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Autologous Hematopoietic Cell Transplantation for Core-binding Factor Acute Myeloid Leukemia

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: NCT01050036
Recruitment Status : Recruiting
First Posted : January 15, 2010
Last Update Posted : July 24, 2018
Information provided by (Responsible Party):
Je-Hwan Lee, Asan Medical Center

Brief Summary:

Primary study objective is the evaluation of efficacy of autologous hematopoietic cell transplantation (HCT) with core-binding factor (CBF) positive acute myeloid leukemia (AML) in the first CR (CR1) in terms of relapse incidence (cumulative incidence of relapse, CIR) and disease-free survival (DFS).

Secondary study objectives are the engraftment rate / time to engraftment, transplantation-related mortality (TRM) rate, event-free survival (EFS) rate, and Overall survival (OS).

Condition or disease Intervention/treatment Phase
Leukemia, Myeloid Procedure: autologous hematopoietic cell transplantation Not Applicable

  Show Detailed Description

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 39 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Phase 2 Study of Autologous Hematopoietic Cell Transplantation for Core-binding Factor Positive Acute Myeloid Leukemia in the First Complete Remission
Study Start Date : January 2010
Estimated Primary Completion Date : December 2018
Estimated Study Completion Date : December 2018

Arm Intervention/treatment
Experimental: HCT recipients
  1. Patient with CBF AML will be eligible in his/her 1st complete remission (CR1) status. Patients who have relapsed or have achieved 2nd complete remission should not be included in this study.
  2. 1st postremission therapy after CR1 will be performed with high-dose cytarabine (HDAC) chemotherapy, consisting of intravenous cytarabine 3 g/m2 infusion during 3 hours twice a day on days 1, 3, and 5.
  3. After achieving CR1, patient will be invited to this protocol and will be able to decide whether to join or not after listening to the information.
Procedure: autologous hematopoietic cell transplantation
  1. Autologous peripheral blood stem cell (PBSCs) harvesting

    • After the second cycle of high-dose ara-C(HDAC) consolidation chemotherapy
    • Mobilization: recombinant human G-CSF(Filgrastim) 5mcg/kg s.c. daily starting on 10 days after start of the second cycle of HDAC chemotherapy
    • Harvest procedure: peripheral blood mononuclear cells will be collected. Target CD34+ cell dose is over 5x10E6/kg.
  2. Conditioning regimen for autologous HCT

    • Busulfan 3.2 mg/kg/day i.v. daily on days -7 to -5 (for 3 days)
    • Etoposide 400mg/m2/day i.v. daily on days -3 to -2 (for 2 days)
  3. Autologous cell infusion and waiting for engraftment
Other Names:
  • Busulfex
  • Etoposid
  • Grasin

Primary Outcome Measures :
  1. cumulative incidence of relapse [ Time Frame: 3 years ]
    cumulative incidence of relapse

Secondary Outcome Measures :
  1. Disease-free survival [ Time Frame: 3 years ]
    defined as the interval between the day when complete remission is confirmed with bone marrow biopsy report (not by the day when bone marrow biopsy was performed) and the day when the relapse is confirmed by bone marrow biopsy or the presence of peripheral blood.

  2. engraftment rate [ Time Frame: 100 days ]
    defined as the interval between day 0 (PBSCs infusion) and the first day of evidence for engraftment.

  3. transplantation-related mortality [ Time Frame: 100 days ]
    transplantation-related mortality

  4. Event-free survival [ Time Frame: 3 years ]
    defined as the interval between day 0-PBSCs infusion and the event.

  5. Overall survival [ Time Frame: 3 years ]
    measured from day 0 to the date of last follow-up or death.

Information from the National Library of Medicine

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Ages Eligible for Study:   15 Years to 65 Years   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Patients with CBF positive AML in CR1. CBF AML includes t(8;21)(q22;q22) [AML1(RUNX1)/ETO(CBFα2T1)], inv(16)(q13q22) (CBFβ/MYH11), t(16;16)(p13;q22) (CBFβ/MYH11) Using RT-PCR, FISH, or standard karyotype analysis technique.
  • Patients who plan to receive the second cycle of HDAC consolidation chemotherapy.
  • 15 years old or older and 65 years or younger
  • Adequate performance status (Karnofsky score of 70 or more).
  • Adequate hepatic and renal function (AST, ALT, and bilirubin < 3.0 x upper normal limit, and creatinine < 2.0 mg/dL).
  • Adequate cardiac function (left ventricular ejection fraction over 40% on heart scan or echocardiography)
  • Signed and dated informed consent must be obtained from patient.

Exclusion Criteria:

  • Presence of significant active infection
  • Presence of uncontrolled bleeding
  • Any coexisting major illness or organ failure
  • Patients with psychiatric disorder or mental deficiency severe as to make compliance with the treatment unlike, and making informed consent impossible
  • Nursing women, pregnant women, women of childbearing potential who do not want adequate contraception
  • Patients with a diagnosis of prior malignancy unless disease-free for at least 5 years following therapy with curative intent (except curatively treated nonmelanoma skin cancer, in situ carcinoma, or cervical intraepithelial neoplasia)

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): NCT01050036

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Contact: Mijin Jeon, RN, CNS 82-2-3010-6689
Contact: Mi Ryang Jang, RN 82-2-3010-7084

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Korea, Republic of
Wonkwang University Hospital Recruiting
Iksan, Chollabuk-do, Korea, Republic of, 570749
Contact: Yeonghui Park, RN    82-63-859-2640   
Principal Investigator: Hyeok Shim, M.D. & PhD.         
Gyeongsang National University Hospital Recruiting
Jinju, Gyeongsangnam-do, Korea, Republic of, 660702
Contact: Hyeok Choi, RN    82-55-750-9454   
Principal Investigator: Gyeong Won Lee, M.D. & PhD.         
Hallym University Sacred Heart Hospital Recruiting
Anyang, Kyeongki-do, Korea, Republic of, 431796
Contact: Hyo Jung Kim, M.D. & PhD.    82-31-380-3704   
Principal Investigator: Dae Young Zang, M.D. & PhD.         
Sub-Investigator: Hyo Jung Kim, M.D. & PhD.         
Kosin University, Gospel Hospital Recruiting
Busan, Korea, Republic of, 602702
Contact: Aeran Lee, RN    82-51-990-5820   
Principal Investigator: Yang Soo Kim, M.D. & PhD.         
Sub-Investigator: Seong-Hoon Shin, M.D. & PhD.         
Sub-Investigator: Ho-Sup Lee, M.D. & PhD.         
Busan Paik Hospital, Inje University College of Medicine Recruiting
Busan, Korea, Republic of, 614735
Contact: Hyejung Eum, RN    82-51-890-6987   
Principal Investigator: Young-Don Joo, M.D. & PhD.         
Sub-Investigator: Won-Sik Lee, M.D. & PhD.         
Daegu Fatima Hospital Recruiting
Daegu, Korea, Republic of, 701600
Contact: Jung-eun Lee, RN    82-53-940-7687   
Principal Investigator: Jung-lim Lee, M.D. & PhD.         
Sub-Investigator: Sun-ah Lee, M.D. & PhD.         
Yeongnam University Hospital Recruiting
Daegu, Korea, Republic of, 705717
Contact: Young Mi Chun, RN    82-53-620-3069   
Principal Investigator: Myung Soo Hyun, M.D. & PhD.         
Sub-Investigator: Min Kyoung Kim, M.D. & PhD.         
Daegu Catholic University Medical Center Recruiting
Daegu, Korea, Republic of, 705718
Contact: Hun Mo Ryoo, M.D. & PhD    82-53-650-4034   
Contact: Sung Hwa Bae, M.D. & PhD    82-53-650-4388   
Principal Investigator: Hun Mo Ryoo, M.D. & PhD         
Sub-Investigator: Sung Hwa Bae, M.D. & PhD         
Gachon University Gil Hospital Recruiting
Inchon, Korea, Republic of, 405-760
Contact: Jin Hee Park, M.D. & PhD.         
Principal Investigator: Jin Hee Park, M.D. & PhD.         
Asan Medical Center Recruiting
Seoul, Korea, Republic of, 138-736
Contact: Mi Ryang Jang, RN    82-2-3010-7084   
Contact: Mijin Jeon, RN    82-2-3010-6689   
Sub-Investigator: Kyoo-Hyung Lee, M.D. & PhD         
Sub-Investigator: Je-Hwan Lee, M.D. & PhD         
Sub-Investigator: Jung-Hee Lee, M.D. & PhD.         
Principal Investigator: Dae-Young Kim, M.D.         
Ulsan University Hospital, University of Ulsan College of Medicine Recruiting
Ulsan, Korea, Republic of, 682714
Contact: Mi-young Kim, RN    82-52-250-8537   
Contact: Eun-hee Lee, RN, CNS    82-52-250-8516   
Principal Investigator: Jae-Hoo Park, M.D. & PhD.         
Sub-Investigator: Young Joo Min, M.D. & PhD.         
Sub-Investigator: Hawk Kim, M.D. & PhD.         
Sponsors and Collaborators
Asan Medical Center
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Principal Investigator: Je-Hwan Lee, MD, PhD Asan Medical Center


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Responsible Party: Je-Hwan Lee, Professor, Asan Medical Center Identifier: NCT01050036     History of Changes
Other Study ID Numbers: AMC-H-54
First Posted: January 15, 2010    Key Record Dates
Last Update Posted: July 24, 2018
Last Verified: July 2018

Keywords provided by Je-Hwan Lee, Asan Medical Center:
Leukemia, Myeloid
Primary complete remission
Favorable karyotype
Autologous hematopoietic cell transplantation
Hematopoietic cell transplantation

Additional relevant MeSH terms:
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Leukemia, Myeloid
Leukemia, Myeloid, Acute
Neoplasms by Histologic Type