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Main
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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
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Register:
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JPRN |
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Last refreshed on:
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17 October 2023 |
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Main ID: |
JPRN-jRCTs031180398 |
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Date of registration:
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20/03/2019 |
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Prospective Registration:
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No |
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Primary sponsor: |
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Public title:
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A phase II study of RIC-SCT for CGD
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Scientific title:
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Reduced intensity conditioning allogeneic stem cell transplantation with dose-adjusted busulfan and anti-thymocyte globulin for chronic granulomatous disease: a multicenter phase II trial - CGD-RIST2 |
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Date of first enrolment:
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30/05/2018 |
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Target sample size:
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22 |
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Recruitment status: |
Recruiting |
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URL:
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https://jrct.niph.go.jp/latest-detail/jRCTs031180398 |
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Study type:
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Interventional |
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Study design:
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single arm study, open(masking not used), no treatment control/standard of care control, single assignment, treatment purpose
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Phase:
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2
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Countries of recruitment
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Japan
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None (Japan only)
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Contacts
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Name:
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Hirotoshi
Sakaguchi |
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Address:
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2-10-1, Okura, Setagaya-ku
157-8535
Tokyo
Japan |
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Telephone:
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+81-3-3416-0181 |
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Email:
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sakaguchi-hi@ncchd.go.jp |
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Affiliation:
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National Center for Child Health and Development |
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Name:
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Hirotoshi
Sakaguchi |
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Address:
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2-10-1, Okura, Setagaya-ku
157-8535
Tokyo
Japan |
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Telephone:
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+81-3-3416-0181 |
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Email:
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sakaguchi-hi@ncchd.go.jp |
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Affiliation:
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National Center for Child Health and Development |
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Key inclusion & exclusion criteria
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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
Exclusion criteria: 1. Intracranial hemorrhage
2. Severe psychological disorders
3. Pregnant or suspected pregnancy
4. Receiving interferon gamma therapy within 4 weeks of the start of conditioning therapy
5. Not eligible for this study at the discretion of the investigator.
Age minimum:
Not applicable
Age maximum:
< 25age old
Gender:
Both
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Health Condition(s) or Problem(s) studied
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Primary immunodeficiency
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Chronic granulomatous disease
Primary immunodeficiency
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D006105
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Intervention(s)
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Conditioning regimen with targeted-busulfan and fludarabin
Total body irradiation (3Gy) at day -1
Stem cell transplantation at day 0
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Primary Outcome(s)
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Event (graft failure/death) at day 100 after transplantation
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Secondary Outcome(s)
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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
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Secondary ID(s)
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UMIN000030647
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Source(s) of Monetary Support
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National Center for Child Health and Development
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Ethics review
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Status: Approval
Approval date: 26/07/2018
Contact:
rinken@ncchd.go.jp
Certified Review Board of National Center for Child Health and Development
+81-3-3416-0181
rinken@ncchd.go.jp
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Results
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Results available:
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Date Posted:
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Date Completed:
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URL:
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