Safety and Efficacy Assessment Treatment Trials of Emixustat Hydrochloride

Study Overview
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Brief Summary
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The purpose of this study is to determine if emixustat hydrochloride (ACU-4429) reduces the rate of progression of geographic atrophy compared to placebo in subjects with dry age-related macular degeneration.

Official Title
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A Phase 2b/3 Multicenter, Randomized, Double-Masked, Dose-Ranging Study Comparing the Efficacy and Safety of Emixustat Hydrochloride (ACU-4429) With Placebo for the Treatment of Geographic Atrophy Associated With Dry Age-Related Macular Degeneration

Conditions
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Geographic Atrophy

Intervention / Treatment
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* Drug: ACU-4429
* Drug: Placebo


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Males or females, age ≥55 years. Clinical diagnosis of GA associated with AMD Able and willing to provide written informed consent. Able to reliably administer oral medication by self or with available assistance. Exclusion Criteria: Active CNV or presence of an active ocular disease. Known serious allergy to the fluorescein sodium for injection in angiography. Pre-specified laboratory abnormalities at screening. Treatment with any investigational study drug within 30 days of screening or device (within 60 days of screening) History of other disease, metabolic dysfunction, physical examination finding, or clinical laboratory finding Female subjects who are pregnant or lactating. Female subjects of childbearing potential and male subjects who are not surgically sterile who are not willing to practice a medically accepted method of birth control from screening through 30 days after completion of the study. Unstable or poorly controlled medical or ophthalmic conditions

Ages Eligible for Study
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Minimum Age: 55 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: ACU-4429 2.5 mg<br>2.5 mg tablet | Drug: ACU-4429<br>* Take orally once daily for 24 months<br>* Other names: emixustat hydrochloride;|
| Experimental: ACU-4429 5 mg<br>5 mg tablet | Drug: ACU-4429<br>* Take orally once daily for 24 months<br>* Other names: emixustat hydrochloride;|
| Experimental: ACU-4429 10 mg<br>10 mg tablet | Drug: ACU-4429<br>* Take orally once daily for 24 months<br>* Other names: emixustat hydrochloride;|
| Placebo Comparator: Placebo<br>Includes identical tablets with only inactive ingredients (0 mg). | Drug: Placebo<br>* Take orally once daily for 24 months<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change from baseline in the total area of the GA lesion(s) |  | 24 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change from baseline in BCVA score |  | 24 months | 
| Frequency of AEs, discontinuations due to AEs, or dose modifications; severity and seriousness of AEs |  | 24 months | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Geographic atrophy, GA, Dry AMD