Clinical trial NCT05802329: Phase I Study to Assess the Safety and Efficacy of OCU200 for Center-Involved Diabetic Macular Edema (DME)
Official title: A Phase 1 Study To Assess The Safety And Efficacy Of OCU200 For Center-Involved Diabetic Macular Edema
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE1
Conditions: Center Involved Diabetic Macular Edema
Diabetic Macular Edema
Keywords: anti-VEGF
Transferrin
Tumstatin
DME
Lead sponsor: Ocugen
Sponsor class: INDUSTRY
First posted: 2023-04-06
Enrollment: 28
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: yes
Allocation: NON_RANDOMIZED
Intervention model: SEQUENTIAL
Intervention model description: 3+3 design with parallel and sequential dosing.
Primary purpose: TREATMENT

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.

Eligibility criteria: 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 with documented incomplete resolution.

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

Primary outcomes:
measure: Study Drug-related adverse events (SDAE)
timeFrame: 20 weeks
measure: treatment-emergent adverse events (TEAEs)
timeFrame: 20 weeks
measure: serious adverse events (SAEs)
timeFrame: 20 weeks
Secondary outcomes:
measure: Best-corrected visual acuity (BCVA)
timeFrame: 20 Weeks (Changes from baseline)
measure: Intraocular pressure (IOP)
timeFrame: 20 weeks(Changes from baseline)
measure: Slit-lamp biomicroscopy
timeFrame: 20 Weeks(Changes from baseline)
measure: Indirect ophthalmoscopy
timeFrame: 20 Weeks (Changes from baseline)
measure: Color fundus photography
timeFrame: 20 Weeks(Changes from baseline)
measure: Spectral Domain Optical Coherence Tomography (SD-OCT)
timeFrame: 20 Weeks(Changes from baseline)
measure: Spectral Domain Optical Coherence Tomography Angiography (SD-OCTA)
timeFrame: 20 weeks (Changes from baseline)
measure: Wide-field Fluorescein Angiography (wf-FA)
timeFrame: 20 weeks (Changes from baseline)
measure: Anti-OCU200 antibody formation
timeFrame: 20 weeks
measure: OCU200 Pharmacokinetics parameters
timeFrame: 20 weeks
Other outcomes:
measure: Improvement of diabetic retinopathy severity scale (DRSS)
timeFrame: 20 weeks
measure: Change from baseline in BCVA letters.
timeFrame: 20 weeks
measure: Changes in CST.
timeFrame: 20 weeks
measure: Best Corrected Visual Acuity
timeFrame: 20 Weeks
measure: Reduction of CST
timeFrame: 20 Weeks
measure: mean increase ≥ 5 BCVA letters
timeFrame: 20 Weeks
measure: Requirement of rescue intervention
timeFrame: 20 weeks
Locations:
city: Deerfield Beach
country: United States
facility: Advanced Research LLC
state: Florida
city: Erie
country: United States
facility: Erie Retina Research, LLC
state: Pennsylvania
city: Bellaire
country: United States
facility: Retina Consultants of Texas Research Centers
state: Texas
city: Southlake
country: United States
facility: Retina Consultants of America
state: Texas
city: La Crosse
country: United States
facility: Gundersen Health System
state: Wisconsin
Study officials:
affiliation: Ocugen
name: Huma Qamar, MD, MPH, CMI
role: STUDY_DIRECTOR
References: