Ophthalmology Clinical Trials

The Department of Ophthalmology participates in numerous clinical trials involving novel therapeutic and diagnostic methods for various eye diseases, including:






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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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

PI: Aakriti Shukla, MD
Contact: Ioannis Michalopoulos or Marzhan Atakulova, MD or Lam Lu eyetrials@cuimc.columbia.edu 
Sponsor: Brown Family Fund, The Glaucoma Foundation
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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: Edylin Bautista  eyetrials@cuimc.columbia.edu
Sponsor: EyePoint Pharmaceuticals, Inc.
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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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