Clinical Trials
The physicians at Vitreo Retinal Associates are actively involved in multiple clinical trials involving inherited retinal disease (Stargardt disease, Usher syndrome, Achromatopsia) and are soon to be enrolling for a trial involving RPE65-associated Leber congenital amaurosis or Retinitis Pigmentosa.
Dr. Christine Kay on Gene Therapy and Stem Cell Therapy:
Current Clinical Trials:
The doctors at Vitreo Retinal 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.
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, MD Pangere Center for Inherited Retinal Diseases
Principal Investigator: Christine Kay, MD Vitreoretinal Associates of Gainesville
Principal Investigator: Byron L Lam, MD Bascom Palmer Eye Institute
Principal Investigator: Mark A Pennesi, MD, PhD Casey Eye Institute
Principal Investigator: Joseph J Carroll, PhD Medical College of Wisconsin
United States, Florida
Gainesville, Florida, United States, 32607
Miami, Florida, United States, 33136
Massachusetts Eye and Ear Infirmary Not yet recruiting
Casey Eye Institute, Oregon Health and Sciences University Not yet recruiting
Portland, Oregon, United States, 97239
University of Pennsylvania Not yet recruiting
Philadelphia, Pennsylvania, United States, 19104
Hadassah-Hebrew University Medical Center Not yet recruiting
Jerusalem, Israel, 9112
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
https://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
4) “Rate of Progression in USH2A Related Retinal Degeneration” (RUSH2A)
Sponsored by Foundation Fighting Blindness Clinical Research Institute (FFB CRI) Consortium
ClinicalTrials.gov Identifier: NCT03146078
This natural history study of patients with USH2A mutations will accelerate the development of outcome measures for clinical trials. Sensitive, objective outcome measures of retinal degeneration will greatly facilitate the development of treatments for Usher syndrome patients. Together these approaches are expected to have an impact on understanding USH2A-related retinal degeneration, developing experimental treatment protocols, and assessing their effectiveness.
5) A Phase 2b Randomized, Double-masked, Controlled Trial to Establish the Safety and Efficacy of Zimura™ (Complement C5 Inhibitor) Compared to Sham in Subjects With Autosomal Recessive Stargardt Disease
Sponsored by Ophthotech
ClinicalTrials.gov Identifier: NCT03364153
This randomized, double-masked, sham-controlled clinical trial will assess the efficacy and safety of Zimura (avacincaptad pegol), a complement C5 inhibitor. It will have an anatomic endpoint, the mean rate change in the area of ellipsoid zone defect as measured by en face spectral domain OCT, as the primary endpoint.
6) Phase 3 Study of ALK-001 in Geographic Atrophy (SAGA)
Sponsored by Alkeus Pharmaceuticals
Trial overview:
This is a double-masked, multicenter, randomized, placebo-controlled clinical trial, evaluating the efficacy and safety of ALK-001 in participants with Geographic Atrophy (GA) secondary to age-related macular degeneration (AMD). Up to 200 participants will receive ALK-001 while up to 100 participants will receive a placebo.
Background:
There is no treatment available for Geographic Atrophy secondary to AMD. AMD is characterized by an age-related degeneration of the retina.
The root cause for this degeneration or why some people develop AMD while others do not, is unknown. Over 20 years ago, it was hypothesized that the dimerization of vitamin A may be a significant contributor to the etiology of AMD. The eye indeed uses vitamin A as a cofactor to sense light, and a striking chemical signature of the aging and degenerating retina is the accumulation of vitamin A dimers in the retinal pigment epithelium (RPE) and the underlying Bruch’s membrane. In rodent models, high levels of vitamin A dimers correlate with poor retinal health, and a variety of mechanisms have been proposed by which vitamin A dimers may induce retinal toxicity. It has been argued that these mechanisms participate in the development and progression of AMD.
ALK-001, the study drug, is a modified form of vitamin A. When taken once a day as a capsule, it replaces natural vitamin A in the body with one that forms vitamin A dimers more slowly. This study will measure the extent to which treatment with ALK-001 slows the progression of Geographic Atrophy.
7) A Clinical Trial of Retinal Gene Therapy for X-linked Retinitis Pigmentosa (XIRIUS)
A Dose Escalation (Phase 1), and Dose Expansion (Phase 2/3) Clinical Trial of Retinal Gene Therapy for X-linked Retinitis Pigmentosa Using an Adeno-Associated Viral Vector (AAV8) Encoding Retinitis Pigmentosa GTPase Regulator (RPGR)
Sponsored by NightStaRx Therapeutics (Biogen)
ClinicalTrials.gov Identifier: NCT03116113
This is a Phase 1/2/3, first-in-human, multi-centre, dose-escalation interventional study of AAV8-RPGR in male subjects with genetically confirmed XLRP. Intervention involves a vitrectomy surgery with subretinal injection of AAV8 expressing RPGR gene. Part I is a dose-selection study; Part II is a dose-expansion study, comparing 2 doses, and a third untreated group to allow for a controlled comparison of efficacy and safety.
8) Phase 1/2 Study to Evaluate the Safety, Tolerability, and Efficacy of QR-1123 in Subjects With Autosomal Dominant Retinitis Pigmentosa (adRP) Due to the P23H Mutation in the RHO Gene
Sponsored by ProQR Therapeutics
ClinicalTrials.gov Identifier: NCT04123626
Background: QR-1123 is an antisense oligonucleotide, designed to specifically target the mutant P23H messenger ribonucleic acid (mRNA) in order to reduce the expression of the P23H protein selectively, while preserving expression of the wild type (WT) rhodopsin (RHO) protein. It is hypothesized that the reduction of mutant P23H mRNA will reduce the deleterious effects of the dominant-negative protein and should result in increased function of WT rhodopsin protein in photoreceptors. Restoration of WT RHO function is expected to improve vision in patients with adRP due to the P23H mutation.
Brief Summary: This study evaluates the safety, tolerability and efficacy of QR-1123 injection in the eye (intravitreal; IVT) injections (one eye/unilateral) in subjects receiving a single dose or repeat doses. Single injections will be assessed in an open label way, and repeat injections will be assessed in a double-masked, randomized, sham-controlled fashion.
9) Rate of Progression in EYS Related Retinal Degeneration (Pro-EYS)
Sponsored by Jaeb Center for Health Research
Collaborator: Foundation Fighting Blindness
ClinicalTrials.gov Identifier: NCT04127006
Brief Summary: The overall goal of this project funded by the Foundation Fighting Blindness is to characterize the natural history of disease progression in patients with EYS mutations in order to accelerate the development of outcome measures for clinical trials. Sensitive, reliable outcome measures of retinal degeneration will greatly facilitate development of treatments for retinitis pigmentosa due to EYS mutations.
10) A Phase 3 Safety and Efficacy Study of Intravitreal Administration of Zimura (Complement C5 Inhibitor) in Geographic Atrophy (GA)
Sponsored by Iveric bio, Inc.
ClinicalTrials.gov Identifier: NCT04435366
Trial Overview:
A Phase 3 Multicenter, Randomized, Double Masked, Sham- Controlled Clinical Trial to Assess the Safety and Efficacy of Intravitreal Administration of Zimura (Complement C5 Inhibitor) in Patients With Geographic Atrophy Secondary to Age-Related Macular Degeneration