Serving Gainesville,

Lake City, and Ocala, FL

Gainesville – 352-371-2800

 

 Lake City – 386-754-0999

 

 Ocala – 352-867-0045

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Current Clinical Trials:

The doctors at Vitreoretinal Associates are currently involved in the following clinical trials:

 

1) Phase 1/2 Safety and Efficacy Trial of AAV Gene Therapy in Patients with CNGB3 Achromatopsia

 

Sponsored by AGTC

ClinicalTrials.gov identifier: NCT02599922

 

More information can be found via ClinicalTrials.gov at www.clinicaltrials.gov/ct2/show/NCT02599922 and from AGTC at www.agtc.com/patients-and-caregivers.

 

There is currently no treatment for achromatopsia, only tools that can reduce symptoms of light sensitivity.

 

AGTC has demonstrated proof-of-concept in naturally occurring dog model of CNGB3-associated achromatopsia disease. In affected animals, delivery of an AAV vector carrying a normal copy of the defective gene restores vision as measured by the ability to navigate a maze.

 

You can find a video about how gene therapy works at this link:

https://www.youtube.com/watch?v=xOQFJJOBGM0

 

This is a non-randomized, open-label, Phase 1/2 study of the safety and efficacy of rAAV2tYF-PR1.7-hCNGB3 administered to one eye by subretinal injection in individuals with achromatopsia caused by mutations in the CNGB3 gene. The primary study endpoint will be safety and the secondary study endpoint will be efficacy.

 

 

Investigators

 

Principal Investigator:Gerald A Fishman, MDPangere Center for Inherited Retinal Diseases

Principal Investigator:Christine Kay, MDVitreoretinal Associates of Gainesville

Principal Investigator:Byron L Lam, MDBascom Palmer Eye Institute

Principal Investigator:Mark A Pennesi, MD, PhDCasey Eye Institute

Principal Investigator:Joseph J Carroll, PhDMedical College of Wisconsin

 

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2) Phase 1/2 Safety and Efficacy Trial of AAV Gene Therapy in Patients with CNGA3 Achromatopsia

 

Sponsored by AGTC

ClinicalTrials.gov identifier: NCT02935517

 

More information can be found via ClinicalTrials.gov at https://www.clinicaltrials.gov/ct2/show/NCT02935517?term=cnga3&rank=2

 

AGTC has demonstrated proof-of-concept in a naturally occurring sheep model of CNGA3-associated achromatopsia.

 

This is a non-randomized, open-label, Phase 1/2 study of the safety and efficacy of rAAV2tYF-PR1.7-hCNGA3 administered to one eye by subretinal injection in individuals with achromatopsia caused by mutations in the CNGB3 gene. The primary study endpoint will be safety and the secondary study endpoint will be efficacy.

 

Locations

 

United States, Florida

VitreoRetinal AssociatesNot yet recruiting

Gainesville, Florida, United States, 32607

Bascom Palmer Eye InstituteNot yet recruiting

Miami, Florida, United States, 33136

United States, Massachusetts

Massachusetts Eye and Ear InfirmaryNot yet recruiting

Boston, Massachusetts, United States, 02114

United States, Oregon

Casey Eye Institute, Oregon Health and Sciences UniversityNot yet recruiting

Portland, Oregon, United States, 97239

United States, Pennsylvania

University of PennsylvaniaNot yet recruiting

Philadelphia, Pennsylvania, United States, 19104

Israel

Hadassah-Hebrew University Medical CenterNot yet recruiting

Jerusalem, Israel, 91120

 

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3) Phase 2 Tolerability and Effects of ALK-001 on Stargardt Disease.

 

Sponsored by Alkeus

ClinicalTrials.gov identifier: NCT02402660

 

https://www.clinicaltrials.gov/ct2/show/NCT02402660?term=alkeus+stargardt&rank=1

 

http://www.alkeuspharma.com/stargardt.html

 

 

Summary: The purpose of this study is to determine the long term safety and tolerability of ALK-001 (C20-D3-retinyl acetate), and to explore the effects of ALK-001 on the progression of Stargardt disease in patients between the ages of 12 and 60 years old.

 

Locations

 

United States, California

University of California Los Angeles - Jules Stein Eye Institute

Los Angeles, California, United States, 90095

Principal Investigator: Michael Gorin, MD, PhD          

Sub-Investigator: Steven Nusinowitz, PhD          

United States, Florida

Vitreoretinal Associates

Gainesville, Florida, United States, 32607

Principal Investigator: Christine Kay, MD          

Sub-Investigator: Jing Zhang, MD          

University of Miami - Bascom Palmer Eye Institute

Miami, Florida, United States, 33136

Principal Investigator: Byron Lam, MD          

United States, Maryland

Johns Hopkins - Wilmer Eye Institute

Baltimore, Maryland, United States, 21287

Principal Investigator: Hendrik Scholl, MD          

Sub-Investigator: Mahmood Shah, MD          

United States, New York

Columbia University Medical Center - Harkness Eye Institute

New York, New York, United States, 10032

Principal Investigator: Stephen Tsang, MD, PhD          

Sub-Investigator: Srilaxmi Bearelly, MD          

United States, Utah

University of Utah - Moran Eye Institute

Salt Lake City, Utah, United States, 84132

Principal Investigator: Paul Bernstein, MD, PhD          

United States, Wisconsin

Medical College of Wisconsin - Eye Institute

Milwaukee, Wisconsin, United States, 53226

Principal Investigator: Kimberly Stepien, MD    

 

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4) “Rate of Progression in USH2A Related Retinal Degeneration” (RUSH2A)

 

Sponsored by Foundation Fighting Blindness Clinical Research Institute (FFB CRI) Consortium

Not yet enrolling

 

Summary: This is a natural history study evaluating patients with Usher syndrome caused by mutations in the Ush 2A gene. This is a multi-center national clinical trial.

 

 

Trust your eyes to our experienced eye care professionals - in practice since 1981.

352-371-2800

At Vitreoretinal Associates, we are actively involved in multiple clinical trials involving inherited retinal disease (Stargardt disease, Usher syndrome, Achromatopsia) and soon to be enrolling for a trial involving RPE65-associated Leber congenital amaurosis or Retinitis Pigmentosa.

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