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    Summary
    EudraCT Number:2020-003392-17
    Sponsor's Protocol Code Number:BUSEQ-IPC2020-006
    National Competent Authority:France - ANSM
    Clinical Trial Type:EEA CTA
    Trial Status:Prematurely Ended
    Date on which this record was first entered in the EudraCT database:2020-12-22
    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 number2020-003392-17
    A.3Full title of the trial
    SEQUENTIAL AND PERSONALIZED BUSULFAN ADMINISTRATION BY PHARMACOKINETICS IN ALLOGENIC GRAFT CONDITIONING FOR PATIENTS WITH MALIGNANT HEMOPATHIA NOT ELIGIBLE FOR STANDARD MYELOABLATIVE PACKAGIN
    ADMINISTRATION DU BUSULFAN SEQUENTIELLE ET PERSONNALISEE PAR LA PHARMACOCINETIQUE DANS LE CONDITIONNEMENT DE GREFFE ALLOGENIQUE CHEZ LES PATIENTS ATTEINTS D’HEMOPATHIES MALIGNES NON ELIGIBLES AU CONDITIONNEMENT MYELOABLATIF STANDARD
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    PERSONALIZED BUSULFAN ADMINISTRATION IN GRAFT CONDITIONING FOR PATIENTS WITH MALIGNANT HEMOPATHIA
    ADMINISTRATION DU BUSULFAN PERSONNALISEE DANS LE CONDITIONNEMENT DE GREFFE CHEZ LES PATIENTS ATTEINTS D’HEMOPATHIES MALIGNES
    A.3.2Name or abbreviated title of the trial where available
    BUSEQ
    BUSEQ
    A.4.1Sponsor's protocol code numberBUSEQ-IPC2020-006
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT04451200
    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 supportMinistère des solidarités et de la santé
    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 pointProject manager
    B.5.3 Address:
    B.5.3.1Street Address232, Bd Sainte Marguerite - BP 156
    B.5.3.2Town/ cityMarseille
    B.5.3.3Post code13273
    B.5.3.4CountryFrance
    B.5.4Telephone number+330491223631
    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.2.1.2Country 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.3.4Pharmaceutical form Solution for infusion
    D.3.4.1Specific paediatric formulation No
    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 INNBUSULFAN
    D.3.9.2Current sponsor codeBUSULFAN
    D.3.9.4EV Substance CodeSUB05993MIG
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    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
    Acute Leukemia
    Mielodysplasic Syndrome
    Myeloproliferative Neoplasm
    Leucémie aiguë, syndrome myélodysplasique ou néoplasie myéloproliférative
    E.1.1.1Medical condition in easily understood language
    Blood cancer
    Cancer sanguin
    E.1.1.2Therapeutic area Diseases [C] - Cancer [C04]
    MedDRA Classification
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    to reduce NRM (non-relapse mortality) by optimizing the administration of busulfan by:
    • Sequential delivery of a total myeloablative dose
    • The use of a personalized approach based on pharmacokinetics (PK)
    L’objectif est de réduire la NRM (non-relapse mortality) en optimisant l’administration du busulfan par :
    • La délivrance séquentielle d’une dose totale myéloablative
    • L’utilisation d’une approche personnalisée basée sur la pharmacocinétique (PK)
    E.2.2Secondary objectives of the trial
    Evaluate the toxicities linked to BuSeq conditioning
    Evaluate graft taking after BuSeq type conditioning
    Evaluate the anti-tumor efficacy of BuSeq conditioning
    Study the pharmacokinetics of the sequential administration of busulfan
    Evaluer les toxicités liées au conditionnement BuSeq
    Evaluer la prise de greffe après conditionnement de type BuSeq
    Evaluer l’efficacité anti tumorale du conditionnement BuSeq
    Etudier la pharmacocinétique de l’administration séquentielle du busulfan
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Adult patient up to 65 years old
    Acute leukemia, myelodysplastic syndrome or myeloproliferative neoplasia eligible for an allogeneic transplant
    Allograft from an identical HLA related donor, Haplo-identical or unrelated (HLA compatibility from 8/10 to 10/10 according to HLA-A, -B, -C, -DR, -DQ allelics)
    Signed consent to participate
    Affiliation to a social security regimen or beneficiary of this regimen
    Patient not eligible for standard myeloablative conditioning due to age> = 45 years and / or the presence of an HCT-CI comorbidity score> = 3
    Patient adulte jusqu’à 65 ans inclus
    Leucémie aiguë, syndrome myélodysplasique ou néoplasie myéloproliférative éligible à une greffe allogénique
    Allogreffe à partir d’un donneur apparenté HLA identique, Haplo-identique ou non apparenté (compatibilité HLA de 8/10 à 10/10 selon HLA-A, -B, -C, -DR, -DQ alléliques)
    Consentement de participation signé
    Affiliation à un régime de sécurité sociale ou bénéficiaire d’un tel régime
    Patient non éligible à un conditionnement myéloablatif standard du fait de l’âge >= 45 ans et/ou la présence d’un score de comorbidité HCT-CI >= 3
    E.4Principal exclusion criteria
    Pregnant woman, without effective contraception or breastfeeding
    Person in emergency situation, patient deprived of liberty or placed under the authority of a tutor,
    Impossibility of undergoing medical follow-up of the trial for geographic, social or psychological reasons
    Contraindications to performing an allogeneic transplant
    Previous allograft
    Placental blood allograft
    Femme enceinte, sans contraception efficace ou allaitant
    Personne en situation d’urgence, faisant l’objet d’une mesure de protection légale, ou hors d’état d’exprimer son consentement
    Impossibilité de se soumettre au suivi médical de l'essai pour des raisons géographiques, sociales ou psychiques
    Contre-indications à la réalisation d’une greffe allogénique
    Allogreffe antérieure
    Allogreffe de sang placentaire
    E.5 End points
    E.5.1Primary end point(s)
    non-relapse mortality evaluation (Time Frame: 100 days post graft )
    Mortalité non liée à la rechute (NRM) à 100 jours après allogreffe
    E.5.1.1Timepoint(s) of evaluation of this end point
    100 days post graft
    100 jours après allogreffe
    E.5.2Secondary end point(s)
    • Incidence of grade 3 or 4 toxicities, related to the study procedure, in the month following the transplant, including veno-occlusive disease and hemorrhagic cystitis
    • Incidence and kinetics of hematological reconstitution (neutrophils> 0.5 G / L on D + 30 and D + 100 and platelets> 20 G / L on D + 30 and D + 100)
    • Incidence of transfusion needs for red blood cells at D + 30 and D + 100
    • Lymphocyte chimerism on D + 30, D + 60 and D + 100
    • Incidence of acute GVH at D100 and chronic at 1 year
    • NRM at 1 year and 5 years
    • Incidence of relapse at 1 year
    • Progression-free survival at 1 year and 5 years
    • Overall survival at 1 year and 5 years
    • Survival without disease and without GVH at 1 year and 5 years
    • For the PK of busulfan: Difference between the theoretical target AUC and that measured a posteriori
    Incidence des toxicités de grade 3 ou 4, liées à la procédure de l’étude, dans le mois suivant la greffe, notamment maladie veno-occlusive et cystite hémorragique
    Incidence et cinétique de la reconstitution hématologique (neutrophiles > 0.5 G/L à J+30 et J+100 et plaquettes > 20 G/L à J+30 et J+100)
    Incidence des besoins transfusionnels de globules rouges à J+30 et J+100
    Chimérisme lymphocytaire à J+30, J+60 et J+100
    Incidence de GVH aigue à J100 et chronique à 1 an
    NRM à 1 an et à 5 ans
    Incidence de la rechute à 1 an
    Survie sans progression à 1 an et à 5 ans
    Survie globale à 1 an et à 5 ans
    Survie sans maladie et sans GVH à 1 an et à 5 ans
    Pour la PK du busulfan : Différence entre l’AUC cible théorique et celle mesurée a posteriori
    E.5.2.1Timepoint(s) of evaluation of this end point
    Days 30, 60 and 90 post-graft. 1 and 5 years post-graft
    jours 30, 60 et 90 post-greffe. 1 an et 5ans après la greffe.
    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 No
    E.6.4Safety No
    E.6.5Efficacy No
    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 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 concerned2
    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
    fin de la recherche corresponds a la dernière visite du dernier patient
    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 months97
    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: 82
    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 state82
    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 Decision2020-12-06
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2021-02-17
    P. End of Trial
    P.End of Trial StatusPrematurely Ended
    P.Date of the global end of the trial2023-07-19
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