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    Summary
    EudraCT Number:2004-004286-15
    Sponsor's Protocol Code Number:BCX1777-T-04-201
    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:2005-11-28
    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 number2004-004286-15
    A.3Full title of the trial
    A Phase II, Multi-Center, Open-Label, Repeat-Dose Study of Forodesine Hydrochloride (BCX-1777) Infusion in Patients with Advanced T-Cell Leukemia with an Option of Long-Term Forodesine Hydrochloride (BCX-1777) Use
    A.3.2Name or abbreviated title of the trial where available
    N/A
    A.4.1Sponsor's protocol code numberBCX1777-T-04-201
    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 SponsorBioCryst Pharmaceuticals Inc.
    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 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 No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameForodesine Hydrochloride
    D.3.2Product code BCX-1777
    D.3.4Pharmaceutical form Intravenous infusion
    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.3.9.2Current sponsor codeBCX-1777
    D.3.9.3Other descriptive nameForodesine Hydrochloride
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number5
    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 Yes
    D.3.11.13.1Other medicinal product typeEnzyme inhibitor
    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
    Advanced T-cell Leukaemia (either T-cell Acute Lymphoblastic Leukaemia [T-ALL] or T-cell Prolymphocytic Leukaemia [T-PLL]) or Aleukaemic Leukaemia.
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 7.0
    E.1.2Level hlgt
    E.1.2Classification code 10024324
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To determine the sustained effectiveness (for a minimum of 28 days) of
    intravenous (IV) forodesine hydrochloride infusions in patients with
    advanced T-cell leukemia (precursor T-lymphoblastic leukemia/lymphoma or
    T-cell prolymphocytic leukemia [T-PLL])
    E.2.2Secondary objectives of the trial
    •To assess the safety and tolerability of multiple infusion cycles of forodesine
    hydrochloride in this patient population
    •To evaluate the effects of forodesine hydrochloride on plasma levels
    of 2-deoxyguanosine (dGuo) and red blood cell (RBC) purine nucleoside
    phosphorylase (PNP) activity and to correlate levels with efficacy parameters
    •To describe the steady-state pharmacokinetics (PK) and pharmacodynamics (PD)
    of forodesine hydrochloride following repeat administration
    •To evaluate the maintenance of response and safety of forodesine hydrochloride
    in long-term treatment
    E.2.3Trial contains a sub-study Information not present in EudraCT
    E.3Principal inclusion criteria
    •Documented T-cell leukemia (precursor T-lymphoblastic leukemia/lymphoma or T-PLL)
    •Failure to have responded to one or more standard regimens for their disease.
    •Performance status of <=2 by Eastern Cooperative Oncology Group (ECOG) criteria
    •Age - 18 years or older
    •Life expectancy of at least 3 months
    •Adequate liver function (aspartate transaminase [AST] and/or alanine transaminase
    [ALT] not >3 times upper limits of normal)
    •Adequate kidney function (calculated creatinine clearance >50 mL/min)
    •Negative urine pregnancy test within 2 to 7 days prior to the start of study treatment in females of childbearing potential
    •Females of childbearing potential and males must be willing and able to use an
    adequate method of contraception to avoid pregnancy for the duration of the study in such a manner that the risk of pregnancy is minimized
    •Signed informed consent/assent form prior to start of any study specific procedures
    E.4Principal exclusion criteria
    •Patients with known human immunodeficiency virus (HIV) infection or human T-cell
    leukemia virus type 1 (HTLV-1)
    •Patients with known Hepatitis B and/or Hepatitis C active infection
    •Tumor-related central nervous system (CNS) leukemia requiring active treatment
    •Active serious infection not controlled by oral or IV antibiotics
    •Treatment with any investigational antileukemic agent or chemotherapy agent within 7 days prior to study entry, unless full recovery from side effects has occurred
    •Rapidly progressive disease with compromised organ function judged to be life threatening by the Investigator
    •Concurrent treatment with other anticancer agents (corticosteroid use will not be
    excluded, but patient must remain on a stable dose)
    •Pregnant and/or lactating female
    •Cutaneous T-cell lymphoma (CTCL) diagnosis (including Sezary Syndrome)
    E.5 End points
    E.5.1Primary end point(s)
    The proportion of patients with a sustained response will constitute the primary outcome measure for the trial.
    A responding patient is one with a partial or complete response.
    A patient with a sustained response is one responding to treatment for at least 28 days, beginning with the first laboratory measurement that shows a clinically significant response and concludes with the Cycle-6 (or Cycle-8) assessments. For precursor T-lymphoblastic leukemia/lymphoma or T-PLL patients with circulating tumor cells, a sustained response will be based on the peripheral blood assessment; for patients who have no disease in the peripheral blood, a sustained response will be based on the bone marrow assessment. For patients with extramedullary disease, a sustained response will also be based on assessments made by additional test methods (i.e. CT scans). During the 28-day period, a partial response or better status must be maintained.
    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 Information not present in EudraCT
    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 No
    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.8.3 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 concerned6
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA17
    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 Information not present in EudraCT
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    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
    The end of the trial will be following the recruitment of 50 evaluable patients and the closure of the database containing the data. It is possible that the database will be closed for analysis of the primary and secondary endpoints whilst some patients may still be receiving BCX-1777 under the compassionate use arm of the study. Length of time to receive BCX-1777 under compassionate use is not limited.
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years2
    E.8.9.1In the Member State concerned months
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years3
    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) Yes
    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 Information not present in EudraCT
    F.3.3.1Women of childbearing potential not using contraception For clinical trials recorded in the database before the 10th March 2011 this question read: "Women of childbearing potential" and did not include the words "not using contraception". An answer of yes could have included women of child bearing potential whether or not they would be using contraception. The answer should therefore be understood in that context. This trial was recorded in the database on 2005-11-28. Yes
    F.3.3.2Women of child-bearing potential using contraception Information not present in EudraCT
    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 state15
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 35
    F.4.2.2In the whole clinical trial 80
    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)
    patients who would continue to benefit from the receipt of BCX-1777 treatment will be able to receive the test compound under a compassionate use program.
    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 Decision2005-11-02
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2005-07-19
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2007-03-27
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