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 Low Dose; Drug: OCU200 Medium Dose; Drug: OCU200 High Dose

• 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 3 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.

A total of 9 to 22 participants will be enrolled in the dose-escalation portion of the study to receive OCU200 alone (9 to 18 participants enrolled according to the 3+3 design and up to 4 participants replaced due to parallel cohort initiation). For the combination therapy cohort, a total of 3 to 6 participants will be enrolled to receive OCU200 (MTD) + Lucentis.

A total of up to 28 participants will be included in this study.

The DSMB will continue to review all available safety data two weeks post-dosing of participants.

Cohort 2 Medium Dose (1 mg/mL): 3+3 participants will receive intravitreal injection of OCU200 medium dose concentration. Following the same algorithm as Cohort 1.

Cohort 3 High Dose (2 mg/mL): 3+3 participants will receive intravitreal injection of OCU200 high dose concentration. Following the same algorithm as Cohort 2.

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: 2025-03
• Last Update Submitted: 2025-03-17
• Last Update Posted: 2025-03-20
• Primary Completion: 2025-09-30
• Study Completion: 2025-10-31

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 28

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 anti-VEGF intravitreal injection

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 (Low Dose)	OCU200 Low Dose	3+3 participants will receive intravitreal injection of OCU200 low dose concentration.
Cohort 2 (Medium Dose)	OCU200 Medium Dose	3+3 participants will receive intravitreal injection of OCU200 medium dose concentration.
Cohort 3 (High Dose)	OCU200 High Dose	3+3 participants will receive intravitreal injection of OCU200 high dose concentration.
Cohort 4 (High Dose/MTD + Lucentis)	OCU200 High Dose	3 + 3 additional participants will be enrolled to receive OCU200 (High Dose or MTD) + Lucentis (in a sequential manner on dosing dates).

Primary Outcome Measures

Name	Time	Method
Study Drug-related adverse events (SDAE)	20 weeks	Counts, frequencies and percentages of SDAEs.
treatment-emergent adverse events (TEAEs)	20 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)	20 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)	20 Weeks (Changes from baseline)	Measured as the ETDRS letter score on the EVA tester or E-ETDRS charts.
Intraocular pressure (IOP)	20 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).
Slit-lamp biomicroscopy	20 Weeks(Changes from baseline)	Changes in visual function.
Indirect ophthalmoscopy	20 Weeks (Changes from baseline)	If visual acuity is so poor that the participant is unable to count fingers or perceive hand motion, light perception will be tested with the indirect ophthalmoscope as the light source.
Color fundus photography	20 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)	20 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)	20 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)	20 weeks (Changes from baseline)	wf-FA will be conducted at screening and EOS visits to assess central and peripheral vasculature.
Anti-OCU200 antibody formation	20 weeks	Blood samples will be collected for the assessment.
OCU200 Pharmacokinetics parameters	20 weeks	Blood samples will be collected for the assessment.

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