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Safety and Efficacy of Emixustat in Stargardt Disease (SeaSTAR)

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ClinicalTrials.gov Identifier: NCT03772665
Recruitment Status : Recruiting
First Posted : December 11, 2018
Last Update Posted : June 11, 2019
Sponsor:
Information provided by (Responsible Party):
Acucela Inc.

Tracking Information
First Submitted Date  ICMJE November 30, 2018
First Posted Date  ICMJE December 11, 2018
Last Update Posted Date June 11, 2019
Actual Study Start Date  ICMJE November 7, 2018
Estimated Primary Completion Date November 2021   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: December 9, 2018)
Mean rate of change in total area of macular atrophy, as measured by fundus autofluorescence (FAF) [ Time Frame: 24 months ]
Original Primary Outcome Measures  ICMJE Same as current
Change History Complete list of historical versions of study NCT03772665 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: December 9, 2018)
  • Number of participants with treatment-related adverse events as assessed by common terminology for adverse events v5.0 [ Time Frame: 24 months ]
  • Mean rate of change in retinal sensitivity as measured by microperimetry [ Time Frame: 24 months ]
  • Mean change in contrast sensitivity [ Time Frame: 24 months ]
  • Mean change in reading speed [ Time Frame: 24 months ]
  • 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.
  • Mean rate of change in total area of decreased autofluorescence on FAF [ Time Frame: 24 months ]
  • Mean rate of change in area of ellipsoid zone loss on optical coherence tomography (OCT) [ Time Frame: 24 months ]
  • Mean change from baseline in mean outer nuclear layer thickness on OCT [ Time Frame: 24 months ]
  • 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
  • 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
  • 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
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 Safety and Efficacy of Emixustat in Stargardt Disease
Official Title  ICMJE 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
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.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE 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
Condition  ICMJE Stargardt Disease
Intervention  ICMJE
  • Drug: Emixustat
    Once daily oral tablet taken for 24 months
    Other Name: emixustat hydrochloride
  • Drug: Placebo
    Once daily oral tablet taken for 24 months
Study Arms  ICMJE
  • Experimental: Emixustat
    10 mg
    Intervention: Drug: Emixustat
  • Placebo Comparator: Placebo
    Includes identical tablets with only inactive ingredients (0 mg).
    Intervention: Drug: Placebo
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 Recruiting
Estimated Enrollment  ICMJE
 (submitted: December 9, 2018)
162
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE March 2022
Estimated Primary Completion Date November 2021   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 16 Years and older   (Child, Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Clinical Trials Helpdesk (206) 805 8310 ClinicalTrials@acucela.com
Listed Location Countries  ICMJE Canada,   Denmark,   France,   Germany,   Italy,   Netherlands,   Spain,   United Kingdom,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03772665
Other Study ID Numbers  ICMJE 4429-301
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: Undecided
Responsible Party Acucela Inc.
Study Sponsor  ICMJE Acucela Inc.
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: Jeff Gregory, MD VP of Clinical Development, Acucela
PRS Account Acucela Inc.
Verification Date June 2019

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