- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT03033108
Pharmacodynamic Study of Emixustat Hydrochloride in Subjects With Macular Atrophy Secondary to Stargardt Disease
April 27, 2021 updated by: Kubota Vision Inc.
A Phase 2a Multicenter, Randomized, Masked Study Evaluating the Pharmacodynamics of Emixustat Hydrochloride in Subjects With Macular Atrophy Secondary to Stargardt Disease
This is a pharmacodynamics study of emixustat hydrochloride in subjects with macular atrophy secondary to Stargardt disease.
Study Overview
Status
Completed
Conditions
Intervention / Treatment
Detailed Description
This is a multicenter, randomized, masked study to characterize the pharmacodynamics, safety and tolerability of emixustat in subjects with macular atrophy secondary to Stargardt disease.
Study Type
Interventional
Enrollment (Actual)
23
Phase
- Phase 2
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Locations
-
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Texas
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Dallas, Texas, United States, 75231
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-
Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
18 years and older (Adult, Older Adult)
Accepts Healthy Volunteers
No
Genders Eligible for Study
All
Description
Inclusion Criteria, including, but not limited to:
- Clinical diagnosis of macular atrophy (MA) secondary to Stargardt disease (STGD) in one or both eyes
- At least 2 pathogenic mutations of the ABCA4 gene
- Early Treatment Diabetic Retinopathy Study BCVA of ≥ 20 letters (approximately ≥ 20/400 Snellen) in the study eye
- Adequate clarity of ocular media and adequate pupillary dilation to permit good quality imaging of MA in the study eye
- Able and willing to provide written informed consent before undergoing any study-related procedures
- Able to reliably administer oral medication by self or with available assistance
Exclusion Criteria, including, but not limited to:
- Macular atrophy associated with a condition other than STGD in either eye.
- Presence in either eye of an active ocular disease that in the opinion of the Investigator compromises or confounds visual function.
- History of any intraocular or ocular surface surgery in either eye within 3 months of screening.
- Current or previous participation in an interventional study to treat STGD using gene therapy or stem cell therapy at any time, or participation in an interventional study of a vitamin A derivative ≤3 months prior to screening.
- Pre-specified laboratory abnormalities at screening
- Presence of other medical or ophthalmic disease, physical examination finding, or clinical laboratory finding that in the opinion of the Investigator may contraindicate the use of an investigational drug and place the subject at risk
- Current or history of cancer (except for adequately treated basal cell or squamous cell carcinoma of the skin) within 1 year of screening
- History of myocardial infarction, stroke, unstable ischemic heart disease, uncontrolled cardiac arrhythmia, or hospitalization for congestive heart failure within 6 months of screening.
- Anticipated hospitalization for a medical/surgical procedure(s) that could result in interruption/premature cessation of study treatment or participation.
- Electrocardiogram with a clinically significant abnormal finding
- Female subjects who are pregnant or lactating
- Female subjects of childbearing potential or male subjects who are not surgically sterile who are not willing to practice a medically accepted method of birth control with their sexual partner from screening through 30 days after the final dose of study drug
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: Randomized
- Interventional Model: Parallel Assignment
- Masking: Quadruple
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
Experimental: Emixustat Dose 1
lowest dose of once-daily oral emixustat
|
Once daily, tablet for oral administration
Other Names:
|
Experimental: Emixustat Dose 2
middle dose of once-daily oral emixustat
|
Once daily, tablet for oral administration
Other Names:
|
Experimental: Emixustat Dose 3
highest dose of once-daily oral emixustat
|
Once daily, tablet for oral administration
Other Names:
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Change in Electrical Response of the Retina to a Flash of Light, as Measured by Electroretinogram
Time Frame: Baseline and 1 month
|
Percent suppression compared to baseline of rod b-wave amplitude recovery after a photobleaching light.
|
Baseline and 1 month
|
Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Percentage of Subjects With Adverse Events, by Severity and Seriousness
Time Frame: 1 month
|
Assessment of safety profile
|
1 month
|
Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Sponsor
Investigators
- Study Director: Acucela Medical Director, MD, Kubota Vision Inc.
Publications and helpful links
The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start (Actual)
January 1, 2017
Primary Completion (Actual)
November 1, 2017
Study Completion (Actual)
December 1, 2017
Study Registration Dates
First Submitted
January 20, 2017
First Submitted That Met QC Criteria
January 24, 2017
First Posted (Estimate)
January 26, 2017
Study Record Updates
Last Update Posted (Actual)
May 19, 2021
Last Update Submitted That Met QC Criteria
April 27, 2021
Last Verified
April 1, 2021
More Information
Terms related to this study
Additional Relevant MeSH Terms
Other Study ID Numbers
- 4429-204
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
No
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
Clinical Trials on Stargardt Disease
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Alkeus Pharmaceuticals, Inc.RecruitingStargardt Disease | Stargardt Macular Degeneration | Stargardt Macular Dystrophy | Autosomal Recessive Stargardt Disease 1 (ABCA4-related)United States
-
Alkeus Pharmaceuticals, Inc.Enrolling by invitationStargardt Disease | Stargardt Macular Degeneration | Stargardt Macular Dystrophy | Autosomal Recessive Stargardt Disease 1 (ABCA4-related)United States
-
West China HospitalActive, not recruiting
-
Belite Bio, IncActive, not recruitingStargardt Disease 1United States, Australia, Belgium, China, France, Germany, Hong Kong, Netherlands, Switzerland, Taiwan, United Kingdom
-
Institute of Molecular and Clinical Ophthalmology...RecruitingStargardt Disease | Stargardt Disease 1 | Fundus Flavimaculatus | Macular Degeneration, Stargardt | Macular Dystrophy With Flecks, Type 1Switzerland
-
IVERIC bio, Inc.Active, not recruitingStargardt Disease 1Italy, United States, Israel, United Kingdom, Germany, Spain, Hungary, France
-
Stargazer Pharmaceuticals, Inc.Completed
-
Queen's University, BelfastUniversity of SussexUnknownRetinitis Pigmentosa | Low Vision | Albinism | Stargardt Disease 1 | Stargardt Disease 3 | Stargardt Disease 4United Kingdom
-
Ophthalmos Research and Education InstituteCompletedStargardt Disease 1 | Dry AMDFrance, Germany, Italy
-
Pomeranian Medical University SzczecinUnknownRetinal Degeneration | Retinitis Pigmentosa | Age Related Macular Degeneration | Stargardt Disease 1Poland
Clinical Trials on Emixustat
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Kubota Vision Inc.Food and Drug Administration (FDA)CompletedStargardt DiseaseUnited States, United Kingdom, Germany, Spain, Brazil, Canada, Denmark, France, Italy, Netherlands, South Africa
-
Kubota Vision Inc.CompletedProliferative Diabetic RetinopathyUnited States
-
Kubota Vision Inc.Otsuka Pharmaceutical Co., Ltd.CompletedGeographic AtrophyUnited States
-
Kubota Vision Inc.CompletedAge-Related Macular Degeneration | Geographic AtrophyUnited States