UMIN-CTR Clinical Trial
| Unique ID issued by UMIN | UMIN000022688 |
|---|---|
| Receipt number | R000026149 |
| Scientific Title | A pilot study of reduced intensity conditioning allogeneic stem cell transplantation with dose-adjusted busulfan for chronic granulomatous disease |
| Date of disclosure of the study information | 2016/06/13 |
| Last modified on | 2021/06/14 12:05:52 |
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Basic information
Public title
A pilot study of reduced intensity conditioning allogeneic stem cell transplantation with dose-adjusted busulfan for chronic granulomatous disease
Acronym
A pilot study of RIC-SCT for CGD
Scientific Title
A pilot study of reduced intensity conditioning allogeneic stem cell transplantation with dose-adjusted busulfan for chronic granulomatous disease
Scientific Title:Acronym
A pilot study of RIC-SCT for CGD
Region
| Japan |
Condition
Condition
Chronic granulomatous disease
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 reduced intensity conditioning stem cell transplantation with dose-adjusted busulfan for chronic granulomatous disease.
Basic objectives2
Safety,Efficacy
Basic objectives -Others
Trial characteristics_1
Trial characteristics_2
Developmental phase
Assessment
Primary outcomes
Event free survival at day 100
Key secondary outcomes
1. Engraftment probability at day 30
2. Mortality at day 100
3. Incidence of infectious disease at week 8
4. Reconstruction of immune status
5. Time from transplantation to onset of acute GVHD
6. Reconstruction of neutrophil function
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 targeted-busulfan and fludarabin
Total body irradiation (3Gy) at day -1
Stem cell transplantation at day 0
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 chronic granulomatous disease
2) Planned to have stem cell transplantation
3) Donor for transplantation is one of the follows;
a. HLA 5/6 (A, B, DR) or more matched related donor
b. HLA 5/6 (A, B, DR) or more matched unrelated donor
4) Age at transplantation <25 years
5) ECOG Performance status: 0-2
6) 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
7) Obtained informed consent from a guardian of the patient
Key exclusion criteria
1. Intracranial hemorrhage
2. Severe psychological disorders
3. Pregnant or suspected pregnancy
4. 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
03-3416-1811
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
03-3416-1811
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-ku, Tokyo
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
Other administrative information
Date of disclosure of the study information
| 2016 | Year | 06 | Month | 13 | 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
Completed
Date of protocol fixation
| 2016 | Year | 05 | Month | 26 | Day |
Date of IRB
| 2016 | Year | 05 | Month | 26 | Day |
Anticipated trial start date
| 2016 | Year | 06 | Month | 10 | Day |
Last follow-up date
| 2018 | Year | 06 | Month | 28 | Day |
Date of closure to data entry
| 2018 | Year | 06 | Month | 28 | Day |
Date trial data considered complete
| 2018 | Year | 06 | Month | 30 | Day |
Date analysis concluded
| 2018 | Year | 07 | Month | 27 | Day |
Other
Other related information
Management information
Registered date
| 2016 | Year | 06 | Month | 10 | Day |
Last modified on
| 2021 | Year | 06 | Month | 14 | Day |
Link to view the page
Value
https://center6.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000026149
