A Phase 3 interventional study of Emixustat and Placebo in Stargardt Disease, sponsored by Kubota Vision Inc.. Completed at 29 sites in 11 countries. Open to participants aged 16 Years and older. Per ClinicalTrials.gov, last updated 2024-05-30.
Sponsored by Kubota Vision Inc. · Phase 3, Interventional, and Treatment
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
Stargardt disease is a rare, inherited degenerative disease of the retina affecting approximately 1 in 8000 to 10 000 people and is the most common type of hereditary macular dystrophy. There are no approved treatments for STGD. This disease is characterized by an excessive accumulation of lipofuscin at the level of the retinal pigment epithelium (RPE). Lipofuscin is made of lipids, proteins, and toxic bis retinoids (such as N retinylidene N retinylethanolamine [A2E]). Accumulation of the toxic bis retinoids found in lipofuscin is thought to cause RPE cell dysfunction and eventual apoptosis, resulting in photoreceptor death and loss of vision.
Stargardt disease has several sub types, where autosomal recessive STGD (STGD1) accounts for the majority (>95%) of all cases. STGD1 is typically diagnosed in the first 3 decades of life and is caused by mutations of the adenosine triphosphate binding cassette subfamily A member 4 (ABCA4) gene. The ABCA4 gene product transports N retinylidene phosphatidylethanolamine (a precursor of toxic bis retinoids) from the lumen side of photoreceptor disc membranes to the cytoplasmic side where the retinal is hydrolyzed from phosphatidylethanolamine. Mutations of the ABCA4 gene result in accumulation of this precursor in disc membranes that are eventually phagocytized by RPE cells, where the precursors are converted into toxic bis retinoids such as A2E. In addition to being a precursor to A2E, all trans retinal has also been implicated in the pathogenesis of STGD through its role in light-mediated toxicity.
Emixustat hydrochloride (emixustat) has been developed by Acucela Inc. for retinal diseases including Stargardt disease (STGD). Emixustat is a potent inhibitor of RPE65 isomerization activity and reduces visual chromophore (11 cis retinal) production in a dose-dependent and reversible manner. Because 11 cis-retinal and its photoproduct (all trans retinal) are substrates for biosynthesis of retinoid toxins (eg, A2E), chronic treatment with emixustat retards the rate at which these toxins accumulate.
67 studies on the registry are indexed under Stargardt Disease; 26 are open to participants now.
This study's enrollment of 194 is above the median of 28 across 44 interventional studies indexed under Stargardt Disease.
Browse Stargardt Disease studies →Kubota Vision Inc. is the lead sponsor of 21 studies on the registry; 2 are open to participants now.
Counted across the registry records on this site, refreshed daily.
Exclusion Criteria:
10 mg
Drug: Emixustat
Includes identical tablets with only inactive ingredients (0 mg).
Drug: Placebo
Once daily oral tablet taken for 24 months
Also known as: emixustat hydrochloride
Once daily oral tablet taken for 24 months
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)
Time frame: 24 months
| Milestone | Emixustat | Placebo |
|---|---|---|
| Started | 128 | 66 |
| Completed | 127 | 66 |
| Not completed | 1 | 0 |
| Withdrew: Protocol violation | 1 | 0 |
Mean rate of change in total area of macular atrophy, as measured by fundus autofluorescence (FAF)
| Rate of change from BL (mm^2/yr) | Emixustat | Placebo |
|---|---|---|
| Mean Rate of Change in Total Area of Macular Atrophy, as Measured by Fundus Autofluorescence (FAF) | 1.280 ± 0.0783 | 1.309 ± 0.1002 |
Collected over 24 months. Non-serious events are listed at a 0% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Emixustat | 0/127 (0%) | 9/127 (7.1%) | 1/127 (0.8%) |
| Placebo | 0/66 (0%) | 2/66 (3%) | 0/66 (0%) |
| Event | Emixustat | Placebo |
|---|---|---|
| neoplasmNeoplasms benign, malignant and unspecified (incl cysts and polyps) | 2/127 | 1/66 |
| agina pectorisCardiac disorders | 1/127 | 1/66 |
| Infections and infestationsInfections and infestations | 1/127 | 1/66 |
| atrial fibrillationCardiac disorders | 1/127 | 0/66 |
| Visual disorderEye disorders | 1/127 | 0/66 |
| Injury, posioning and proceduralInjury, poisoning and procedural complications | 1/127 | 0/66 |
| Nervous system disorderNervous system disorders | 1/127 | 0/66 |
| Vascular disorderVascular disorders | 1/127 | 0/66 |
| Event | Emixustat | Placebo |
|---|---|---|
| Visual acuity reducedEye disorders | 1/127 | 0/66 |
ITT
| Age, Categorical(Participants) | Emixustat | Placebo | Total |
|---|---|---|---|
| <=18 years | 5 | 2 | 7 |
| Between 18 and 65 years | 122 | 64 | 186 |
| >=65 years | 1 | 0 | 1 |
| Age, Continuous(years) | Emixustat | Placebo | Total |
|---|---|---|---|
| Mean | 43.9 ± 15.03 | 43.0 ± 14.07 | 43.6 ± 14.75 |
| Sex: Female, Male(Participants) | Emixustat | Placebo | Total |
|---|---|---|---|
| Female | 59 | 29 | 88 |
| Male | 69 | 37 | 106 |
| Race and Ethnicity Not Collected(Participants) | Emixustat | Placebo | Total |
|---|---|---|---|
| Count of participants | — | — | 0 |
| Region of Enrollment(participants) | Emixustat | Placebo | Total |
|---|---|---|---|
| Canada | 3 | 2 | 5 |
| Netherlands | 3 | 2 | 5 |
| United States | 34 | 19 | 53 |
| Brazil | 20 | 9 | 29 |
| Denmark | 6 | 4 | 10 |
| Italy | 24 | 9 | 33 |
| South Africa | 11 | 7 | 18 |
| United Kingdom | 6 | 3 | 9 |
| France | 8 | 4 | 12 |
| Germany | 8 | 5 | 13 |
| Spain | 5 | 2 | 7 |
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Kubota Vision Inc.