Clinical trial NCT01802866: Safety and Efficacy Assessment Treatment Trials of Emixustat Hydrochloride
Official title: 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
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
PHASE3
Conditions: Geographic Atrophy
Keywords: Geographic atrophy
GA
Dry AMD
Lead sponsor: Kubota Vision Inc.
Sponsor class: INDUSTRY
Collaborators: name: Otsuka Pharmaceutical Co., Ltd.
First posted: 2013-03-04
Enrollment: 508
Sex: ALL
Minimum age (years): 55
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: 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.


Eligibility criteria: Inclusion Criteria:

1. Males or females, age ≥55 years.
2. Clinical diagnosis of GA associated with AMD
3. Able and willing to provide written informed consent.
4. Able to reliably administer oral medication by self or with available assistance.

Exclusion Criteria:

1. Active CNV or presence of an active ocular disease.
2. Known serious allergy to the fluorescein sodium for injection in angiography.
3. Pre-specified laboratory abnormalities at screening.
4. Treatment with any investigational study drug within 30 days of screening or device (within 60 days of screening)
5. History of other disease, metabolic dysfunction, physical examination finding, or clinical laboratory finding
6. Female subjects who are pregnant or lactating.
7. 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.
8. Unstable or poorly controlled medical or ophthalmic conditions

Primary outcomes:
measure: Change from baseline in the total area of the GA lesion(s)
timeFrame: 24 months
Secondary outcomes:
measure: Change from baseline in BCVA score
timeFrame: 24 months
measure: Frequency of AEs, discontinuations due to AEs, or dose modifications; severity and seriousness of AEs
timeFrame: 24 months
Other outcomes:
Locations:
city: Phoenix
country: United States
state: Arizona
Study officials:
affiliation: Kubota Vision Inc.
name: Acucela Medical Monitor
role: STUDY_DIRECTOR
References:
citation: Yeong JL, Loveman E, Colquitt JL, Royle P, Waugh N, Lois N. Visual cycle modulators versus placebo or observation for the prevention and treatment of geographic atrophy due to age-related macular degeneration. Cochrane Database Syst Rev. 2020 Dec 17;12(12):CD013154. doi: 10.1002/14651858.CD013154.pub2.
citation: Rosenfeld PJ, Dugel PU, Holz FG, Heier JS, Pearlman JA, Novack RL, Csaky KG, Koester JM, Gregory JK, Kubota R. Emixustat Hydrochloride for Geographic Atrophy Secondary to Age-Related Macular Degeneration: A Randomized Clinical Trial. Ophthalmology. 2018 Oct;125(10):1556-1567. doi: 10.1016/j.ophtha.2018.03.059. Epub 2018 Apr 30.