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Safety and Efficacy of Emixustat in Stargardt Disease (SeaSTAR)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03772665
Recruitment Status : Active, not recruiting
First Posted : December 11, 2018
Last Update Posted : September 5, 2021
Sponsor:
Information provided by (Responsible Party):
Kubota Vision Inc.

Study Description
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Brief Summary:

The purpose of this study is to determine if emixustat hydrochloride reduces the rate of progression of macular atrophy compared to placebo in subjects with Stargardt disease.

Funding Source -- FDA OOPD


Condition or disease Intervention/treatment Phase
Stargardt Disease Drug: Emixustat Drug: Placebo Phase 3

Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 194 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Intervention Model Description: This is a multicenter, randomized, double-masked, placebo-controlled study to evaluate the efficacy and safety of emixustat compared to placebo in subjects who have Macular Atrophy secondary to Stargardt disease.
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Masking Description: Double-Masked
Primary Purpose: Treatment
Official Title: A Phase 3 Multicenter, Randomized, Double-Masked Study Comparing the Efficacy and Safety of Emixustat Hydrochloride With Placebo for the Treatment of Macular Atrophy Secondary to Stargardt Disease
Actual Study Start Date : November 7, 2018
Estimated Primary Completion Date : April 2022
Estimated Study Completion Date : June 2022

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Experimental: Emixustat
10 mg
 Drug: Emixustat
Once daily oral tablet taken for 24 months
Other Name: emixustat hydrochloride
Placebo Comparator: Placebo
Includes identical tablets with only inactive ingredients (0 mg).
 Drug: Placebo
Once daily oral tablet taken for 24 months


Outcome Measures
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Primary Outcome Measures :
  1. Mean rate of change in total area of macular atrophy, as measured by fundus autofluorescence (FAF) [ Time Frame: 24 months ]

Secondary Outcome Measures :
  1. Number of participants with treatment-related adverse events as assessed by common terminology for adverse events v5.0 [ Time Frame: 24 months ]
  2. Mean rate of change in retinal sensitivity as measured by microperimetry [ Time Frame: 24 months ]
  3. Mean change in contrast sensitivity [ Time Frame: 24 months ]
  4. Mean change in reading speed [ Time Frame: 24 months ]
  5. Mean change in best-corrected Early Treatment Diabetic Retinopathy Study (ETDRS) visual acuity letter score [ Time Frame: 24 months ]
    Scores range from 0 to 100, with 100 being the best visual acuity.

  6. Mean rate of change in total area of decreased autofluorescence on FAF [ Time Frame: 24 months ]
  7. Mean rate of change in area of ellipsoid zone loss on optical coherence tomography (OCT) [ Time Frame: 24 months ]
  8. Mean change from baseline in mean outer nuclear layer thickness on OCT [ Time Frame: 24 months ]
  9. Mean change in patient assessment of how vision affects their ability to perform everyday tasks, using the Visual Function Questionnaire 25-item (VFQ-25) composite score [ Time Frame: 24 months ]
    Score ranges from 0 to 100, with 100 representing better ability

  10. Mean change in patient assessment of the ability to perform reading activities independently, using the Functional Reading Independence Index (FRII) score [ Time Frame: 24 months ]
    Score ranges from 0 to 4, with 4 representing higher independence

  11. Mean change in patient assessment of general health, using the EQ-5D-5L (a 5-dimension, 5-level, generic measure of health) index value [ Time Frame: 24 months ]
    Values range from 0 to 1, with 1 representing better health


Eligibility Criteria
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Ages Eligible for Study:   16 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • A clinical diagnosis of macular atrophy secondary to Stargardt disease (STGD)
  • Macular atrophy measured to fall within a defined size range
  • Two mutations of the ABCA4 gene. If only one mutation, a typical STGD appearance of the retina.
  • Visual acuity in the study eye of at least 20/320

Exclusion Criteria:

  • Macular atrophy secondary to a disease other than STGD
  • Mutations of genes, other than ABCA4, that are associated with retinal degeneration
  • Surgery in the study eye in the past 3 months
  • Prior participation in a gene therapy or stem cell clinical trial for STGD
  • Recent participation in a clinical trial for STGD evaluating a complement inhibitor or vitamin A derivative
  • Use of certain medications in the past 4 weeks that might interfere with emixustat
  • An abnormal electrocardiogram (ECG)
  • Certain abnormalities on laboratory blood testing
  • Female subjects who are pregnant or nursing
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): NCT03772665


Locations
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Sponsors and Collaborators
Kubota Vision Inc.
Investigators
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Study Director: Jeff Gregory, MD VP of Clinical Development, Acucela
More Information
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Responsible Party: Kubota Vision Inc.
ClinicalTrials.gov Identifier: NCT03772665    
Other Study ID Numbers: 4429-301
R01FD006849 ( U.S. FDA Grant/Contract )
First Posted: December 11, 2018    Key Record Dates
Last Update Posted: September 5, 2021
Last Verified: September 2021
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Kubota Vision Inc.:
Stargardt Disease
STGD
ABCA4
Additional relevant MeSH terms:
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Stargardt Disease
Macular Degeneration
Eye Diseases, Hereditary
Eye Diseases
 Retinal Degeneration
Retinal Diseases
Genetic Diseases, Inborn