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    The EU Clinical Trials Register currently displays   43839   clinical trials with a EudraCT protocol, of which   7280   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:2018-004564-59
    Sponsor's Protocol Code Number:Pontrack01
    National Competent Authority:Germany - BfArM
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2019-05-15
    Trial results
    Expand All   Collapse All
    A. Protocol Information
    A.1Member State ConcernedGermany - BfArM
    A.2EudraCT number2018-004564-59
    A.3Full title of the trial
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A.3.2Name or abbreviated title of the trial where available
    A.4.1Sponsor's protocol code numberPontrack01
    A.5.4Other Identifiers
    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 SponsorHeidelberg University
    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 supportIncyte Biosciences International Sàrl
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationHeidelberg University
    B.5.2Functional name of contact pointCoordinating Investigator
    B.5.3 Address:
    B.5.3.1Street AddressTheodor-Kutzer-Ufer 1-3
    B.5.3.2Town/ cityMannheim
    B.5.3.3Post code68167
    B.5.4Telephone number00496213836966
    B.5.5Fax number00496213836969
    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 Yes
    D. name Iclusig
    D. of the Marketing Authorisation holderIncyte Biosciences Distribution B.V.
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community Yes
    D.2.5.1Orphan drug designation numberEU/3/09/716
    D.3 Description of the IMP
    D.3.4Pharmaceutical form Film-coated tablet
    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 INNponatinib
    D.3.9.1CAS number 1114544-31-8
    D.3.9.3Other descriptive namePONATINIB HYDROCHLORIDE
    D.3.9.4EV Substance CodeSUB121686
    D.3.10 Strength
    D.3.10.1Concentration unit g gram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number15
    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. 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 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
    chronic myeloid leukemia in chronic phase
    E.1.1.1Medical condition in easily understood language
    chronic myeloid leukemia in chronic phase
    E.1.1.2Therapeutic area Diseases [C] - Blood and lymphatic diseases [C15]
    MedDRA Classification
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    The main purpose of the study is the assessment of patients who are in MR4 or deeper after 2 years of ponatinib treatment.
    E.2.2Secondary objectives of the trial
    Secondary objectives of the study are
    Molecular status (no MMR, MMR, MR4, and MR4.5) at the evaluation times defined in the visit schedule
    Time from inclusion to first MR4 and to first MR4.5
    Assessment of safety profile, tolerability and adverse events under ponatinib treatment
    Assessment of health-related quality of life (QoL) profiles under ponatinib treatment
    Identification of clinical and biological factors associated with the achievement of MR4 or better under ponatinib (e.g. risk scores (Sokal/Euro/EUTOS/ELTS), gender, duration of TKI treatment, molecular level at study entry)
    Evaluation of medico-economic impact of ponatinib therapy with the possibility of a TFR approach
    Evaluation of overall survival and progression-free survival
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
     Female or male ≥ 18 years of age
     Patients with CML in chronic phase (CP)
     BCR-ABLIS between 0,5-0,01 and demonstrated by the last PCR before inclusion
     not achieving MR4 (defined as < 0,01% BCR-ABLIS) or not achieving a stable MR4 (defined as no continuous in MR4 during the last 12 months before inclusion) after ≥ 3 years of treatment with nilotinib, dasatinib and / or bosutinib in first or second line
     Philadelphia -chromosome and/or BCR-ABL (either b3a2 and /or b2a2) fusion gene positive CML
     Patients must have had an eye examination including fundoscopy by an ophthalmologist within 8 weeks prior to first treatment
    E.4Principal exclusion criteria
     Failure of any TKI at any time during CML treatment according to current ELN criteria (including any detection of mutations or additional cytogenetic aberrations)
     Prior diagnosis of accelerated phase (AP) or blast phase (BP) at any time in the history of the disease
     Previously planned or performed allogenic SCT
     At time of inclusion (results of screening)
    • High cardiac risk according to ESC score (≥ 10%)
    • Clinically significant resting bradycardia (<50 bpm)
    • QTcF interval on baseline electrocardiogram (ECG) evaluation, defined as QTcF of >450 ms in males or > 470 ms in females.
    • Treatment with inhibitors of CYP3A4 or medications that have been well documented to prolong the QT interval (see chapter V.4.5)
    • Uncontrolled hypertension (diastolic blood pressure ≥ 90 mm Hg; systolic ≥ 140 mm Hg). Patients with hypertension should be under treatment on study entry to effect blood pressure control
    • Ankle-brachial-index (ABI) < 0,9 or >1,4 or alternatively signs of arterial occlusion in duplex sonography
    • No adequate hepatic function
    (total serum bilirubin > 1.5 × ULN, unless due to Gilbert’s syndrome; Alanine aminotransferase (ALAT) > 2.5 × ULN,
    or > 5 × ULN if leukemic infiltration of the liver is present; aspartate aminotransferase (ASAT) > 2.5 × ULN,
    or > 5 × ULN if leukemic infiltration of the liver is present)
    • Positive hepatitis B virus serology test
    • No adequate pancreatic function
    (serum lipase and amylase >1.5 × ULN)
    • No adequate renal function [estimated creatinine clearance (eGFR) of < 50 ml/min, Cockcroft and Gault Score]
    • Other severe or uncontrolled medical conditions(e.g. Infection)
    • Women who are pregnant or breast feeding
    • positive serum pregnancy test (of woman with childbearing potential, see page 59)
    • woman of childbearing potential not agreeing to use an higly-effective form of contraception or fertile male not agreeing to use an acceptable birth control method (for definition see appendix) with sexual partners throughout study participation until 90 days after EoT.
    • Legally incapacitated
    • No ability to comprehend and sign the informed consent.
    • Held in an institution by legal or official order
    • Not able to take oral therapy
    • No willingness or ability to comply with scheduled visits, treatment plans, laboratory tests, and other study procedures.
    • Participation in other clinical trials
     History of
    • myocardial infarction (MI)
    • clinically significant (as determined by the treating physician) atrial or ventricular arrhythmias
    • coronary heart disease
    • congenital long QT syndrome or family history of
    • use of a ventricular paced pacemaker
    • other clinically significant heart disease (e.g. unstable angina, congestive heart failure) or impaired cardiac function
    • hyperlipidaemia.
    • cerebrovascular accident (CVA, e.g. stroke) or transient ischemic attack (TIA)
    • peripheral vascular infarction, including visceral infarction or other vascular occlusive events
    • any revascularization procedure, (e.g. placement of stents, bypasses)
    • venous thromboembolism, including deep venous thrombosis or pulmonary embolism, within 6 months prior to enrolment
    • retinal venous occlusions
    • Moderate or severe acute or chronic liver disease
    • alcohol abuse.
    • severe hypertriglyceridemia
    • either acute pancreatitis within 1 year before study entry or chronic pancreatitis
    • renal artery stenosis
    • moderate , severe or end-stage chronic renal disease unrelated to tumor
    • severe diabetes with end organ damage (e.g. microalbuminuria) or poorly controlled diabetes, defined as HbA1c values over the previous year of > 7.5% (59 mmol/mol) on more than 3 occasions. Patients with pre-existing, well-controlled diabetes are not excluded.
    • bleeding issues
    • any other malignancy except if neither clinically significant nor requires active intervention.
    E.5 End points
    E.5.1Primary end point(s)
    Treatment with Ponatinib for 2 years;
    see primary objectives
    E.5.1.1Timepoint(s) of evaluation of this end point
    endpoint for each patient after 2 years of treatment with Ponatinib
    E.5.2Secondary end point(s)
    Treatment with Ponatinib for 2 years;
    see secondary objectives
    E.5.2.1Timepoint(s) of evaluation of this end point
    Inclusion of the last patient into the study plus 2 years of treatment
    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 Yes
    E.6.13.1Other scope of the trial description
    Therapeutic exploratory (phase II)
    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 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 concerned15
    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 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
    Despite LP/LV is planned for Q4/2023 and data base lock in Q1/2026, a descriptive follow-up analyses is planned until Q2/2026. Therefore end of study is defined as June 30, 2026.
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years4
    E.8.9.1In the Member State concerned months0
    E.8.9.1In the Member State concerned days0
    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: 55
    F.1.3Elderly (>=65 years) No
    F.1.3.1Number of subjects for this age range: 5
    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 state60
    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)
    A descriptive post study FU for 2 y is planned for all patients.
    For patients who have achieved an MR4, it is recommended to continue with ponatinib. In addition, it is planned to stop the treatment according to current recommendations in these patients and to include these patients in a TFR registry .
    For all other patients, treatment decision should be made on an individual bases according to response.
    AEs/ SAEs will be followed until the findings have been clarified or became stable.

    G. Investigator Networks to be involved in the Trial
    G.4 Investigator Network to be involved in the Trial: 1
    G.4.1Name of Organisation Deutsche CML Studiengruppe /Deutsche CML Allianz
    G.4.3.4Network Country Germany
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2019-08-29
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2019-08-19
    P. End of Trial
    P.End of Trial StatusOngoing
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