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Trial record 1 of 1 for:    AAA IPC 2011-003 | Recruiting Studies
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Evaluation of 3 Different Doses of IV Busulfan (AAA)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. Identifier: NCT01985061
Recruitment Status : Recruiting
First Posted : November 15, 2013
Last Update Posted : July 13, 2018
Information provided by (Responsible Party):
Institut Paoli-Calmettes

Brief Summary:

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

Condition or disease Intervention/treatment Phase
Myelodysplastic Syndrome Acute Myeloid Leukemia Drug: BX2 Drug: BX3 Drug: BX4-Suspended Phase 2

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 177 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official 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
Actual Study Start Date : December 2013
Estimated Primary Completion Date : December 2018
Estimated Study Completion Date : December 2018

Arm Intervention/treatment
Active Comparator: BX2
Fludarabine (Fludara®): 30 mg/m2 on D-6, D-5, D-4, D-3 and D-2 Busulfan IV (Busilvex®) : 3.2 mg/kg/d on D-4 and D-3 Thymoglobuline®: 2.5 mg/kg/d on D-3 and D-2
Drug: BX2
Other Name: Busulfan Intravenous 2 days at 3.2 mg/kg/d

Experimental: BX3
Fludarabine (Fludara®): 30 mg/m² on D-6, D-5, D-4, D-3 and D-2 Busulfan IV (Busilvex®) : 3.2 mg/kg/d on D-5, D-4 and D-3 Thymoglobuline® : 2.5 mg/kg/d on D-3 and D-2
Drug: BX3
Other Name: Busulfan Intravenous 3 days

Active Comparator: BX4-Suspended
Fludarabine (Fludara®): 30 mg/m²on D-6, D-5, D-4, D-3 and D-2 Busulfan IV (Busilvex®) : 3.2 mg/kg/d on D-6, D-5, D-4 and D-3 Thymoglobuline® : 2.5 mg/kg/d on D-3 and D-2
Drug: BX4-Suspended
Other Name: Busulfan intravenous 4 days

Primary Outcome Measures :
  1. Time to progression or death [ Time Frame: up to 2 years ]
    2-year progression free survival rates

Secondary Outcome Measures :
  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 ]

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years to 65 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  1. Patients with poor prognosis myeloid malignancies:

    • Myelodysplastic syndrome,
    • Acute Myeloid Leukemia (AML) beyond Complete Response (CR1),
    • CR1 AML with poor risk cytogenetics
  2. 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.
  3. Availability of a HLA identical sibling or matched unrelated donor (10/10)
  4. Affiliation to social security
  5. Written Informed Consent

Exclusion Criteria:

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT01985061

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Contact: Dominique GENRE, MD 33491223778
Contact: Jihane PAKRADOUNI, PharmD,PhD 33491223778

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Institut Paoli Calmettes Recruiting
Marseille, France, 13009
Contact: Dominique GENRE, MD    33491223778   
Contact: Jihane PAKRADOUNI, PharmD, PhD    33491223778   
Principal Investigator: Didier BLAISE, MD PhD         
Sponsors and Collaborators
Institut Paoli-Calmettes
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Principal Investigator: Didier BLAISE, MD PhD Institut Paoli-Calmettes

Additional Information:
Publications automatically indexed to this study by Identifier (NCT Number):
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Responsible Party: Institut Paoli-Calmettes Identifier: NCT01985061    
Other Study ID Numbers: AAA-IPC2011-003
First Posted: November 15, 2013    Key Record Dates
Last Update Posted: July 13, 2018
Last Verified: July 2018
Keywords provided by Institut Paoli-Calmettes:
Myelodysplasic syndrome
Acute Myeloid leukemia
Additional relevant MeSH terms:
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Leukemia, Myeloid
Leukemia, Myeloid, Acute
Myelodysplastic Syndromes
Neoplasms by Histologic Type
Bone Marrow Diseases
Hematologic Diseases
Fludarabine phosphate
Antineoplastic Agents
Antimetabolites, Antineoplastic
Molecular Mechanisms of Pharmacological Action
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs
Alkylating Agents
Antineoplastic Agents, Alkylating
Myeloablative Agonists