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

NCT02753400 | Completed Phase 2 Results

• 1 other identifier: 4429-203
• Study Type: interventional
• Target: 24
• Locations: 1 country
• Sites: 1

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.

Conditions

Proliferative Diabetic Retinopathy

Outcome Measures

Primary Outcomes (1)

• 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.

  Baseline and 12 weeks

Study Arms (2)

Emixustat hydrochloride

EXPERIMENTAL

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.

Drug: emixustat hydrochloride; Other: Placebo

Placebo

PLACEBO COMPARATOR

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.

Other: Placebo

Interventions

emixustat hydrochloride DRUG

Tablet for oral administration

Also known as: ACU-4429, Emixustat HCl

Emixustat hydrochloride

Placebo OTHER

Placebo tablets for oral administration contain only inactive ingredients

Emixustat hydrochloride; Placebo

Eligibility Criteria

• Age: 18 Years - 85 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Kubota Vision Inc.: lead

Study Sites (1)

Retina Institute of California

Arcadia, California, United States

Location

Related Publications (1)

• 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.

  PMID: 32852613 DERIVED

MeSH Terms

Interventions

emixustat

Results Point of Contact

• Title: Clinical Trials Helpdesk
• Organization: Kubota Vision Inc.

clinicaltrials@kubotavision.com

2068058310

Study Officials

• Responsible Medical Officer

  Kubota Vision Inc.

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 19, 2016
• First Posted: April 27, 2016
• Study Start: May 1, 2016
• Primary Completion: November 1, 2017
• Study Completion: December 1, 2017
• Last Updated: May 19, 2021
• Results First Posted: April 27, 2021

Record last verified: 2021-04

Locations

US (1)