UMIN-CTR Clinical Trial
| Unique ID issued by UMIN | UMIN000037944 |
|---|---|
| Receipt number | R000043239 |
| 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 |
| Date of disclosure of the study information | 2019/10/01 |
| Last modified on | 2019/09/05 17:57:19 |
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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
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
haplo@hyo-med.ac.jp
Sponsor or person
Institute
Division of Hematology, Department of Internal Medicine, Hyogo College of Medicine
Institute
Department
Personal name
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
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
Value
https://center6.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000043239
