UMIN-CTR Clinical Trial

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
Email	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
Email	kato-mt@ncchd.go.jp

Sponsor
Institute	National Center for Child Health and Development
Institute
Department

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
Email	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
URL(English)	https://upload.umin.ac.jp/cgi-open-bin/icdr_e/ctr_view.cgi?recptno=R000026149