UMIN-CTR Clinical Trial

Basic information
Public title	A study of haploidentical transplantation with post-transplant cyclophosphamide and prophylactic donor lymphocyte infusions.
Acronym	A study of haploidentical transplantation with post-transplant cyclophosphamide and prophylactic donor lymphocyte infusions.
Scientific Title	A study of haploidentical transplantation with post-transplant cyclophosphamide and prophylactic donor lymphocyte infusions.
Scientific Title:Acronym	A study of haploidentical transplantation with post-transplant cyclophosphamide and prophylactic donor lymphocyte infusions.
Region	Japan

Condition
Condition	AML,ALL,MDS,NHL,CML Neuroblastoma,Rhabdomyosarcoma,Ewing sarcoma
Classification by specialty	Hematology and clinical oncology Pediatrics
Classification by malignancy	Malignancy
Genomic information	NO

Objectives
Narrative objectives1	To investigate the safety and efficacy of HLA haploidentical hematopoietic stem cell transplantation using post transplant cyclophosphamide and prophylactic donor lymphocyte infusions.
Basic objectives2	Safety,Efficacy
Basic objectives -Others
Trial characteristics_1
Trial characteristics_2
Developmental phase

Assessment
Primary outcomes	treatment related mortality within 100days
Key secondary outcomes	1. overall survival, event free survival, relapse rate at 100 days 2. overall survival, event free survival, relapse rate at 1 year 3. neutrophilans platlet engraftment 4. engraftment failure 5. complete chimerism 6. acute GVHD 7. chronic GVHD 8. infectious event 9. grade 3-4 toxicity within 100 days

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	GVHD prophilaxis Cyclophosphamide 50mg/kg iv day3,4(or 5) and prophylactic donor lymphocyte infusions (day21,day35 and day60)
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	1 months-old <=
Age-upper limit	192 months-old >
Gender	Male and Female
Key inclusion criteria	1. patients who have hematologic malignancies or refractory childhood solid tumors and who are eligible for allogenic transplantation. 2. Age 0-15 years 3. 2nd CR or subsequent CR, nonCR. 1st CR as defined at least one of folloing: a) Acute lymphoblastic leukemia Intermediate or Poor risk as defined by NCCN guidelines b) Acute Myelogenous Leukemia Greater than 1 cycle of induction therapy required to achieve remission, Intedmediate or 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. e) patients who have refractory or relapsed childhood solid tumors (neuroblastoma or rhabdmyosarcoma or Ewing sarcoma) 4. Patients who have no HLA-matched or 7/8 matched related donor. 5. Patients who have no HLA-matched or 7/8 matched unrelated donor. 6. Patients who given written informed consent to participate in the study.
Key exclusion criteria	1. Patients who are positive for HBs antigen, HCV antibody, or HIV antibody. 2. Patients with active other malignancies. 3. Women who are pregnant, of childbearing potential, or lactating. 4. Patients who experienced serious hypersensitivity or anaphylaxis to cyclophosphamide. 5. patients who are not eligble for this study at the discretion of the investigator.
Target sample size	8

Research contact person
Last name of lead principal investigator	1st name Middle name Last name Akihiro Yachie
Organization	Kanazawa University Hospital
Division name	Pediatrics
Zip code
Address	13-1, takaramachi, kanazawa, ishikawa
TEL	076-265-2313
Email	pedialib@med.kanazawa-u.ac.jp

Public contact
1st name of contact person	1st name Middle name Last name Hideaki Maeba
Organization	Kanazawa University Hospital
Division name	Pediatrics
Zip code
Address	13-1, takaramachi, kanazawa, ishikawa
TEL	076-265-2313
Homepage URL
Email	maebahideakijp@yahoo.co.jp

Sponsor
Institute	Department of Pediatrics, Kanazawa University Hospital
Institute
Department

Funding Source
Organization	non
Organization
Division
Category of Funding Organization	Other
Nationality of Funding Organization

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

IRB Contact (For public release)
Organization
Address
Tel
Email

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	2018 Year 03 Month 02 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	Terminated
Date of protocol fixation	2018 Year 01 Month 01 Day
Date of IRB	2018 Year 01 Month 01 Day
Anticipated trial start date	2018 Year 01 Month 01 Day
Last follow-up date	2018 Year 12 Month 03 Day
Date of closure to data entry	2018 Year 12 Month 03 Day
Date trial data considered complete	2018 Year 12 Month 03 Day
Date analysis concluded	2018 Year 12 Month 03 Day

Other
Other related information

Management information
Registered date	2018 Year 01 Month 11 Day
Last modified on	2019 Year 03 Month 14 Day

Link to view the page
URL(English)	https://upload.umin.ac.jp/cgi-open-bin/icdr_e/ctr_view.cgi?recptno=R000034502