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

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

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:
  • emixustat hydrochloride
Experimental: Emixustat Dose 2
middle dose of once-daily oral emixustat
Once daily, tablet for oral administration
Other Names:
  • emixustat hydrochloride
Experimental: Emixustat Dose 3
highest dose of once-daily oral emixustat
Once daily, tablet for oral administration
Other Names:
  • emixustat hydrochloride

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.

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

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.

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