Ophthalmology Clinical Trials
The Department of Ophthalmology participates in numerous clinical trials involving novel therapeutic and diagnostic methods for various eye diseases, including:
SCOPE – A Study of Disease Progression in Genetically Defined Patients with Geographic Atrophy Secondary to Age-Related Macular Degeneration
SCOPE [NCT03894020] is an observational study that is evaluating how dry AMD progresses in people with certain genetic profiles. Candidates are screened through a process called genotyping using saliva samples provided by the potential candidates. Genotyping is a process where a person’s DNA is evaluated using biological tests to determine differences in a person’s genetic code that might indicate a predisposition to a disease like dry AMD.
HORIZON – A Phase II, Open-Label, Outcomes-Assessor Masked, Multicenter, Randomized, Controlled Study To Evaluate The Safety And Efficacy Of Two Doses Of Gt005 Administered As A Single Sub Retinal Injection In Subjects With Geographic Atrophy Secondary To Dry Age-Related Macular Degeneration
HORIZON [NCT04566445] is a randomized Phase II clinical trial evaluating the safety and effectiveness of two doses of GT005 administered as a single subretinal injection. HORIZON is enrolling up to 180 people who have GA secondary to AMD. Participants in HORIZON may be assigned to one of two treatment arms or the control arm of the trial and will be followed for 48 weeks.
EXPLORE – A Phase 2, Outcomes Assessor-Masked, Multicenter, Randomized Study To Evaluate The Safety And Efficacy Of Two Doses Of GT005 Administered As A Single Sub Retinal Injection In Subjects With Geographic Atrophy Secondary To Age-Related Macular Degeneration.
EXPLORE [NCT04437368] is a randomized Phase II clinical trial evaluating the safety and effectiveness of two doses of GT005 administered as a single subretinal injection. EXPLORE is enrolling up to 75 people who have GA secondary to AMD who have rare variants in their Complement Factor I (CFI) gene associated with low levels of the CFI protein in their blood. Participants in EXPLORE may be assigned to one of two treatments arm or the control arm of the trial and will be followed for 48 weeks.
EXCEL – An Open-label, Phase 1/2 Trial of Gene Therapy 4D-125 in Males with X-linked Retinitis Pigmentosa (XLRP) Caused by Mutations in the RPGR Gene
XLRP is a rare, X-linked genetic disorder primarily affecting males. Approximately 70% of cases are due to mutations in the gene encoding retinitis pigmentosa GTPase regulator (RPGR). Loss of RPGR function in the retinal cells initially manifests with night blindness in the first decade, followed by loss of visual acuity and blindness in the 3rd – 4th decade. There are currently no approved therapies or treatments for XLRP. Data from the observational phase (Natural History Cohort) of this trial will further characterize and evaluate natural disease progression in this patient population.
Tinlarebant – A Phase 3 Randomized, Double-Masked, Placebo Controlled Study to Evaluate the Safety and Efficacy of Tinlarebant in Treatment of Stargardt Disease in Adolescent Subjects
To evaluate the effect of Tinlarebant compared to placebo on lesion size growth in the study eye from Baseline to Month 24 in adolesccent subjects with Stargardt Disease caused by documented mutations in the ABCA4 gene.
SOLSTICE – A Long-term Follow-up Study to Evaluate the Safety and Efficacy of Retinal Gene Therapy in Subjects with Choroideremia Previously Treated with Adeno-Associated Viral Vector Encoding Rab Escort Protein-1 (AAV2-REP1) and in Subjects with X-Linked Retinitis Pigmentosa Previously Treated with Adeno-Associated Viral Vector Encoding RPGR(AAV8-RPGR) in an Antecedent Study.
The purpose of this study is to see whether the experimental gene therapy (AAV2-REP1) is safe and effective for people with CHM over a long-term period of 5 years post treatment. This study also includes participants diagnosed with X-linked retinitis pigmentosa (XLRP) that have been treated in the past with an experimental gene therapy called Adeno-Associated Viral Vector (AAV8) Encoding Retinitis Pigmentosa GTPase Regulator RPGR (AAV8-RPGR).
MAPS – Structural and Functional Progression of Glaucomatous Damage to the Macula.
The overall goal of MAPS is to improve the sensitivity and specificity of glaucoma diagnosis and progression detection through the evaluation of structural and functional damage to the macula by developing a longitudinal dataset from healthy controls and patients with early functional glaucomatous damage and testing the hypothesis that monitoring central progression is more accurate and less time-consuming than currently employed methods.
Vitamin B3/Pyruvate – Nutritional Supplements and Performance During Visual Field Testing
The Columbia Ophthalmology Glaucoma Division and Clinical Trials Unit is conducting a clinical research study to test if nutritional supplements are a safe and effective therapy for preventing worsening of visual field loss related to glaucoma and for restoring visual function in glaucoma.
The supplements, nicotinamide (vitamin B3) and pyruvate, have been used for conditions such as dementia, diabetes, and high blood pressure. In a pilot study (De Moraes CG et al. JAMA Ophthalmology. 2022 Jan 1;140(1):11-18, https://pubmed.ncbi.nlm.nih.gov/34792559/) at Columbia Ophthalmology, patients on these supplements experienced visual field improvement as compared to those on placebo.
Participants in this randomized clinical trial will receive either a combination of nicotinamide and pyruvate or placebo for a period of 20 months. This study consists of in-person visits to theNYC offices (880 3rd Avenue or 635 West 165th Street) and periodic phone calls for the purposes of the study and for usual glaucoma care. Participants will receive the study supplements and study-related tests and exams at no cost.
You may be eligible for the study if you meet the following criteria:
- Are between 35-85 years of age
- Are diagnosed with open-angle glaucoma
- Are willing to take that study supplements for a period of 20 months
Pigmentary Glaucoma – Determining Genetic Causality of Pigmentary Glaucoma
Pigmentary glaucoma, a type of open angle glaucoma, typically affects young, near-sighted individuals. Although current research suggests that pigmentary glaucoma runs in families, little is known about genetic basis of the condition. This study seeks to better understand how genetic variation contributes to an individual’s chance to develop pigmentary glaucoma and the likelihood of disease progression after diagnosis. Expanding our understanding of the genetic basis of pigmentary glaucoma may allow for more refined methods of diagnosis as well as targeted treatment options for this condition.
Basic Human Research Study Of Novel Glaucoma Endpoints And Identification Of Optimal Patient Populations For Neuroprotection Trials.
To obtain longitudinal structural and functional measurements in glaucoma patients to identify novel and optimal endpoints for measuring disease progression in neuroprotection. To identify glaucoma patient sub-populations who may be at risk for rapid disease progression, and to identify risk factors for rapid disease progression.
PEDIATRIC CATARACT – A Phase 3/4, Prospective, Randomized, Active Treatment-Controlled, Parallel-Design, Multicenter Study to Evaluate the Safety of DEXYCU for the Treatment of Inflammation Following Ocular Surgery for Childhood Cataract.
This is a prospective, randomized, active treatment-controlled, parallel-design multicenter trial in subjects 0 to 3 years of age who are undergoing ocular surgery for childhood cataract. The primary objective is to evaluate the safety of dexamethasone injection (DEXYCU) in the treatment of inflammation following ocular surgery for for childhood cataract.
ASCENT- A Randomized, Partially Masked, Controlled, Phase 3 Clinical Study toEvaluate the Efficacy and Safety of RGX-314 Gene Therapy in Participants with nAMD
This is a phase 3, multicenter, partially masked, randomized, active-controlled, parallel arm study in participants with nAMD to investigate the efficacy and safety of two doses of RGX-314 administered as a single subretinal injection in the study eye. Participants in the control arm will receive aflibercept administered intravitreally in the study eye over 54 weeks.