UMIN-CTR Clinical Trial
| Unique ID issued by UMIN | UMIN000029275 |
|---|---|
| Receipt number | R000033461 |
| Scientific Title | A pilot phase I study of azacitidine for the first relapsed patients with infant acute lymphoblastic leukemia and MLL gene rearrangement |
| Date of disclosure of the study information | 2017/10/01 |
| Last modified on | 2023/03/05 15:51:59 |
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Basic information
Public title
A pilot phase I study of azacitidine for the first relapsed patients with infant acute lymphoblastic leukemia and MLL gene rearrangement
Acronym
AZA-MLL-P16
Scientific Title
A pilot phase I study of azacitidine for the first relapsed patients with infant acute lymphoblastic leukemia and MLL gene rearrangement
Scientific Title:Acronym
AZA-MLL-P16
Region
| Japan |
Condition
Condition
Children with first relapsed infant MLL gene rearrangement positive acute lymphoblastic leukemia
Classification by specialty
| Hematology and clinical oncology | Pediatrics |
Classification by malignancy
Malignancy
Genomic information
NO
Objectives
Narrative objectives1
The primary aim of the study is to evaluate a safety of azacitidine (AZA) for children with relapsed infant MLL gene rearrangement positive acute lymphoblastic leukemia. An efficacy of AZA will be evaluated as a secondary aim.
Basic objectives2
Safety,Efficacy
Basic objectives -Others
Trial characteristics_1
Confirmatory
Trial characteristics_2
Developmental phase
Phase I
Assessment
Primary outcomes
Occurrence rate of dose limiting toxicity (DLT)
Key secondary outcomes
1. Complete remission (CR) rate at the end of the test drug (AZA) administration.
2. CR rate at the end of the trial.
Base
Study type
Interventional
Study design
Basic design
Single arm
Randomization
Non-randomized
Randomization unit
Blinding
Open -no one is blinded
Control
Uncontrolled
Stratification
NO
Dynamic allocation
NO
Institution consideration
Blocking
NO
Concealment
Intervention
No. of arms
1
Purpose of intervention
Treatment
Type of intervention
| Medicine |
Interventions/Control_1
Either DL1 or DL0 would be given as a trial therapy.
DL1: AZA 2.5mg/kg/dose, 30min DIV, 7 days
DL0: AZA 2mg/kg/dose, 30min DIV, 7 days
Following 7-day of AZA, one course of chemotherapy selected by the physician will be given.
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
| 6 | years-old | >= |
Gender
Male and Female
Key inclusion criteria
Patients should meet all the criteria listed below:
(1) Patients must be diagnosed as B-cell precursor acute lymphoblastic leukemia (ALL) with MLL gene rearrangement confirmed by FISH.
(2) Patients must be a first relapsed case.
(3) Patients must be less than 1 year old at the time of initial ALL diagnosis.
(4) Patients must be in complete remission following one course of standard induction chemotherapy for relapsed ALL.
(5) All patients' parents or legal guardians must sign a written informed consent.
(6) Patients must have performance score (PS) of 0 to 3.
(7) Patients' peripheral blood cell counts must meet the following criteria;
1. ANC 500/mcL or higher, after 48 hours or longer cessation of G-CSF.
2. Platelet count 50,000/mcL or higher, without platelet transfusion within 3 days.
(8) Patients must have sufficient organ function (liver, kidney, heart, lung) at the trial entry;
1. SpO2 96% or higher in room air, no abnormal findings in chest X-ray.
2. No abnormal ECG and UCG findings which require interventions.
3. AST and ALT value within 5 times of normal upper limit.
4. Serum bilirubin value within 2 times of normal upper limit.
5. Creatinine value within 2 times of normal upper limit.
Key exclusion criteria
Patients should be excluded if either of the below criteria is met:
(1) Patients with uncontrollable infections.
(2) Patients with congenital heart disease which require any interventions.
(3) Patients with somatic chromosomal abnormalities.
(4) Patients with active hemorrhage at study entry.
(5) Patients with CNS symptoms at study entry.
(6) Patients with other malignant diseases.
(7) Patients with isolated extramedullary relapse.
(8) Patients with CNS relapse.
(9) Patients not eligible for the study entry decided by the primary/co-investigators of the study.
Target sample size
12
Research contact person
Name of lead principal investigator
| 1st name | Daisuke |
| Middle name | |
| Last name | Tomizawa |
Organization
National Center for Child Health and Development
Division name
Children's Cancer Center
Zip code
157-8535
Address
2-10-1 Okura, Setagaya-ku, Tokyo, Japan
TEL
03-3416-0181
tomizawa-d@ncchd.go.jp
Public contact
Name of contact person
| 1st name | Daisuke |
| Middle name | |
| Last name | Tomizawa |
Organization
National Center for Child Health and Development
Division name
Children's Cancer Center
Zip code
157-8535
Address
2-10-1 Okura, Setagaya-ku, Tokyo, Japan
TEL
03-3416-0181
Homepage URL
tomizawa-d@ncchd.go.jp
Sponsor or person
Institute
National Center for Child Health and Development
Institute
Department
Personal name
Funding Source
Organization
A grant from National Center for Child Health and Development #27-4 "Studies on developing novel therapy for rare subtype of refractory leukemia.lymphoma in children"
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
Institutioanl Review Board of National Center for Child Health and Development
Address
2-10-1 Okura, Setagaya-ku, Tokyo
Tel
03-3416-0181
hagino-k@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
国立研究開発法人 国立成育医療研究センター（東京都）
Other administrative information
Date of disclosure of the study information
| 2017 | Year | 10 | Month | 01 | Day |
Related information
URL releasing protocol
https://jrct.niph.go.jp/latest-detail/jRCTs031180063
Publication of results
Unpublished
Result
URL related to results and publications
https://jrct.niph.go.jp/latest-detail/jRCTs031180063
Number of participants that the trial has enrolled
1
Results
Primary endpoint of this trial was incidence of dose limiting toxicity (DLT) of the investigational drug azacytidine. No DLT was observed in DL1 for the registered patient. However, at least three patients are required to evaluate the incidence of DLT in DL1; therefore, DLT of azacitidine could not be evaluated in this study.
Results date posted
| 2023 | Year | 03 | Month | 05 | Day |
Results Delayed
Results Delay Reason
Date of the first journal publication of results
Baseline Characteristics
Total three patients were screened for registration of the trial, and one patient registered and was eligible.
This patient was assigned to the dose level 1 (DL1). Baseline characteristics of the patient was as follows.
Age at diagnosis: 0 year old at initial diagnosis,
one year old at relapse
Sex: Female
Diagnosis: MLL gene-rearranged B-cell
precursor acute lymphoblastic leukemia (B-ALL),
first bone marrow relapse
Participant flow
This trial is a Phase I clinical trial, and was initiated on September 11, 2017. One patient registered to the trial (assigned to DL1), and completed the trial therapy. Trial registration ended on March 31, 2022. Because the follow up period (6 months after patient registration) of the registered patient was already completed, follow-up period of the total study ended on March 31, 2022 as well.
Along with the enactment and enforcement of the Clinical Trials Act in Japan, this trial obtained approval as a specified clinical trial from the certified review board on November 19, 2018, and was released in jRCT on December 27, 2018.
Adverse events
No severe adverse events were reported.
Outcome measures
Primary endpoint of this trial was incidence of dose limiting toxicity (DLT) of the investigational drug azacytidine.
Secondary endpoints included maintenance rate of complete remission (CR) at the end of azacitidine completion and at the end of the trial period.
Plan to share IPD
IPD sharing Plan description
Progress
Recruitment status
Completed
Date of protocol fixation
| 2017 | Year | 09 | Month | 11 | Day |
Date of IRB
| 2017 | Year | 08 | Month | 31 | Day |
Anticipated trial start date
| 2017 | Year | 10 | Month | 01 | Day |
Last follow-up date
| 2022 | Year | 09 | Month | 30 | Day |
Date of closure to data entry
Date trial data considered complete
Date analysis concluded
Other
Other related information
Management information
Registered date
| 2017 | Year | 09 | Month | 25 | Day |
Last modified on
| 2023 | Year | 03 | Month | 05 | Day |
Link to view the page
Value
https://center6.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000033461
