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NCT Number: NCT01985061

Evaluation of 3 Different Doses of IV Busulfan

Albeit the safety of the stem cell transplantation procedure has been greatly improved, further refining the intensity of the conditioning is an important issue to explore, especially in patients with poor prognosis, the goal being to maintain the very favorable safety profile and improve the disease control. This is the goal our prospective trial; we aim to prospectively evaluate in a prospective multicenter trial the efficacy of different conditioning regimens in patients with high-risk myeloid malignancies.

The study is a phase II trial randomizing patients between a prospective active control arm (BX2) and two experimental arms (BX3 and BX4). A standard group was kept in this clinical trial in order to avoid the limitations induced by the comparison with historical controls in the context of continuously improving practice. Each experimental arm will be conducted in parallel according to a standard phase II trial design.

In addition, this trial will associate four ancillary studies to the main clinical objective: 1/ a prospective assessment of the quality of life of the patients over a period of 2 years 2/ an analysis of the cost effectiveness of the procedure, assessed over a period of 2 years 3/ an observational busulfan pharmacokinetic study 4/ a busulfan pharmacogenomic study

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Key information

Conditions

Myelodysplastic Syndrome Acute Myeloid Leukemia Bone Marrow Diseases Hematologic Diseases Hemic and Lymphatic Diseases Leukemia Leukemia, Myeloid Leukemia, Myeloid, Acute Myelodysplastic Syndromes Neoplasms Neoplasms by Histologic Type

Age range

18 year–65 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Institut Paoli Calmettes

Marseille, 13009, France

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Patients with poor prognosis myeloid malignancies:
  • Myelodysplastic syndrome,
  • Acute Myeloid Leukemia (AML) beyond Complete Response (CR1),
  • CR1 AML with poor risk cytogenetics
  • Adult patients: aged ≥ 55 years up to 65 or < 55 years not eligible for myeloablative conditioning regimen based on Total Body Irradiation (TBI) or double alkylating agent combinations.
  • Availability of a HLA identical sibling or matched unrelated donor (10/10)
  • Affiliation to social security
  • Written Informed Consent

Exclusion criteria

  • History of previous Allo-Hematological Stem Cell Transplantation (HSCT)
  • HIV positivity
  • Signs of chronic active hepatitis B and/or C
  • Evolutive psychiatric disease
  • Concomitant neoplastic disease
  • Pregnant or lactating woman or without contraception (for child bearing potential wom-en)
  • Usual contra-indications for Allo-HSCT

Treatment and study plan

BX2

Drug

Other names: Busulfan Intravenous 2 days at 3.2 mg/kg/d

BX3

Drug

Other names: Busulfan Intravenous 3 days

BX4-Suspended

Drug

Suspended

Other names: Busulfan intravenous 4 days

Primary outcomes

  1. Time to progression or death

    Time frame: up to 2 years

    2-year progression free survival rates

Secondary outcomes

  1. Time to neutrophil>0.5G/l and platelets>50G/l

    Time frame: up to 2 months

    hematologic recovery

  2. Graft versus host disease

    Time frame: up to 2 years

  3. relapse

    Time frame: up to 2 years

  4. Occurrence of grade 3-4 adverse events according the CTC-AE v4.0 scale

    Time frame: up to 6 months

    safety

Sponsors and collaborators

Lead sponsor

Institut Paoli-Calmettes

Other

Registry information

Official study title

Prospective and Multicentre Evaluation of 3 Different Doses of IV Busulfan Associated With Fludarabine and Thymoglobuline in the Conditioning of Allogeneic Stem Cell Transplantation (SCT) From a Matched Related or Unrelated Donor in Patients With Poor Prognosis Myeloid Malignancies

Acronym: AAA

Important dates

Study start
2013
Primary completion
2026
Study completion
2026
First posted
Nov 15, 2013
Registry last updated
Jun 15, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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