Clinical trial NCT03033108: Pharmacodynamic Study of Emixustat Hydrochloride in Subjects With Macular Atrophy Secondary to Stargardt Disease
Official title: A Phase 2a Multicenter, Randomized, Masked Study Evaluating the Pharmacodynamics of Emixustat Hydrochloride in Subjects With Macular Atrophy Secondary to Stargardt Disease
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Stargardt Disease
Macular Atrophy
Lead sponsor: Kubota Vision Inc.
Sponsor class: INDUSTRY
First posted: 2017-01-26
Enrollment: 23
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: This is a pharmacodynamics study of emixustat hydrochloride in subjects with macular atrophy secondary to Stargardt disease.

Detailed description: This is a multicenter, randomized, masked study to characterize the pharmacodynamics, safety and tolerability of emixustat in subjects with macular atrophy secondary to Stargardt disease.

Eligibility criteria: Inclusion Criteria, including, but not limited to:

* Clinical diagnosis of macular atrophy (MA) secondary to Stargardt disease (STGD) in one or both eyes
* At least 2 pathogenic mutations of the ABCA4 gene
* Early Treatment Diabetic Retinopathy Study BCVA of ≥ 20 letters (approximately ≥ 20/400 Snellen) in the study eye
* Adequate clarity of ocular media and adequate pupillary dilation to permit good quality imaging of MA in the study eye
* Able and willing to provide written informed consent before undergoing any study-related procedures
* Able to reliably administer oral medication by self or with available assistance

Exclusion Criteria, including, but not limited to:

* Macular atrophy associated with a condition other than STGD in either eye.
* Presence in either eye of an active ocular disease that in the opinion of the Investigator compromises or confounds visual function.
* History of any intraocular or ocular surface surgery in either eye within 3 months of screening.
* Current or previous participation in an interventional study to treat STGD using gene therapy or stem cell therapy at any time, or participation in an interventional study of a vitamin A derivative ≤3 months prior to screening.
* Pre-specified laboratory abnormalities at screening
* Presence of other medical or ophthalmic disease, physical examination finding, or clinical laboratory finding that in the opinion of the Investigator may contraindicate the use of an investigational drug and place the subject at risk
* Current or history of cancer (except for adequately treated basal cell or squamous cell carcinoma of the skin) within 1 year of screening
* History of myocardial infarction, stroke, unstable ischemic heart disease, uncontrolled cardiac arrhythmia, or hospitalization for congestive heart failure within 6 months of screening.
* Anticipated hospitalization for a medical/surgical procedure(s) that could result in interruption/premature cessation of study treatment or participation.
* Electrocardiogram with a clinically significant abnormal finding
* Female subjects who are pregnant or lactating
* Female subjects of childbearing potential or male subjects who are not surgically sterile who are not willing to practice a medically accepted method of birth control with their sexual partner from screening through 30 days after the final dose of study drug

Primary outcomes:
measure: Change in Electrical Response of the Retina to a Flash of Light, as Measured by Electroretinogram
timeFrame: Baseline and 1 month
Secondary outcomes:
measure: Percentage of Subjects With Adverse Events, by Severity and Seriousness
timeFrame: 1 month
Other outcomes:
Locations:
city: Dallas
country: United States
state: Texas
Study officials:
affiliation: Kubota Vision Inc.
name: Acucela Medical Director, MD
role: STUDY_DIRECTOR
References:
citation: Kubota R, Birch DG, Gregory JK, Koester JM. Randomised study evaluating the pharmacodynamics of emixustat hydrochloride in subjects with macular atrophy secondary to Stargardt disease. Br J Ophthalmol. 2022 Mar;106(3):403-408. doi: 10.1136/bjophthalmol-2020-317712. Epub 2020 Nov 19.