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SeaSTAR: Safety and Efficacy of Emixustat in Stargardt Disease

Sponsor
Kubota Vision Inc. (Industry)
Overall Status
Completed
CT.gov ID
NCT03772665
Collaborator
(none)
194
Enrollment
29
Locations
2
Arms
43.5
Actual Duration (Months)
6.7
Patients Per Site
0.2
Patients Per Site Per Month

Study Details

Study Description

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
Phase 3

Study Design

Study Type:
Interventional
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.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 :
Nov 7, 2018
Actual Primary Completion Date :
Jun 13, 2022
Actual Study Completion Date :
Jun 23, 2022

Arms and Interventions

Arm Intervention/Treatment
Experimental: Emixustat

10 mg

Drug: Emixustat
Once daily oral tablet taken for 24 months
Other Names:
  • 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

    Primary Outcome Measures

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

    Secondary Outcome Measures

    1. Number of participants with treatment-related adverse events as assessed by common terminology for adverse events v5.0 [24 months]

    2. Mean rate of change in retinal sensitivity as measured by microperimetry [24 months]

    3. Mean change in contrast sensitivity [24 months]

    4. Mean change in reading speed [24 months]

    5. Mean change in best-corrected Early Treatment Diabetic Retinopathy Study (ETDRS) visual acuity letter score [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 [24 months]

    7. Mean rate of change in area of ellipsoid zone loss on optical coherence tomography (OCT) [24 months]

    8. Mean change from baseline in mean outer nuclear layer thickness on OCT [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 [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 [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 [24 months]

      Values range from 0 to 1, with 1 representing better health

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    16 Years and Older
    Sexes Eligible for Study:
    All
    Accepts Healthy Volunteers:
    No
    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

    Locations

    Site City State Country Postal Code
    1 Retina-Vitreous Associates Medical Group Beverly Hills California United States 90211
    2 UCSF Dept. of Ophthalmology San Francisco California United States 94143-0730
    3 Emory University Atlanta Georgia United States 30322
    4 The Wilmer Eye Institute Johns Hopkins University Baltimore Maryland United States 21287
    5 University of Michigan Kellogg Eye Center Ann Arbor Michigan United States 48105
    6 Mayo Clinic Rochester Rochester Minnesota United States 55905
    7 Duke Eye Center Durham North Carolina United States 27710
    8 Casey Eye Institute - OHSU Portland Oregon United States 97239
    9 Retina Foundation of the Southwest Dallas Texas United States 75231
    10 University of Utah John Moran Eye Center Salt Lake City Utah United States 84132
    11 Medical College of Wisconsin-Eye Institute Milwaukee Wisconsin United States 53226
    12 Santa Casa de Misericórdia de Belo Horizonte Belo Horizonte Minas Gerais Brazil 30150-320
    13 Hospital Sao Paulo São Paulo Brazil 04024-002
    14 The Hospital for Sick Children Toronto Ontario Canada MSG 1X8
    15 Rigshospitalet-Glostrup Glostrup Hovedstaden Denmark DK-2600
    16 Service D'Ophtalmologie Chi Creteil Créteil Île-de-France France 94000
    17 CHNO Quinze-Vingts - CIC Paris Île-de-France France 75012
    18 Universitätsklinikum Tübingen, Department für Augenheilkunde Tübingen Baden-Württemberg Germany 72076
    19 Universitäts-Augenklinik Bonn Bonn Germany 53127
    20 AOU Università della Campania Luigi Vanvitelli Naples Campania Italy 80131
    21 Università Cattolica del Sacro Cuore - Fondazione Policlinico Gemelli Rome Lazio Italy 00168
    22 IRCCS Ospedale San Raffaele Milan Lombardy Italy 20132
    23 UOC Oculistica Asst Fatebene Pratelli Sacco Universita delgi Studi di Milano Milan Lombardy Italy 20157
    24 SODC di Oculistica AOU Careggi Florence Tuscany Italy 50134
    25 Radboud University Medical Center Nijmegen Gelderland Netherlands 6500
    26 Pretoria Eye Institute Pretoria Gauteng South Africa 0082
    27 Fundacion Jimenez Diaz University Hospital Madrid Spain 28040
    28 Oxford Eye Hospital,Oxford University Hospitals NHS Foundation Trust Oxford Oxfordshire United Kingdom OXD3 9DU
    29 Moorfields Eye Hospital NHS Foundation Trust London United Kingdom EC1V 2PD

    Sponsors and Collaborators

    • Kubota Vision Inc.

    Investigators

    • Study Director: Jeff Gregory, MD, VP of Clinical Development, Acucela

    Study Documents (Full-Text)

    None provided.

    More Information

    Publications

    None provided.
    Responsible Party:
    Kubota Vision Inc.
    ClinicalTrials.gov Identifier:
    NCT03772665
    Other Study ID Numbers:
    • 4429-301
    • R01FD006849
    First Posted:
    Dec 11, 2018
    Last Update Posted:
    Jul 1, 2022
    Last Verified:
    Jun 1, 2022
    Individual Participant Data (IPD) Sharing Statement:
    Undecided
    Plan to Share IPD:
    Undecided
    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:
    Stargardt Disease
    Macular Degeneration

    Study Results

    No Results Posted as of Jul 1, 2022