Ophthalmology Clinical Trials

This is a prospective, observational study designed to evaluate the long-term safety and efficacy of ABBV-RGX-314. Eligible participants are those who were previously enrolled in one of the parent clinical studies (eg, RGX-314-001, RGX-314-2103, RGX-314-2104 [also known as M23-416] and RGX-314-3101 [also known as M23-409]) for the treatment of nAMD in which they received a single subretinal administration of ABBV-RGX-314 in their study eye.

PI: Tongalp Tezel, MD
Contact: Maribel Rodriguez, MD mr3208@cumc.columbia.edu  
Sponsor: ABBVIE, Inc.

ClinicalTrials.gov: NCT06088979

IRB status: Approved
Enrollment: Closed

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 the NYC offices (880 3rd Avenue or 622 West 168th 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.

PI: Aakriti Shukla, MD
Contact: Ioannis Michalopoulos  im2482@cumc.columbia.edu 
Sponsor: Brown Family Fund, The Glaucoma Foundation

ClinicalTrials.gov: NCT05695027

IRB status: Approved
Enrollment: Closed

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This is a prospective, multi-centre, long-term, follow-up study for participants who received GT005 in an antecedent clinical study (GT005-02 or GT005-03).The purpose of this study is to evaluate the long-term safety of GT005, the long-term durability of GT005 on geographic atrophy progression, and the long-term durability of GT005 on functional measures.

PI: Tongalp Tezel, MD
Contact: Maribel Rodriguez, MD   mr3208@cumc.columbia.edu 
Sponsor: Novartis Pharmaceuticals Corporation

ClinicalTrials.gov: NCT05481827

IRB status: Approved
Enrollment: Closed

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  ma3448@cumc.columbia.edu 
Sponsor: Brown Family Fund

IRB status: Approved
Enrollment: Open

Retinoblastoma is the most common pediatric malignant intraocular tumor and originates from the retina. Treatment of eyes with advanced intraocular retinoblastoma remains a challenge. The historic standard of care for patients with unilateral disease is enucleation and for those with bilateral disease, a variety of modalities have been tried. These include radiation therapy, systemic chemotherapy, periocular administration of chemotherapy, selective intra-arterial chemotherapy, and intravitreal chemotherapy. Unfortunately, all of these modalities are associated with significant morbidity and investigators are looking for new ways to treat these patients either with novel directed drug delivery methods or with new less toxic agents. This study will evaluate the safety and efficacy of topotecan delivered directly to the eye using a novel sustained-release topotecan episcleral delivery system (also referred to as a Chemoplaque) in patients with active de novo or recurrent intraocular retinoblastoma in at least one eye following completion of first-line therapy. The study intervention involves the insertion and removal of the Chemoplaque, examinations under anaesthesia (EUAs), visits to clinic to monitor for adverse events throughout, and post plaque removal toxicity evaluation. EUAs, clinic visits and laboratory tests are standard of care for retinoblastoma patients. 

PI: Brian Marr, MD
Contact: Patricia Lemberg, MD email: pl2853@cumc.columbia.edu
Sponsor: Targeted Therapy Technologies, LLC.

ClinicalTrials.gov: NCT04156347

IRB status: Approved
Enrollment: Open

The study is a prospective observational study of patients with STGD1, the aim of which is to further enhance understanding of disease progression and structural and functional markers that can be used to evaluate the efficacy and safety of therapeutic interventions, especially in light of advancements in imaging technology.

PI: Vlad Diaconita, MD
Contact: Maribel Rodriguez, MD   mr3208@cumc.columbia.edu 
Sponsor: SpliceBio, SL.

ClinicalTrials.gov: NCT06435000

IRB status: Approved
Enrollment: Open

First-in-human trials are conducted primarily to determine the safe dose range for further clinical development of promising drug candidates. This first-in-human study is being conducted to identify doses of SB-007 that are safe and demonstrate a potential efficacy signal in subjects with moderate or advanced STGD1.

PI: Vlad Diaconita, MD
Contact: Maribel Rodriguez, MD   mr3208@cumc.columbia.edu 
Sponsor: SpliceBio, SL.

ClinicalTrials.gov: NCT06942572

IRB status: Approved
Enrollment: Open

AccordionThis prospective observational, low-interventional, non-drug study aims to further characterize anatomical and functional biomarkers in DR. The study will enable observation of routine treatments or assessments for DR. Some of these assessments are performed as part of the standard of care. There will be additional assessments outside of routine and therefore the study is classified as low-interventional.

PI: Vlad Diaconita, MD
Contact: Maribel Rodriguez, MD   mr3208@cumc.columbia.edu 
Sponsor: Roche, Inc.

IRB status: Approved
Enrollment: Open

This study is evaluating an investigational medication called tinlarebant, which is being developed to help slow vision loss in people with Stargardt disease, a rare inherited eye condition that can lead to progressive vision impairment. Tinlarebant works by reducing the buildup of harmful vitamin A byproducts in the retina that may contribute to damage of the light-sensitive cells in the eye. In previous studies, tinlarebant showed promising results in slowing the progression of retinal damage and was generally well tolerated. Researchers are continuing to study its safety and effectiveness to determine whether it may help preserve vision in people living with Stargardt disease.

PI: Aliaa Hamed Abdelhakim, MD, PhD
Contact: Edylin Bautista, MD   mmb2225@cumc.columbia.edu 
Sponsor: Belite Bio, Inc.

ClinicalTrials.gov: NCT05244304

IRB status: Approved
Enrollment: Open

This study is evaluating an investigational gene therapy called 4D-150 for people with wet age-related macular degeneration (wet AMD), a condition that can cause vision loss due to abnormal blood vessel growth and leakage in the retina. Current treatments often require frequent eye injections to help preserve vision. 4D-150 is designed to provide long-lasting treatment with a single injection by enabling the eye to produce proteins that block the signals responsible for abnormal blood vessel growth. Researchers are studying whether one dose of 4D-150 can maintain vision and reduce the need for ongoing injections while remaining safe and well tolerated.

PI: Tongalp Tezel, MD
Contact: Edylin Bautista, MD   mmb2225@cumc.columbia.edu 
Sponsor: 4D Molecular Therapeutics

ClinicalTrials.gov: NCT07064759

IRB status: Approved
Enrollment: Open

This Phase 1 study is evaluating the safety and tolerability of ER-100, an investigational gene therapy for people with open-angle glaucoma (OAG) or non-arteritic anterior ischemic optic neuropathy (NAION). ER-100 is designed to help protect optic nerve cells and preserve vision. Researchers hope this study will provide important information about the potential of ER-100 as a future treatment for optic nerve diseases.

PI: Aakriti Garg Shukla, MD, MSc
Contact: Ioannis Michalopoulos     im2482@cumc.columbia.edu
Sponsor: Life Biosciences, Inc.

ClinicalTrials.gov: NCT07290244

IRB status: Approved
Enrollment: Open

This study is evaluating FWY003, an investigational oral treatment for geographic atrophy (GA) caused by age-related macular degeneration (AMD). Researchers are studying whether FWY003 can slow the progression of GA, help preserve vision, and provide a convenient alternative to currently available treatments. The study will also assess the safety and tolerability of FWY003 in adults with GA.

 PI: Tongalp Tezel, MD
Contact: Sokleab Em     se2389@cumc.columbia.edu
Sponsor: Novartis

ClinicalTrials.gov: NCT07441642

IRB status: Approved
Enrollment: Open

This study is evaluating an investigational gene therapy for people with BEST1-related retinal disease, a rare inherited eye condition that can cause progressive vision loss. Researchers are studying whether this treatment can safely deliver a healthy copy of the BEST1 gene to retinal cells and help improve or preserve vision. Participants will undergo regular eye examinations and specialized imaging tests to monitor retinal health and assess the safety and potential effectiveness of the therapy over time.

PI: Jason D Horowitz, MD
Contact: Maribel Rodriguez, MD      mr3208@cumc.columbia.edu  
Sponsor: Opus Genetics

IRB status: Approved
Enrollment: Open

The LIGHTHOUSE study is evaluating ATSN-201, an investigational gene therapy for X-linked retinoschisis (XLRS), a rare inherited retinal disease that can cause vision loss. The goal of the study is to assess the safety of ATSN-201 and determine whether it can help preserve or improve vision by addressing the genetic cause of the condition.

PI: Aliaa Hamed Abdelhakim, MD, PhD
Contact: Maribel Rodriguez, MD      mr3208@cumc.columbia.edu  
Sponsor: Atsena Therapeutics, Inc

ClinicalTrials.gov: NCT05878860

IRB status: Pending approval
Enrollment: Will open after approval

The NORTHSTAR Study is a Phase 3 clinical trial evaluating ALK-001 (gildeuretinol acetate) for people with Stargardt disease, an inherited retinal disease that can lead to vision loss. The goal of the study is to determine whether ALK-001 can safely slow disease progression and help preserve vision.

PI: Stanley Chang, MD
Contact: Maribel Rodriguez, MD      mr3208@cumc.columbia.edu  
Sponsor: Alkeus Pharmaceuticals

IRB status: Pending approval
Enrollment: Will open after approval

The NAAVIGATE Study is a Phase 2b/3 clinical trial evaluating surabgene lomparvovec (ABBV-RGX-314), an investigational gene therapy for people with diabetic retinopathy. The goal of the study is to determine whether a single treatment can safely improve diabetic retinopathy and reduce the risk of vision-threatening complications over time.

PI: Tongalp Tezel, MD
Contact: Maribel Rodriguez, MD      mr3208@cumc.columbia.edu  
Sponsor: Abbvie

IRB status: Pending approval
Enrollment: Will open after approval