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    Summary
    EudraCT Number:2014-000758-12
    Sponsor's Protocol Code Number:BX-PK-IPC2013-016
    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:2014-04-10
    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 number2014-000758-12
    A.3Full title of the trial
    Study of pharmacokinetics of intravenous busulfan (Busilvex ®) in the conditioning allogeneic transplantation in patients with high-risk hematological disease.
    Etude de pharmacocinétique du Busulfan intra-veineux (Busilvex®) dans le conditionnement d’une greffe allogénique chez des patients à haut risque porteurs d’hémopathie.
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Determine the efficacy and tolerability of appropriate doses of busulfan to administer in patients with high risk of hematological disease.
    Déterminer l'efficacité et la tolérance de doses adaptées de Busulfan à administrer chez des patients à haut risque atteints d'hémopathie.
    A.3.2Name or abbreviated title of the trial where available
    BX-PK-IPC2013-016
    A.4.1Sponsor's protocol code numberBX-PK-IPC2013-016
    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 SponsorINSTITUT PAOLI CALMETTES
    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 supportAgence de la Biomédecine
    B.4.2CountryFrance
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationINSTITUT PAOLI CALMETTES
    B.5.2Functional name of contact pointDIRECTOR OF DRCI
    B.5.3 Address:
    B.5.3.1Street Address232 Bd de Sainte-Marguerite - BP156
    B.5.3.2Town/ cityMarseille cedex 9
    B.5.3.3Post code13273
    B.5.3.4CountryFrance
    B.5.4Telephone number33 4 91223778
    B.5.5Fax number33 4 91223601
    B.5.6E-maildrci.up@ipc.unicancer.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 Yes
    D.2.1.1.1Trade name BUSULFAN
    D.2.1.1.2Name of the Marketing Authorisation holderPierre FABRE
    D.2.1.2Country which granted the Marketing AuthorisationFrance
    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 nameBUSILVEX
    D.3.2Product code L01AB01
    D.3.4Pharmaceutical form Solution for infusion
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous use
    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
    hematological malignancies : Acute Myeloblastic Leukemia, Chronic Lymphoïd Leukemia, Multiple myéloma, Acute lymphoblastic leukemia.
    Hemopathies malignes
    E.1.1.1Medical condition in easily understood language
    Hematological malignancies
    Hemopathies malignes
    E.1.1.2Therapeutic area Diseases [C] - Cancer [C04]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 17.0
    E.1.2Level LLT
    E.1.2Classification code 10063625
    E.1.2Term Acute lymphoblastic leukemia recurrent
    E.1.2System Organ Class 100000004864
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 17.0
    E.1.2Level LLT
    E.1.2Classification code 10000878
    E.1.2Term Acute myeloblastic leukemia
    E.1.2System Organ Class 100000004864
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    Determine the effectiveness of a myeloablative conditioning combining fludarabine, thymoglobulin, and intravenous busulfan in appropriate dose based on pharmacokinetic study made the first day of administration of busulfan in preparation for allogeneic HLA-compatible family or non-family.
    Il s’agit de déterminer l’efficacité d’un conditionnement myéloablatif associant Fludarabine, Thymoglobuline, et Busulfan intra-veineux à dose adaptée, en fonction d’une pharmacocinétique réalisée au premier jour d’administration (ou J-6 du conditionnement) du busulfan, en préparation à une greffe allogénique HLA compatible familiale ou non familiale.
    E.2.2Secondary objectives of the trial
    - Study of pharmacokinetics of intravenous busulfan performed on day 1 of administration (or J-6 packaging), and allowing the adaptation of doses remaining three days of administration of busulfan, and J-2 to monitor patient exposure.

    - Determine the tolerance according to the usual criteria of allograft, that is to say chimerism (M1, M2 and M3), hematologic recovery, the rate of response to treatment, the incidence of graft against the host (GVH) acute and chronic, relapse and mortality unrelated to relapse, overall survival, and tolerance to 6 months.
    - Etude de pharmacocinétique du Busulfan intra-veineux réalisée au 1er jour d’administration (ou J-6 du conditionnement), et permettant l’adaptation de doses sur les trois jours restant d’administration du Busulfan, et à J-2 afin de contrôler l’exposition du patient.

    - Déterminer la tolérance selon les critères habituels de greffe allogénique, c’est-à-dire le chimérisme (M1, M2 et M3), la récupération hématologique, le taux de réponse au traitement, l’incidence du greffon contre l’hôte (GVH) aigüe et chronique, de la rechute et de la mortalité non liée à la rechute, la survie globale, et la tolérance à 6 mois.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Consent to participate signed
    2. Affiliation to a social security scheme, or beneficiary of such a system,
    3. Related or unrelated donor HLA identical: All patients age ≥ 55 years with hematologic malignancies and considered eligible for an allogeneic transplant from a donor genotype and phenotype identical identical 10/10,
    4. The basic pathology should be considered "chemo-sensitive" complete or partial remission (CR, PR) or stable disease (SD).
    1. Consentement de participation signé,
    2. Affiliation à un régime de sécurité sociale, ou bénéficiaire d’un tel régime,
    3. Donneur apparenté ou non apparenté HLA identique : Tous les patients d’un âge ≥ 55 ans atteints d’une hémopathie maligne et jugés éligibles pour une greffe allogénique à partir d’un donneur géno-identique et phéno-identique 10/10,
    4. La pathologie de base doit être considérée « chimio-sensible »: rémission complète ou partielle (RC, RP) ou maladie stable (SD).
    E.4Principal exclusion criteria
    1. Pregnant or likely to be (without effective contraception) or breastfeeding woman
    2. Person in an emergency situation, a major person under a legal protection measure (major guardianship, curatorship or judicial protection), or unable to consent,
    3. Inability to undergo medical monitoring test for geographical, social or psychological reasons,
    4. Cons-indications for allogeneic transplantation,
    5. Age <55 years
    6. Previous allograft
    7. Convert cancerous progressive disease concomitantly
    8. Evolutionary psychiatric condition,
    9. Seropositive for human immunodeficiency virus or hepatitis C scalable requiring treatment,
    10. Women of childbearing potential and men, in the absence of effective contraception during treatment and for 12 months after stopping treatment.
    1. Femme enceinte ou susceptible de l’être (sans contraception efficace) ou allaitant,
    2. Personne en situation d’urgence, personne majeure faisant l’objet d’une mesure de protection légale (majeur sous tutelle, curatelle ou sauvegarde de justice), ou hors d’état d’exprimer son consentement,
    3. Impossibilité de se soumettre au suivi médical de l'essai pour des raisons géographiques, sociales ou psychiques,
    4. Contre-indications à une greffe allogénique,
    5. Age < 55 ans,
    6. Allogreffe antérieure,
    7. Autre maladie cancéreuse évolutive de façon concomitante,
    8. Affection psychiatrique évolutive,
    9. Sérologie positive pour le virus de l’immunodéficience humaine ou hépatite C évolutive nécessitant un traitement,
    10. Femmes en âge de procréer ou homme, en l'absence de contraception efficace pendant le traitement et jusqu'à 12 mois après l'arrêt du traitement.
    E.5 End points
    E.5.1Primary end point(s)
    Evaluate the progression free survival at 2 years
    Evaluer la survie sans progression à 2 ans
    E.5.1.1Timepoint(s) of evaluation of this end point
    date of progression disease
    date de progression
    E.5.2Secondary end point(s)
    - Plasma concentration of busulfan,
    - Death is not related to the disease (NRM),
    - Effect of acute and chronic GVHD GVHD,
    - Haematological recovery,
    - Engraftment and chimerism full 100 donor
    - Incidence of relapse,
    - Overall Survival.
    - Concentration plasmatique de Busulfan,
    - Décès non liée à la maladie (NRM),
    - Incidence de GVHD aigue et GVHD chronique,
    - Récupération hématologique,
    - Prise de greffe et de chimérisme donneur complet à 100,
    - Incidence de la rechute,
    - Survie globale.
    E.5.2.1Timepoint(s) of evaluation of this end point
    Pharmacokinetic study
    Date of death
    disease status and MPG up to 24 months
    engraftment and full donor chimerism 100% aus months 1, 2 and 3
    Etude de pharmacocinétique
    date de décès
    statut de la maladie et de la GVH jusqu'à 24 mois
    prise de greffe et de chimérisme donneur complet à 100% aus mois 1, 2 et 3
    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 No
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic Yes
    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.8.3 The trial involves single site in the Member State concerned Yes
    E.8.4 The trial involves multiple sites in the Member State concerned No
    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
    LVLS
    Dernière visite du dernier patient inclus
    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 months48
    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.1.2.1Number of subjects for this age range: 33
    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 state33
    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)
    None
    Non
    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 Decision2014-05-30
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2014-04-09
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2020-05-05
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