Unique ID issued by UMIN | UMIN000027010 |
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Receipt number | R000030105 |
Scientific Title | Allogeneic hematopoietic stem cell transplantation from HLA6/8-4/8 matched related donor for poor prognostic or refractory hematologic malignancy and solid tumor - Ibaraki Children's Hospital phase II study |
Date of disclosure of the study information | 2017/04/17 |
Last modified on | 2017/07/25 23:29:22 |
Allogeneic hematopoietic stem cell transplantation from HLA6/8-4/8 matched related donor for poor prognostic or refractory hematologic malignancy and solid tumor -
Ibaraki Children's Hospital phase II study
ICH-HAPLO-16
Allogeneic hematopoietic stem cell transplantation from HLA6/8-4/8 matched related donor for poor prognostic or refractory hematologic malignancy and solid tumor -
Ibaraki Children's Hospital phase II study
ICH-HAPLO-16
Japan |
Acute leukemia, malignant lymphoma, and solid tumor
Hematology and clinical oncology | Pediatrics | Child |
Malignancy
YES
To evaluate the safety and efficacy of haploidentical family member, reduced toxicity hematopoietic stem cell transplantation against refractory hematologic malignancy and solid tumor in an attempt to reduce the late adverse effect and improve the prognosis
Safety,Efficacy
Exploratory
Pragmatic
Phase II
Disease free survival rate at day 60 after transplantation
Engraftment rate at 30 days after transplantation
Achievement rate of complete chimerism at 30 days after transplantation
Cumulative incidence of acute GVHD over a 1-year period
Cumulative incidence of grade 2 to 4 acute GVHD over a 1-year period
Cumulative incidence of grade 3 to 4 acute GVHD over a 1-year period
Cumulative incidence of hemorrhagic cystitis over a 1-year period
Cumulative incidence of thrombotic microangiopathy over a 1-year period
Cumulative incidence of hepatic sinusoidal obstruction syndrome over a 1-year period
Cumulative incidence of idiopathic pneumonia syndrome over a 1-year period
Cumulative therapy-related mortality over a 2-year period
Cumulative incidence of chronic GVHD over a 3-year period
Cumulative incidence of severe chronic GVHD over a 3-year period
Relapse free survival rate at 1 year after transplantation
Overall survival rate at 1 year after transplantation
Relapse free survival rate at 3 years after transplantation
Overall survival rate at 3 years after transplantation
Cumulative incidence of relapse over a 1-year period
Cumulative incidence of relapse over a 3-year period
Interventional
Single arm
Non-randomized
Open -no one is blinded
Historical
NO
NO
1
Treatment
Maneuver |
Patients receive peripheral blood stem cell transplantation or bone marrow transplantation from HLA-4/8 to 6/8 matched family member after preconditioning with 180mg/m^2 of fludarabine, 140-210mg/m^2 of melphalan, and 2.5-5.0mg/kg of rabbit anti-thymocyte globulin, added with 4-6Gy of total body irradiation or 6.4-9.6mg/kg of busulfan depending on the disease condition. The patient was followed with intensive GVHD prophylaxis composed of methotrexate, tacrolimus, and methylprednisolone. The graft was analyzed by flowcytometry. The patients undergo physical examination, serial peripheral blood investigation, bone marrow analysis, and imaging study over chimerism, engraftment, immunological recovery, disease condition, or side effect.
0 | years-old | <= |
20 | years-old | > |
Male and Female
(1)Histologically or cytologically diagnosed acute leukemia, or malignant lymphoma, or solid tumor (2)Age less than 20 years at diagnosis (3)Confirmed Eligible clinical entity (3-1)High Risk acute leukemia or malignant lymphoma (a)Primary induction failure at transplantation (b)In the first complete remission obtained after more than three courses of induction (c)In the first complete remission with detectable minimum residual disease (d)In the first complete remission with unfavorable cytogenetic or molecular features (e)Very early relapse within three years after diagnosis, the second complete remission but poor response or minimum residual disease against re-induction chemotherapy after relapse, the third or more advanced complete remission or not in remission after relapse (3-2)High risk solid tumor (a)MYCN-amplified, 1p-lost, or 11q-lost neuroblastoma (b)Not in remission (3-3)No HLA-match donor in family and no appropriate donor in unrelated donor bank or cord blood bank (3-4)Recurrent acute leukemia or malignant lymphoma after hematopoietic stem cell transplantation (3-5)Recurrent solid tumor (4)ECOG performance status score 0-1, if deterioration due to active disease, score 2 eligible (5)Appropriate organ function fulfilling following condition a)Serum concentration of total bilirubin, less than upper limit of normal range for age b)Serum concentration of creatinine, less than upper limit of normal range for age c)Serum concentration of cystatin C, less than 1.00 mg/L d)Pulse-oximetry saturation more than 97 percent awake and at rest breathing room air e)%Vital capacity, more than 70% by pulmonary function test (if patient more than 6 years old) f)Forced expiratory volume in 1 second%, more than 70% (if patient more than 6 years old) g)Plasma BNP, less than 40 pg/mL h)%Fractional shortening, more than 27% (6)All patients and/or their parents or legal guardians must sign a written informed consent
(1)Germ line chromosomal abnormality other than trisomy 21
(2)Concurrent or prior malignant disease other than the original disease in interest or prior organ transplantation other than hematopoietic cell transplantation
(3)Congenital or acquired immunodeficiency
(4)Uncontrolled fungal, bacterial, or viral infection (including tuberculosis of HIV)
(5)Pregnant, lactating, or highly suspected to be pregnant
(6)Corrected QT interval by Fridericia, more than 0.45 second
(7)Active hemorrhage in central nervous system, fulfilling grade more than 3 on CTCAE version 4.0
(8)Consciousness disturbance, score less than 14 on Glasgow Coma Scale
(9)Obesity, by more than 30% of average weight for age.
(10)Considered to be unsuitable for entry for the study
30
1st name | |
Middle name | |
Last name | Keisuke Kato, M.D. |
Ibaraki Children's Hospital
Division of Pediatric Hematology and Oncology, Specialty Care Service, Pediatrics
Futaba-dai 3-3-1, Mito, Japan
029-254-1151
keikato-ind@umin.ac.jp
1st name | |
Middle name | |
Last name | Keisuke Kato, M.D. |
Ibaraki Children's Hospital
Division of Pediatric Hematology and Oncology, Specialty Care Service, Pediatrics
Futaba-dai 3-3-1, Mito, Japan
029-254-1151
keikato-ind@umin.ac.jp
Ibaraki Children's Hospital
Ibaraki Children's Hospital
Other
NO
茨城県立こども病院小児血液腫瘍科
2017 | Year | 04 | Month | 17 | Day |
Unpublished
Enrolling by invitation
2016 | Year | 10 | Month | 18 | Day |
2016 | Year | 10 | Month | 18 | Day |
2017 | Year | 04 | Month | 16 | Day |
2017 | Year | 07 | Month | 25 | Day |
Value
https://center6.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000030105
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