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

Diabetic Macular Edema Clinical Trial

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

Status Not yet recruiting

Clinical Trial Summary

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

Clinical Trial 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 following algorithm will be followed for dose-escalation:

Cohort 1 Low Dose (0.5 mg/mL): 3+3 participants will receive intravitreal injection of OCU200 low dose concentration.

1 of 3 results will occur from the first 3 Cohort 3 participants (first dose):

1. If none of the first 3 participants on high dose concentration are determined by the DSMB to have a DLT, then the high dose concentration will be the determined MTD.

2. If 2 or more of the first 3 participants on the high dose concentration are determined by the DSMB to have a DLT, then the medium dose concentration (1 mg/mL) will be the determined MTD. Any dosing on Cohort 4 will be paused and continuation will be re-assessed.

3. If exactly 1 of the first 3 participants on high dose concentration is determined by the DSMB to have a DLT, then 3 additional participants will be enrolled on high dose concentration. Any dosing on Cohort 4 will be paused and continuation will be reassessed.

1. If none of the 3 additional participants are determined by the DSMB to have a DLT, then the high dose concentration will be the determined MTD.

2. If 1 or more of the 3 additional participants are determined by the DSMB to have a DLT, then the medium dose concentration will be the determined MTD. Upon positive DSMB recommendation following dose 1, participants will subsequently receive a 2nd dose (6 weeks later) according to their assigned cohort. The DSMB will review 2 weeks of safety data post 2nd dosing. If a DLT occurs after the 2nd dose of any subject, the dosing interval or dose concentration will be re-assessed.

The following algorithm will be followed for combination therapy cohort:

Cohort 4 MTD + Lucentis: 3 + 3 additional participants will be enrolled to receive OCU200 (High Dose or MTD) + Lucentis (in a sequential manner on dosing dates).

The sentinel participant in Cohort 4 will only be dosed after DSMB review of safety data for the sentinel subject in Cohort 3 or the determination of OCU200 MTD.

If the sentinel participant does not have a DLT as determined by DSMB, two additional participants will be dosed. 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. ;

Related Conditions & MeSH terms

• Diabetic Macular Edema
• Edema
• Macular Edema

• NCT number: NCT05802329
• Study type: Interventional
• Source: Ocugen
• Contact: Shane Spence
• Phone: 484-237-3384
• Email: shane.spence@ocugen.com
• Status: Not yet recruiting
• Phase: Phase 1
• Start date: April 2023
• Completion date: September 2023