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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.

Tracking Information
First Submitted Date  ICMJE January 20, 2017
First Posted Date  ICMJE January 26, 2017
Results First Submitted Date  ICMJE March 31, 2021
Results First Posted Date  ICMJE April 27, 2021
Last Update Posted Date May 19, 2021
Actual Study Start Date  ICMJE January 2017
Actual Primary Completion Date November 2017   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: April 27, 2021)
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.
Original Primary Outcome Measures  ICMJE
 (submitted: January 24, 2017)
Change in Electrical Response of the Retina to a Flash of Light, as Measured by Electroretinogram [ Time Frame: 1 month ]
Percent suppression compared to baseline of rod b-wave amplitude recovery after a photobleaching light.
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: January 24, 2017)
Percentage of Subjects With Adverse Events, by Severity and Seriousness [ Time Frame: 1 month ]
Assessment of safety profile
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Pharmacodynamic Study of Emixustat Hydrochloride in Subjects With Macular Atrophy Secondary to Stargardt Disease
Official Title  ICMJE A Phase 2a Multicenter, Randomized, Masked Study Evaluating the Pharmacodynamics of Emixustat Hydrochloride in Subjects With Macular Atrophy Secondary to Stargardt Disease
Brief Summary This is a pharmacodynamics study of emixustat hydrochloride in subjects with macular atrophy secondary to Stargardt disease.
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  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Condition  ICMJE
  • Stargardt Disease
  • Macular Atrophy
Intervention  ICMJE Drug: Emixustat
Once daily, tablet for oral administration
Other Name: emixustat hydrochloride
Study Arms  ICMJE
  • Experimental: Emixustat Dose 1
    lowest dose of once-daily oral emixustat
    Intervention: Drug: Emixustat
  • Experimental: Emixustat Dose 2
    middle dose of once-daily oral emixustat
    Intervention: Drug: Emixustat
  • Experimental: Emixustat Dose 3
    highest dose of once-daily oral emixustat
    Intervention: Drug: Emixustat
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: January 17, 2018)
23
Original Estimated Enrollment  ICMJE
 (submitted: January 24, 2017)
30
Actual Study Completion Date  ICMJE December 2017
Actual Primary Completion Date November 2017   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03033108
Other Study ID Numbers  ICMJE 4429-204
Has Data Monitoring Committee No
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Responsible Party Kubota Vision Inc.
Study Sponsor  ICMJE Kubota Vision Inc.
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: Acucela Medical Director, MD Kubota Vision Inc.
PRS Account Kubota Vision Inc.
Verification Date April 2021

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP