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Name:
UMIN ID:

Recruitment status Open public recruiting
Unique ID issued by UMIN UMIN000026159
Receipt No. 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 2017/02/16

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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
0 years-old <=
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
Middle name
Last name Motohiro Kato
Organization National Center for Child Health and Development
Division name Children's Cancer Center
Zip code
Address 2-10-1, Okura, Setagaya, Tokyo
TEL 03-3416-0181
Email kato-mt@ncchd.go.jp

Public contact
Name of contact person
1st name
Middle name
Last name Motohiro Kato
Organization National Center for Child Health and Development
Division name Children's Cancer Center
Zip code
Address 2-10-1, Okura, Setagaya, Tokyo
TEL 03-3416-0181
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
Address
Tel
Email

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 Open public recruiting
Date of protocol fixation
2017 Year 02 Month 28 Day
Date of IRB
Anticipated trial start date
2017 Year 03 Month 01 Day
Last follow-up date
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
2017 Year 02 Month 16 Day


Link to view the page
URL(English) https://upload.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000030060

Research Plan
Registered date File name

Research case data specifications
Registered date File name

Research case data
Registered date File name


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