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Sequential and Personalized PK-guided Busulfan Administration in the Frame of the Conditiong Regimen for Allo-HSCT in Patients With Malignant Hemopathies Ineligible for the Standard Myeloablative Conditioning (BUSEQ)

Primary Purpose

Acute Leukemia, Mielodysplasic Syndrome, Myeloproliferative Neoplasm

Status
Not yet recruiting
Phase
Phase 2
Locations
Study Type
Interventional
Intervention
Busulfan Injection
Sponsored by
Institut Paoli-Calmettes
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Acute Leukemia

Eligibility Criteria

18 Years - 65 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

  • Adult patient up to 65 years old
  • Acute leukemia, myelodysplastic syndrome or myeloproliferative neoplasia eligible for an allogeneic transplant
  • Chemosensitive disease, in complete or partial or stable remission
  • 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

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

Sites / Locations

    Arms of the Study

    Arm 1

    Arm Type

    Experimental

    Arm Label

    busulfan treatment

    Arm Description

    Personalized BU administration

    Outcomes

    Primary Outcome Measures

    non-relapse mortality evaluation
    non-relapse mortality evaluation

    Secondary Outcome Measures

    incidence of grade 3 or 4 toxicities
    To evaluate toxicities linked to sequential bususlfan administration
    graft taking after sequential busulfan conditioning
    incidence of hematological reconstituation
    incidence of transfusion needs for red blood cells
    number of transfusions after graft
    incidence of transfusion needs for red blood cells
    number of transfusions after graft
    incidence in graft taking after sequential busulfan conditioning
    Lymphocyte chimerism
    anti-tumoral efficacy of sequential busulfan conditioning: incidence of acute GVH
    incidence of acute GVH
    anti-tumoral efficacy of sequential busulfan conditioning: incidence of acute GVH
    incidence of acute GVH
    the anti-tumoral efficacy of sequential busulfan conditioning: incidence of chronic GVH
    incidence of chronic GVH
    the anti-tumoral efficacy of sequential busulfan conditioning: incidence of chronic GVH
    incidence of chronic GVH
    anti-tumoral efficacy of sequential busulfan conditioning: progression-free survival
    progression-free survival
    anti-tumoral efficacy of sequential busulfan conditioning: progression-free survival
    overall survival
    anti-tumoral efficacy of sequential busulfan conditioning: progression-free survival
    overall survival
    anti-tumoral efficacy of sequential busulfan conditioning: Incidence of relapse
    Incidence of relapse
    Differences between the theoretical target AUC and the measured a posteriori
    To study the pharmacokinetics of the sequential administration of busulfan

    Full Information

    First Posted
    June 3, 2020
    Last Updated
    November 16, 2020
    Sponsor
    Institut Paoli-Calmettes
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    1. Study Identification

    Unique Protocol Identification Number
    NCT04451200
    Brief Title
    Sequential and Personalized PK-guided Busulfan Administration in the Frame of the Conditiong Regimen for Allo-HSCT in Patients With Malignant Hemopathies Ineligible for the Standard Myeloablative Conditioning
    Acronym
    BUSEQ
    Official Title
    Sequential and Personalized Pharmacokinetic-guided Busulfan Administration in the Frame of the Conditiong Regimen for Allogeneic Haematopoietic Stem Cell Transplantation in Patients With Malignant Hemopathies Ineligible for the Standard Myeloablative Conditioning
    Study Type
    Interventional

    2. Study Status

    Record Verification Date
    October 2020
    Overall Recruitment Status
    Not yet recruiting
    Study Start Date
    November 2020 (Anticipated)
    Primary Completion Date
    November 2023 (Anticipated)
    Study Completion Date
    December 2028 (Anticipated)

    3. Sponsor/Collaborators

    Responsible Party, by Official Title
    Sponsor
    Name of the Sponsor
    Institut Paoli-Calmettes

    4. Oversight

    Studies a U.S. FDA-regulated Drug Product
    No
    Studies a U.S. FDA-regulated Device Product
    No
    Product Manufactured in and Exported from the U.S.
    No
    Data Monitoring Committee
    No

    5. Study Description

    Brief Summary
    Because the anti-leukemic activity of busulfan, this dug is largely used in graft conditioning but in elderly and/or cormobid patienth an excess of toxicity is observed. This study focus on the possibility of significanty reducing this toxicity by customizing the doses of busulfan to individual PK parameters.

    6. Conditions and Keywords

    Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
    Acute Leukemia, Mielodysplasic Syndrome, Myeloproliferative Neoplasm

    7. Study Design

    Primary Purpose
    Treatment
    Study Phase
    Phase 2
    Interventional Study Model
    Single Group Assignment
    Masking
    None (Open Label)
    Allocation
    N/A
    Enrollment
    82 (Anticipated)

    8. Arms, Groups, and Interventions

    Arm Title
    busulfan treatment
    Arm Type
    Experimental
    Arm Description
    Personalized BU administration
    Intervention Type
    Drug
    Intervention Name(s)
    Busulfan Injection
    Intervention Description
    injections doses will be personalized by PK at days -7 and -4
    Primary Outcome Measure Information:
    Title
    non-relapse mortality evaluation
    Description
    non-relapse mortality evaluation
    Time Frame
    100 days post graft
    Secondary Outcome Measure Information:
    Title
    incidence of grade 3 or 4 toxicities
    Description
    To evaluate toxicities linked to sequential bususlfan administration
    Time Frame
    1 month
    Title
    graft taking after sequential busulfan conditioning
    Description
    incidence of hematological reconstituation
    Time Frame
    day 30 and day 100 post graft
    Title
    incidence of transfusion needs for red blood cells
    Description
    number of transfusions after graft
    Time Frame
    day 30 post graft
    Title
    incidence of transfusion needs for red blood cells
    Description
    number of transfusions after graft
    Time Frame
    day 60 post graft
    Title
    incidence in graft taking after sequential busulfan conditioning
    Description
    Lymphocyte chimerism
    Time Frame
    day 100 post graft
    Title
    anti-tumoral efficacy of sequential busulfan conditioning: incidence of acute GVH
    Description
    incidence of acute GVH
    Time Frame
    1 year
    Title
    anti-tumoral efficacy of sequential busulfan conditioning: incidence of acute GVH
    Description
    incidence of acute GVH
    Time Frame
    5 years
    Title
    the anti-tumoral efficacy of sequential busulfan conditioning: incidence of chronic GVH
    Description
    incidence of chronic GVH
    Time Frame
    1 and 5 years
    Title
    the anti-tumoral efficacy of sequential busulfan conditioning: incidence of chronic GVH
    Description
    incidence of chronic GVH
    Time Frame
    1 year
    Title
    anti-tumoral efficacy of sequential busulfan conditioning: progression-free survival
    Description
    progression-free survival
    Time Frame
    5 years
    Title
    anti-tumoral efficacy of sequential busulfan conditioning: progression-free survival
    Description
    overall survival
    Time Frame
    1 year
    Title
    anti-tumoral efficacy of sequential busulfan conditioning: progression-free survival
    Description
    overall survival
    Time Frame
    5 years
    Title
    anti-tumoral efficacy of sequential busulfan conditioning: Incidence of relapse
    Description
    Incidence of relapse
    Time Frame
    1 year
    Title
    Differences between the theoretical target AUC and the measured a posteriori
    Description
    To study the pharmacokinetics of the sequential administration of busulfan
    Time Frame
    1 month

    10. Eligibility

    Sex
    All
    Minimum Age & Unit of Time
    18 Years
    Maximum Age & Unit of Time
    65 Years
    Accepts Healthy Volunteers
    No
    Eligibility Criteria
    Inclusion Criteria: Adult patient up to 65 years old Acute leukemia, myelodysplastic syndrome or myeloproliferative neoplasia eligible for an allogeneic transplant Chemosensitive disease, in complete or partial or stable remission 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 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
    Central Contact Person:
    First Name & Middle Initial & Last Name or Official Title & Degree
    Dominique Genre, MD
    Phone
    +33491223778
    Email
    drci.up@ipc.unicancer.fr

    12. IPD Sharing Statement

    Plan to Share IPD
    No

    Learn more about this trial

    Sequential and Personalized PK-guided Busulfan Administration in the Frame of the Conditiong Regimen for Allo-HSCT in Patients With Malignant Hemopathies Ineligible for the Standard Myeloablative Conditioning

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