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    Summary
    EudraCT Number:2013-000200-41
    Sponsor's Protocol Code Number:CAMN107A2203
    National Competent Authority:Hungary - National Institute of Pharmacy
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2013-06-21
    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 ConcernedHungary - National Institute of Pharmacy
    A.2EudraCT number2013-000200-41
    A.3Full title of the trial
    A multi-center, open label, non-controlled phase II study to evaluate efficacy and safety of oral nilotinib in pediatric patients with newly diagnosed Ph+ chronic myelogenous leukemia (CML) in chronic phase (CP) or with Ph+ CML in
    CP or accelerated phase (AP) resistant or intolerant to either imatinib or dasatinib
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Open label, phase II study to evaluate efficacy and safety of oral nilotinib in Philadelphia positive (Ph+) chronic myelogenous leukemia (CML) pediatric patients.
    A.4.1Sponsor's protocol code numberCAMN107A2203
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/297/2015
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorNovartis Pharma Services AG
    B.1.3.4CountrySwitzerland
    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 supportNovartis Pharma Services AG
    B.4.2CountrySwitzerland
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationNovartis Hungary Kft.
    B.5.2Functional name of contact pointPublic Information Desk
    B.5.3 Address:
    B.5.3.1Street AddressBartók Béla út 43-47
    B.5.3.2Town/ cityBudapest
    B.5.3.3Post code1114
    B.5.3.4CountryHungary
    B.5.4Telephone number00 36 1 457-6500
    B.5.5Fax number00 36 1 457-6600
    B.5.6E-mailinfoph.hungary@novartis.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 Yes
    D.2.1.1.1Trade name Tasigna
    D.2.1.1.2Name of the Marketing Authorisation holderNovartis Europharm Limited
    D.2.1.2Country which granted the Marketing AuthorisationEuropean Union
    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/3/06/375
    D.3 Description of the IMP
    D.3.1Product namenilotinib
    D.3.2Product code AMN107
    D.3.4Pharmaceutical form Capsule, hard
    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 INNNILOTINIB
    D.3.9.1CAS number 641571-10-0
    D.3.9.2Current sponsor codeAMN107
    D.3.9.4EV Substance CodeSUB25225
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number150
    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.IMP: 2
    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.2.1.1.1Trade name Tasigna
    D.2.1.1.2Name of the Marketing Authorisation holderNovartis Europharm Limited
    D.2.1.2Country which granted the Marketing AuthorisationEuropean Union
    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/3/06/375
    D.3 Description of the IMP
    D.3.1Product namenilotinib
    D.3.2Product code AMN107
    D.3.4Pharmaceutical form Capsule, hard
    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 INNNILOTINIB
    D.3.9.1CAS number 641571-10-0
    D.3.9.2Current sponsor codeAMN107
    D.3.9.4EV Substance CodeSUB25225
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number200
    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.IMP: 3
    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 Yes
    D.2.5.1Orphan drug designation numberEU/3/06/375
    D.3 Description of the IMP
    D.3.1Product namenilotinib
    D.3.2Product code AMN107
    D.3.4Pharmaceutical form Capsule, hard
    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 INNNILOTINIB
    D.3.9.1CAS number 641571-10-0
    D.3.9.2Current sponsor codeAMN107
    D.3.9.4EV Substance CodeSUB25225
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number50
    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
    - leukemia
    - leukemia,pediatric
    - leukemia, myleiod
    - leukemia, mylegenous, chronic
    - leukemia, mylegenous, accelerated
    - BCR-ABL positive
    - myeloproliferative disorder
    - bone marrow disease
    - hematologic diseases
    - neoplastic processes
    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 LLT
    E.1.2Classification code 10009012
    E.1.2Term Chronic myelogenous leukemia
    E.1.2System Organ Class 100000004864
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    1. To assess efficacy of nilotinib in pediatric patients with Ph+ CML-CP resistant or intolerant to either imatinib or dasatinib
    2. To assess efficacy of nilotinib in pediatric patients with Ph+ CML-AP resistant or intolerant to either imatinib or dasatinib
    3. To assess efficacy of nilotinib in pediatric patients with newly diagnosed Ph+ CML-CP
    E.2.2Secondary objectives of the trial
    1. To further characterize efficacy and PK profile of nilotinib in pediatric patients with Ph+ CML
    2. To further characterize safety and tolerability of nilotinib in pediatric patients with Ph+ CML
    3. To assess long term effect on growth, development and maturation of nilotinib treatment in pediatric patients with Ph+ CML
    4. To identify emerging signs of resistance to nilotinib
    5. To describe acceptability of the study drug formulation
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Patients eligible for inclusion in this study have to meet all of the following criteria:
    1. Male or female patients from 1 year of age to less than 18 years of age at study entry
    2. Newly diagnosed Ph+ CML-CP or Ph+ CML-CP or AP resistant or intolerant to either imatinib or dasatinib (as defined in the protocol)
    3. Performance status: Karnofsky ≥50% for patients >10 years of age, and Lansky ≥50 for patients ≤10 years of age.
    4. Adequate renal, hepatic and pancreatic function as defined in the protocol
    5. Patients must have potassium, magnesium, phosphorus and total calcium values ≥LLN (lower limit of normal) or corrected to within normal limits with supplements prior to the first dose of study medication
    6. Written informed consent prior to any screening procedures. However, if requested tests were performed as part of clinical practice within the window required per protocol, this data can be used provided all parameters required per protocol were verified.
    E.4Principal exclusion criteria
    Patients eligible for this study must not meet any of the following criteria:
    1. Female patients who are : (a) pregnant, (b) of childbearing potential without a negative hCG pregnancy test prior to baseline and (c) female of childbearing potential who do not agree to abstinence or, if sexually active, do not agree to the use of highly effective contraception
    2. Patients actively receiving therapy with strong CYP3A4 inhibitors or inducers and the treatment cannot be either discontinued or switched to a different medication at least 14 days prior to starting study drug
    3. Patients who are currently receiving treatment with any medications that have a known risk or possible risk to prolong the QT interval and the treatment cannot be either discontinued or switched to a different medication prior to starting study drug
    4. Acute or chronic liver, pancreatic or severe renal disease considered unrelated to CML
    5. History of pancreatitis within 12 months of starting study drug or past medical history of chronic pancreatitis
    6. Active or systemic bacterial, fungal, or viral infection as documented by positive cultures, radiological imaging techniques, or septic shock syndrome. Such infections should have been resolved for ≥2 weeks before including the patient in the study
    7. Impaired cardiac function as defined in the protocol
    8. Patients with documented T315I mutation in BCR-ABL
    9. Previous treatment with more than one TKI for imatinib or dasatinib resistant/intolerant Ph+ CML patients. Previous treatment with any TKI for newly diagnosed Ph+ CML patients is not permitted unless the patient has received imatinib for less than 2 weeks prior to the first dose of study drug and discontinued at least 5 days prior to the first dose of nilotinib.
    10. Patients who have received myelosuppressive chemotherapy within 3 weeks, imatinib within 5 days, or dasatinib within 3 days prior to the first dose of study drug
    11. Patients who have not recovered from all acute toxicities from all prior myelosuppressive chemotherapy prior to starting study drug
    12. Patients receiving hydroxyurea greater than 21 days for the treatment of Ph+ CML either prior to initiation of nilotinib or with maximum duration planned to exceed one week post initiation of nilotinib
    13. Patients who have received hematopoietic growth factors within 7 days prior to starting study drug
    14. Patients who have received Pegfilgrastim (Neulasta®) within 14 days prior to starting study drug
    15. In case of Stem Cell Transplant (SCT) or Rescue without total body irradiation (TBI):
    Evidence of either active graft vs. host disease or less than 3 months since SCT.
    16. In case of radiation therapy: less than 2 weeks if local palliative, less than 3 months after total body irradiation (TBI), or craniospinal radiation therapy or if at least 50% radiation of pelvis; less than 6 weeks after other substantial BM radiation
    17. Patients with known Hepatitis B, Hepatitis C, or HIV infection
    18. Patients who, in the opinion of the investigator, are unlikely to comply with the protocol or safety monitoring requirements
    19. Patients who are breast feeding
    20. Patients who have a known hypersensitivity to the active ingredient or any of the excipients including lactose
    21. Impairment of gastrointestinal (GI) function or GI disease that may significantly alter the absorption of study drug (e.g., ulcerative disease, uncontrolled nausea, vomiting, diarrhea, mal-absorption, small bowel resection, or gastric bypass surgery)
    22. Severe and/or uncontrolled concurrent medical disease that in the opinion of the investigator could cause unacceptable safety risks or compromise compliance with the protocol (e.g., diabetes, infection, febrile neutropenia etc.).
    E.5 End points
    E.5.1Primary end point(s)
    1. Rate of Major Molecular Response (MMR)
    2. Rate of Complete Hematological Response (CHR)
    3. Rate of Major Molecular Response (MMR)
    4. Rate of Complete Cytogenetic Response (CCyR)
    E.5.1.1Timepoint(s) of evaluation of this end point
    1. At 6 cycles
    2. By 3 cycles
    3. By 12 cycles
    4. At 12 cycles
    E.5.2Secondary end point(s)
    1. Rate of MCyR and CCyR
    2. Rate of each cytogenetic response category
    3. Rate of MMR and CHR
    4. Time to response, duration of response, time to disease progression, overall survival, event free survival
    5. Population pharmacokinetic parameters of nilotinib
    6. Pharmacodynamics (BCR-ABL transcript levels determined with standard protocols in peripheral blood)
    7. Safety and tolerability: incidence and severity of adverse events, as assessed by patient symptoms, physical exam assessments, abnormal laboratory tests, echocardiograms and electrocardiograms
    8. Assessment of development (growth and sexual maturation), and
    thyroid function
    9. Mutational assessment of BCR-ABL
    10.Questionnaire on acceptability (including palatability) of dose forms used after first dose, cycle 1 and cycle 12
    E.5.2.1Timepoint(s) of evaluation of this end point
    1. By 6, 12, 18, 24, 36, 48, and 66 cycles
    2. By 6, 12, 18, 24, 36, 48, and 66 cycles
    3. By 3, 6, 9, 12, 18, 24, 36, 48, and 66 cycles
    4. Up to 66 cycles
    5. Up to 66 cycles
    6. Continuous
    7. Up to 66 cycles
    8. Up to 66 cycles
    9. Up to 66 cycles
    10. After 1st dose, cycle 1, and cycle 12
    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 Yes
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic Yes
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    E.6.13Others Yes
    E.6.13.1Other scope of the trial description
    acceptability of study drug formulation (including palatability)
    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 trial3
    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 concerned1
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA13
    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
    Austria
    Belgium
    Canada
    France
    Germany
    Hungary
    Italy
    Japan
    Korea, Republic of
    Malaysia
    Netherlands
    Russian Federation
    Spain
    Thailand
    Turkey
    United Kingdom
    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
    End of study is defined as the timepoint when all patients have completed the Study Evaluation Completion (SEC) visit. The SEC will either be a minimum of 30 days post Cycle 66 or a minimum of 30 days post early discontinuation.
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years5
    E.8.9.1In the Member State concerned months5
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years5
    E.8.9.2In all countries concerned by the trial months3
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 Yes
    F.1.1Number of subjects for this age range: 59
    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) Yes
    F.1.1.4.1Number of subjects for this age range: 1
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 18
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 41
    F.1.2Adults (18-64 years) No
    F.1.2.1Number of subjects for this age range: 0
    F.1.3Elderly (>=65 years) No
    F.1.3.1Number of subjects for this age range: 0
    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 state5
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 19
    F.4.2.2In the whole clinical trial 59
    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)
    At the end of the study, patients will be eligible to continue to a roll-over or alternative study protocol in order to collect further safety data and allow patients to receive treatment with nilotinib for as long as they continue to demonstrate benefit, do not experience unacceptable toxicities, or until nilotinib is commercially available in pediatric patients.
    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 Decision2013-09-12
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2013-08-28
    P. End of Trial
    P.End of Trial StatusOngoing
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