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    Summary
    EudraCT Number:2014-001641-24
    Sponsor's Protocol Code Number:PYR-311
    National Competent Authority:Germany - BfArM
    Clinical Trial Type:EEA CTA
    Trial Status:Prematurely Ended
    Date on which this record was first entered in the EudraCT database:2014-07-30
    Trial results
    Index
    A. PROTOCOL INFORMATION
    B. SPONSOR INFORMATION
    C. APPLICANT IDENTIFICATION
    D. IMP IDENTIFICATION
    D.8 INFORMATION ON PLACEBO
    E. GENERAL INFORMATION ON THE TRIAL
    F. POPULATION OF TRIAL SUBJECTS
    G. INVESTIGATOR NETWORKS TO BE INVOLVED IN THE TRIAL
    N. REVIEW BY THE COMPETENT AUTHORITY OR ETHICS COMMITTEE IN THE COUNTRY CONCERNED
    P. END OF TRIAL
    Expand All   Collapse All
    A. Protocol Information
    A.1Member State ConcernedGermany - BfArM
    A.2EudraCT number2014-001641-24
    A.3Full title of the trial
    A Phase 3 Randomized, Double-Blind, Placebo-Controlled, Multi-Center Study to Evaluate the Safety and Efficacy of Pyridorin® (pyridoxamine dihydrochloride) in Subjects With Nephropathy Due to Type 2 Diabetes (PIONEER)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Safety and Efficacy of Pyridorin® in Subjects with Nephropathy Due to Type 2 Diabetes
    A.4.1Sponsor's protocol code numberPYR-311
    A.5.4Other Identifiers
    Name:US IND numberNumber:58,684
    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 SponsorNephroGenex
    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 supportNephroGenex, Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationMedpace Finland OY
    B.5.2Functional name of contact pointClinical Trials Information
    B.5.3 Address:
    B.5.3.1Street AddressKarjalankatu 2, 4 Krs
    B.5.3.2Town/ cityHelsinki
    B.5.3.3Post code00520
    B.5.3.4CountryFinland
    B.5.4Telephone number+49892524055
    B.5.5Fax number+49896702 100
    B.5.6E-mailregsubmissions@medpace.com
    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 namePyridorin®
    D.3.2Product code Pyridorin®
    D.3.4Pharmaceutical form Capsule
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNPyridorin
    D.3.9.1CAS number 524-36-7
    D.3.9.3Other descriptive namePYRIDOXAMINE DIHYDROCHLORIDE
    D.3.9.4EV Substance CodeSUB15060MIG
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number300
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee 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 No
    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
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboCapsule
    D.8.4Route of administration of the placeboOral use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Nephropathy Due to Type 2 Diabetes
    E.1.1.1Medical condition in easily understood language
    Kidney damage due to high amounts of sugar in the blood.
    E.1.1.2Therapeutic area Not possible to specify
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 18.1
    E.1.2Level PT
    E.1.2Classification code 10061835
    E.1.2Term Diabetic nephropathy
    E.1.2System Organ Class 10038359 - Renal and urinary disorders
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To determine the efficacy of Pyridorin 300 mg BID compared to placebo in reducing the rate of progression of renal disease due to type 2 diabetes mellitus.
    E.2.2Secondary objectives of the trial
    Comparisons of Pyridorin 300 mg BID to placebo for:
    Time to the composite endpoint event of:
    ─ A SCr increase of ≥100% from baseline that occurs during follow-up; or
    ─ ESRD.
    The safety of Pyridorin compared to placebo, as assessed by adverse events, 12-lead electrocardiograms (ECGs), vital signs, physical examination, clinical chemistries, glycosylated hemoglobin (HbA1c), and hematology.

    Comparisons of Pyridorin 300 mg BID to placebo for:
    - Change in serum cystatin-C from baseline to Week 52 and from baseline to Week 104 (Month 24);
    - Change in urine protein/creatinine ratio (PCR) from baseline to Week 52 and from baseline to Week 104;
    - Change in urinary transforming growth factor-beta (TGF-β) excretion from baseline to Week 52 and from baseline to Week 104; and
    - Change in SCr from baseline to Week 52 and from baseline to Week 104.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Patients who have given voluntary written informed consent to participate in this study prior to conducting Screening (Visit 1) procedures;
    2. Patients 18 years of age or older with a diagnosis of type 2 diabetes;
    3. Women of childbearing potential (WOCBP) who agree to use appropriate birth control (double-barrier methods, hormonal contraceptives, or intrauterine device) for the duration of the study (women of childbearing potential is defined as all women who are not surgically sterile or are not at least 1 year post menopausal). All women of childbearing potential must have a negative serum pregnancy test at Visit 1;
    4. All men (unless surgically sterile, as defined below) who have sexual intercourse with a WOCBP must agree and commit to use a highly effective method of contraception for the duration of the study (defined as the time of the signing of the informed consent form through the conclusion of patient participation). Highly effective methods of contraception include:
    i. Male subjects agreeing that they and their female spouse/partners will use adequate contraception (2 forms of birth control, one of which must be barrier method) or be of non-childbearing potential.
    ii. To be considered surgically sterilized, a male partner must have had a vasectomy at least 24 weeks before Visit 1;
    5. At Visit 1, patients must have a history of overt diabetic nephropathy, as defined by the following:
    • A SCr measurement greater than or equal to 1.3(greater than or equal to 1.25) mg/dL (111 micro mol/L) for females or greater than or equal to 1.5(greater than or equal to 1.45) mg/dL (128 micro mol/L) for males;
    • At visit 1 or 1.1 24-hour urine collection PCR greater than or equal to 1200 mg/g (136 mg/mmol) and, if applicable for PS phase, at Visit 1S or 1.1S a 24-hour urine collection PCR greater than or equal to 600 mg/g (68mg/mmol);
    •For eligibility determination , laboratory reported values of PCR will be rounded up to 2 significant digits (e.g. ≥1150 mg/g to 1200 mg/g; ≥595 mg/g to 600 mg/g),
    6. Patients must have a SCr measurement less than 3.0 mg/dL (265 micro mol/L);
    7. Patients must have an eGFR of greater than or equal to 20 mL/min/1.73m2, using the 4-variable Modification of Diet in Renal Disease equation in which eGFR = 175 x (SCr(mg/dL))-1.154 x (Age(years))-0.203 x (0.742 if female) x (1.212 if African American);
    8. Patient must have a second screening SCr measurement at Visit 1.1 or 1.1S taken 1 week (± 2 days) after screening (Visit 1 or 1S). The value of the second screening SCr measurement must be less than 3.0 mg/dL (265 micro mol/L) for both genders and within 25% of the first screening measurement;
    9. Patients must be taking a single ACE-I or ARB at a constant dose for at least 26 weeks prior to Visit 1, where the dose of the ACE-I or the ARB is considered appropriate for that patient and it is anticipated that the same dose can and will be maintained throughout the course of the study;
    10. Patients taking any blood pressure medications in addition to an ACE-I or ARB, including diuretics, must be on a stable dose for 13 weeks
    prior to Visit 1 (and Visit 1S if applicable) with a seated blood pressure of less than or equal to 150/90 mmHg;
    11. Patients not taking any blood pressure medications, including diuretics, other than an ACE-I or ARB must have a seated blood pressure less than or equal to 150/90 mmHg at Visit 1(and Visit 1S if applicable) and a seated blood pressure considered appropriate for the patient and one that can be sustained throughout the study.
    E.4Principal exclusion criteria
    1. Patients with type 1 diabetes or MODY (a monogenic form of diabetes);
    2. Patients with a diagnosis of chronic kidney disease other than diabetic renal disease with or without hypertensive renal disease;
    3. Patients receiving a renin inhibitor or an aldosterone antagonist or a combination of an ACE-I and an ARB within 26 weeks of Visit 1;
    4. Patients with a history of solid organ transplantation;
    5. Patients with a history of myocardial infarction, coronary re-vascularization procedures (including percutaneous transluminal coronary angioplasty), stroke, or transient ischemic attack within 30 days prior to Visit 1;
    6. Patients with a diagnosis of New York Heart Association Class III or IV congestive heart failure at any time;
    7. Patients with a history of being treated for neoplastic disease (except basal or squamous cell carcinoma of the skin) within 5 years prior to Visit 1;
    8. Patients with any history of dialysis within 2 years prior to Visit 1;
    9. Patients in whom dialysis or renal transplantation is anticipated by their physician within 1 year after Visit 1;
    10. Patients who used SCr-altering drugs within 30 days prior to Visit 1 (See Section 7.2.2);
    11. Patients who require systemic immunosuppression therapy for greater than 2 weeks (except for inhalant steroids);
    12. Patients with clinically significant liver disease or transaminase (alanine aminotransferase and aspartate aminotransferase) levels greater than 2.5 × the upper limit of normal (ULN) measured at Visit 1;
    13. Patients with bilirubin levels greater than 1.5 × ULN measured at Visit 1;
    14. Patients with a history of allergic or other adverse response to vitamin B preparations;
    15. Patients who require greater than 50 mg of vitamin B6 daily;
    16. Patients who have an active history of dysphagia or swallowing disorders;
    17. Patients with a history of hypersensitivity to Pyridorin or any of the excipients (non-active ingredients) in the Pyridorin formulation;
    18. Patients who have taken pyridoxamine or any other investigational drug within 30 days prior to Visit 1 or Visit 1S, or have participated in a previous Pyridorin study or another interventional clinical study within 30 days prior to Visit 1 or Visit 1S;
    19. Patients with an active history of drug or alcohol abuse;
    20. Patients unlikely to comply with the study protocol (eg, an inability and unwillingness to participate in adequate training, an uncooperative attitude, inability to return for follow-up visits, or unlikelihood of completing the study);
    21. Women who are lactating, pregnant, or intend to become pregnant during the course of the study; and
    22. Persons employed with the sponsor, CRO, or one of the study investigative sites must be excluded from participation, even if they are not involved directly in the conduct of the study.
    E.5 End points
    E.5.1Primary end point(s)
    Time to the composite endpoint consisting of the earliest event amongst:
    ─ A SCr increase of ≥50% from baseline that occurs during follow-up;
    or
    ─ ESRD.
    E.5.1.1Timepoint(s) of evaluation of this end point
    These endpoints are evaluated with each central lab draw for SCr.
    E.5.2Secondary end point(s)
    Time to the composite endpoint consisting of the earliest event amongst:
    ─ A SCr increase of ≥100% from baseline that occurs during follow-up;
    or
    ─ ESRD.
    E.5.2.1Timepoint(s) of evaluation of this end point
    These endpoints are evaluated with each central lab draw for SCr.
    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 No
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic No
    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.7.1.3.1Other trial type description
    E.7.2Therapeutic exploratory (Phase II) No
    E.7.3Therapeutic confirmatory (Phase III) Yes
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled Yes
    E.8.1.1Randomised Yes
    E.8.1.2Open No
    E.8.1.3Single blind No
    E.8.1.4Double blind Yes
    E.8.1.5Parallel group No
    E.8.1.6Cross over No
    E.8.1.7Other No
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo Yes
    E.8.2.3Other No
    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 concerned5
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA67
    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
    Australia
    France
    Germany
    Hong Kong
    Hungary
    Israel
    Spain
    United States
    E.8.7Trial has a data monitoring committee Yes
    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
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years3
    E.8.9.1In the Member State concerned months1
    E.8.9.1In the Member State concerned days15
    E.8.9.2In all countries concerned by the trial years3
    E.8.9.2In all countries concerned by the trial months5
    E.8.9.2In all countries concerned by the trial days21
    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: 300
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 300
    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 Yes
    F.3.3.7.1Details of other specific vulnerable populations
    Participants who cannot read
    F.4 Planned number of subjects to be included
    F.4.1In the member state25
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 180
    F.4.2.2In the whole clinical trial 600
    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)
    After participation in the trial, patients will be treated with the current standard therapy in their country.
    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 Decision2014-09-29
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2014-09-15
    P. End of Trial
    P.End of Trial StatusPrematurely Ended
    P.Date of the global end of the trial2016-04-04
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