UMIN-CTR Clinical Trial

Basic information
Public title	ATG-based HLA haploidentical stem cell transplantation (HCM regimen) for recurrent or refractory round-cell-type sarcoma in AYA generation: prospective phase II study
Acronym	ATG-based HLA haploidentical stem cell transplantation (HCM regimen) for recurrent or refractory round-cell-type sarcoma in AYA generation
Scientific Title	ATG-based HLA haploidentical stem cell transplantation (HCM regimen) for recurrent or refractory round-cell-type sarcoma in AYA generation: prospective phase II study
Scientific Title:Acronym	ATG-based HLA haploidentical stem cell transplantation (HCM regimen) for recurrent or refractory round-cell-type sarcoma in AYA generation
Region	Japan

Condition
Condition	round-cell-type sarcoma
Classification by specialty	Hematology and clinical oncology
Classification by malignancy	Malignancy
Genomic information	NO

Objectives
Narrative objectives1	To evaluate whether HLA haploidentical stem cell transplantation (Hyogo regimen) can improve the survival prognosis of AYA-generation patients with recurrent or refractory round-cell-type sarcoma
Basic objectives2	Efficacy
Basic objectives -Others
Trial characteristics_1	Exploratory
Trial characteristics_2	Explanatory
Developmental phase	Phase II

Assessment
Primary outcomes	Median survival time (MST) in registration period (8 years) and follow-up period (2 years) If the lower limit of 95% CI of the MST is over 0.82 years, which is the MST of relapsed rhabdomyosarcoma (JCO. 1999; 17: 3487-3493), this study is defined as "positive study".
Key secondary outcomes	Safety 1) Engraftment rate on day 35. Engraftment is defined as neutrophil > 500/microL with >90% donor origin. Rejection is defined when engraftment is not achieved within day 35 after transplantation. 2) Acute GVHD within day 100 3) Complications including thrombotic microangiopathy (TMA) 4) Other adverse events assessed using CTCAE Efficacy 5) Response rate assessed using iRECIST. Assessment using RECIST ver. 1.1 is also documented for comparizon with other studies 6) Relapse rate 7) Disease-free survival (DFS) or progression-free survival (PFS)

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	Maneuver
Interventions/Control_1	The basic structure of the conditioning regimen was applied from our haploidentical stem cell transplantation (Hyogo regimen) for hematological malignancies, consisting of fludarabine at 30 mg/m2/day for 6 days (days -9 and -4), melphalan at 70 mg/m2/day for 2 days (days -3 and -2), rabbit anti-thymocyte globulin (ATG, thymoglobulin, Sanofi) at 1.25 mg/kg/day for 2 days (days -2 and -1), and 3 Gy of total body irradiation. To prevent anaphylaxis caused by ATG and stem cell infusions, methylprednisolone (mPSL) was administered at 2 mg/kg/day and 500 mg/body/day on days -2 and -1 and days 0 and 1. GVHD prophylaxis consists of continuous infusion of tacrolimus with a target concentration of 10-12 ng/mL from day -3 and mPSL 1 mg/kg/day after day 2. The stem cell source used was peripheral blood stem cells, which were infused on days 0 and 1, targeting 5-10*10e6/kg of CD 34+ cells, without being frozen.
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	39 years-old >=
Gender	Male and Female
Key inclusion criteria	1) Recurrent or refractory rhabdomyosarcoma with alveolar or pleomorphic type 2) Recurrent or refractory Ewing family sarcoma 3) desmoplastic small round cell tumor (any stage)
Key exclusion criteria	1) serum Cre >2.0 mg/dl 2) serum T-bil >2.0 mg/dl 3) serum AST and/or ALT >200 mg/dl 4) EF <40% by ultracardiograpgy 5) SpO2 <90% in ambient air 6) PS (ECOG score) 3-4 Not always excluded if data are elevated due to original disease and are expected to improve by treatment
Target sample size	16

Research contact person
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	663-8501
Address	1-1 Mukogawa-cho, Nishinomiya City, Hyogo 663-8501, Japan
TEL	0798456886
Email	rinri@hyo-med.ac.jp

Public contact
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	663-8501
Address	1-1 Mukogawa-cho, Nishinomiya City, Hyogo 663-8501, Japan
TEL	0798456886
Homepage URL
Email	haplo@hyo-med.ac.jp

Sponsor
Institute	Division of Hematology, Department of Internal Medicine, Hyogo College of Medicine
Institute
Department

Funding Source
Organization	partially supplied by Hyogo College of Medicine
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	IRB, Hyogo College of Medicine
Address	1-1, Mukogawa-cho, Nishinomiya city, Hyogo, Japan
Tel	0798456066
Email	rinri@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	2019 Year 10 Month 01 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	Preinitiation
Date of protocol fixation	2019 Year 09 Month 05 Day
Date of IRB
Anticipated trial start date	2019 Year 10 Month 10 Day
Last follow-up date	2029 Year 10 Month 31 Day
Date of closure to data entry
Date trial data considered complete
Date analysis concluded

Other
Other related information

Management information
Registered date	2019 Year 09 Month 05 Day
Last modified on	2019 Year 09 Month 05 Day

Link to view the page
URL(English)	https://upload.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000043239