UMIN-CTR Clinical Trial

Basic information
Public title	Phase 2a study of peptide cocktail vaccine and interferon-beta for patients with refractory pediatric central nervous system tumor
Acronym	NCCV+IFN-beta p2a
Scientific Title	Phase 2a study of peptide cocktail vaccine and interferon-beta for patients with refractory pediatric central nervous system tumor
Scientific Title:Acronym	NCCV+IFN-beta p2a
Region	Japan

Condition
Condition	refractory pediatric central nervous system tumors
Classification by specialty	Hematology and clinical oncology Neurosurgery
Classification by malignancy	Malignancy
Genomic information	NO

Objectives
Narrative objectives1	To evaluate the efficacy of peptide anticancer cocktail vaccine "NCCV Cocktail-1" combined with interferon-beta in patients with refractory pediatric central nervous system tumor.
Basic objectives2	Efficacy
Basic objectives -Others
Trial characteristics_1
Trial characteristics_2
Developmental phase

Assessment
Primary outcomes	Response rate after 16 weeks from the start of the treatment
Key secondary outcomes	Progression free survival Overall survival Clinical benefit rate after 16 weeks Adverse events

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 Vaccine
Interventions/Control_1	NCCV Cocktail-1 is administered by subcutaneous injection weekly for the first 8 weeks and then administered biweekly. IFN-beta is administrated by intravenous administration five times a week for the first 2 weeks and then administrated once a week.
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	30 years-old >
Gender	Male and Female
Key inclusion criteria	1) Histologically proven glioma or medulloblastoma and diffuse intrinsic pontine glioma diagnosed on images. 2) aged 30 or less 3) Medical history of standard therapy 4) In the imaging examination within 28 days, at least one neoplastic lesion was confirmed and all lesions was no change or was progressive compared with the comparable latest imaging examination more than 21 days ago from the latest image 5) ECOG Performance Status of 0-2, or 3 due to neurological disorder or limb loss. 6) Adequate organ function. i.e. meet all criterion as follows. i. Absolute neutrophil count >= 1,000 /mm3 ii. Platelet count >= 50,000 /mm3 iii. Total bilirubin <= 1.5 mg/dL iv. AST <= 200 IU/L v. ALT <= 200 IU/L vi. serum creatinine <= 0.8 mg/dL (1 years-4 years) <= 1.2 mg/dL (5 years-9 years) <= 1.5 mg/dL (10 years-39 years) 7) Written informed consent; over 20 years old: from patient own between 16 to 19 years old: from legal acceptable representative and patient 15 years and under: from legal acceptable representative 8) HLA-A*24:02 positive. 9) In tumor cells, any of the cancer antigens of KOC1, FOXM1, or KIF20A have been confirmed by immunostaining except diffuse intrinsic pontine glioma
Key exclusion criteria	1) Chemotherapy within 3 weeks prior to enrollment. 2) Radiation therapy within 3 weeks prior to enrollment. 3) Major surgery within 1 week prior to enrollment. 4) Allogeneic marrow transplant within 1 year to enrollment, or on-going GVHD. 5) Malignant pleural effusion, ascites or cardiac effusion required invasive treatment. 6) Synchronous or asynchronous (within 5 years) other cancer except carcinoma in situ or intramucosal carcinoma. 7) Active infection requires systemic therapy. 8) Active gastrointestinal bleeding required blood transfusion. 9) Any severe and/or uncontrolled medical conditions. severe interstitial pneumonia history. 10) History of severe interstitial pneumonia. 11) Severe psychiatric disorder. 12) Known serious drug allergy. 13) Woman during pregnancy, or impossible to discontine breast-feeding for 120 days after the final dose of study treatment. Patient or the partner without intent to use birth control. 14) Inadequate physical condition judged by investigator.
Target sample size	13

Research contact person
Name of lead principal investigator	1st name Middle name Last name Junichi Hara
Organization	Osaka City General Hospital
Division name	Department of Pediatric Hematology/Oncology
Zip code
Address	2-13-22 Miyakojima-hondori Miyakojima-ku Osaka 534-0021 JAPAN
TEL	06-6929-1221
Email	j-hara@med.osakacity-hp.or.jp

Public contact
Name of contact person	1st name Middle name Last name Chika Nitani
Organization	Osaka City General Hospital
Division name	Department of Pediatric Hematology/Oncology
Zip code
Address	2-13-22 Miyakojima-hondori Miyakojima-ku Osaka 534-0021 JAPAN
TEL	06-6929-1221
Homepage URL
Email	c-tanaka@med.osakacity-hp.or.jp

Sponsor
Institute	Osaka City General Hospital
Institute
Department

Funding Source
Organization	Japan Agency for Medical Research and Development
Organization
Division
Category of Funding Organization	Japanese Governmental office
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

Other administrative information
Date of disclosure of the study information	2018 Year 05 Month 30 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	Terminated
Date of protocol fixation	2017 Year 12 Month 15 Day
Date of IRB	2019 Year 03 Month 31 Day
Anticipated trial start date	2018 Year 05 Month 01 Day
Last follow-up date	2019 Year 03 Month 31 Day
Date of closure to data entry
Date trial data considered complete
Date analysis concluded

Other
Other related information

Management information
Registered date	2018 Year 05 Month 30 Day
Last modified on	2019 Year 06 Month 11 Day

Link to view the page
URL(English)	https://upload.umin.ac.jp/cgi-open-bin/icdr_e/ctr_view.cgi?recptno=R000037366