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Pharmacodynamic Study of Emixustat Hydrochloride in Subjects With Macular Atrophy Secondary to Stargardt Disease

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ClinicalTrials.gov Identifier: NCT03033108
Recruitment Status : Completed
First Posted : January 26, 2017
Results First Posted : April 27, 2021
Last Update Posted : May 19, 2021
Sponsor:
Information provided by (Responsible Party):
Kubota Vision Inc.

Study Description
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Brief Summary:
This is a pharmacodynamics study of emixustat hydrochloride in subjects with macular atrophy secondary to Stargardt disease.

Condition or disease Intervention/treatment Phase
Stargardt Disease Macular Atrophy Drug: Emixustat Phase 2

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 Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 23 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A Phase 2a Multicenter, Randomized, Masked Study Evaluating the Pharmacodynamics of Emixustat Hydrochloride in Subjects With Macular Atrophy Secondary to Stargardt Disease
Actual Study Start Date : January 2017
Actual Primary Completion Date : November 2017
Actual Study Completion Date : December 2017

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Experimental: Emixustat Dose 1
lowest dose of once-daily oral emixustat
 Drug: Emixustat
Once daily, tablet for oral administration
Other Name: emixustat hydrochloride
Experimental: Emixustat Dose 2
middle dose of once-daily oral emixustat
 Drug: Emixustat
Once daily, tablet for oral administration
Other Name: emixustat hydrochloride
Experimental: Emixustat Dose 3
highest dose of once-daily oral emixustat
 Drug: Emixustat
Once daily, tablet for oral administration
Other Name: emixustat hydrochloride


Outcome Measures
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Primary Outcome Measures :
  1. 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.


Secondary Outcome Measures :
  1. Percentage of Subjects With Adverse Events, by Severity and Seriousness [ Time Frame: 1 month ]
    Assessment of safety profile


Eligibility Criteria
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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03033108


Locations
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United States, Texas
Dallas, Texas, United States, 75231
Sponsors and Collaborators
Kubota Vision Inc.
Investigators
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Study Director: Acucela Medical Director, MD Kubota Vision Inc.
  Study Documents (Full-Text)

Documents provided by Kubota Vision Inc.:
Study Protocol and Statistical Analysis Plan  [PDF] July 13, 2017

More Information
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Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

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Responsible Party: Kubota Vision Inc.
ClinicalTrials.gov Identifier: NCT03033108    
Other Study ID Numbers: 4429-204
First Posted: January 26, 2017    Key Record Dates
Results First Posted: April 27, 2021
Last Update Posted: May 19, 2021
Last Verified: April 2021
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No
Additional relevant MeSH terms:
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Stargardt Disease
Macular Degeneration
Anetoderma
Atrophy
Pathological Conditions, Anatomical
Eye Diseases, Hereditary
Eye Diseases
 Retinal Degeneration
Retinal Diseases
Genetic Diseases, Inborn
Connective Tissue Diseases
Skin Abnormalities
Skin Diseases