Phase I Study to Assess the Safety and Efficacy of OCU200 for Center-Involved Diabetic Macular Edema (DME) (DME)

November 25, 2025 updated by: Ocugen

A Phase 1 Study To Assess The Safety And Efficacy Of OCU200 For Center-Involved Diabetic Macular Edema

A Phase 1 study to assess the safety and efficacy of OCU200 for center-involved diabetic macular edema

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Detailed Description

This is a multicenter, open-label, dose ranging study with 4 cohorts in the dose-escalation portion of the study. An accelerated 3+3 design with parallel and sequential dosing will be used.

Under the escalation design, 12 subjects will be enrolled if there are no DLTs and up to 24 subjects under the condition that exactly 1 of the 3 subjects of every cohort if determined to have a DLT.

Each subject will receive a total of 2 intravitreal injections of OCU200 6 weeks apart.

The DSMB will review the sentinel subject 1 week safety data post dosing in every cohort of all 3 subjects.

Cohort 1: 3+3 participants will receive intravitreal injection of OCU200.

Cohort 2: 3+3 participants will receive intravitreal injection of OCU200.

Cohort 3: 3+3 participants will receive intravitreal injection of OCU200.

Cohort 4: 3+3 participants will receive intravitreal injection of OCU200.

Study Type

Interventional

Enrollment (Estimated)

24

Phase

  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact Backup

Study Locations

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

Yes

Description

Inclusion Criteria:

  1. Diagnosis of Type 1 or Type 2 Diabetes Mellitus
  2. Decreased visual acuity attributable primarily to DME

  3. Central-involved DME with central retinal subfield thickness (CST) values, as assessed with spectral-domain optical coherence tomography (SD-OCT) of:

    1. ≥ 320 but ≤ 450µm if male or ≥ 305 but ≤ 435µm if female on Heidelberg Spectralis
    2. ≥ 305 but ≤ 435µm if male or ≥ 290 but ≤ 420µm if female on Zeiss Cirrus
  4. BCVA ≤ 78 and ≥ 24 letters on ETDRS chart
  5. Sufficient ocular media clarity, pupillary dilation and participant cooperation to permit acquisition of good quality retinal imaging
  6. No history of prior anti-VEGF injection or history of at least 2 consecutive intravitreal anti-VEGF injection (less than 7 weeks apart) with incomplete resolution of CST within 1 year.

Note: The last anti-VEGF injection must be administered at least six weeks (45 days) prior to the study treatment (Day 1) in the study eye.

Exclusion Criteria:

  1. Presence of any condition that prevent clear visualization of retina (e.g., significant cataract, vitreous hemorrhage)
  2. Uncontrolled hypertension
  3. Uncontrolled glaucoma
  4. Concurrent disease in the study eye, other than central-involved DME
  5. Intravitreal or periocular steroid treatment within 3 months prior to the screening visit
  6. Any ocular surgery within 3 months prior to the screening visit in the study eye
  7. Uncontrolled/poorly controlled diabetes (Glycated hemoglobin (HbA1c) ≥ 10%)
  8. History of retinal detachment in the study eye or other retinal vascular disease in the study eye
  9. Focal or pan-retinal laser photocoagulation in the study eye within 3 months prior to the screening visit
  10. Presence of any inherited retinal disease or history of proliferative diabetic retinopathy
  11. History of Renal disease including stage 3b or worse

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Non-Randomized
  • Interventional Model: Sequential Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Cohort 1
3+3 participants will receive intravitreal injection of OCU200 (0.5 mg/mL concentration).
Drug: OCU200
Intravitreal Injection
Experimental: Cohort 2
3+3 participants will receive intravitreal injection of OCU200 (1 mg/ML concentration).
Drug: OCU200
Intravitreal Injection
Experimental: Cohort 3
3+3 participants will receive intravitreal injection of OCU200 (2 mg/mL concentration).
Drug: OCU200
Intravitreal Injection
Experimental: Cohort 4
3+3 participants will receive intravitreal injection of OCU200 (5 mg/mL concentration).
Drug: OCU200
Intravitreal Injection

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Study Drug-related adverse events (SDAE)
Time Frame: 24 weeks
Counts, frequencies and percentages of SDAEs.
24 weeks
Treatment-emergent adverse events (TEAEs)
Time Frame: 24 weeks
Counts, frequencies and percentages TEAEs.
24 weeks
Serious adverse events (SAEs)
Time Frame: 24 weeks
Counts, frequencies and percentages of SAEs.
24 weeks

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Best-corrected visual acuity (BCVA)
Time Frame: 24 Weeks (Changes from baseline)
Measured as the ETDRS letter score on the EVA tester or E-ETDRS charts.
24 Weeks (Changes from baseline)
Color fundus photography
Time Frame: 24 Weeks(Changes from baseline)
Color fundus photographs will be taken to evaluate retinal anatomy and grade diabetic retinopathy severity scale (DRSS).
24 Weeks(Changes from baseline)
Spectral Domain Optical Coherence Tomography (SD-OCT)
Time Frame: 24 Weeks(Changes from baseline)
SD-OCT will be utilized to assess retinal thickness. OCT images and scans will be transmitted to a central reading center for independent analysis.
24 Weeks(Changes from baseline)
Spectral Domain Optical Coherence Tomography Angiography (SD-OCTA)
Time Frame: 24 weeks (Changes from baseline)
SD-OCTA will be utilized to assess retinal vasculature and images will be transmitted to a central reader for independent analysis.
24 weeks (Changes from baseline)
Wide-field Fluorescein Angiography (wf-FA)
Time Frame: 24 weeks (Changes from baseline)
wf-FA will be conducted at screening and EOS visits to assess central and peripheral vasculature.
24 weeks (Changes from baseline)
Intraocular pressure (IOP)
Time Frame: 24 weeks(Changes from baseline)
IOP measurement by applanation or rebound tonometry.
24 weeks(Changes from baseline)

Other Outcome Measures

Outcome Measure
Measure Description
Time Frame
Improvement of diabetic retinopathy severity scale (DRSS)
Time Frame: 24 weeks
Proportion of participants with ≥ 2-step improvement of diabetic retinopathy severity scale (DRSS)
24 weeks
Reduction of CST
Time Frame: 24 Weeks
The proportion of participants having a ≥ 10% reduction of CST at Week 13 and Week 19
24 Weeks
Change from baseline in BCVA letters.
Time Frame: 24 weeks
Dose response as assessed by mean change from baseline in BCVA letters and mean number of injections by study visit
24 weeks

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Ocugen

Investigators

  • Study Director: Huma Qamar, MD, MPH, CMI, Ocugen

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

January 13, 2024

Primary Completion (Estimated)

July 31, 2026

Study Completion (Estimated)

July 31, 2026

Study Registration Dates

First Submitted

March 27, 2023

First Submitted That Met QC Criteria

March 27, 2023

First Posted (Actual)

April 6, 2023

Study Record Updates

Last Update Posted (Estimated)

December 3, 2025

Last Update Submitted That Met QC Criteria

November 25, 2025

Last Verified

November 1, 2025

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • OCU200-101

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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