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Active, not recruiting Phase 2Phase 3 Interventional

A Phase 2/3 Trial to Assess the Efficacy and Safety of OCU410ST for Stargardt Disease

ClinicalTrials.gov ID: NCT05956626

Public ClinicalTrials.gov record NCT05956626. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.

ClinicalTrials.gov public records Last synced Sep 6, 2026, 7:51 AM EDT

Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.

Official title

A PHASE 1 STUDY TO ASSESS THE SAFETY AND EFFICACY OF OCU410ST FOR STARGARDT DISEASE AND PHASE 2/3 PIVOTAL CONFIRMATORY CLINICAL TRIAL TO ASSESS THE SAFETY AND EFFICACY OF OCU410ST FOR STARGARDT DISEASE

Brief summary

Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.

Phase 2/3 Pivotal Confirmatory Clinical Trial is a randomized, outcome assessor-masked, multicenter study, that will enroll fifty-one (51) subjects. Subjects will be enrolled in a 2:1 ratio to either the treatment group (n=34 subjects) or to an untreated control group (n=17 subjects). Phase 1 is complete and closed for enrollment. It was a multicenter, open-label, dose ranging/dose escalation study that enrolled 9 subjects. OCU410ST Phase 1- Retinal Structure and Visual Function Data Results * Safety: Favorable safety and tolerability profile No SAE deemed related to OCU410ST including intraocular inflammation, exudation, endophthalmitis, anterior ischemic optic neuropathy (AIONs) or vasculitis. * Primary Endpoint: Structural Improvement Atrophic lesions grew slower by 48% at 12M in evaluable treated eyes when compared to untreated eyes * Secondary Endpoint: Visual Function (BCVA) 100% evaluable treated eyes demonstrated stabilization or improvement when compared to untreated eyes in visual function OCU410ST Phase 1- Structural and Functional Outcomes at 12M Data Results * The GARDian3 clinical trial for ABCA4- related retinopathies including Stargardt disease builds upon encouraging results and positive data from the Phase 1 GARDian trial, which demonstrated 48% slower lesion growth at 12-month follow-up in evaluable treated eyes compared to untreated eyes * Additionally, evaluable treated eyes showed a statistically significant (p=0.031) and clinically meaningful improvement of nearly 2-line/9-letter gain in best corrected visual acuity (BCVA) at 12-month follow-up when compared to untreated eyes.

Study identification

NCT ID
NCT05956626
Recruitment status
Active, not recruiting
Study type
Interventional
Phase
Phase 2, Phase 3
Lead sponsor
Ocugen
Industry
Enrollment
51 participants

Conditions and interventions

Interventions

Drug

Eligibility (public fields only)

Age range
5 Years and older
Sex
All
Healthy volunteers
Healthy volunteers not accepted

This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.

Study timeline

Start date
Aug 24, 2023
Primary completion
Sep 27, 2026
Completion
Sep 27, 2026
Last update posted
Jul 30, 2026

2023 – 2026

United States locations

U.S. sites
14
U.S. states
10
U.S. cities
14
Facility City State ZIP Site status
Associated Retina Consultants Phoenix Arizona 85020
Retinal Consultants Medical Group Sacramento California 95825
Vitreo Retinal Associates, P.A. Gainesville Florida 32607
Bascom Palmer Eye Institute Miami Florida 33136
Advanced Research, LLC Pompano Beach Florida 33064
Retina Partners Midwest, P.C. Carmel Indiana 46032
Boston Children's Hospital Boston Massachusetts 02115
Mississippi Retina Associates Jackson Mississippi 39202
The Retina Institute St Louis Missouri 63128
Duke Eye Center Durham North Carolina 27710
Erie Retina Research, LLC Erie Pennsylvania 16505
Retina Consultants of Texas Bellaire Texas 77401
Retina Foundation of the Southwest Dallas Texas 75231
Valley Retina Institute McAllen Texas 78503

Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.

About this trial record page

What this page shows
Public field values for ClinicalTrials.gov record NCT05956626, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
What this page does not do
No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
Where the data comes from
Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
Last refresh
Last update posted Jul 30, 2026 · Synced Sep 6, 2026

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Open the official record

The complete protocol, eligibility criteria, and contact information for NCT05956626 live on ClinicalTrials.gov.

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