Pharmacodynamic Study of Emixustat Hydrochloride in Subjects With Macular Atrophy Secondary to Stargardt Disease

NCT ID: NCT03033108

Last Updated: 2021-05-19

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2
• Total Enrollment: 23 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2017-01-31
• Study Completion Date: 2017-12-31

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.

Conditions

Stargardt Disease; Macular Atrophy

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: QUADRUPLE

Study Groups

Emixustat Dose 1

lowest dose of once-daily oral emixustat

Group Type: EXPERIMENTAL

Emixustat

Intervention Type: DRUG

Once daily, tablet for oral administration

Emixustat Dose 2

middle dose of once-daily oral emixustat

Group Type: EXPERIMENTAL

Emixustat

Intervention Type: DRUG

Once daily, tablet for oral administration

Emixustat Dose 3

highest dose of once-daily oral emixustat

Group Type: EXPERIMENTAL

Emixustat

Intervention Type: DRUG

Once daily, tablet for oral administration

Interventions

Emixustat

Once daily, tablet for oral administration

Intervention Type: DRUG

Other Intervention Names

emixustat hydrochloride

Eligibility Criteria

Inclusion Criteria

• 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

• 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

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Kubota Vision Inc.

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Acucela Medical Director, MD

Role: STUDY_DIRECTOR

Kubota Vision Inc.

Locations

Dallas, Texas, United States

Countries

United States

References

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.

Reference Type: DERIVED

PMID: 33214244 (View on PubMed)

Provided Documents

Document Type: Study Protocol and Statistical Analysis Plan

View Document

Other Identifiers

4429-204

Identifier Type: -

Identifier Source: org_study_id