Safety and Efficacy of Emixustat in Stargardt Disease

NCT03772665 | Completed Phase 3 Results DMC FDA Drug

• 2 other identifiers: 4429-301R01FD006849
• Study Type: interventional
• Target: 194
• Locations: 11 countries
• Sites: 29

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

Conditions

Stargardt Disease

Keywords

Stargardt Disease; STGD; ABCA4

Outcome Measures

Primary Outcomes (1)

• Mean Rate of Change in Total Area of Macular Atrophy, as Measured by Fundus Autofluorescence (FAF)

  Mean rate of change in total area of macular atrophy, as measured by fundus autofluorescence (FAF)

  24 months

Study Arms (2)

Emixustat

EXPERIMENTAL

10 mg

Drug: Emixustat

Placebo

PLACEBO COMPARATOR

Includes identical tablets with only inactive ingredients (0 mg).

Drug: Placebo

Interventions

Emixustat DRUG

Once daily oral tablet taken for 24 months

Also known as: emixustat hydrochloride

Emixustat

Placebo DRUG

Once daily oral tablet taken for 24 months

Placebo

Eligibility Criteria

• Age: 16 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Kubota Vision Inc.: lead
• Food and Drug Administration (FDA): collaborator

Study Sites (29)

Retina-Vitreous Associates Medical Group

Beverly Hills, California, 90211, United States

Location

UCSF Dept. of Ophthalmology

San Francisco, California, 94143-0730, United States

Location

Emory University

Atlanta, Georgia, 30322, United States

Location

The Wilmer Eye Institute Johns Hopkins University

Baltimore, Maryland, 21287, United States

Location

University of Michigan Kellogg Eye Center

Ann Arbor, Michigan, 48105, United States

Location

Mayo Clinic Rochester

Rochester, Minnesota, 55905, United States

Location

Duke Eye Center

Durham, North Carolina, 27710, United States

Location

Casey Eye Institute - OHSU

Portland, Oregon, 97239, United States

Location

Retina Foundation of the Southwest

Dallas, Texas, 75231, United States

Location

University of Utah John Moran Eye Center

Salt Lake City, Utah, 84132, United States

Location

Medical College of Wisconsin-Eye Institute

Milwaukee, Wisconsin, 53226, United States

Location

Santa Casa de Misericórdia de Belo Horizonte

Belo Horizonte, Minas Gerais, 30150-320, Brazil

Location

Hospital Sao Paulo

São Paulo, 04024-002, Brazil

Location

The Hospital for Sick Children

Toronto, Ontario, MSG 1X8, Canada

Location

Rigshospitalet-Glostrup

Glostrup Municipality, Capital Region, DK-2600, Denmark

Location

Service D'Ophtalmologie Chi Creteil

Créteil, Île-de-France Region, 94000, France

Location

CHNO Quinze-Vingts - CIC

Paris, Île-de-France Region, 75012, France

Location

Universitätsklinikum Tübingen, Department für Augenheilkunde

Tübingen, Baden-Wurttemberg, 72076, Germany

Location

Universitäts-Augenklinik Bonn

Bonn, 53127, Germany

Location

AOU Università della Campania Luigi Vanvitelli

Naples, Campania, 80131, Italy

Location

Università Cattolica del Sacro Cuore - Fondazione Policlinico Gemelli

Rome, Lazio, 00168, Italy

Location

IRCCS Ospedale San Raffaele

Milan, Lombardy, 20132, Italy

Location

UOC Oculistica Asst Fatebene Pratelli Sacco Universita delgi Studi di Milano

Milan, Lombardy, 20157, Italy

Location

SODC di Oculistica AOU Careggi

Florence, Tuscany, 50134, Italy

Location

Radboud University Medical Center

Nijmegen, Gelderland, 6500, Netherlands

Location

Pretoria Eye Institute

Pretoria, Gauteng, 0082, South Africa

Location

Fundacion Jimenez Diaz University Hospital

Madrid, 28040, Spain

Location

Oxford Eye Hospital,Oxford University Hospitals NHS Foundation Trust

Oxford, Oxfordshire, OXD3 9DU, United Kingdom

Location

Moorfields Eye Hospital NHS Foundation Trust

London, EC1V 2PD, United Kingdom

Location

MeSH Terms

Conditions

Stargardt Disease

Interventions

emixustat

Condition Hierarchy (Ancestors)

Eye Diseases, Hereditary; Eye Diseases; Macular Degeneration; Retinal Degeneration; Retinal Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Results Point of Contact

• Title: Dr. Jeff Gregory
• Organization: Acucela, dba Kubota Vision

clinicaltrials@acucela.com

2068058310

Study Officials

• Jeff Gregory, MD

  VP of Clinical Development, Acucela

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Masking Details: Double-Masked
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: 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.

Study Record Dates

• First Submitted: November 30, 2018
• First Posted: December 11, 2018
• Study Start: January 7, 2019
• Primary Completion: June 13, 2022
• Study Completion: June 23, 2022
• Last Updated: May 30, 2024
• Results First Posted: August 3, 2023

Record last verified: 2024-05

Locations

BR (2); CA (1); DE (2); ZA (1); FR (2); ES (1); US (11); IT (5); GB (2); NL (1); DK (1)