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Study to Assess the Safety and Efficacy of OCU410 for Geographic Atrophy (ArMaDa)

Primary Purpose

Geographic Atrophy

Status
Recruiting
Phase
Phase 1
Locations
United States
Study Type
Interventional
Intervention
OCU410
Sponsored by
Ocugen
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Geographic Atrophy

Eligibility Criteria

50 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria: Subjects 50 years of age or older. BCVA of approximately 24 letters or more using Early Treatment Diabetic Retinopathy Study (ETDRS) chart (20/320 Snellen equivalent). Fundus autofluorescence (FAF) imaging shows: Total GA area ≥2.5 and ≤17.5 mm2 (1 and 7 disk areas [DA], respectively) If GA is multifocal, at least one focal lesion must be ≥1.25 mm2 (0.5 DA), with the overall aggregate area of GA The entire GA lesion must be completely visualized on the macula-centered image and must be able to be imaged in its entirety, and not contiguous with any areas of peripapillary atrophy Absence of any pattern of hyper-autofluorescence in the junctional zone of GA Exclusion Criteria: Previous treatment with a gene-therapy or cell therapy product Previous treatment with any investigational drug or device within one year. The history of any investigational product with a washout period of up to six months will be evaluated on a case-by-case basis. Previous treatment with Syfovre (Pegcetacoplan injection) GA due to causes other than AMD such as Stargardt disease, cone rod dystrophy or toxic maculopathies like Plaquenil maculopathy. however, benign conditions of the vitreous or peripheral retina are not exclusionary (i.e., pavingstone degeneration).

Sites / Locations

  • Retina Consultants of TexasRecruiting
  • Retina Foundation of the SouthwestRecruiting

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm 4

Arm 5

Arm 6

Arm Type

Experimental

Experimental

Experimental

Experimental

Experimental

No Intervention

Arm Label

Phase1 Dose Escalation- Low Dose (2.5×10E10 vg/mL):

Phase1 Dose Escalation- Medium Dose (5×10E10 vg/mL):

Phase1 Dose Escalation- High Dose (1.5×10E11 vg/mL):

Phase 2 Dose Expansion: Maximum tolerated dose (MTD) from Phase 1-Randomized Arm

Phase 2 Dose Expansion: Lower Dose from Phase 1-Randomized Arm

Control Arm

Arm Description

Low Dose (2.5×10E10 vg/mL): Subjects will receive a subretinal injection of OCU410 in the low dose concentration.

Medium Dose (5×10E10 vg/mL): Subjects will receive a subretinal injection of OCU410 in the medium dose concentration.

High Dose (1.5×10E11 vg/mL): Subjects will receive a subretinal injection in the high dose concentration.

Maximum tolerated dose (MTD) from Phase 1: Subjects will receive a subretinal injection in the MTD concentration.

Subjects will receive a subretinal injection of OCU410 in a Lower Dose concentration.

No Intervention Control Arm: Subject will not receive any active study intervention

Outcomes

Primary Outcome Measures

Safety (Participants With Ocular and Non-ocular AEs (Adverse Events) and SAEs (Serious Adverse Events))
The primary endpoint is safety, determined by the number of ocular and non-ocular Study Drug-related adverse events (SDAE), treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs).
Change in anatomy of ocular structures using Slit Lamp Biomicroscopy
We will use Slit-lamp Biomicroscopy to visualize the anatomy of ocular structures before and after sub-retinal injections and follow-up visits.
Change in anatomy of ocular structures using Indirect ophthalmoscopy
We will use Indirect ophthalmoscopy to visualize the anatomy of ocular structures before and after sub-retinal injections and follow-up visits.
Change from baseline in BCVA (Best Corrected Visual Acuity)
Visual function of the study eye was assessed using the Early Treatment Diabetic Retinopathy Study (ETDRS) Best Corrected Visual Acuity (BCVA) letter score. A higher score represents better vision.
Change in Low Luminance Visual Acuity
Measured by letter score. A higher score represents better vision
Change in the Intraocular Pressure (mmHg)
Measured by applanation or rebound tonometry with confirmation with Goldmann tonometer if IOP is outside normal range (8-21mmHg).

Secondary Outcome Measures

Humoral and cellular immune response
Blood samples will be collected for the assessment. The secondary safety endpoints include change from baseline in Humoral and cellular immune response in response to OCU410 administration
Shedding of viral vector
Blood samples will be collected for the assessment to determine AAV vector shedding in systemic circulation after OCU410 administration
Laboratory parameters including serum chemistry and hematology
Blood samples will be collected for the assessment to determine a change from baseline after OCU410 administration.

Full Information

First Posted
June 30, 2023
Last Updated
October 24, 2023
Sponsor
Ocugen
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1. Study Identification

Unique Protocol Identification Number
NCT06018558
Brief Title
Study to Assess the Safety and Efficacy of OCU410 for Geographic Atrophy
Acronym
ArMaDa
Official Title
A Phase 1/2 Study to Assess the Safety And Efficacy Of OCU410 For Geographic Atrophy Secondary To Dry Age-Related Macular Degeneration
Study Type
Interventional

2. Study Status

Record Verification Date
October 2023
Overall Recruitment Status
Recruiting
Study Start Date
August 23, 2023 (Actual)
Primary Completion Date
September 23, 2025 (Anticipated)
Study Completion Date
September 23, 2025 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Ocugen

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes

5. Study Description

Brief Summary
This is a Phase 1/2 Study to Assess the Safety and Efficacy of OCU410 for Geographic Atrophy Secondary to Dry Age-Related Macular Degeneration (AMD). This is a multicenter study, which will be conducted in two phases and will enroll up to a total of 63 subjects.
Detailed Description
Name of Sponsor/Company: Ocugen, Inc. 11 Great Valley Parkway Malvern, PA 19355 Name of Investigational Product: OCU410 Name of Active Ingredient: Adeno-associated viral vector 5 human RORA (AAV5-hRORA) Protocol Number: OCU410-101 Phase: 1/2 Country: US Title of Study: A Phase 1/2 Study to Assess the Safety and Efficacy of OCU410 for Geographic Atrophy Secondary to Dry Age-Related Macular Degeneration. Study Center(s): Approximately five clinical study centers in the US. Background: Age-related Macular Degeneration (AMD) is an ocular disease where macular degenerative occurs. AMD manifests in two forms, Dry (nonexudative, atrophic) AMD and Wet (exudative, neovascular) AMD. Geographic atrophy (GA) is an advanced stage of dry AMD that affects nearly 1 million people in the US and 5 million people worldwide, with its prevalence increasing exponentially with age. It leads to progressive and irreversible loss of visual function due to the growth of atrophic lesions that destroy the retinal cells responsible for vision. OCU410 Product Information: Ocugen, Inc., has developed a proprietary modifier gene therapy platform, OCU410, as the second agent in a novel class of NHR-based gene modifier therapy for patients with dry AMD. The proposed indication for OCU410 (AAV5-hRORA) is for the treatment of GA secondary to dry AMD. The drug product is a sterile ophthalmic suspension for subretinal injection. OCU410 therapy regulates gene pathways contributing to GA by restoring homeostasis in the eye and thereby serving as a therapeutic candidate for dry AMD. The modifier gene therapy platform is a new way of addressing a genetic disease arising through a multitude of genetic mutations in various genes but leading to the same end result (phenotype) of a diseased condition. This study will be conducted in two phases enrolling up to 63 subjects. Treated subjects will receive a single subretinal injection of OCU410 in the study eye. Phase 1 is a multicenter, open-label, dose-ranging/dose-escalating study with a 3+3 design enrolling up to 18 subjects. Phase 2 is a randomized dose-expansion cohort in which 45 subjects will be randomized in a 1:1:1 ratio in to one of the 2 treatment arms or the untreated control arm.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Geographic Atrophy

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 1, Phase 2
Interventional Study Model
Sequential Assignment
Model Description
Study will be conducted in 2 phases: Phase 1 will be 3+3 design will be used for sequential dose-escalation cohorts in which subjects will receive a single subretinal injection of OCU410 in the study eye. Phase 2 will be a dose-expansion phase of the study, where the subjects will be randomized in 1:1:1
Masking
Outcomes Assessor
Masking Description
The following team members will be masked: Bio-Statistician, Data Programmer, Imaging Reading Center Team, Head of Clinical Development and Medical Affairs.
Allocation
Randomized
Enrollment
63 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
Phase1 Dose Escalation- Low Dose (2.5×10E10 vg/mL):
Arm Type
Experimental
Arm Description
Low Dose (2.5×10E10 vg/mL): Subjects will receive a subretinal injection of OCU410 in the low dose concentration.
Arm Title
Phase1 Dose Escalation- Medium Dose (5×10E10 vg/mL):
Arm Type
Experimental
Arm Description
Medium Dose (5×10E10 vg/mL): Subjects will receive a subretinal injection of OCU410 in the medium dose concentration.
Arm Title
Phase1 Dose Escalation- High Dose (1.5×10E11 vg/mL):
Arm Type
Experimental
Arm Description
High Dose (1.5×10E11 vg/mL): Subjects will receive a subretinal injection in the high dose concentration.
Arm Title
Phase 2 Dose Expansion: Maximum tolerated dose (MTD) from Phase 1-Randomized Arm
Arm Type
Experimental
Arm Description
Maximum tolerated dose (MTD) from Phase 1: Subjects will receive a subretinal injection in the MTD concentration.
Arm Title
Phase 2 Dose Expansion: Lower Dose from Phase 1-Randomized Arm
Arm Type
Experimental
Arm Description
Subjects will receive a subretinal injection of OCU410 in a Lower Dose concentration.
Arm Title
Control Arm
Arm Type
No Intervention
Arm Description
No Intervention Control Arm: Subject will not receive any active study intervention
Intervention Type
Genetic
Intervention Name(s)
OCU410
Intervention Description
Subretinal administration of OCU410
Primary Outcome Measure Information:
Title
Safety (Participants With Ocular and Non-ocular AEs (Adverse Events) and SAEs (Serious Adverse Events))
Description
The primary endpoint is safety, determined by the number of ocular and non-ocular Study Drug-related adverse events (SDAE), treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs).
Time Frame
12 months (Screening to 12 months post OCU410 administration)
Title
Change in anatomy of ocular structures using Slit Lamp Biomicroscopy
Description
We will use Slit-lamp Biomicroscopy to visualize the anatomy of ocular structures before and after sub-retinal injections and follow-up visits.
Time Frame
12 months (Screening to 12 months post OCU410 administration)
Title
Change in anatomy of ocular structures using Indirect ophthalmoscopy
Description
We will use Indirect ophthalmoscopy to visualize the anatomy of ocular structures before and after sub-retinal injections and follow-up visits.
Time Frame
12 months (Screening to 12 months post OCU410 administration)
Title
Change from baseline in BCVA (Best Corrected Visual Acuity)
Description
Visual function of the study eye was assessed using the Early Treatment Diabetic Retinopathy Study (ETDRS) Best Corrected Visual Acuity (BCVA) letter score. A higher score represents better vision.
Time Frame
12 months (Screening to 12 months post OCU410 administration)
Title
Change in Low Luminance Visual Acuity
Description
Measured by letter score. A higher score represents better vision
Time Frame
12 months (Screening to 12 months post OCU410 administration)
Title
Change in the Intraocular Pressure (mmHg)
Description
Measured by applanation or rebound tonometry with confirmation with Goldmann tonometer if IOP is outside normal range (8-21mmHg).
Time Frame
12 months (Screening to 12 months post OCU410 administration)
Secondary Outcome Measure Information:
Title
Humoral and cellular immune response
Description
Blood samples will be collected for the assessment. The secondary safety endpoints include change from baseline in Humoral and cellular immune response in response to OCU410 administration
Time Frame
12 months (Screening to 12 months post OCU410 administration)
Title
Shedding of viral vector
Description
Blood samples will be collected for the assessment to determine AAV vector shedding in systemic circulation after OCU410 administration
Time Frame
12 months (Screening to 12 months post OCU410 administration)
Title
Laboratory parameters including serum chemistry and hematology
Description
Blood samples will be collected for the assessment to determine a change from baseline after OCU410 administration.
Time Frame
12 months (Screening to 12 months post OCU410 administration)
Other Pre-specified Outcome Measures:
Title
Structural Outcome: Change Using Qualitative and quantitative assessments of autofluorescence pattern (FAF)
Description
Changes in the intensity of FAF will be evaluated from the baseline measurements, to assess the loss of retinal layers.
Time Frame
12 months (Screening to 12 months post OCU410 administration)
Title
Changes in National Eye Institute Visual Function Questionnaire 25 (NEI-VFQ25)
Description
The National Eye Institute Visual Function Questionnaire 25 (NEI-VFQ25) questionnaires will be completed to assess the impact of vision on quality of subject's life.
Time Frame
12 months (Screening to 12 months post OCU410 administration)
Title
Change From Baseline in Mean Threshold Sensitivity (MAIA)
Description
Mean threshold sensitivity of all points will be determined to assess the macular functional response and determine GA progression.
Time Frame
12 months (Screening to 12 months post OCU410 administration)
Title
Change from Baseline in drusen volume using SD-OCT
Description
Measurement of change in drusen volume will be determined using Spectral Domain OCT measurements.
Time Frame
12 months (Screening to 12 months post OCU410 administration)

10. Eligibility

Sex
All
Minimum Age & Unit of Time
50 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Subjects 50 years of age or older. BCVA of approximately 24 letters or more using Early Treatment Diabetic Retinopathy Study (ETDRS) chart (20/320 Snellen equivalent). Fundus autofluorescence (FAF) imaging shows: Total GA area ≥2.5 and ≤17.5 mm2 (1 and 7 disk areas [DA], respectively) If GA is multifocal, at least one focal lesion must be ≥1.25 mm2 (0.5 DA), with the overall aggregate area of GA The entire GA lesion must be completely visualized on the macula-centered image and must be able to be imaged in its entirety, and not contiguous with any areas of peripapillary atrophy Absence of any pattern of hyper-autofluorescence in the junctional zone of GA Exclusion Criteria: Previous treatment with a gene-therapy or cell therapy product Previous treatment with any investigational drug or device within one year. The history of any investigational product with a washout period of up to six months will be evaluated on a case-by-case basis. Previous treatment with Syfovre (Pegcetacoplan injection) GA due to causes other than AMD such as Stargardt disease, cone rod dystrophy or toxic maculopathies like Plaquenil maculopathy. however, benign conditions of the vitreous or peripheral retina are not exclusionary (i.e., pavingstone degeneration).
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Umair Qazi, MD, MPH
Phone
202 817 0787
Email
umair.qazi@ocugen.com
First Name & Middle Initial & Last Name or Official Title & Degree
Roshan George, MD, MPH
Phone
(845) 664-1505
Email
roshan.george@ocugen.com
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Huma Qamar, MD, MPH, CMI
Organizational Affiliation
Ocugen
Official's Role
Study Director
Facility Information:
Facility Name
Retina Consultants of Texas
City
Bellaire
State/Province
Texas
ZIP/Postal Code
77401
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Rebbecca Taing
Phone
800-833-5921
Email
rebbecca.taing@retinaconsultantstexas.com
First Name & Middle Initial & Last Name & Degree
Charles Wykoff, M.D; Ph.D
Facility Name
Retina Foundation of the Southwest
City
Dallas
State/Province
Texas
ZIP/Postal Code
75231
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Kimberly Cummings, CCRC
Phone
214-363-3911
Ext
128
Email
evasquez@retinafoundation.org
First Name & Middle Initial & Last Name & Degree
Karl Csaky, M.D; Ph.D

12. IPD Sharing Statement

Plan to Share IPD
No

Learn more about this trial

Study to Assess the Safety and Efficacy of OCU410 for Geographic Atrophy

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