UMIN-CTR Clinical Trial
| Unique ID issued by UMIN | UMIN000026159 |
|---|---|
| Receipt number | R000030060 |
| Scientific Title | A pilot study for hematopoietic stem cell transplantation with post-transplantation cyclophosphamide and anti-thymoglobulin from HLA-mismatched related donor for non-malignant diseases (Halo-NM) |
| Date of disclosure of the study information | 2017/03/01 |
| Last modified on | 2020/12/13 09:10:35 |
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Basic information
Public title
A pilot study for hematopoietic stem cell transplantation with post-transplantation cyclophosphamide and anti-thymoglobulin from HLA-mismatched related donor for non-malignant diseases (Halo-NM)
Acronym
A pilot study for SCT from HLA-mismatched donor for non-malignant diseases
Scientific Title
A pilot study for hematopoietic stem cell transplantation with post-transplantation cyclophosphamide and anti-thymoglobulin from HLA-mismatched related donor for non-malignant diseases (Halo-NM)
Scientific Title:Acronym
A pilot study for SCT from HLA-mismatched donor for non-malignant diseases
Region
| Japan |
Condition
Condition
Non-malignant diseases (bone marrow failure, immunodeficiency, inborn errors of metabolism)
Classification by specialty
| Hematology and clinical oncology | Pediatrics |
Classification by malignancy
Others
Genomic information
NO
Objectives
Narrative objectives1
To assess the efficacy and safety of hematopoietic stem cell transplantation from HLA-mismatched related donor with post-transplant cyclophosphamide and anti-thymocyte globulin.
Basic objectives2
Safety,Efficacy
Basic objectives -Others
Trial characteristics_1
Trial characteristics_2
Developmental phase
Assessment
Primary outcomes
Incidence of events (acute GVHD grade III or more, graft failure, death) by day 30
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
Uncontrolled
Stratification
Dynamic allocation
Institution consideration
Blocking
Concealment
Intervention
No. of arms
1
Purpose of intervention
Treatment
Type of intervention
| Medicine |
Interventions/Control_1
Conditioning regimen with anti-thymocyte globulin.
Stem cell transplantation at day 0.
Cyclophosphamide at day 3 and 4.
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
| Not applicable |
Age-upper limit
| 25 | years-old | > |
Gender
Male and Female
Key inclusion criteria
1) Diagnosed as bone marrow failure, immunodeficiency, or inborn errors of metabolism
2) Fanconi anemia is excluded for bone marrow failure.
3) No HLA 5/6 (A, B, DR) or more matched related donor
4) No HLA 6/6 (A, B, DR) matched unrelated donor
5) Transplantation from 2- or more-antigen mismatched related donor
6) Age at transplantation <25 years
7) ECOG Performance status: 0-2
8) Sufficient organ function, as follows;
a. T-Bil <= 1.5 mg/dl
b. Serum creatinine <= 0.8 mg/dl (age <5y), <= 1.2 mg/dl (age 5-9y), <= 1.5 mg/dl (10y or older)
c. Ejection fraction 45% or better, and QTfc <0.45 sec
9) Obtained informed consent from a guardian of the patient
Key exclusion criteria
1. Effusion with grade 2 or worse at CTCAE ver 4.0
2. Uncontrolled infection
3. Severe psychological disorders
4. Pregnant or suspected pregnancy
5. Not eligible for this study at the discretion of the investigator.
Target sample size
9
Research contact person
Name of lead principal investigator
| 1st name | Motohiro |
| Middle name | |
| Last name | Kato |
Organization
National Center for Child Health and Development
Division name
Children's Cancer Center
Zip code
157-8535
Address
2-10-1, Okura, Setagaya, Tokyo
TEL
03-3416-0181
kato-mt@ncchd.go.jp
Public contact
Name of contact person
| 1st name | Motohiro |
| Middle name | |
| Last name | Kato |
Organization
National Center for Child Health and Development
Division name
Children's Cancer Center
Zip code
157-8535
Address
2-10-1, Okura, Setagaya, Tokyo
TEL
03-3416-0181
Homepage URL
kato-mt@ncchd.go.jp
Sponsor or person
Institute
National Center for Child Health and Development
Institute
Department
Personal name
Funding Source
Organization
National Center for Child Health and Development
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
National Center for Child Health and Development
Address
2-10-1, Okura, Setagaya, Tokyo JAPAN
Tel
03-3416-0181
rinri@ncchd.go.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
国立成育医療研究センター（東京都）、東京医科歯科大学病院（東京都）
Other administrative information
Date of disclosure of the study information
| 2017 | Year | 03 | 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
Main results already published
Date of protocol fixation
| 2017 | Year | 02 | Month | 28 | Day |
Date of IRB
| 2017 | Year | 02 | Month | 27 | Day |
Anticipated trial start date
| 2017 | Year | 03 | Month | 01 | Day |
Last follow-up date
| 2020 | Year | 07 | Month | 23 | Day |
Date of closure to data entry
Date trial data considered complete
Date analysis concluded
Other
Other related information
Management information
Registered date
| 2017 | Year | 02 | Month | 16 | Day |
Last modified on
| 2020 | Year | 12 | Month | 13 | Day |
Link to view the page
Value
https://center6.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000030060
