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    Summary
    EudraCT Number:2008-002219-42
    Sponsor's Protocol Code Number:BCX1777-108
    National Competent Authority:Germany - BfArM
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2008-08-21
    Trial results View 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 number2008-002219-42
    A.3Full title of the trial
    A Phase I/II Pharmacokinetic Study of Intravenous and Oral Forodesine in Children with Relapsed or Refractory T-cell or B-cell Precursor Acute Lymphoblastic Leukaemia or T-cell Non- Hodgkin’s Lymphoma
    A.4.1Sponsor's protocol code numberBCX1777-108
    A.7Trial is part of a Paediatric Investigation Plan Information not present in EudraCT
    A.8EMA Decision number of Paediatric Investigation Plan
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorMundipharma Research Limited
    B.1.3.4CountryUnited Kingdom
    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 support
    B.4.2Country
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisation
    B.5.2Functional name of contact point
    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 Yes
    D.2.5.1Orphan drug designation numberEU/3/06/421 (ALL only not to T-NHL)
    D.3 Description of the IMP
    D.3.1Product nameForodesine Sterile Solution, 10mg/ml
    D.3.2Product code BCX-1777
    D.3.4Pharmaceutical form Solution for injection
    D.3.4.1Specific paediatric formulation Information not present in EudraCT
    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 INNForodesine hydrochloride
    D.3.9.1CAS number 284490-13-7
    D.3.9.2Current sponsor codeBCX-1777
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number10
    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) Information not present in EudraCT
    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 Information not present in EudraCT
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) Information not present in EudraCT
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product Information not present in EudraCT
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy Information not present in EudraCT
    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 Information not present in EudraCT
    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 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/3/06/421
    D.3 Description of the IMP
    D.3.1Product nameForodesine Sterile Solution, 10mg/ml
    D.3.2Product code BCX-1777
    D.3.4Pharmaceutical form Oral solution
    D.3.4.1Specific paediatric formulation Information not present in EudraCT
    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 INNForodesine hydrochloride
    D.3.9.1CAS number 284490-13-7
    D.3.9.2Current sponsor codeBCX-1777
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number10
    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) Information not present in EudraCT
    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 Information not present in EudraCT
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) Information not present in EudraCT
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product Information not present in EudraCT
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy Information not present in EudraCT
    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 Information not present in EudraCT
    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
    T-cell or B-cell precursor (BCP) acute lymphoblastic leukaemia (T-ALL or BCP-ALL) or T-cell Non Hodgkin’s lymphoma (T-NHL)
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 9.1
    E.1.2Level LLT
    E.1.2Classification code 10000844
    E.1.2Term Acute lymphoblastic leukaemia
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 9.1
    E.1.2Level HLGT
    E.1.2Classification code 10025321
    E.1.2Term Lymphomas non-Hodgkin's T-cell
    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 evaluate the pharmacokinetic (PK) and pharmacodynamic (PD) parameters of the proposed intravenous and oral forodesine doses in children with relapsed or refractory T-cell or B-cell precursor (BCP) acute lymphoblastic leukaemia (T-ALL or BCP-ALL) or T-cell Non-Hodgkin’s Lymphoma (T-NHL)

    2. Attempt to assess the oral bioavailability of forodesine and the level of accumulation after repeated oral and intravenous dosing

    3. Attempt to assess whether twice daily dosing alters the oral bioavailability of forodesine compared with once daily dosing
    E.2.2Secondary objectives of the trial
    1. Evaluate the safety of six different dose schedules of forodesine in paediatric subjects

    2. Collect preliminary efficacy data on forodesine in children with relapsed or refractory haematological malignancies at six different dose schedules

    3. Collect preliminary data on the penetration and efficacy of forodesine in the
    cerebrospinal fluid (CSF).
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Subjects who are to be included in the study, are those who meet all of the following criteria:
    1. Males and females aged ≥ 2 years to ≤ 18 years

    2. Males and females ≥ 13 kg

    3. Unequivocal histological diagnosis of T-ALL, BCP-ALL or T-NHL (World Health
    Organisation [WHO] classification) at initial diagnosis

    4. T-ALL or T-NHL subjects:
    a. 1st relapse after HSCT, or
    b. ≥2 relapses, or
    c. newly diagnosed subjects who are refractory to 1 standard induction
    chemotherapy regimen, or
    d. 1st relapse subjects who are refractory to 1 standard induction chemotherapy
    regimen

    BCP-ALL subjects:
    a. ≥ 2 relapses, or
    b. newly diagnosed subjects who are refractory to 2 standard induction chemotherapy regimens, or
    c. 1st relapse subjects who are refractory to 2 standard induction chemotherapy
    regimens

    Note: Relapse is defined as ≥ 25% marrow blasts or reappearance of any nodal lesion(s) after documented complete response (CR).

    Two regimens can be defined as either two different treatment regimens or the same
    treatment regimen given to a subject who has relapsed after a durable response to the first treatment. A previous regimen can include a HSCT.

    5. KPS or LPS (as appropriate for subject’s age) scores ≥60

    6. Anticipated life expectancy of at least 6 weeks

    7. Adequate kidney and liver function in the opinion of the Investigator and the Medical Monitor e.g. creatinine levels ≤ 2.0 times upper limit of normal, liver function tests (aspartate aminotransferase [AST] and/or alanine aminotransferase [ALT] ≤ 3 times upper limit of normal and total bilirubin ≤ 5 times upper limit of normal

    8. Female subjects of childbearing potential (i.e. have reached the age of menarche) must have a negative urine pregnancy test recorded prior to the first dose of study medication, be non-lactating, and be willing to use adequate and highly effective method of contraception throughout the study and for one month after the last dose of study medication, if sexually active. A highly effective method of birth control is defined as those which result in a low failure rate (i.e. less than 1% per year) when used consistently and correctly such as sterilisation, implants, injectables, combined oral contraceptives, some IUDs (hormonal), sexual abstinence or vasectomised partner

    9. Sexually active male subjects must be willing and able to use a barrier form of
    contraception (i.e. condoms) or sexual abstinence throughout the study and for one
    month after the last dose of study medication

    10. Signed informed consent form and assent if appropriate according to local laws and regulations prior to start of any study specific procedures
    E.4Principal exclusion criteria
    Subjects who are to be excluded from the study are those who meet any of the following criteria:

    1. Subjects with clinical evidence of active symptomatic CNS disease or isolated CNS
    disease.

    2. Prior treatment with any antileukaemic agent, chemotherapy or leukophoresis treatment within 7 days (within 4–5 days for 6-mercaptopurine [MP] and within 2 days for low-dose methotrexate) prior to study entry. Corticosteroid use will not be excluded.

    3. Concurrent treatment with other anticancer agents (with the exception of CNS therapy, see Section 9.5.9.2 of the protocol).

    4. Subjects with a history of HIV and/or HTLV-1 infection
    Note: HIV and HTLV-1 testing will be performed at the Screening Visit for subjects who have not been tested within 3 months of screening. Results for HIV and HTLV-1 infection do not need to be available prior to initiation of treatment due to the aggressive nature of the disease. Subjects with a positive result will be evaluated on a case by case basis.

    5. Subjects with known active HBV, HCV, CMV and/or EBV infection
    Note: HBV, HCV, CMV and EBV testing will be performed at the Screening Visit for
    subjects who have not been tested within 3 months of screening. Results for HBV, HCV, CMV and EBV do not need to be available prior to initiation of treatment due to the aggressive nature of the disease. Subjects with active disease will be evaluated on a case by case basis.

    6. Subjects with active serious infection.

    7. Females who are pregnant (positive β-hCG test) or lactating.

    8. Lack of full recovery from adverse drug reactions due to prior therapy, independent of when that therapy was given.

    9. Subjects who have chronic gastrointestinal disease or conditions that may hamper
    compliance and/or absorption of the product; however, study drug administration via
    nasogastric or gastrostomy tube is allowed

    10. Any history of hypersensitivity or intolerance to any component of the study medication.

    11. Subjects who have received an investigational medicinal product in a clinical study within 30 days of study entry (defined as the start of the Screening Period). Current participation in another clinical trial is not permitted unless the sole purpose of the trial is for long term follow up/survival data.

    12. Previous enrolment in this clinical study.

    13. Rapidly progressive disease with compromised organ function judged to be life-threatening by the investigator.
    E.5 End points
    E.5.1Primary end point(s)
    The primary objective of the proposed study is to provide adequate PK/PD data for the development of the intravenous and oral formulation in paediatric use i.e. to determine the optimal dose for a future Phase II/III study and to demonstrate safety of forodesine in paediatric patients.

    Preliminary efficacy data on forodesine in children with relapsed or refractory
    haematological malignancies at six different dose schedules will also be assessed.
    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 Yes
    E.6.10Pharmacogenetic Information not present in EudraCT
    E.6.11Pharmacogenomic Information not present in EudraCT
    E.6.12Pharmacoeconomic Information not present in EudraCT
    E.6.13Others Information not present in EudraCT
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) Yes
    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 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 Yes
    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 Yes
    E.8.2.3.1Comparator description
    Different dosages of the same formulation will be compared.
    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 EEA21
    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 Information not present in EudraCT
    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
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years1
    E.8.9.1In the Member State concerned months9
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years1
    E.8.9.2In all countries concerned by the trial months9
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 Yes
    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) Yes
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.2Adults (18-64 years) No
    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 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 Yes
    F.3.3.6.1Details of subjects incapable of giving consent
    Subjects under the age of 16 will sign an assent form which is only valid with a fully signed Parental informed consent form.
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state6
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 28
    F.4.2.2In the whole clinical trial 28
    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 Decision2008-10-27
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2009-01-26
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2010-10-14
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