UMIN-CTR Clinical Trial

Basic information
Official scientific title of the study	A phase II study of haploidentical transplantation with post-transplantation cyclophosphamide following myeloablative conditioning
Title of the study (Brief title)	JSCT-Haplo14 MAC
Region	Japan

Condition
Condition	ALL,AML,MDS,CML,NHL,MM
Classification by specialty	Hematology and clinical oncology
Classification by malignancy	Malignancy
Genomic information	NO

Objectives
Narrative objectives1	To investigate safety and efficacy of HLA haploidentical peripheral blood stem cell transplantation using 150mg/m2 of fludarabine, 12.8mg/kg of intravenous busulfan and 4Gy of total body irradiationin or 90mg/m2 of fludarabine and 12Gy of total body irradiation in patients who have no HLA-matched or 7/8 matched, related or unrelated donor.
Basic objectives2	Safety,Efficacy
Basic objectives -Others
Trial characteristics_1
Trial characteristics_2
Developmental phase

Assessment
Primary outcomes	Event-free survival at 1 year after transplantation.
Key secondary outcomes	1. Engraftment at 100 days. 2. Non-relapse mortality at 100 days, 1 year and 2 year. 3. Relapse at 100 days, 1 year and 2 year. 4. Event-free survival at 2 year. 5. Overall survival at 1 year and 2 year. 6. Grade 3-4 non-hematologic toxicity within 100 days. 7. Infectious event within 1 year. 8. Acute and Chronic Graft-versus-Host Disease within 2 year. 9. Fever from day 0 to day7.

Base
Study type	Interventional

Study design
Basic design	Single arm
Randomization	Non-randomized
Randomization unit
Blinding	Open -no one is blinded
Control	Uncontrolled
Stratification
Dynamic allocation
Institution consideration
Blocking
Concealment

Intervention
No. of arms	1
Purpose of intervention	Treatment
Type of intervention	Medicine
Interventions/Control_1	The preparative regimen will consist of : Fludarabine 30mg/m2 IV Days -6, -5, -4, -3, -2 Busulfan 3.2mg/kg IV Days -6, -5, -4, -3 Total body irradiation (TBI) 4Gy Day -1 or Fludarabine 30mg/m2 IV Days -6, -5, -4 TBI 12Gy Day -3, -2, -1 The GVHD prophylaxis regimen will consist of : Cyclophosphamide 50mg/kg IV days 3, 4 Tacrolimus IV beginning Day 5 with dose adjusted to maintain a level of 5-15 ng/ml Mycophenolate mofetil 15mg/kg po TID beginning Day5, maximum dose 1 g po TID.
Interventions/Control_2
Interventions/Control_3
Interventions/Control_4
Interventions/Control_5
Interventions/Control_6
Interventions/Control_7
Interventions/Control_8
Interventions/Control_9
Interventions/Control_10

Eligibility
Age-lower limit	15 years-old <=
Age-upper limit	65 years-old >=
Gender	Male and Female
Key inclusion criteria	1.Patients who have hematologic malignancies and who are eligible for allogeneic stem cell transplantation following myeloablative conditioning 2.Age: 15-60 years old. 3.2nd CR or subsequent CR, non CR. 1st CR as defined at least one of the following: a) Acute lymphoblastic leukemia Poor risk as defined by NCCN guidelines b) Acute Myelogenous Leukemia Greater than 1 cycle of induction therapy required to achieve remission High risk as defined by NCCN guidelines Monosomal karyotype ckit mutations with t(8;21), inv(16), t(16;16) t(7;11)(p15;p15) Preceding myelodysplastic syndrome c) Myelodysplastic syndrome high, very high as defined by WHO classification-based Prognostic Scoring System Patients who have receive ten units or more platelet transfusions per week, or two units or more RBC transfusions per month. 4.Patients who have no prior allogeneic or autologous hematopoietic stem cell transplant 5.Performance status: 0-2 6.Patients with adequate physical function (Cardiac, Hepatic, Renal, Pulmonary). 7.Patients who have no HLA-matched or 7/8 matched, related donor. 8.Patients who have no HLA-matched or 7/8 matched, related donor. 9.Patients who give written informed consent to participate in the study. 10.Patients who are expected to live for 3 months or more.
Key exclusion criteria	1. Patients who are positive for HBs antigen, HCV antibody, or HIV antibody. 2. Patients with active other malignancies. 3. Patients with active infectious disease. 4. Women who are pregnant, of childbearing potential, or lactating. 5. Patients who experienced serious hypersensitivity or anaphylaxis to cyclophosphamide, fludarabine, tacrolimus, mycophenolate mofetil. 6. Positive anti-donor HLA antibody. 7. Patients who need chemotherapy within 13 days before transplantation. 8. Patients who are not eligible for this study at the discretion of the investigator.
Target sample size	38

Research contact person
Name of lead principal investigator	Junichi Sugita
Organization	Hokkaido University Hospital
Division name	Department of Hematology
Address	060-8638 Sappor
TEL	011-
Email	jsct-office@umin.ac.jp

Public contact
Name of contact person	Junichi Sugita
Organization	JSCT
Division name	Haplo14 DC
Address	104-0031
TEL	03-6225-2025
Homepage URL
Email	jsct-office@umin.ac.jp

Sponsor
Institute	JSCT
Institute
Department

Funding Source
Organization	Resarch Foundation for Community Medicine
Organization
Division
Category of Funding Organization	Non profit foundation
Nationality of Funding Organization

Other related organizations
Co-sponsor
Name of secondary funｄer(s)

Secondary IDs
Secondary IDs	NO
Study ID_1
Org. issuing International ID_1
Study ID_2
Org. issuing International ID_2
IND to MHLW

Institutions
Institutions

Other administrative information
Date of disclosure of the study information	2014 Year 06 Month 27 Day

Progress
Recruitment status	Main results already published
Date of protocol fixation	2014 Year 07 Month 12 Day
Anticipated trial start date	2014 Year 08 Month 01 Day
Last follow-up date
Date of closure to data entry
Date trial data considered complete
Date analysis concluded	2017 Year 09 Month 27 Day

Related information
URL releasing protocol
Publication of results	Published
URL releasing results
Results
Other related information

Management information
Registered date	2014 Year 06 Month 27 Day
Last modified on	2018 Year 03 Month 22 Day

Link to view the page
URL(English)	https://upload.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000016759