UMIN-CTR Clinical Trial

Basic information
Official scientific title of the study	Phase 1/2 clinical trial to evaluate the safety and efficacy of daily intravitreal injections of KUS121 for three days in patients with non-arteritic central retinal artery occlusion
Title of the study (Brief title)	Phase 1/2 clinical trial of intravitreal injections of KUS121 in patients with central retinal artery occlusion
Region	Japan

Condition
Condition	non-arteritic central retinal artery occlusion
Classification by specialty	Ophthalmology
Classification by malignancy	Others
Genomic information	NO

Objectives
Narrative objectives1	KUS121, one of inhibitor of VCP (valosin-containing protein) ATPase has a protective effect on retinal cells in vitro and in animal models for ocular diseases. In this clinical trial, we investigate safety and eficacy of intravitreal injection of KUS121 on CRAO patients.
Basic objectives2	Safety
Basic objectives -Others
Trial characteristics_1	Exploratory
Trial characteristics_2
Developmental phase	Phase I,II

Assessment
Primary outcomes	Safety: adverse events and side effects
Key secondary outcomes	adverse events related to the procedure of intravitreal injection pharmacokinetics visual acuity visula field Sensitivity of the retina electroretinography retinal thickness

Base
Study type	Interventional

Study design
Basic design	Single arm
Randomization	Non-randomized
Randomization unit
Blinding	Open -no one is blinded
Control	Historical
Stratification
Dynamic allocation
Institution consideration
Blocking
Concealment

Intervention
No. of arms	1
Purpose of intervention	Treatment
Type of intervention	Medicine
Interventions/Control_1	daily intravitreal injections KUS121 25 microgram for 3 days daily intravitreal injections KUS121 50 microgram for 3 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	Not applicable
Gender	Male and Female
Key inclusion criteria	1) Age >= 20 years 2) Non-arteritic central retinal artery occlusion (CRAO) 3) Best-corrected visual acuity (BCVA) <= 0.1 and better than hand motion 4) Laboratory data obtained prior to enrollment meeting the following criteria: Hemoglobin >= 10 g/dL WBC count >= 2,000 /uL Platelet count >= 100000 /uL AST <= 50 IU/L ALT <= 50 IU/L Total bilirubin <= 1.5 mg/dL Creatinine <= 1.5 mg/dL 5) Both men and women reporting the possibility of pregnancy were required to agree to be on contraception from the first injection until 4 weeks after the last injection. 6) Written informed consent of the patient
Key exclusion criteria	1) Patients who reported one or more of the following conditions: severe complications from cardiac, hepatic, or renal disease; poorly controlled diabetes mellitus 2) Eyes with the following conditions: Acute ocular or periocular infection Uncontrolled intra ocular pressure despite medication Retinal venous occlusion Abnormality in the macula Opacity in the lens or the vitreous such that the fundus is not visible 3) Contralateral eye: BCVA <= 0.1, or visual field loss >= 1/2 of isopter I4e 4) Bilateral onset of CRAO 5) Systemic use of immunosuppressant or steroid medications 6) Participation in other studies during the period 16 weeks prior 7) Possibility or history of hypersensitivity reaction to fluorescein. 8) Inability to understand consent requirements, or unwillingness or inability to participate in all of the follow-up examinations 9) Decision by the principal investigator or the physician in charge that patient was unsuitable for participation in the present study
Target sample size	9

Research contact person
Name of lead principal investigator	Hanako Ikeda
Organization	Kyoto University Hospital
Division name	Institute for Advancement of Clinical and Translational Science
Address	Shogoin-kawaharacho 54, Sakyo-ku, Kyoto, JAPAN
TEL	075-751-3248
Email	hanakoi@kuhp.kyoto-u.ac.jp

Public contact
Name of contact person	Hanako Ikeda
Organization	Kyoto University Hospital
Division name	Institute for Advancement of Clinical and Translational Science
Address	Shogoin-kawaharacho 54, Sakyo-ku, Kyoto, JAPAN
TEL	075-751-3248
Homepage URL
Email	hanakoi@kuhp.kyoto-u.ac.jp

Sponsor
Institute	Kyoto University Hospital Institute for Advancement of Clinical and Translational Science
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)	Kyoto Drug Discovery & Development Co., Ltd.

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	京都大学　医学部附属病院

Other administrative information
Date of disclosure of the study information	2016 Year 09 Month 15 Day

Progress
Recruitment status	Completed
Date of protocol fixation	2016 Year 08 Month 24 Day
Anticipated trial start date	2016 Year 10 Month 28 Day
Last follow-up date	2018 Year 03 Month 20 Day
Date of closure to data entry
Date trial data considered complete	2018 Year 06 Month 30 Day
Date analysis concluded	2018 Year 08 Month 31 Day

Related information
URL releasing protocol
Publication of results	Unpublished
URL releasing results
Results
Other related information

Management information
Registered date	2016 Year 09 Month 08 Day
Last modified on	2018 Year 03 Month 13 Day

Link to view the page
URL(English)	https://upload.umin.ac.jp/cgi-open-bin/icdr_e/ctr_view.cgi?recptno=R000026911