Trial · NCT03772665

Safety and Efficacy of Emixustat in Stargardt Disease

NCT03772665, Phase 3, Completed, Kubota Vision Inc.; results first posted 2023-08-03

3 sourcestrials-2026-11-12-0.5.0

Cite this record

ConductScience Library. Safety and Efficacy of Emixustat in Stargardt Disease. Trials, record NCT03772665. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03772665

On this page

Trial

As registered on ClinicalTrials.gov, in the graph's capture of the registry.

Title: Safety and Efficacy of Emixustat in Stargardt Disease
NCT number: NCT03772665
Status: Completed
Phases: Phase 3
Enrollment: 194 participants, actual
Lead sponsor: Kubota Vision Inc.
Conditions: Stargardt Disease
Interventions: Emixustat, Placebo
First posted: 2018-12-11
Last updated: 2024-05-30
Results first posted: 2023-08-03

Source ClinicalTrials.gov

Registered outcomes1

Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.

List	Outcome	Time frame	Description
Primary	Mean Rate of Change in Total Area of Macular Atrophy, as Measured by Fundus Autofluorescence (FAF)	24 months	Mean rate of change in total area of macular atrophy, as measured by fundus autofluorescence (FAF)

Source ClinicalTrials.gov

Outcomes1

Each outcome the trial posted results for, in ClinicalTrials.gov's words.

Type	Outcome	Time frame	Population	Units	Measured as
PRIMARY	Mean Rate of Change in Total Area of Macular Atrophy, as Measured by Fundus Autofluorescence (FAF)	24 months	ITT	Rate of change from BL (mm^2/yr)	MEAN

Source ClinicalTrials.gov

Results by group2

Each measurement the trial posted, group by group, in the words and numbers it posted.

Outcome	Group	Category	Value	Spread	Units
Mean Rate of Change in Total Area of Macular Atrophy, as Measured by Fundus Autofluorescence (FAF)	Emixustat		1.280	Standard Error 0.0783	Rate of change from BL (mm^2/yr)
Mean Rate of Change in Total Area of Macular Atrophy, as Measured by Fundus Autofluorescence (FAF)	Placebo		1.309	Standard Error 0.1002	Rate of change from BL (mm^2/yr)

Source ClinicalTrials.gov

Participant flow6

How many started, completed or left each group, period by period.

Period	Milestone	Group	Participants
Overall Study	STARTED	Emixustat	128
Overall Study	STARTED	Placebo	66
Overall Study	COMPLETED	Emixustat	127
Overall Study	COMPLETED	Placebo	66
Overall Study	NOT COMPLETED	Emixustat	1
Overall Study	NOT COMPLETED	Placebo	0

Source ClinicalTrials.gov

Adverse events18

Each adverse event the trial posted, group by group: the participants affected out of those at risk.

Type	Event	Organ system	Group	Affected	At risk	Events
other	Visual acuity reduced	Eye disorders	Emixustat	1	127	1
other	Visual acuity reduced	Eye disorders	Placebo	0	66	0
serious	agina pectoris	Cardiac disorders	Emixustat	1	127	1
serious	agina pectoris	Cardiac disorders	Placebo	1	66	1
serious	atrial fibrillation	Cardiac disorders	Emixustat	1	127	1
serious	atrial fibrillation	Cardiac disorders	Placebo	0	66	0
serious	neoplasm	Neoplasms benign, malignant and unspecified (incl cysts and polyps)	Emixustat	2	127	2
serious	neoplasm	Neoplasms benign, malignant and unspecified (incl cysts and polyps)	Placebo	1	66	1
serious	Infections and infestations	Infections and infestations	Emixustat	1	127	1
serious	Infections and infestations	Infections and infestations	Placebo	1	66	1

1 to 10 of 18Source ClinicalTrials.gov

See all 18 adverse events

Adverse event totals6

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	Emixustat	0	127
Total, all-cause mortality	Placebo	0	66
Total, other adverse events	Emixustat	1	127
Total, other adverse events	Placebo	0	66
Total, serious adverse events	Emixustat	9	127
Total, serious adverse events	Placebo	2	66

Source ClinicalTrials.gov

Papers0

The papers the trial lists on ClinicalTrials.gov.

None in this release.

Grants1

The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.

Project	Title	Principal investigators	Organization	Funder	Fiscal years
R01FD006849	A Phase 3 Study of Emixustat Hydrochloride for Stargardt Disease IND 101,084 (02/11/2019)	KUBOTA, RYO	ACUCELA, INC.	FOOD AND DRUG ADMINISTRATION	2020 to 2022

Source NIH RePORTER

Trial comparisons2

Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.

Comparison	Source
Emixustat vs Placebo: deaths from any cause	Trial comparisons
Emixustat vs Placebo: serious adverse events	Trial comparisons

Tests0

The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.

None in this release.

Data dates

The day each source was copied for this release.

ClinicalTrials.gov: 2026-11-12; ClinicalTrials.gov
Results, from AACT: 2026-03-23; AACT
NIH grant links: 2026-11-13; NIH RePORTER

Sources 3

ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
ClinicalTrials.govNo license stated
NIH RePORTER's clinical studies and project filesPublic domain

Each source's terms

ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
ClinicalTrials.govnone stated
NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)

ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.

For software

Read this trial from software.

The record and each of its lists are JSON, twenty rows a page. No key.

GET https://conductscience.org/api/trials/v1/NCT03772665

GET https://conductscience.org/api/trials/v1/NCT03772665/registered

API reference For AI assistants