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

Phase 1

Recruiting

• Conditions: Center Involved Diabetic Macular Edema; Diabetic Macular Edema
• Interventions: Drug: OCU200

• Registration Number: NCT05802329
• Lead Sponsor: Ocugen

Brief Summary

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

Detailed Description

This is a multicenter, open-label, dose ranging study with 4 cohorts in the dose-escalation portion of the study and 1 cohort in the combination therapy portion of the study. An accelerated 3+3 design with parallel and sequential dosing will be used.

Under the escalation design, there will be 12 subjects enrolled if there are no DLTs and up to 24 subjects under the condition that exactly 1 of the 3 subjects of every cohort is determined to have a DLT. For the combination therapy cohort, a total of 3 to 6 subjects will be enrolled to receive OCU200 (MTD) + Lucentis. A total of up to 30 subjects will be included in this study.

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 (0.5 mg/mL): 3+3 participants will receive intravitreal injection of OCU200 concentration.

Cohort 2 (1 mg/mL): 3+3 participants will receive intravitreal injection of OCU200 concentration.

Cohort 3 (2 mg/mL): 3+3 participants will receive intravitreal injection of OCU200 concentration.

Cohort 4 (5 mg/mL): 3+3 participants will receive intravitreal injection of OCU200 concentration.

Cohort 5 MTD + Lucentis: 3+3 participants will receive intravitreal injection of OCU200 (MTD) + Lucentis

Study Timeline

• Study First Submitted: 2023-03-27
• Study First Submitted QC: 2023-03-27
• Study First Posted: 2023-04-06
• Study Start: 2024-01-13
• Status Verified: 2025-08
• Last Update Submitted: 2025-08-01
• Last Update Posted: 2025-08-06
• Primary Completion: 2026-07-31
• Study Completion: 2026-07-31

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 30

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 if male or ≥ 305 µm if female on Heidelberg Spectralis
   2. ≥ 305 if male or ≥ 290 if female on Zeiss Cirrus
   3. BCVA ≤ 78 and ≥ 24 letters on ETDRS chart

4. Sufficient ocular media clarity, pupillary dilation and participant cooperation to permit acquisition of good quality retinal imaging

5. No history of prior anti-VEGF injection for treatment of DME or history of at least 2 consecutive intravitreal anti-VEGF injection (less than 7 weeks apart) for the treatment of DME with documented incomplete resolution of central subfield thickening within 1 year prior to the screening visit.

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.

Subjects previously receiving treatment in the fellow eye: The last anti-VEGF injection must be administered at least two weeks prior to Day 1(Treatment) and the treatment can be resumed, per PI if clinically deemed necessary post two weeks from the day of OCU200 injection.

Untreated fellow eye requiring SOC treatment for DME during the study for clinically diagnosed reasons: Treatment may be permitted upon receiving prior approval from the Sponsor.

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
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 proliferative diabetic retinopathy

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SEQUENTIAL

Arm && Interventions

Group	Intervention	Description
Cohort 1	OCU200	3+3 participants will receive intravitreal injection of OCU200 concentration.
Cohort 2	OCU200	3+3 participants will receive intravitreal injection of OCU200 concentration.
Cohort 3	OCU200	3+3 participants will receive intravitreal injection of OCU200 concentration.
Cohort 4	OCU200	3+3 participants will receive intravitreal injection of OCU200 concentration.
Cohort 5	OCU200	MTD + Lucentis: 3+3 participants will receive intravitreal injection of OCU200 (MTD) + Lucentis

Primary Outcome Measures

Name	Time	Method
Study Drug-related adverse events (SDAE)	24 weeks	Counts, frequencies and percentages of SDAEs.
treatment-emergent adverse events (TEAEs)	24 weeks	Counts, frequencies and percentages TEAEs. TEAEs are defined as an event that was not present prior to administration of the dose of study drug and present after the dose, or if it represents the exacerbation of an event that was present prior to the dose.
serious adverse events (SAEs)	24 weeks	Counts, frequencies and percentages of SAEs including Resulted in Death, Life-threatening, Hospitalization, Disabling/incapacitating, Congenital anomaly or birth defect and Medically significant AEs ( AE that did not meet any of the above criteria but could have jeopardized the subject and might have required medical or surgical intervention to prevent one of the outcomes listed above).

Secondary Outcome Measures

Name	Time	Method
Best-corrected visual acuity (BCVA)	24 Weeks (Changes from baseline)	Measured as the ETDRS letter score on the EVA tester or E-ETDRS charts.
Intraocular pressure (IOP)	24 weeks(Changes from baseline)	IOP measurement by applanation or rebound tonometry. Confirmation with Goldmann tonometer if IOP reading is outside the normal range (8-21mmHg).
Color fundus photography	24 Weeks(Changes from baseline)	Color fundus photographs will be taken to evaluate retinal anatomy and grade diabetic retinopathy severity scale (DRSS).
Spectral Domain Optical Coherence Tomography (SD-OCT)	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.
Spectral Domain Optical Coherence Tomography Angiography (SD-OCTA)	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.
Wide-field Fluorescein Angiography (wf-FA)	24 weeks (Changes from baseline)	wf-FA will be conducted at screening and EOS visits to assess central and peripheral vasculature.

Trial Locations

Locations (5)

Advanced Research LLC; 🇺🇸 Deerfield Beach, Florida, United States

Erie Retina Research, LLC; 🇺🇸 Erie, Pennsylvania, United States

Retina Consultants of Texas Research Centers; 🇺🇸 Bellaire, Texas, United States

Retina Consultants of America; 🇺🇸 Southlake, Texas, United States

Gundersen Health System; 🇺🇸 La Crosse, Wisconsin, United States