UMIN-CTR Clinical Trial
| Unique ID issued by UMIN | UMIN000005936 |
|---|---|
| Receipt number | R000007016 |
| Scientific Title | Phase II study of allogeneic hematopoietic stem cell transplantation using a preparative regimens consisting of iv Bu + Flu + L-PAM for patients with juvenile myelomonocytic leukemia (JMML) |
| Date of disclosure of the study information | 2011/07/07 |
| Last modified on | 2011/07/07 14:45:05 |
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Basic information
Public title
Phase II study of allogeneic hematopoietic stem cell transplantation using a preparative regimens consisting of iv Bu + Flu + L-PAM for patients with juvenile myelomonocytic leukemia (JMML)
Acronym
JMML-11
Scientific Title
Phase II study of allogeneic hematopoietic stem cell transplantation using a preparative regimens consisting of iv Bu + Flu + L-PAM for patients with juvenile myelomonocytic leukemia (JMML)
Scientific Title:Acronym
JMML-11
Region
| Japan |
Condition
Condition
Juvenile myelomonocytic leukemia (JMML)
Classification by specialty
| Pediatrics |
Classification by malignancy
Malignancy
Genomic information
NO
Objectives
Narrative objectives1
To evaluate the safety and the efficacy of allogeneic hematopoietic stem cell transplantation using a preparative regimen consisting of iv Bu + Flu + L-PAM for patients with newly diagnosed juvenile myelomonocytic leukemia (JMML).
Basic objectives2
Safety,Efficacy
Basic objectives -Others
Trial characteristics_1
Exploratory
Trial characteristics_2
Developmental phase
Phase II
Assessment
Primary outcomes
Event-free survival 1 year after stem cell transplantation
Key secondary outcomes
Safety of the regimen.
The adverse events and the efficacy of GVHD (acute/chronic)
Overall survival 3 years after stem cell transplantation
Event-free survival 3 years after stem cell transplantation
Non-relapse mortality within 100 days after stem cell transplantation
The rate of late effects after stem cell transplantation
The effect of individualized iv Bu dose modification on the engraftment rate and adverse event rate
The effect of chimerism assays on engraftment rate and adverse event rate within 100 days after stem cell transplantation
To elucidate the efficacy of MRD assessment using DNA mutation status of PTPN11 or RAS on relapse rate, eFS at 1 year and 3 years after stem cell transplantation.
Engraftment rate 100 days after stem cell transplantation
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
The effect of allogeneic hematopoietic stem cell transplantation assessing event-free survival 1 year after transplantation.
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
| 1 | years-old | <= |
Age-upper limit
| 14 | years-old | >= |
Gender
Male and Female
Key inclusion criteria
Diagnosis of JMML, that was confirmed by the pathological central review conducted by the MDS Committee of the Japanese Society of Pediatric Hematology.
Diagnosis between 6 months old and 14 years old.
No previous treatment including unaccepted chemotherapy, radiotherapy and allogeneic hematopoietic stem cell transplantation.
Adequate end organ function.
ECOG performance status score should be 0-2.
Total bilirubin less than 2mg/dl, creatinin below the upper normal limit defined according to the age, creatinin clearance (or cystatin) within normal range.
Normal EKG.
Ejection fraction of left ventricle more than 50%.
Saturation of oxygen more than 95%.
Legal guardians must sign an informed consent and have been told of its possible benefits and toxic side effects.
An adequate donor is available.
Key exclusion criteria
Diagnosis of Noonan syndrome, disorders with germline RAS mutations and ALPS-like diseases with somatic RAS mutations.
Intracranial hemorrhage.
Heart failure requiring treatment.
Chronic renal failure.
Active uncontrolled infections.
Uncontrolled diabetes mellitus.
Liver cirrhosis.
Psychiatric problems.
Target sample size
43
Research contact person
Name of lead principal investigator
| 1st name | |
| Middle name | |
| Last name | Atsushi Manabe |
Organization
St. Luke's International Hospital
Division name
department of Pediatrics
Zip code
Address
9-1, Akashi-cho, Chuo-ku, Tokyo, 104-8560
TEL
03-3541-5151
Public contact
Name of contact person
| 1st name | |
| Middle name | |
| Last name | Akiko Saito |
Organization
NPO-OSCR
Division name
Data Center
Zip code
Address
National Nagoya Hospital, 4-1-1, San-nomaru, Naka-ku, Nagoya
TEL
052-951-1111
Homepage URL
http://www.jplsg.jp/
officejp@nnh.hosp.go.jp
Sponsor or person
Institute
Japanese Pediatric Leukemia/Lymphoma Study Group (JPLSG)
Institute
Department
Personal name
Funding Source
Organization
Ministry of Health, Labour and Welfare
Organization
Division
Category of Funding Organization
Nationality of Funding Organization
Japan
Other related organizations
Co-sponsor
Name of secondary funder(s)
IRB Contact (For public release)
Organization
Address
Tel
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
| 2011 | Year | 07 | Month | 07 | Day |
Related information
URL releasing protocol
http://www.jplsg.jp/
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
| 2011 | Year | 07 | Month | 01 | Day |
Date of IRB
Anticipated trial start date
| 2011 | Year | 07 | Month | 01 | Day |
Last follow-up date
| 2018 | Year | 05 | Month | 01 | Day |
Date of closure to data entry
| 2018 | Year | 05 | Month | 01 | Day |
Date trial data considered complete
| 2018 | Year | 05 | Month | 01 | Day |
Date analysis concluded
| 2018 | Year | 05 | Month | 01 | Day |
Other
Other related information
Management information
Registered date
| 2011 | Year | 07 | Month | 06 | Day |
Last modified on
| 2011 | Year | 07 | Month | 07 | Day |
Link to view the page
Value
https://center6.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000007016
