (pre-dose and 1, 2, 3, 4, 6, 8 and 24 hours post-dose)

  13. Apparent Oral Clearance (CL/F) of Bosutinib

    Clearance of a drug is a measure of the rate at which a drug is metabolized or eliminated by normal biological processes. Clearance obtained after oral dose (apparent oral clearance) is influenced by the fraction of the dose absorbed. Drug clearance is a quantitative measure of the rate at which a drug substance is removed from the blood.

    Measured: Day 15 (pre-dose and 1, 2, 3, 4, 6, 8 and 24 hours post-dose)

  14. Apparent Volume of Distribution (Vz/F) of Bosutinib

    Volume of distribution is defined as the theoretical volume in which the total amount of drug would need to be uniformly distributed to produce the desired plasma concentration of a drug. Apparent volume of distribution after oral dose (Vz/F) is influenced by the fraction absorbed.

    Measured: Day 1 (pre-dose and 1, 3, 5 and 24 hours post-dose), Day 15 (pre-dose and 1, 2, 3, 4, 6, 8 and 24 hours post-dose)

  15. Terminal Elimination Half-Life (t1/2) of Bosutinib

    t1/2 is the time measured for the plasma concentration to decrease by one half.

    Measured: Day 1 (pre-dose and 1, 3, 5 and 24 hours post-dose), Day 15 (pre-dose and 1, 2, 3, 4, 6, 8 and 24 hours post-dose)

  16. Observed Accumulation Ratio (Rac) of Bosutinib

    Observed accumulation ratio (Rac) was calculated as AUC from time 0 to 24 hours (Day 15) divided by AUC from time 0 to 24 hours (Day 1).

    Measured: Day 15 (pre-dose and 1, 2, 3, 4, 6, 8 and 24 hours post-dose)

  17. Change From Baseline in Kidney Disease Quality of Life (KDQoL)-36 Scale Scores at Month 25

    The KDQoL-36 is a 36-item questionnaire on kidney disease-specific measure of patient-reported quality of life with 5 subscales: physical and mental functioning (items 1-12); burden of kidney disease subscale (items 13-16); symptoms and problems (items 17-28); effects of kidney disease on daily life subscale (items 29-36). The raw scores are transformed linearly to a range of 0 to 100, with higher scores indicating better quality of life.

    Measured: Baseline and end of ITPV (Month 25)

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted3 Nov 2010 (estimated)
  2. Study startDec 2010
  3. Primary completionJul 2014
  4. Study completionAug 2014

Resources

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
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Masking
Quadruple blind
Who is masked
Participant, Care provider, Investigator, Outcomes assessor
Enrolment
172 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT01233869
Sponsor's protocol ID
B1871019
Other IDs
3160A7-2211, 2010-023017-65
Status last verified
Feb 2016
First submitted
28 Oct 2010
Last updated
11 Mar 2016
Data monitoring committee
Yes

Publications

  • St Pierre K, Cashmore BA, Bolignano D, Zoccali C, Ruospo M, Craig JC, Strippoli GF, Mallett AJ, Green SC, Tunnicliffe DJ. Interventions for preventing the progression of autosomal dominant polycystic kidney disease. Cochrane Database Syst Rev. 2024 Oct 2;10(10):CD010294. doi: 10.1002/14651858.CD010294.pub3.

    PubMed 39356039

  • Tesar V, Ciechanowski K, Pei Y, Barash I, Shannon M, Li R, Williams JH, Levisetti M, Arkin S, Serra A. Bosutinib versus Placebo for Autosomal Dominant Polycystic Kidney Disease. J Am Soc Nephrol. 2017 Nov;28(11):3404-3413. doi: 10.1681/ASN.2016111232. Epub 2017 Aug 24.

    PubMed 28838955

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Bosutinib in people with Polycystic Kidney, Autosomal Dominant, run by Pfizer at 67 sites in 17 countries.

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 3 inclusion and 3 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 11 Mar 2016. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 67 sites in United States, Poland, Czechia, Romania, Hungary and 12 more countries. 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. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this 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.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 28 Oct 2015). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

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?

Change From Baseline (CFB) in Total Kidney Volume (TKV) at Month 25 (time frame: Baseline and Month 25 (end of Initial Treatment Period Visit [ITPV])). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT01233869, which also lists 2 linked publications.

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 11 Mar 2016.

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