UMIN-CTR Clinical Trial
| Unique ID issued by UMIN | UMIN000019532 |
|---|---|
| Receipt number | R000022592 |
| Scientific Title | Clinical Phase II Study of hematopoietic stem ctell transplantation for ataxia telangiectasia and related diseases |
| Date of disclosure of the study information | 2015/12/01 |
| Last modified on | 2024/11/05 08:10:52 |
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Basic information
Public title
Clinical Phase II Study of hematopoietic stem ctell transplantation for ataxia telangiectasia and related diseases
Acronym
HSCT for ataxia telangiectasia and related diseases
Scientific Title
Clinical Phase II Study of hematopoietic stem ctell transplantation for ataxia telangiectasia and related diseases
Scientific Title:Acronym
HSCT for ataxia telangiectasia and related diseases
Region
| Japan |
Condition
Condition
ataxia telangiectasia
DNA damage response disorder
Classification by specialty
| Medicine in general | Pediatrics |
Classification by malignancy
Others
Genomic information
YES
Objectives
Narrative objectives1
effectiveness and safety of hematopoietic stem cell transplantation for ataxia telangiectasia and related disease is evaluated.
Basic objectives2
Safety
Basic objectives -Others
Trial characteristics_1
Confirmatory
Trial characteristics_2
Developmental phase
Assessment
Primary outcomes
overall survival for 1 year
Key secondary outcomes
percentage of adverse effect
Base
Study type
Interventional
Study design
Basic design
Single arm
Randomization
Non-randomized
Randomization unit
Blinding
Open -no one is blinded
Control
Historical
Stratification
Dynamic allocation
Institution consideration
Blocking
Concealment
Intervention
No. of arms
1
Purpose of intervention
Treatment
Type of intervention
| Medicine |
Interventions/Control_1
fludarabine 30 mg/m2x5, cyclophosphamide 5mg/kgx4, ATG 1.25mg/kgx4
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
| Not applicable |
Gender
Male and Female
Key inclusion criteria
Ataxia Telanogiectasia
Nijmegen Syndrome
LIG4 Syndrome
Cernunnos deficiency
PRKDC mutated SCID
RIDDLE Syndrome
other DDR associated diseases
Key exclusion criteria
multiple organs failure
intra cranial hemorrhage
heart failure
renal failure
uncontrolled infection
uncontrolled diabetes
lever cirrhosis
serious psychological problem
Target sample size
5
Research contact person
Name of lead principal investigator
| 1st name | Masatoshi |
| Middle name | |
| Last name | Takagi |
Organization
Tokyo Medical and Dental University, Graduate Schoool
Division name
Dept. of Pediatrics and Developmental Biology, Dept. of Community Pediatrics, Perinatal and Maternal Medicine
Zip code
113-8519
Address
1-5-45 Yushima, Bunkyo-ku, Tokyo 113-8510, Japan
TEL
03-5803-5249
m.takagi.ped@tmd.ac.jp
Public contact
Name of contact person
| 1st name | Kohsuke |
| Middle name | |
| Last name | Imai |
Organization
Tokyo Medical and Dental University
Division name
Dept. of Community Pediatrics, Perinatal and Maternal Medicine
Zip code
113-8519
Address
1-5-45 Yushima, Bunkyo-ku, Tokyo 113-8510, Japan
TEL
03-5803-4705
Homepage URL
kimai.ped@tmd.ac.jp
Sponsor or person
Institute
Graduate Schoool,
Dept. of Community Pediatrics,
Perinatal and Maternal Medicine,
Tokyo Medical and Dental University
Institute
Department
Personal name
Funding Source
Organization
Japan Agency for Medical Reserch and Development
Organization
Division
Category of Funding Organization
Other
Nationality of Funding Organization
Japan
Other related organizations
Co-sponsor
Name of secondary funder(s)
IRB Contact (For public release)
Organization
Tokyo Medical and Dental University
Address
Yushima1-5-45, Bunkyo-ku, Tokyo
Tel
+81358035247
m.takagi.ped@tmd.ac.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
東京医科歯科大学医学部附属病院（東京都）
Other administrative information
Date of disclosure of the study information
| 2015 | Year | 12 | 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
Completed
Date of protocol fixation
| 2015 | Year | 11 | Month | 20 | Day |
Date of IRB
| 2015 | Year | 11 | Month | 19 | Day |
Anticipated trial start date
| 2015 | Year | 12 | Month | 01 | Day |
Last follow-up date
| 2024 | Year | 11 | Month | 05 | Day |
Date of closure to data entry
| 2024 | Year | 11 | Month | 05 | Day |
Date trial data considered complete
| 2024 | Year | 11 | Month | 05 | Day |
Date analysis concluded
| 2024 | Year | 12 | Month | 31 | Day |
Other
Other related information
Management information
Registered date
| 2015 | Year | 10 | Month | 28 | Day |
Last modified on
| 2024 | Year | 11 | Month | 05 | Day |
Link to view the page
Value
https://center6.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000022592
