Proliferative Diabetic RetinopathyPhase 2NCT02753400
Study to Evaluate Effects of Emixustat Hydrochloride in Subjects With Proliferative Diabetic Retinopathy
A Phase 2 study testing emixustat hydrochloride in people with Proliferative Diabetic Retinopathy, run by Kubota Vision Inc. at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 85 years
- Conditions the study is for
- Proliferative Diabetic Retinopathy
- What is being tested?
- emixustat hydrochloride
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 24 participants
- Where
- 1 siteUnited States
- Study dates
- May 2016to Dec 2017
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 85 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
Show all 13Show fewer
- 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
Trial locations and contacts
1 site in 1 country.
About this study
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
To evaluate the effects of oral emixustat hydrochloride (emixustat) on aqueous humor biomarkers associated with proliferative diabetic retinopathy (PDR) from baseline to week 12.
Read the 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.
- Conditions
- Proliferative Diabetic Retinopathy
- Being tested
- emixustat hydrochloride, Placebo
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
emixustat hydrochlorideDrug
Also called ACU-4429, Emixustat HCl
Tablet for oral administration
PlaceboOther
Placebo tablets for oral administration contain only inactive ingredients
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: 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. Receives: Other: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: Baseline and 12 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Apr 2016 (estimated)
- Study startMay 2016
- Primary completionNov 2017
- Study completionDec 2017
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Proliferative Diabetic RetinopathyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 24 participants (actual)
Sponsor and investigators
- Lead sponsor
- Kubota Vision Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Responsible Medical Officer, Study director, Kubota Vision Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02753400
- Sponsor's protocol ID
- 4429-203
- Status last verified
- Apr 2021
- First submitted
- 19 Apr 2016
- Last updated
- 19 May 2021
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- Individual data sharing
- Undecided
Publications
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.
PubMed 32852613
Study at a glance
From the registry record, last updated by the sponsor on 19 May 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT02753400
- Acronym / sponsor ID
- 4429-203
- Conditions
- Proliferative Diabetic Retinopathy
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 24 participants (actual)
- Lead sponsor
- Kubota Vision Inc.Industry (pharma, biotech, devices)
- Study start
- May 2016 (actual)
- Primary completion
- Nov 2017 (actual)
- Study completion
- Dec 2017 (actual)
- First posted
- 27 Apr 2016 (estimated)
- Results
- Posted 27 Apr 2021
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Emixustat hydrochloride | Experimental | Drug: emixustat hydrochloride, Other: PlaceboWeek 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. |
| Placebo | Placebo comparator | Other: PlaceboFour 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. |
Who may qualify
- Ages
- 18 to 85 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- 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
Key exclusion criteria (13 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| emixustat hydrochlorideAlso: ACU-4429, Emixustat HClTablet for oral administration | Drug | Emixustat hydrochloride |
| PlaceboPlacebo tablets for oral administration contain only inactive ingredients | Other | Emixustat hydrochloride, Placebo |
Primary outcome
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
Time frame: Baseline and 12 weeks
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.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 May 2021.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 32852613Kubota 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.
- Individual participant dataSharing planned: Undecided
Related trials for Proliferative Diabetic Retinopathy
Recruiting
To Investigate the Feasibility and Acceptability of Trained Non-medical Professionals Performing Laser Treatment for Proliferative Diabetic Retinopathy (PDR), and the Potential Impact on Service Capacity. Assess Feasibility of Conducting a Full Trial Comparing Trained ECPs With Ophthalmologists
Proliferative Diabetic Retinopathy (PDR)
RecruitingPhase 2
A Study to Test Whether BI 764524 Helps People With an Eye Condition Called Diabetic Retinopathy
Diabetic Retinopathy
RecruitingObservational study
Longitudinal Observational Study of Diabetic Retinopathy Progression in Type 2 Diabetes Patients
Diabetic Retinopathy, Diabetes Mellitus, Type 2
Not yet recruitingPhase 4
Continuous Delivery of Ranibizumab Via the Port Delivery System for Proliferative Diabetic Retinopathy-related Vitreous Hemorrhage With Pars Plana Vitrectomy and Limited PRP.
Proliferative Diabetic Retinopathy, Diabetic Retinopathy
Active, not recruitingPhase 2
A Study to Evaluate the Efficacy and Safety of Orally Administered VX-01
Diabetic Retinopathy, NPDR - Non Proliferative Diabetic Retinopathy
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Responsible Medical Officer study director
Kubota Vision Inc.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing emixustat hydrochloride in people with Proliferative Diabetic Retinopathy, run by Kubota Vision Inc. at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 13 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 19 May 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 27 Apr 2021). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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 (time frame: Baseline and 12 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02753400, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 19 May 2021.