Pharmacokinetic and Pharmacodynamic Study of Emixustat in Subjects With Geographic Atrophy Associated With Dry Age-Related Macular Degeneration

Kubota Vision

Status and phase: Completed; Phase 1

Conditions: Age-Related Macular Degeneration; Geographic Atrophy

Treatments: Drug: Emixustat HCl Tablet; Other: Placebo Tablet

Study type: Interventional

Funder types: Industry

Identifiers

NCT02130531

4429-101

Details and patient eligibility

About

The purpose of this study is to characterize emixustat hydrochloride pharmacokinetic and pharmacodynamic parameters in subjects with geographic atrophy associated with dry age-related macular degeneration.

Full description

This is a multi-center, randomized, masked, multiple-dose, crossover study of the pharmacokinetics of emixustat hydrochloride (HCl) in subjects with geographic atrophy (GA) associated with dry age-related macular degeneration (AMD). Subjects will receive study drug during multiple dose periods in a crossover design, and serve as their own controls. There is a 21-28 day washout period between dose periods. The final Study Exit visit is 21 to 28 days after the subject's last dose of study drug at the end of the final dosing period.

Enrollment: 26 patients

Sex: All

Ages: 65+ years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Males or females age ≥65 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

• Geographic atrophy associated with a condition other than AMD
• History of, active or high risk of developing choroidal neovascularization (CNV) in either eye
• 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
• Prior participation in any clinical study of emixustat
• History of other disease, metabolic dysfunction, chronic immunosuppression, physical examination finding
• Male subjects who are not surgically sterile and are not willing to practice a medically accepted method of birth control with their female partner of childbearing potential from screening through 21 days after the last dose of study drug

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Crossover Assignment

Masking: Triple Blind

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26 participants in 2 patient groups

Cohort 1

Experimental group

Description:

Two dose periods in any sequence (subjects will be assigned to 1 of 2 possible sequences): * Emixustat HCl Tablet Strength B (mid dose); 1 tablet once daily \& 1 placebo tablet once daily for 7 days * Emixustat HCl Tablet Strength A (low dose); 1 tablet twice daily for 7 days

Treatment:

Other: Placebo Tablet

Drug: Emixustat HCl Tablet

Cohort 2

Experimental group

Description:

Three dose periods in any sequence (subjects will be assigned to 1 of 6 possible sequences): * Emixustat HCl Tablet Strength A (low dose); 1 tablet daily for 7 days * Emixustat HCl Tablet Strength B (mid dose); 1 tablet daily for 7 days * Emixustat HCl Tablet Strength C (high dose); 1 tablet daily for 7 days

Treatment:

Drug: Emixustat HCl Tablet