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

NCT05802329 | Recruiting Phase 1 DMC FDA Drug

• 1 other identifier: OCU200-101
• Study Type: interventional
• Target: 24
• Locations: 1 country
• Sites: 5

Brief Summary

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

Conditions

Center Involved Diabetic Macular Edema; Diabetic Macular Edema

Keywords

anti-VEGF; Transferrin; Tumstatin; DME

Outcome Measures

Primary Outcomes (3)

• Study Drug-related adverse events (SDAE)

  Counts, frequencies and percentages of SDAEs.

  24 weeks

• Treatment-emergent adverse events (TEAEs)

  Counts, frequencies and percentages TEAEs.

  24 weeks

• Serious adverse events (SAEs)

  Counts, frequencies and percentages of SAEs.

  24 weeks

Secondary Outcomes (6)

• Best-corrected visual acuity (BCVA)

  24 Weeks (Changes from baseline)

• Intraocular pressure (IOP)

  24 weeks(Changes from baseline)

• Color fundus photography

  24 Weeks(Changes from baseline)

• Spectral Domain Optical Coherence Tomography (SD-OCT)

  24 Weeks(Changes from baseline)

• Spectral Domain Optical Coherence Tomography Angiography (SD-OCTA)

  24 weeks (Changes from baseline)

Other Outcomes (3)

• Improvement of diabetic retinopathy severity scale (DRSS)

  24 weeks

• Change from baseline in BCVA letters.

  24 weeks

• Reduction of CST

  24 Weeks

Study Arms (4)

Cohort 1

EXPERIMENTAL

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

Drug: OCU200

Cohort 2

EXPERIMENTAL

3+3 participants will receive intravitreal injection of OCU200 (1 mg/ML concentration).

Drug: OCU200

Cohort 3

EXPERIMENTAL

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

Drug: OCU200

Cohort 4

EXPERIMENTAL

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

Drug: OCU200

Interventions

OCU200 DRUG

Intravitreal Injection

Cohort 1; Cohort 2; Cohort 3; Cohort 4

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Diagnosis of Type 1 or Type 2 Diabetes Mellitus
• Decreased visual acuity attributable primarily to DME
• Central-involved DME with central retinal subfield thickness (CST) values, as assessed with spectral-domain optical coherence tomography (SD-OCT) of:
• ≥ 320 but ≤ 450µm if male or ≥ 305 but ≤ 435µm if female on Heidelberg Spectralis
• ≥ 305 but ≤ 435µm if male or ≥ 290 but ≤ 420µm if female on Zeiss Cirrus
• BCVA ≤ 78 and ≥ 24 letters on ETDRS chart
• Sufficient ocular media clarity, pupillary dilation and participant cooperation to permit acquisition of good quality retinal imaging
• 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.

You may not qualify if:

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

Sponsors & Collaborators

• Ocugen: lead

Study Sites (5)

Advanced Research LLC

Deerfield Beach, Florida, 33064, United States

RECRUITING

Erie Retina Research, LLC

Erie, Pennsylvania, 16507, United States

RECRUITING

Retina Consultants of Texas Research Centers

Bellaire, Texas, 77401, United States

RECRUITING

Retina Consultants of America

Southlake, Texas, 76092, United States

RECRUITING

Gundersen Health System

La Crosse, Wisconsin, 54601, United States

RECRUITING

Study Officials

• Huma Qamar, MD, MPH, CMI

  Ocugen

  STUDY DIRECTOR

Central Study Contacts

Roshan A George, MD, MPH

CONTACT

845-664-1505; roshan.george@ocugen.com

Oscar Cuzzani, MD, PhD

CONTACT

Oscar.Cuzzani@ocugen.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: 3+3 design with parallel and sequential dosing.

Study Record Dates

• First Submitted: March 27, 2023
• First Posted: April 6, 2023
• Study Start: January 13, 2024
• Primary Completion (Estimated): July 31, 2026
• Study Completion (Estimated): July 31, 2026
• Last Updated: December 3, 2025

Record last verified: 2025-11

Data Sharing

• IPD Sharing: Will not share

Locations

US (5)