UMIN-CTR Clinical Trial

Basic information
Public title	Clinical trial to evaluate the safety of Temsirolimus combined with Vincristine and Irinotecan in children with recurrent/refractory solid tumors
Acronym	Clinical trial to evaluate the safety of Temsirolimus combined with Vincristine and Irinotecan in children with recurrent/refractory solid tumors
Scientific Title	Clinical trial to evaluate the safety of Temsirolimus combined with Vincristine and Irinotecan in children with recurrent/refractory solid tumors
Scientific Title:Acronym	Clinical trial to evaluate the safety of Temsirolimus combined with Vincristine and Irinotecan in children with recurrent/refractory solid tumors
Region	Japan

Condition
Condition	childhood recurrent/refractory solid malignant tumors
Classification by specialty	Pediatrics
Classification by malignancy	Malignancy
Genomic information	NO

Objectives
Narrative objectives1	Feasibility study for the safety of temusilorimus dose in Japanese children with malignancy.
Basic objectives2	Safety
Basic objectives -Others
Trial characteristics_1	Exploratory
Trial characteristics_2	Pragmatic
Developmental phase	Phase I

Assessment
Primary outcomes	Dose limiting toxicity of temusirolimus with the combination therapy of vincristin and irinotecan
Key secondary outcomes	Response rates and adverse eventsin vincristin/irinotecan/temusirolimus combination study

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	Vincristin Irinotecan Temsilorimus
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 days-old <=
Age-upper limit	18 years-old >
Gender	Male and Female
Key inclusion criteria	recurrent/ reflactory childhood solid malignant tumors: neuroblastomaneuroblastoma, rhabdomyosarcoma, undifferentiated sarcoma, Ewing family tumor, retinoblastoma, nephroblastoma, hepatoblastoma, osteosarcoma, others (including brain tumors)
Key exclusion criteria	active double cancer severe infection abnormal ECG findings intestinal pneumonia etc.
Target sample size	6

Research contact person
Name of lead principal investigator	1st name Eiso Middle name Last name Hiyama
Organization	Hiroshima University
Division name	Natural Center for Basic Research and Development
Zip code	734-8551
Address	1-2-3, Kasumi, Minami-ku, Hiroshima, Japan
TEL	082-257-5951
Email	eiso@hiroshima-u.ac.jp

Public contact
Name of contact person	1st name Sho Middle name Last name Kurihara
Organization	Hiroshima University Hospital
Division name	Pediatric Surgery
Zip code	734-8551
Address	1-2-3, Kasumi, Minami-ku, Hiroshima
TEL	082-257-5416
Homepage URL	http://home.hiroshima-u.ac.jp/eiso/
Email	jplt@hiroshima-u.ac.jp

Sponsor
Institute	Natural Center for Basic Research and Development, Hiroshima University
Institute
Department

Funding Source
Organization	Japan Agency for Medical Research and Development Health Labour Sciences Research Grant
Organization
Division
Category of Funding Organization	Government offices of other countries
Nationality of Funding Organization	Japan

Other related organizations
Co-sponsor	Japanese Study Group for Pediatric Liver Tumor
Name of secondary funder(s)

IRB Contact (For public release)
Organization	Hiroshima University hospital IRB
Address	1-2-3, Kasumi, Minami-ku, Hiroshima, 734-8551, Japan
Tel	082-257-5596
Email	hugcp@hiroshima-u.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	広島大学病院（広島県）

Other administrative information
Date of disclosure of the study information	2015 Year 05 Month 07 Day

Related information
URL releasing protocol	http://home.hiroshima-u.ac.jp/eiso/
Publication of results	Unpublished

Result
URL related to results and publications	http://home.hiroshima-u.ac.jp/eiso/
Number of participants that the trial has enrolled	3
Results	This study was ended becasue the objected cases were enrolled and no DLTs appeared. The safety of dose of temsirolimus (35 mg/m2) with the combination of Vincristin and Irrinotecan was confirmed.
Results date posted	2022 Year 04 Month 13 Day
Results Delayed
Results Delay Reason
Date of the first journal publication of results
Baseline Characteristics	1)Patients with metastatic disease, 2) cases with recurrence at least 1 month and less than 18 years of age, regardless of stage, or refractory pediatric solid tumors. However, patients who are not considered viable for more than 3 months or who are judged to be unable to tolerate chemotherapy will be excluded. It is imperative that written informed consent has been obtained from the patient's legally acceptable representative, and from the patient's legally acceptable representative, as well as the legally acceptable representative, for minors aged 16 years or older. In addition, to combine with irinotecan, participants will be previously tested for UGT1A1 polymorphisms and selected after confirming that they are not homovariants and compound heterozygotes for UGT1A1*28/*28 or *6/*6.
Participant flow	The physician confirms that the target patient meets all eligibility criteria and does not meet any of the exclusion criteria, fills out all of the required items in the registration eligibility verification form, and submits the registration to the registrar by FAX or direct handover.
Adverse events	Neutrophil count decreased in Grade4 >1.7 days (168 hours) 2.1 Platelet count < 20,000 platelets per mm3 on two blood tests within a course or requiring two platelet transfusions in 7 days 3. Cytopenia that does not meet initiation criteria more than 14 days after the expected starting date of the next course (day22) Nonhematologic toxicities 1. Non-haematological toxicities that may interfere with course initiation beyond Day 15 (day36) counting from the expected starting date of the next course (day22) (but not DLTs for allergic reactions and anaphylaxis related to vincristine, irinotecan or temsirolimus, even if leading to study treatment discontinuation) 2. Except for non-hematologic toxicity of Grade3, 4 Three enrolled patients will receive two courses each of the above VIT therapies to assess the presence or absence of DLT in each course.
Outcome measures	Among the above three cases, 1) VIT therapy is judged to be feasible if DLT expression is 0. 2) If there is one or two DLT episodes, three additional patients will be enrolled and if there are two or fewer DLT episodes out of six, VIT therapy will be considered feasible. 3) The clinical trial will be discontinued when at least 3 patients develop DLT. However, if there are other patients on treatment who have already been enrolled, treatment can be continued for up to 2 courses as long as safety is ensured.
Plan to share IPD
IPD sharing Plan description

Progress
Recruitment status	Completed
Date of protocol fixation	2013 Year 07 Month 03 Day
Date of IRB	2013 Year 07 Month 04 Day
Anticipated trial start date	2013 Year 07 Month 09 Day
Last follow-up date	2015 Year 03 Month 31 Day
Date of closure to data entry	2015 Year 03 Month 31 Day
Date trial data considered complete	2015 Year 04 Month 30 Day
Date analysis concluded	2015 Year 05 Month 31 Day

Other
Other related information

Management information
Registered date	2015 Year 05 Month 07 Day
Last modified on	2022 Year 04 Month 13 Day

Link to view the page
URL(English)	https://center6.umin.ac.jp/cgi-open-bin/icdr_e/ctr_view.cgi?recptno=R000012545