UMIN-CTR Clinical Trial

Basic information
Public title	Phase I study of piggyBac transposon mediated chimeric antigen receptor gene modified T cells for CD19 positive acute lymphoblastic leukemia
Acronym	piggyBac transposon mediated chimeric antigen receptor gene modified T cells for CD19 positive acute lymphoblastic leukemia
Scientific Title	Phase I study of piggyBac transposon mediated chimeric antigen receptor gene modified T cells for CD19 positive acute lymphoblastic leukemia
Scientific Title:Acronym	piggyBac transposon mediated chimeric antigen receptor gene modified T cells for CD19 positive acute lymphoblastic leukemia
Region	Japan

Condition
Condition	refractory/relapsed CD19 positive acute lymphoblastic leukemia
Classification by specialty	Hematology and clinical oncology
Classification by malignancy	Malignancy
Genomic information	NO

Objectives
Narrative objectives1	To evaluate tolerability and safety of piggyBac transposon mediated chimeric antigen receptor modified T cells (CAR-T) for CD19 positive acute lymphoblastic leukemia
Basic objectives2	Safety
Basic objectives -Others
Trial characteristics_1
Trial characteristics_2
Developmental phase	Phase I

Assessment
Primary outcomes	Incidence of DLT until 4 weeks after CAR-T infusion
Key secondary outcomes

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	Maneuver
Interventions/Control_1	Administration of CAR-T cells produced from autologous peripheral blood
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	60 years-old >=
Gender	Male and Female
Key inclusion criteria	1) CD19 positive ALL 2) chemo-refractory/relapsed after allogeneic stem cell transplantation 3) 1-60 years old* 4) Written informed consent from patient or legal representative 5) Fulfill all of the following criteria: i) Performance status (PS) score: 0-3 ii) AST=<5 x upper normal limit (UNL) iii) ALT=<5 x UNL iv) total bilirubin =<3 x UNL v) serum creatinine =<3 x UNL vi) SpO2>=90% (room air) *Cohort 1: 16-60 years old, Cohort 2: 1-15 years old
Key exclusion criteria	1) predonisolone use >=0.5 mg/kg 2) BM blast >=20% 3) CNS-3 4) isolated extramedullary leukemia 5) severe or uncontrollable infection 6) severe or uncontrollable GVHD 7) severe or uncontrollable heart failure 8) previous use of CAR-T therapy 9) previous use of CD3/CD19 bispesific antibody 10) Psychoses 11) History of allergic reaction to albumin 12) History of allergic reaction to mouse derived protein
Target sample size	12

Research contact person
Name of lead principal investigator	1st name Yoshiyuki Middle name Last name Takahashi
Organization	Nagoya University Graduate School of Medicine
Division name	Department of Pediatrics
Zip code	466-8550
Address	65 Tsurumai-cho, Showa-ku, Nagoya, Aichi
TEL	052-744-2942
Email	ytakaha@med.nagoya-u.ac.jp

Public contact
Name of contact person	1st name Nobuhiro Middle name Last name Nishio
Organization	Nagoya University Hospital
Division name	Center for Advanced Medicine and Clinical Research
Zip code	466-8550
Address	65 Tsurumai-cho, Showa-ku, Nagoya, Aichi
TEL	052-744-2942
Homepage URL
Email	nnishio@med.nagoya-u.ac.jp

Sponsor
Institute	Nagoya University
Institute
Department

Funding Source
Organization	AMED
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	The Second Certified Special Committee for Regenerative Medicine, Osaka University
Address	4F Center of Medical Innovation and Translational Research 2-2 Yamadaoka, Suita, Osaka
Tel	06-6210-8293
Email	nintei@dmi.med.osaka-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

Other administrative information
Date of disclosure of the study information	2018 Year 01 Month 25 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	Open public recruiting
Date of protocol fixation	2017 Year 09 Month 20 Day
Date of IRB	2018 Year 02 Month 09 Day
Anticipated trial start date	2018 Year 03 Month 01 Day
Last follow-up date	2021 Year 12 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 01 Month 24 Day
Last modified on	2020 Year 07 Month 27 Day

Link to view the page
URL(English)	https://upload.umin.ac.jp/cgi-open-bin/icdr_e/ctr_view.cgi?recptno=R000035020