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. 

PI: Tongalp Tezel, MD 
Contact: Maribel Rodriguez, MD eyetrials@cuimc.columbia.edu
Sponsor: Gyroscope Therapeutics
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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. 

PI: Tongalp Tezel, MD 
Contact: Maribel Rodriguez, MD eyetrials@cuimc.columbia.edu 
Sponsor: Gyroscope Therapeutics 
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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. 

PI: Tongalp Tezel, MD
Contact: Maribel Rodriguez, MD eyetrials@cuimc.columbia.edu  
Sponsor: Gyroscope Therapeutics
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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. 

PI: Irene Maumenee, MD 
Contact:  Marzhan Atakulova, MD eyetrials@cuimc.columbia.edu  
Sponsor: 4D Therapeutics 
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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. 

PI: Tarun Sharma, MD
Contact: Edylin Bautista, MD eyetrials@cuimc.columbia.edu  
Sponsor: Belite Bio, Inc.
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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). 

PI: Jason Horowitz, MD   
Contact: Maribel Rodriguez, MD eyetrials@cuimc.columbia.edu 
Sponsor: Biogen, Inc.
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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. 

PI: Jeffrey Liebmann, MD  
Contact: Lam Lu or Giannis Michalopoulos eyetrials@cuimc.columbia.edu 
Sponsor: Brown Family Fund
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Vitamin B3/Pyruvate – Nutritional Supplements and Performance During Visual Field Testing 

The purpose of this clinical trial is to investigate the efficacy of over-the-counter nutritional supplements (vitamin B3 and pyruvate) compared to placebo can help people who have glaucoma perform better during visual field testing. The visual field test is a type of examination that is done to see if your peripheral vision (or “side vision”) is normal. It is used by your doctor to diagnose, determine the severity of, and monitor your glaucoma. The visual field test uses a light spot that is repeatedly presented in different areas of your peripheral vision. 

PI: Aakriti Shukla, MD
Contact: Giannis Michalopoulos or Marzhan Atakulova, MD or Lam Lu eyetrials@cuimc.columbia.edu 
Sponsor: Brown Family Fund
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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. 

PI: Rando Allikmets, PhD 
Contact: Marzhan Atakulova, MD  eyetrials@cuimc.columbia.edu 
Sponsor: Brown Family Fund
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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. 

PI: Jeffrey Liebmann, MD
Contact: Lam Lu or Ioannis Michalopoulos eyetrials@cuimc.columbia.edu 
Sponsor: Novartis International AG 

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.

PI: Steven Rosenberg, MD
Contact: eyetrials@cuimc.columbia.edu 
Sponsor: EyePoint Pharmaceuticals, Inc.
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THEIA-A: A Phase 3 Global, Randomized, Double-masked,Placebo-controlled, 3-arm Study to Evaluate the Efficacy, ClinicalOutcomes, and Safety of Vutrisiran in Adult and AdolescentPatients with Stargardt Disease Type 1 (STGD1).

This is a randomized, double-masked global Phase 3 study designed to evaluate the efficacy, safety of vutrisiran in adolescent (aged ≥12 and <18 years at the time of
consent/assent) and adult (≥18 years at the time of consent) patients with macular atrophy secondary to Stargardt Disease.

PI: Aliaa Abdelhakim, PhD, MD
Contact: eyetrials@cuimc.columbia.edu 
Sponsor: Alnylam Pharmaceuticals, Inc.
 

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. 

PI: Tongalp Tezel, MD
Contact: Maribel Rodriguez, MD eyetrials@cuimc.columbia.edu  
Sponsor: REGENXBIO Inc.
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