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    Summary
    EudraCT Number:2012-003814-15
    Sponsor's Protocol Code Number:2012-784
    National Competent Authority:France - ANSM
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2013-06-11
    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 ConcernedFrance - ANSM
    A.2EudraCT number2012-003814-15
    A.3Full title of the trial
    A multicenter phase II pilot open label study to evaluate the efficacy and safety of BKM120 in the treatment of patients with advanced or metastatic differentiated thyroid cancers
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A multicenter phase II pilot open label study to evaluate the efficacy and safety of BKM120 in the treatment of patients with advanced or metastatic differentiated thyroid cancers
    A.3.2Name or abbreviated title of the trial where available
    BKM 120
    A.4.1Sponsor's protocol code number2012-784
    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 SponsorHOSPICES CIVILS DE LYON
    B.1.3.4CountryFrance
    B.3.1 and B.3.2Status of the sponsorNon-Commercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportNOVARTIS
    B.4.2CountryFrance
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationHOSPICES CIVILS DE LYON
    B.5.2Functional name of contact pointZUBLENA
    B.5.3 Address:
    B.5.3.1Street Address3 Quai des Celestins
    B.5.3.2Town/ cityLYON
    B.5.3.3Post code69002
    B.5.3.4CountryFrance
    B.5.4Telephone number04 72 11 54 11
    B.5.5Fax number04 72 11 51 90
    B.5.6E-mailirene.zublena@chu-lyon.fr
    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.3.2Product code BKM120
    D.3.4Pharmaceutical form Capsule, soft
    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.3.9.3Other descriptive nameBKM120 HYDROCHLORIDE SALT
    D.3.9.4EV Substance CodeSUB32821
    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
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Follicular or poorly differentiated cancer refractory to radio iodine
    E.1.1.1Medical condition in easily understood language
    Thyroid cancer
    E.1.1.2Therapeutic area Diseases [C] - Cancer [C04]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 19.1
    E.1.2Level PT
    E.1.2Classification code 10066474
    E.1.2Term Thyroid cancer
    E.1.2System Organ Class 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    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 efficacy of BKM120 as measured by the progression free survival (PFS rate) at 6 months in patients with progressive, metastatic, refractory, follicular or poorly differentiated thyroid cancers.

    E.2.2Secondary objectives of the trial
    • to evaluate the efficacy of BKM120 according to:
    o The PFS rate at 12 months
    o The objective tumor response rate (Complete Response + Partial Response according to RECIST (version 1.1.)) after 6 months of treatment
    o The overall survival (defined as the time from start of study treatment to the date of death due to any cause) at 6 months.
    • to assess the safety and tolerance of BKM120
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1- Histologically confirmed differentiated follicular or poorly differentiated thyroid cancer
    2- Patients refractory to radio iodine: i.e.; absence of radioiodine uptake in at least one target lesion on a post-therapeutic whole body scan, presence of a target lesion after a cumulative radio-iodine activity of at least 600 mCi, patients with radio-iodine uptake who have progression of the disease within 12 months after radioactive iodine (RAI) treatment
    3- Metastatic or locally invasive disease
    4- Patients must have at least one site of measurable disease per RECIST (version 1.1.)
    5- Documented progression as per RECIST (version 1.1.) based on 2 comparative imagings performed within the last 12 months (+20%)
    6- Patients may have received two previous treatment with tyrosine kinase inhibitors but must be off treatment within at least 4 weeks
    7- Patient has signed the informed consent before any trial related activities and according to local guidelines
    8- Patient (male or female) is ≥ 18 years at the day of consenting to the study
    9- Patient has an Eastern Cooperative Oncology Group (ECOG) performance status ≤ 2 which the investigator believes is stable at the time of screening
    10- Patient has adequate bone marrow and organ function as defined by the following laboratory values:
    • Absolute Neutrophil Count (ANC) ≥ 1.0 x 109/L
    • Platelets ≥ 100 x 109/L
    • Hemoglobin ≥ 9.0 g/dL
    • INR ≤ 1,5
    • Potassium, calcium, (corrected for serum albumin) and magnesium within normal limits (WNL) for the institution
    • Serum Creatinine ≤ 1.5 x ULN
    • Alanine aminotransferase (ALT) and aspartate aminotransferase (AST) within normal range (or ≤ 3.0 x ULN if liver metastases are present)
    • Total Serum bilirubin within normal range (or ≤ 1.5 x ULN if liver metastases are present; or total bilirubin ≤ 3.0 x ULN with direct bilirubin within normal range in patients with well documented Gilbert’s Syndrome which is defined as presence of several episodes of unconjugated hyperbilirubinemia with normal results from CBC count (including normal reticulocyte count and blood smear), normal liver function test results, and absence of other contributing disease processes at the time of diagnosis (see APPENDIX 1)
    • Fasting plasma glucose (FPG) ≤ 120 mg/dL or ≤ 6.7 mmol/L
    • HbA1c ≤ 8 %
    11- Patient has no legal protection measure
    12- Patient has a health coverage (affiliation to social security system or similar)
    E.4Principal exclusion criteria
    1- Other histological subtypes of thyroid tumors: papillary, anaplastic, medullary, lymphoma or sarcoma
    2- Patient has received previous treatment with PI3K and/or mTOR inhibitors or AKT inhibitors,
    3- Patient has symptomatic central nervous system (CNS) metastases.
    4-Patient has a concurrent malignancy or malignancy within 3 years of study enrollment, (with the exception of adequately treated, basal or squamous cell carcinoma or non-melanomatous skin cancer)
    5- Patient has a score ≥ 12 on the PHQ-9 questionnaire
    6- Patient selects a response of “1, 2 or 3” to question number 9 on the PHQ-9 questionnaire regarding potential for suicidal thoughts or ideation
    7- Patient has a GAD-7 mood scale score ≥ 15
    8- Patient has a medically documented history of or active major depressive episode, bipolar disorder (I or II), obsessive-compulsive disorder, schizophrenia, a history of suicidal attempt or ideation, or homicidal ideation (e.g. risk of doing harm to self or others)
    9- Patient is concurrently using other approved or investigational antineoplastic agent
    10- Patient who received wide field radiotherapy ≤ 4 weeks or limited field radiation for palliation ≤ 2 weeks prior to starting study drug or who have not recovered to grade 1 or better from related side effects of such therapy (exceptions include alopecia, bone marrow and organ functions)
    11- Patient has had major surgery within 14 days prior to starting study drug or has not recovered from major side effects of the surgery
    12- Patient has active cardiac disease or a history of cardiac dysfunction
    13- Patient has a Left Ventricular Ejection Fraction (LVEF) < 50% as determined by Multiple Gated acquisition (MUGA) scan or echocardiogram (ECHO)
    14- Patient has any of the following cardiac conduction abnormalities:
    15- Patient is currently receiving treatment with medication that has a known risk to prolong the QT interval or inducing Torsades de Pointes, and the treatment cannot be discontinued or switched to a different medication prior to randomization.
    16- Patient has impairment of gastrointestinal (GI) function or GI disease that may significantly alter the absorption of BKM120 (e.g., ulcerative diseases, uncontrolled nausea, vomiting, diarrhea, malabsorption syndrome, or small bowel resection)
    17-Patient is currently receiving increasing or chronic treatment (> 5 days) with corticosteroids or another immunosuppressive agent, as chronic administration of corticosteroids (> 5 days) can induce CYP3A4
    18- Patient has any other concurrent severe and/or uncontrolled medical condition that would, in the investigator’s judgment contraindicate patient participation in the clinical study (e.g.,chronic pancreatitis, chronic active hepatitis, etc.)
    19- Patient has a history of non-compliance to medical regimen or inability to grant consent
    20- Patient is currently receiving treatment with drugs known to be moderate and strong inhibitors or inducers of isoenzyme CYP3A, The patient must have discontinued strong inducers for at least one week and must have discontinued strong inhibitors before the treatment is initiated. Switching to a different medication prior to entry in the treatment phase is allowed.
    21- Patient has a known history of HIV (testing not mandatory) infection
    22- Pregnant or nursing (lactating) woman where pregnancy is defined as the state of a female after conception and until the termination of gestation, confirmed by a positive beta hCG laboratory test (> 5 mIU/mL)
    23- Patient who does not apply highly effective contraception during the study and through the duration as defined below after the final dose of study treatment.
    • Women of child-bearing potential, defined as all women physiologically capable of becoming pregnant, must use highly effective contraception during dosing of any study agent + contraception through 4 weeks after final dose of study treatment (i.e. Time for complete elimination of the investigational drug according to its half-life [5 x T1/2]).
    • Highly effective contraception is defined as either:
    a. Total abstinence:
    b. Sterilization:
    c. Male partner sterilization
    d. Use of a combination of any two of the following (a+b):
    a) Placement of an intrauterine device (IUD) or intrauterine system (IUS)
    b) Barrier methods of contraception:
    24- Patient has a known hypersensitivity to any of the excipients of BKM120
    25-Patient has not recovered to grade 1 or better (except alopecia) from related side effects of any prior antineoplastic therapy
    26- Patient is currently receiving warfarin or other coumarin derived anti-coagulant, for treatment, prophylaxis or otherwise. Therapy with heparin, low molecular weight heparin (LMWH), or fondaparinux is allowed
    E.5 End points
    E.5.1Primary end point(s)
    The primary criterion of the study will be progression-free survival (PFS) at 6 months after the onset of the treatment as determined by the radiologist of centers. The progression-free survival is defined as a lack of objective tumor progression and death. Tumor progression is documented by the CT or MRI scans.
    E.5.1.1Timepoint(s) of evaluation of this end point
    6 months after the onset of the treatment
    E.5.2Secondary end point(s)
    PFS at 12 months
    - Objective tumor response (CR and PR) after 6 months of treatment according to RECIST criteria (version 1.1.) and validated by a central review committee.
    - Overall survival (defined as the time from start of study treatment to the date of death due to any cause) at 6 months.
    - The safety and tolerance of BKM120 at 24 months.
    - Thyroglobulin determination and relation with tumors changes.
    - The genetic tumoral alterations including alterations in genes known to be involved in thyroid tumorigenesis (PIK3CA, BRAF, N-RAS) and the predictive value of the tumor molecular profile on the therapeutic effect of the drug.
    E.5.2.1Timepoint(s) of evaluation of this end point
    6,12 and 24 months
    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 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) Yes
    E.7.3Therapeutic confirmatory (Phase III) No
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled No
    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 No
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo No
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial1
    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 concerned8
    E.8.5The trial involves multiple Member States No
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA No
    E.8.6.2Trial being conducted completely outside of the EEA No
    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 years
    E.8.9.1In the Member State concerned months36
    E.8.9.1In the Member State concerned days
    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.1.3Elderly (>=65 years) No
    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 No
    F.3.3.1Women of childbearing potential not using contraception No
    F.3.3.2Women of child-bearing potential using contraception No
    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 state42
    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 Decision2013-03-06
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2013-01-15
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2016-12-15
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