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)

Institut Paoli-Calmettes

Status and phase: Not yet enrolling; Phase 2

Conditions: Acute Leukemia; Mielodysplasic Syndrome; Myeloproliferative Neoplasm

Treatments: Drug: Busulfan Injection

Study type: Interventional

Funder types: Other

Identifiers

NCT04451200

BUSEQ-IPC 2020-006

Details and patient eligibility

About

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.

Enrollment: 82 estimated patients

Sex: All

Ages: 18 to 65 years old

Volunteers: No 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

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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82 participants in 1 patient group

busulfan treatment

Experimental group

Description:

Personalized BU administration

Treatment:

Drug: Busulfan Injection