PERSONALIZED BUSULFAN ADMINISTRATION IN GRAFT CONDITIONING FOR PATIENTS WITH MALIGNANT HEMOPATHIA

SEQUENTIAL AND PERSONALIZED BUSULFAN ADMINISTRATION BY PHARMACOKINETICS IN ALLOGENIC GRAFT CONDITIONING FOR PATIENTS WITH MALIGNANT HEMOPATHIA NOT ELIGIBLE FOR STANDARD MYELOABLATIVE PACKAGIN - BUSEQ

Status

Active, not recruiting

Phases

Phase 2

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2020-003392-17-FR

Enrollment

Registered

2020-12-22

Start date

2021-02-17

Completion date

Unknown

Last updated

2023-08-28

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Acute Leukemia Mielodysplasic Syndrome Myeloproliferative Neoplasm

Interventions

Pharmaceutical Form: Solution for infusion INN or Proposed INN: BUSULFAN Current Sponsor code: BUSULFAN Concentration unit: mg milligram(s) Concentration type: equal

Eligibility

Sex/Gender

All

Inclusion criteria

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 Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 82 F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range

Exclusion criteria

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

Design outcomes

Primary

Measure	Time frame
Main Objective: 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);Secondary Objective: 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;Primary end point(s): non-relapse mortality evaluation (Time Frame: 100 days post graft );Timepoint(s) of evaluation of this end point: 100 days post graft	—

Measure

Time frame

Main Objective: 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);Secondary Objective: 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;Primary end point(s): non-relapse mortality evaluation (Time Frame: 100 days post graft );Timepoint(s) of evaluation of this end point: 100 days post graft

—

Secondary

Measure	Time frame
Secondary 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;Timepoint(s) of evaluation of this end point: Days 30, 60 and 90 post-graft. 1 and 5 years post-graft	—

Measure

Time frame

Secondary 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;Timepoint(s) of evaluation of this end point: Days 30, 60 and 90 post-graft. 1 and 5 years post-graft

—

Countries

France

Contacts

Public ContactProject manager

Institut Paoli-Calmettes

+330491223631

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026