UMIN-CTR Clinical Trial

Basic information
Public title	Phase I / IIa, single-institution, open-label study to explore the safety and efficacy of R-OKY-034F in patients with refractory / intolerable and surgically unresectable pancreatic cancer with standard chemotherapy
Acronym	Phase I / IIa, single-institution, open-label study to explore the safety and efficacy of R-OKY-034F in patients with refractory / intolerable and surgically unresectable pancreatic cancer with standard chemotherapy
Scientific Title	Phase I / IIa trial of R-OKY-034F for pancreatic cancer patients
Scientific Title:Acronym	Phase I / IIa trial of R-OKY-034F for pancreatic cancer patients
Region	Japan

Condition
Condition	Pancreatic cancer
Classification by specialty	Hepato-biliary-pancreatic medicine
Classification by malignancy	Malignancy
Genomic information	NO

Objectives
Narrative objectives1	Determination of dose-limiting toxicity (DLT) of R-OKY-034F by oral administration of R-OKY-034F orally once daily for patients with refractory / intolerable and surgically unresectable pancreatic cancer with standard chemotherapy And estimate the maximum tolerated dose (MTD) and assess safety to determine the recommended dose (RD) for clinical trials after Phase IIb. Also, preliminary search for effectiveness.
Basic objectives2	Safety
Basic objectives -Others
Trial characteristics_1	Exploratory
Trial characteristics_2
Developmental phase	Phase I,II

Assessment
Primary outcomes	<safety> 1) Determination of dose-limiting toxicity (DLT) of R-OKY-034F 2)Determination of the maximum dose (MTD) of R-OKY-034F <Effectiveness> 1) Calculation of response rate and disease control rate for each dose when R-OKY-034F is administered
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	Medicine
Interventions/Control_1	R-OKY-034F once a day for 28 days orally 1, 2.5, 5, 10, 15 mg/kg Post-observation period: 14 days
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	20 years-old <=
Age-upper limit	80 years-old >
Gender	Male and Female
Key inclusion criteria	1) Disease Patients who have been diagnosed with pancreatic ductal cancer pathologically or histologically 2) Clinical stage of disease Image diagnosis by contrast-enhanced CT or contrast-enhanced MRI within 14 days prior to registration, "Diagnoses of Pancreatic cancer medical care guidelines 2016 edition: How is the resectability of pancreatic cancer determined?" Patient Patients who are refractory or intolerant of both gemcitabine-based standard chemotherapy and 5-FU-based standard chemotherapy and have no other standard chemotherapy available 3) Age Patients aged 20 and over and under 80 at the time of enrollment 4) PS: Performance status Patients with PS 0 to 2 5) Evaluation possibility of lesion Patients with one or more measurable lesions defined by RECIST criteria 6) Consent Patients who have obtained informed consent from patients themselves regarding clinical trial participation 7) prognosis Patients with a prognosis of more than 2 months
Key exclusion criteria	1) Duplicate cancer Patients with active double cancer. However, we do not exclude duplicate cancers that do not affect safety and efficacy assessment. 2) Items related to complications * Patients with unstable angina (angina pectoris onset or attack worsening within 3 weeks before enrollment), or patients with a history of myocardial infarction within 6 months * Patients with QtcE prolongation of 450 ms or more * Patients with interstitial lung disease or patients with previous history * Patients with high degree of disability due to respiratory disease (interstitial lung disease, radiation lung injury etc.) (grade 3 or higher on the modified MRC scale) * Patients with high physical activity restriction due to heart failure (NYHA class III or higher) * Patients with intestinal paralysis, intestinal obstruction or severe diarrhea * Patients who have the following abnormal values for kidney function, liver function and bone marrow function at screening test - Moderate or higher renal dysfunction with GFR estimate less than 45 ml / min / 1.73 m2 - Moderate or more liver dysfunction, with either AST or ALT> 100 IU / L - Leukocytes: less than 3,000 / microL - Neutrophils: less than 1,500 /microL - Platelet: less than 75,000 / microL - Hemoglobin: less than 8.0 g / dL - Total bilirubin: 2.5 mg / dL or more * Patients weighing less than 40 kg * Pregnant women * Breastfeeding women (It is not this limitation if it is possible to refrain from breastfeeding for 14 days after the final oral administration from the study drug oral administration start date) * Patients who are complicated by psychosis or psychiatric symptoms and judged as difficult to participate in clinical trials 3) Other * Women and men who can not agree to the practice of highly effective contraception * Patients who the investigator or subinvestigator judged as inappropriate for trial entry
Target sample size	30

Research contact person
Name of lead principal investigator	1st name Tetsuo Middle name Last name Takehara
Organization	Osaka University
Division name	Department of Gastroenterology and Hepatology
Zip code	565-0871
Address	2-15 Yamadaoka, Suita, Osaka
TEL	06-6879-5111
Email	lat1.office@dmi.med.osaka-u.ac.jp

Public contact
Name of contact person	1st name Hideaki Middle name Last name Harada
Organization	Osaka University
Division name	Department of Medical Innovation
Zip code	565-0871
Address	2-2 Yamadaoka, Suita, Osaka
TEL	06-6210-8289
Homepage URL
Email	lat1.office@dmi.med.osaka-u.ac.jp

Sponsor
Institute	Osaka University Hospital
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	Institutional Review Board of Osaka University Hospital
Address	2-15 Yamadaoka, Suita, Osaka
Tel	06-6210-8290
Email	jim-chiken@hp-crc.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	2019 Year 04 Month 03 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	Completed
Date of protocol fixation	2018 Year 09 Month 19 Day
Date of IRB	2018 Year 12 Month 04 Day
Anticipated trial start date	2019 Year 03 Month 20 Day
Last follow-up date	2021 Year 08 Month 31 Day
Date of closure to data entry	2021 Year 10 Month 31 Day
Date trial data considered complete	2021 Year 10 Month 31 Day
Date analysis concluded	2021 Year 12 Month 31 Day

Other
Other related information

Management information
Registered date	2019 Year 04 Month 03 Day
Last modified on	2021 Year 10 Month 04 Day

Link to view the page
URL(English)	https://upload.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000041465