UMIN-CTR Clinical Trial

Basic information
Public title	HLA haploidentical hematopoietic stem cell transplantation, Phase II trial
Acronym	haplo-SCT (Hyogo), Phase II
Scientific Title	HLA haploidentical hematopoietic stem cell transplantation, Phase II trial
Scientific Title:Acronym	haplo-SCT (Hyogo), Phase II
Region	Japan

Condition
Condition	hematologic malignancies, bone marrow failure syndrome, and other hematological disorders which requires hematopoietic stem cell transplantation
Classification by specialty	Hematology and clinical oncology
Classification by malignancy	Malignancy
Genomic information	NO

Objectives
Narrative objectives1	To evaluate the efficacy of hematopoietic stem cell transplantation from HLA haploidentical family members for hematologic disorders
Basic objectives2	Efficacy
Basic objectives -Others
Trial characteristics_1	Exploratory
Trial characteristics_2
Developmental phase	Phase II

Assessment
Primary outcomes	Overall survival at day 100 after transplantation
Key secondary outcomes

Base
Study type	Interventional

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

Intervention
No. of arms	1
Purpose of intervention	Treatment
Type of intervention	Medicine
Interventions/Control_1	The basic structure of the preconditioning regimen consists of fludarabine (FLU) at 30 mg/m2/day for 6 days (days -9 and -4), melphalan (MEL) at 70 mg/ m2/day for 2 days (days -3 and -2), and rabbit anti-thymocyte globulin (ATG, Thymoglobulin, Sanofi) at 1.25 mg/kg/day for 2 days (days -2 and -1). To ensure engraftment, 3 Gy of total body irradiation (TBI) was added. For active diseases, high-dose cytarabine (CA) at 2 g/m2/day for 4 days (days -9 and -6) was added for the purpose of tumor reduction. To prevent anaphylaxis caused by ATG, methylprednisolone (mPSL) was administered at 2 mg/kg on days -2 and -1. GVHD prophylaxis consists of the continuous infusion of TAC with a target concentration of 10-12 ng/mL started from day -3, and 1 mg/kg/day of mPSL started after day 0. Peripheral blood stem cells (PBSC) were used as a stem cell source, which were infused without being frozen. G-CSF was administered from day 3 to the day of engraftment.
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	70 years-old >=
Gender	Male and Female
Key inclusion criteria	Patients with hamatologic disorder who need hematopoietic stem cell transplantation who fulfill the following, 1) age under 70 years old 2) written informed consent 3) no available donor with matched or 1 antigin-mismatched HLA in HLA A, B, and DR. 4) no available donor in BM bank, or no time for waiting the cordination due to urgent transplantation 5) available HLA 2-3 antigen-mismatched donor in family members 6) In a case of high risk diseases, patient can select matched or HLA 1 antigen-mismatched donor (if any) or HLA 2-3 antigen-mismatched donor 7) PS 0-2 8) EF>50%, SpO2>93%, T-Bil<2mg/dl, AST<2.5*institutional upper limit, Cre<1.5*institutional upper limit
Key exclusion criteria	1) allergic for drugs used in this protocol 2) active CNS lesion (exclude original disease) 3) active infection 4) when the physician in chief decides the patient is inappropriate for this protocol
Target sample size	100

Research contact person
Last name of lead principal investigator	1st name Kazuhiro Middle name Last name Ikegame
Organization	Hyogo College of Medicine
Division name	Division of Hematology, Department of Internal Medicine
Zip code	6638501
Address	1-1, Mukogawa-cho, Nishinomiya city, Hyogo
TEL	+81-798-45-6886
Email	haplo@hyo-med.a.cjp

Public contact
1st name of contact person	1st name Kazuhiro Middle name Last name Ikegame
Organization	Hyogo College of Medicine
Division name	Division of Hematology, Department of Internal Medicine
Zip code	638501
Address	1-1, Mukogawa-cho, Nishinomiya city, Hyogo
TEL	+81-798-45-6886
Homepage URL
Email	kame@hyo-med.a.cjp

Sponsor
Institute	Hyogo College of Medicine
Institute
Department

Funding Source
Organization	Ministry of Education, Culture, Science and Technology
Organization
Division
Category of Funding Organization	Japanese Governmental office
Nationality of Funding Organization

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

IRB Contact (For public release)
Organization	IRB, Hyogo College of medicine
Address	1-1, Mukogawa-cho, Nishinomiya city, Hyogo
Tel	0798456886
Email	kame@hyo-med.ac.jp

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	2017 Year 06 Month 18 Day

Related information
URL releasing protocol
Publication of results	Unpublished

Result
URL related to results and publications
Number of participants that the trial has enrolled
Results
Results date posted
Results Delayed
Results Delay Reason
Date of the first journal publication of results
Baseline Characteristics
Participant flow
Adverse events
Outcome measures
Plan to share IPD
IPD sharing Plan description

Progress
Recruitment status	Enrolling by invitation
Date of protocol fixation	2017 Year 01 Month 04 Day
Date of IRB
Anticipated trial start date	2017 Year 04 Month 18 Day
Last follow-up date
Date of closure to data entry
Date trial data considered complete
Date analysis concluded

Other
Other related information

Management information
Registered date	2017 Year 04 Month 18 Day
Last modified on	2019 Year 04 Month 22 Day

Link to view the page
URL(English)	https://upload.umin.ac.jp/cgi-open-bin/icdr_e/ctr_view.cgi?recptno=R000031010