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

NCT03033108 | Completed Phase 2 Results

• 1 other identifier: 4429-204
• Study Type: interventional
• Target: 23
• Locations: 1 country
• Sites: 1

Brief Summary

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

Conditions

Stargardt Disease; Macular Atrophy

Outcome Measures

Primary Outcomes (1)

• Change in Electrical Response of the Retina to a Flash of Light, as Measured by Electroretinogram

  Percent suppression compared to baseline of rod b-wave amplitude recovery after a photobleaching light.

  Baseline and 1 month

Secondary Outcomes (1)

• Percentage of Subjects With Adverse Events, by Severity and Seriousness

  1 month

Study Arms (3)

Emixustat Dose 1

EXPERIMENTAL

lowest dose of once-daily oral emixustat

Drug: Emixustat

Emixustat Dose 2

EXPERIMENTAL

middle dose of once-daily oral emixustat

Drug: Emixustat

Emixustat Dose 3

EXPERIMENTAL

highest dose of once-daily oral emixustat

Drug: Emixustat

Interventions

Emixustat DRUG

Once daily, tablet for oral administration

Also known as: emixustat hydrochloride

Emixustat Dose 1; Emixustat Dose 2; Emixustat Dose 3

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Kubota Vision Inc.: lead

Study Sites (1)

Unknown Facility

Dallas, Texas, 75231, United States

Location

Related Publications (1)

• 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.

  PMID: 33214244 DERIVED

MeSH Terms

Conditions

Stargardt Disease; Anetoderma

Interventions

emixustat

Condition Hierarchy (Ancestors)

Eye Diseases, Hereditary; Eye Diseases; Macular Degeneration; Retinal Degeneration; Retinal Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Connective Tissue Diseases; Skin and Connective Tissue Diseases; Skin Abnormalities; Skin Diseases

Limitations and Caveats

Small sample size and inherent, significant intersubject and intrasubject variability observed in electroretinography.

Results Point of Contact

• Title: Clinical Trials Helpdesk
• Organization: Kubota Vision Inc.

clinicaltrials@kubotavision.com

(206) 805-8310

Study Officials

• Acucela Medical Director, MD

  Kubota Vision Inc.

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 20, 2017
• First Posted: January 26, 2017
• Study Start: January 1, 2017
• Primary Completion: November 1, 2017
• Study Completion: December 1, 2017
• Last Updated: May 19, 2021
• Results First Posted: April 27, 2021

Record last verified: 2021-04

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)