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Study to Evaluate Effects of Emixustat Hydrochloride in Subjects With Proliferative Diabetic Retinopathy

Primary Purpose

Proliferative Diabetic Retinopathy

Status

Completed

Phase

Phase 2

Locations

United States

Study Type

Interventional

Intervention

emixustat hydrochloride

Placebo

About this trial

This is an interventional treatment trial for Proliferative Diabetic Retinopathy

Eligibility Criteria

18 Years - 85 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

Able and willing to provide written informed consent
Documented diagnosis of type 1 or type 2 diabetes mellitus
Meets specific ocular criteria for the study eye including but not limited to, the presence of PDR with or without diabetic macular edema in study eye for which treatment can be deferred for at least 4 weeks after Day 1 visit
Media clarity, pupillary dilation, and subject cooperation sufficient to obtain adequate assessments

Exclusion Criteria:

Any condition that would preclude participation in the study (e.g., unstable medical status including blood pressure, cardiovascular disease or glycemic control)
History of myocardial infarction or other acute cardiac event
History of chronic renal failure requiring dialysis or kidney transplant
Prior participation in any clinical study of emixustat
Treatment with any investigational study drug within 30 days of screening
Known allergy to fluorescein sodium for injection in angiography
Treatment with specific prohibited medications or therapy beginning 4 weeks prior to screening and throughout the duration of the study
History of systemic anti-VEGF or pro-VEGF treatment within 4 months prior to randomization
Pre-specified laboratory abnormalities at screening
Specific ocular characteristics in the study eye
Male subjects who are not surgically sterile and are not willing to practice a medically accepted method of birth control with their female partner of childbearing potential from screening through 30 days following completion of the study
Female subjects of childbearing potential who are not willing to practice a medically accepted method of birth control with their non-surgically sterile male sexual partner from screening through 30 days following completion of the study
Female subjects who are pregnant or lactating

Sites / Locations

Retina Institute of California

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Placebo Comparator

Arm Label

Emixustat hydrochloride

Placebo

Arm Description

Week 1- Four tablets (2 placebo, 2 emixustat HCl Strength A) Week 2- Four tablets (2 placebo, 2 emixustat HCl Strength B) Week 3- Four tablets (2 placebo, 2 emixustat HCl Strength C) Week 4- Four emixustat HCl tablets (Strength C) All tablets are administered orally once daily. After week 4, all subjects will be held at a stable dose for the remainder of the 12-week dosing regimen.

Four placebo tablets are administered orally once daily for 12 weeks; Subjects in the placebo group will be mock-titrated on the same schedule as those in the active arm.

Outcomes

Primary Outcome Measures

Change in Aqueous Humor Concentration of the Following Biomarkers:IL-6, IL-8, IP-10, PDGF-AA, TGFβ-1, MCP-1, IL-1β, and VEGF, to be Reported in pg/mL Values

All values for IL-1β were below the lower limit of detection and were recorded as zero. Tests for IP-10 and MCP-1 failed accuracy and stability testing during assay development and were dropped from the study. The assay for PDGF-AA could not be developed.and results were not reported.

Secondary Outcome Measures

Full Information

NCT ID

NCT02753400

First Posted

April 19, 2016

Last Updated

April 27, 2021

Sponsor

Kubota Vision Inc.

1. Study Identification

Unique Protocol Identification Number

NCT02753400

Brief Title

Study to Evaluate Effects of Emixustat Hydrochloride in Subjects With Proliferative Diabetic Retinopathy

Official Title

A Multicenter, Randomized, Double-Masked, Placebo-Controlled, Pilot Study to Evaluate Effects of Emixustat Hydrochloride on Aqueous Humor Biomarkers Associated With Proliferative Diabetic Retinopathy

Study Type

Interventional

2. Study Status

Record Verification Date

April 2021

Overall Recruitment Status

Completed

Study Start Date

May 2016 (Actual)

Primary Completion Date

November 2017 (Actual)

Study Completion Date

December 2017 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Kubota Vision Inc.

4. Oversight

Data Monitoring Committee

5. Study Description

Brief Summary

To evaluate the effects of oral emixustat hydrochloride (emixustat) on aqueous humor biomarkers associated with proliferative diabetic retinopathy (PDR) from baseline to week 12.

Detailed Description

This is a multicenter, randomized, double-masked, placebo-controlled study to evaluate the effects of emixustat in subjects with PDR. Subjects will be randomly assigned to either emixustat or placebo arms and treated once daily (QD) for 12 weeks. Doses of emixustat will be doubled on a weekly basis until week 4 after which all subjects will be held at a stable dose for the remainder of the 12-week dosing regimen. Subjects in the placebo group will be mock-titrated on the same schedule as those in the emixustat arm.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Proliferative Diabetic Retinopathy

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 2

Interventional Study Model

Parallel Assignment

Masking

ParticipantCare ProviderInvestigatorOutcomes Assessor

Allocation

Randomized

Enrollment

24 (Actual)

8. Arms, Groups, and Interventions

Arm Title

Emixustat hydrochloride

Arm Type

Experimental

Arm Description

Arm Title

Placebo

Arm Type

Placebo Comparator

Arm Description

Four placebo tablets are administered orally once daily for 12 weeks; Subjects in the placebo group will be mock-titrated on the same schedule as those in the active arm.

Intervention Type

Drug

Intervention Name(s)

emixustat hydrochloride

Other Intervention Name(s)

ACU-4429, Emixustat HCl

Intervention Description

Tablet for oral administration

Intervention Type

Other

Intervention Name(s)

Placebo

Intervention Description

Placebo tablets for oral administration contain only inactive ingredients

Primary Outcome Measure Information:

Title

Change in Aqueous Humor Concentration of the Following Biomarkers:IL-6, IL-8, IP-10, PDGF-AA, TGFβ-1, MCP-1, IL-1β, and VEGF, to be Reported in pg/mL Values

Description

Time Frame

Baseline and 12 weeks

10. Eligibility

Sex

All

Minimum Age & Unit of Time

18 Years

Maximum Age & Unit of Time

85 Years

Accepts Healthy Volunteers

Eligibility Criteria

Inclusion Criteria: Able and willing to provide written informed consent Documented diagnosis of type 1 or type 2 diabetes mellitus Meets specific ocular criteria for the study eye including but not limited to, the presence of PDR with or without diabetic macular edema in study eye for which treatment can be deferred for at least 4 weeks after Day 1 visit Media clarity, pupillary dilation, and subject cooperation sufficient to obtain adequate assessments Exclusion Criteria: Any condition that would preclude participation in the study (e.g., unstable medical status including blood pressure, cardiovascular disease or glycemic control) History of myocardial infarction or other acute cardiac event History of chronic renal failure requiring dialysis or kidney transplant Prior participation in any clinical study of emixustat Treatment with any investigational study drug within 30 days of screening Known allergy to fluorescein sodium for injection in angiography Treatment with specific prohibited medications or therapy beginning 4 weeks prior to screening and throughout the duration of the study History of systemic anti-VEGF or pro-VEGF treatment within 4 months prior to randomization Pre-specified laboratory abnormalities at screening Specific ocular characteristics in the study eye Male subjects who are not surgically sterile and are not willing to practice a medically accepted method of birth control with their female partner of childbearing potential from screening through 30 days following completion of the study Female subjects of childbearing potential who are not willing to practice a medically accepted method of birth control with their non-surgically sterile male sexual partner from screening through 30 days following completion of the study Female subjects who are pregnant or lactating

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Responsible Medical Officer

Organizational Affiliation

Kubota Vision Inc.

Official's Role

Study Director

Facility Information:

Facility Name

Retina Institute of California

City

Arcadia

State/Province

California

Country

United States

12. IPD Sharing Statement

Plan to Share IPD

Undecided

Citations:

PubMed Identifier

32852613

Citation

Kubota R, Jhaveri C, Koester JM, Gregory JK. Effects of emixustat hydrochloride in patients with proliferative diabetic retinopathy: a randomized, placebo-controlled phase 2 study. Graefes Arch Clin Exp Ophthalmol. 2021 Feb;259(2):369-378. doi: 10.1007/s00417-020-04899-y. Epub 2020 Aug 27.

Results Reference

derived

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Study to Evaluate Effects of Emixustat Hydrochloride in Subjects With Proliferative Diabetic Retinopathy

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