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The European Union Clinical Trials Register   allows you to search for protocol and results information on:
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    The EU Clinical Trials Register currently displays   43845   clinical trials with a EudraCT protocol, of which   7282   are clinical trials conducted with subjects less than 18 years old.   The register also displays information on   18700   older paediatric trials (in scope of Article 45 of the Paediatric Regulation (EC) No 1901/2006).

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    EudraCT Number:2019-003607-35
    Sponsor's Protocol Code Number:C0221002
    National Competent Authority:Poland - Office for Medicinal Products
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2020-10-20
    Trial results View results
    Expand All   Collapse All
    A. Protocol Information
    A.1Member State ConcernedPoland - Office for Medicinal Products
    A.2EudraCT number2019-003607-35
    A.3Full title of the trial
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Study to Evaluate PF-06730512 in Adults With Focal Segmental Glomerulosclerosis (FSGS)
    A.4.1Sponsor's protocol code numberC0221002
    A.7Trial is part of a Paediatric Investigation Plan No
    A.8EMA Decision number of Paediatric Investigation Plan
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorPfizer Inc.
    B.1.3.4CountryUnited States
    B.3.1 and B.3.2Status of the sponsorCommercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportPfizer Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationPfizer Inc.
    B.5.2Functional name of contact pointClinical Trials.gov Call Centre
    B.5.3 Address:
    B.5.3.1Street Address235 East 42nd Street
    B.5.3.2Town/ cityNew York
    B.5.3.3Post codeNY 10017
    B.5.3.4CountryUnited States
    B.5.4Telephone number+18007181021
    D. IMP Identification
    D.IMP: 1
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation No
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product namePF-06730512
    D.3.2Product code PF-06730512
    D.3.4Pharmaceutical form Powder for solution for infusion
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNPF-06730512
    D.3.9.2Current sponsor codePF-06730512
    D.3.9.3Other descriptive namePF-06730512
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number120
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin No
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) Yes
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D. cell therapy medicinal product No
    D. therapy medical product No
    D. Engineered Product No
    D. ATIMP (i.e. one involving a medical device) No
    D. on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product Yes
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.8 Information on Placebo
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Focal Segmental Glomerulosclerosis (FSGS)
    E.1.1.1Medical condition in easily understood language
    Kidney disease
    E.1.1.2Therapeutic area Diseases [C] - Male diseases of the urinary and reproductive systems [C12]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 21.1
    E.1.2Level PT
    E.1.2Classification code 10067757
    E.1.2Term Focal segmental glomerulosclerosis
    E.1.2System Organ Class 10038359 - Renal and urinary disorders
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To evaluate the efficacy of PF-06730512 compared to baseline in the reduction of proteinuria following 24 weeks of treatment in patients with FSGS.
    E.2.2Secondary objectives of the trial
    - To evaluate the safety and tolerability of PF-06730512 following 24 weeks of treatment in subjects with FSGS.
    - To evaluate the effects of PF-06730512 on proteinuria time course.
    - To summarize complete and partial remission rates following administration of PF-06730512.
    - To evaluate the effect of PF-06730512 on renal function.
    - To evaluate the serum exposure of PF-06730512 in FSGS patients.
    - To evaluate the immunogenicity profile of PF-06730512.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1- Evidence of a personally signed and dated informed consent document indicating that the subject has been informed of all pertinent aspects of the study.

    2- Male or female subject age ≥18 years must have a confirmed diagnosis of FSGS defined as a minimum of 1 glomerulus demonstrating segmental sclerosis on light microscopy within the past 5 years.

    3- Estimated glomerular filtration rate (eGFR) ≥45 ml/min/1.73 m2 based on the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) formula, with repeat measurements permitted, as appropriate.
     - If eGFR is 30-45 ml/min/1.73 m2, a recent biopsy (within 12 months prior to Screening) must demonstrate <50% tubulo-interstitial fibrosis. The Investigator and the subject may decide to perform a biopsy which might fulfill this eligibility criterion.

    4- UPCR >1.5 g protein/g creatinine at Screening.

    5- Based on all previous treatment received for FSGS, subject is eligible if:
    a. Subject has been treated with at least 1, and up to 3 classes of Immunosuppressants* either alone or in combination;
    b. Subject has a contraindication to any class of Immunosuppressant (may include steroids) or is considered to be intolerant to any class of Immunosuppressant (may include steroids) per Investigator judgment.

    6- Subjects on ongoing corticosteroid treatment must have received an adequate course and be on a stable dose for at least 4 weeks prior to the Lead-in Period and expected to remain stable throughout the study. The same requirements apply to ongoing treatment with CNIs and/or MMF so long as subjects have been receiving either or both of these treatments for at least 3 months prior to the Lead-in Period. Refer to Section 5.8.1 for additional information.

    7.Willing and able to comply with scheduled visits, treatment plan, laboratory tests and other study procedures including completion of daily diaries to record symptoms and urine collections.

    8- Female subjects of non-childbearing potential will meet at least 1 of the following criteria (all other female subjects, including female subjects with tubal ligations, are considered to be of childbearing potential):
    a. Achieved postmenopausal status, defined as follows: cessation of regular menses for at least 12 consecutive months with no alternative pathological or physiological cause; status may be confirmed with/and have a serum follicle-stimulating hormone (FSH) level confirming the postmenopausal state;
    b. Have undergone a documented hysterectomy and/or bilateral oophorectomy;
    c. Have medically confirmed ovarian failure.
    E.4Principal exclusion criteria
    1. Diagnosis of collapsing FSGS.

    2. Advanced chronic changes on renal biopsy as evidenced by >50% tubulo-interstitial fibrosis.

    3. Evidence or history of clinically significant hematological, endocrine (including Type 1 diabetes mellitus), pulmonary, gastrointestinal, cardiovascular, hepatic, psychiatric, neurological, or hepatic disease, like cirrhosis or chronic active liver disease.

    4. Organ transplantation.

    5. History of any malignancy except for subjects who had a basal or squamous cell skin cancer which has been treated and fully resolved for a minimum of 5 years.

    6.Body mass index (BMI) >45 kg/m2 (based on an estimated dry weight as per judgment of the investigator, if applicable).

    7. Treatment with rituximab within 6 months prior to start of the Lead-in Period. If Rituximab treatment is longer than 6 months (prior to start of the Lead-in Period), subject is still excluded if no peripheral CD19+ B-cells are identified in the local laboratory prior to first study drug dosing.

    8. The following immunosuppressant medications within the past 28 days prior to start of the Lead in Period: azathioprine (Imuran), cytotoxic agents, acthar.

    9.Excessive alcohol consumption that may impair the subject's ability to participate in the study per investigator judgment in accordance with national guidelines, if available.

    10. Screening sitting blood pressure (BP) ≥155 mm Hg (systolic) or ≥95mm Hg (diastolic), following at least 5 minutes of sitting rest. If BP is ≥ 155 mm Hg (systolic) or ≥95 mm Hg (diastolic), the BP should be repeated 2 more times and the average of the 3 BP values should be used to determine the subject's eligibility. Repeat BP measurements on a different day are allowed to meet this eligibility criterion. Note: Repeat BP measurement should be done within designated screening window and may be considered as unplanned visit.

    11. Active/serious infection including, but not limited to, Hepatitis B or C, human immunodeficiency virus (HIV). This includes subjects with a known history of cytomegalovirus (CMV), CMV mononucleosis, gammaherpesviral mononucleosis, parvovirus infection, or simian virus 40 infection. Note: Laboratory test results for these types of infections may require medical interpretation by the investigator and consultation with the Sponsor or designee.

    12.Subjects with a history of bilateral vesicoureteral reflux.

    13.Subjects with a history of prior treatment with or use of interferon, lithium, pamidronate, mTOR inhibitors (eg, sirolimus), testosterone/anabolic steroids, anthracycline (eg, doxorubicin), heroin.

    14.Subjects with ANY of the following abnormalities in clinical laboratory tests at Screening, as assessed by the study specific laboratory and confirmed by a repeat test, if deemed necessary:
    a.Aspartate aminotransferase (AST) or alanine aminotransferase (ALT) level 1.5× upper limit of normal (ULN);
    b.Total bilirubin level 1.5× ULN; subjects with a history of Gilbert's syndrome may have direct bilirubin measured and would be eligible for this study provided the direct bilirubin level is ≤ ULN.

    15.Investigator site staff members directly involved in the conduct of the study and their family members, site staff members otherwise supervised by the investigator, or subjects who are Pfizer employees, including their family members, directly involved in the conduct of the study.

    16.Previous known participation in a study investigating PF 06730512.

    17.Participation in other studies involving investigational drug(s) within 30 days (or as determined by the local requirement) or 5 half lives prior to study entry and/or during study participation.

    18.A positive urine drug test at Screening (except positive for tetrahydrocannabinol [THC]).

    19.Pregnant female subjects; breastfeeding female subjects; lactating female subjects; male subjects and female subjects of childbearing potential who are unwilling or unable to use 1 highly effective method of contraception as outlined in this protocol for the duration of the study and for at least 9 weeks (63 days) after the last dose of investigational product.

    20. Other acute or chronic medical or psychiatric condition including recent (within the past year) or active suicidal ideation or behavior or laboratory abnormality that may increase the risk associated with study participation or investigational product administration or may interfere with the interpretation of study results and, in the judgment of the investigator, would make the subject inappropriate for entry into this

    21. Subjects with a history of hypersensitivity to the components of the study drug.
    E.5 End points
    E.5.1Primary end point(s)
    Percentage change from baseline to Week 13 in Urinary Protein to Creatinine Ratio (UPCR).
    E.5.1.1Timepoint(s) of evaluation of this end point
    Week 13
    E.5.2Secondary end point(s)
    - Adverse Events, Laboratory Safety Tests (Hematology, Clinical Chemistry, Urinalysis), Body Weight, Blood Pressure, Pulse Rate, oral temperature and Electrocardiogram (ECG).
    - Percentage change from baseline to Weeks 2, 5, 9 and beyond Week 13, as applicable, in UPCR.
    - Percentage change from baseline to Weeks 3, 5, 9 and 13 in estimated glomerular filtration rate (eGFR).
    - Serum concentration of PF-06730512.
    - Incidence of the development of anti-drug antibody (ADA) and neutralizing antibody (NAb).
    E.5.2.1Timepoint(s) of evaluation of this end point
    Weeks 3, 5, 9 and 13 (see above)
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis No
    E.6.2Prophylaxis No
    E.6.3Therapy Yes
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic Yes
    E.6.7Pharmacodynamic No
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    E.6.13Others No
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) No
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other No
    E. trial type description
    E.7.2Therapeutic exploratory (Phase II) Yes
    E.7.3Therapeutic confirmatory (Phase III) No
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled Yes
    E.8.1.1Randomised No
    E.8.1.2Open Yes
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    E.8.1.5Parallel group No
    E.8.1.6Cross over No
    E.8.1.7Other Yes
    E. trial design description
    Sequential study
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo No
    E.8.2.3Other Yes
    E. description
    Same compound, different dose
    E.8.2.4Number of treatment arms in the trial2
    E.8.3 The trial involves single site in the Member State concerned No
    E.8.4 The trial involves multiple sites in the Member State concerned Yes
    E.8.4.1Number of sites anticipated in Member State concerned3
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA27
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA Yes
    E.8.6.2Trial being conducted completely outside of the EEA No
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    United States
    United Kingdom
    E.8.7Trial has a data monitoring committee No
    E.8.8 Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years2
    E.8.9.1In the Member State concerned months2
    E.8.9.1In the Member State concerned days29
    E.8.9.2In all countries concerned by the trial years4
    E.8.9.2In all countries concerned by the trial months11
    E.8.9.2In all countries concerned by the trial days24
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 No
    F.1.1.1In Utero No
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.3Newborns (0-27 days) No
    F.1.1.4Infants and toddlers (28 days-23 months) No
    F.1.1.5Children (2-11years) No
    F.1.1.6Adolescents (12-17 years) No
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 72
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 4
    F.2 Gender
    F.2.1Female Yes
    F.2.2Male Yes
    F.3 Group of trial subjects
    F.3.1Healthy volunteers No
    F.3.2Patients Yes
    F.3.3Specific vulnerable populations Yes
    F.3.3.1Women of childbearing potential not using contraception No
    F.3.3.2Women of child-bearing potential using contraception Yes
    F.3.3.3Pregnant women No
    F.3.3.4Nursing women No
    F.3.3.5Emergency situation No
    F.3.3.6Subjects incapable of giving consent personally No
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state8
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 16
    F.4.2.2In the whole clinical trial 72
    F.5 Plans for treatment or care after the subject has ended the participation in the trial (if it is different from the expected normal treatment of that condition)
    G. Investigator Networks to be involved in the Trial
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2020-10-23
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2020-09-08
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2023-02-14
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