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

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ClinicalTrials.gov Identifier: NCT06018558
Recruitment Status : Recruiting
First Posted : August 30, 2023
Last Update Posted : March 12, 2024
Sponsor:
Information provided by (Responsible Party):
Ocugen

Study Description
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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.


Condition or disease Intervention/treatment Phase
Geographic Atrophy Genetic: OCU410 Phase 1 Phase 2

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.

Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 63 participants
Allocation: Randomized
Intervention Model: Sequential Assignment
Intervention 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: Single (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.
Primary Purpose: Treatment
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
Actual Study Start Date : August 23, 2023
Estimated Primary Completion Date : September 23, 2025
Estimated Study Completion Date : September 23, 2025

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Experimental: Phase1 Dose Escalation- Low Dose (2.5×10E10 vg/mL):
Low Dose (2.5×10E10 vg/mL): Subjects will receive a subretinal injection of OCU410 in the low dose concentration.
 Genetic: OCU410
Subretinal administration of OCU410
Experimental: Phase1 Dose Escalation- Medium Dose (5×10E10 vg/mL):
Medium Dose (5×10E10 vg/mL): Subjects will receive a subretinal injection of OCU410 in the medium dose concentration.
 Genetic: OCU410
Subretinal administration of OCU410
Experimental: Phase1 Dose Escalation- High Dose (1.5×10E11 vg/mL):
High Dose (1.5×10E11 vg/mL): Subjects will receive a subretinal injection in the high dose concentration.
 Genetic: OCU410
Subretinal administration of OCU410
Experimental: Phase 2 Dose Expansion: Maximum tolerated dose (MTD) from Phase 1-Randomized Arm
Maximum tolerated dose (MTD) from Phase 1: Subjects will receive a subretinal injection in the MTD concentration.
 Genetic: OCU410
Subretinal administration of OCU410
Experimental: Phase 2 Dose Expansion: Lower Dose from Phase 1-Randomized Arm
Subjects will receive a subretinal injection of OCU410 in a Lower Dose concentration.
 Genetic: OCU410
Subretinal administration of OCU410
No Intervention: Control Arm
No Intervention Control Arm: Subject will not receive any active study intervention


Outcome Measures
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Primary Outcome Measures :
  1. Safety (Participants With Ocular and Non-ocular AEs (Adverse Events) and SAEs (Serious Adverse Events)) [ Time Frame: 12 months (Screening to 12 months post OCU410 administration) ]
    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).

  2. Change in anatomy of ocular structures using Slit Lamp Biomicroscopy [ Time Frame: 12 months (Screening to 12 months post OCU410 administration) ]
    We will use Slit-lamp Biomicroscopy to visualize the anatomy of ocular structures before and after sub-retinal injections and follow-up visits.

  3. Change in anatomy of ocular structures using Indirect ophthalmoscopy [ Time Frame: 12 months (Screening to 12 months post OCU410 administration) ]
    We will use Indirect ophthalmoscopy to visualize the anatomy of ocular structures before and after sub-retinal injections and follow-up visits.

  4. Change from baseline in BCVA (Best Corrected Visual Acuity) [ Time Frame: 12 months (Screening to 12 months post OCU410 administration) ]
    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.

  5. Change in Low Luminance Visual Acuity [ Time Frame: 12 months (Screening to 12 months post OCU410 administration) ]
    Measured by letter score. A higher score represents better vision

  6. Change in the Intraocular Pressure (mmHg) [ Time Frame: 12 months (Screening to 12 months post OCU410 administration) ]
    Measured by applanation or rebound tonometry with confirmation with Goldmann tonometer if IOP is outside normal range (8-21mmHg).


Secondary Outcome Measures :
  1. Humoral and cellular immune response [ Time Frame: 12 months (Screening to 12 months post OCU410 administration) ]
    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

  2. Shedding of viral vector [ Time Frame: 12 months (Screening to 12 months post OCU410 administration) ]
    Blood samples will be collected for the assessment to determine AAV vector shedding in systemic circulation after OCU410 administration

  3. Laboratory parameters including serum chemistry and hematology [ Time Frame: 12 months (Screening to 12 months post OCU410 administration) ]
    Blood samples will be collected for the assessment to determine a change from baseline after OCU410 administration.


Other Outcome Measures:
  1. Structural Outcome: Change Using Qualitative and quantitative assessments of autofluorescence pattern (FAF) [ Time Frame: 12 months (Screening to 12 months post OCU410 administration) ]
    Changes in the intensity of FAF will be evaluated from the baseline measurements, to assess the loss of retinal layers.

  2. Changes in National Eye Institute Visual Function Questionnaire 25 (NEI-VFQ25) [ Time Frame: 12 months (Screening to 12 months post OCU410 administration) ]
    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.

  3. Change From Baseline in Mean Threshold Sensitivity (MAIA) [ Time Frame: 12 months (Screening to 12 months post OCU410 administration) ]
    Mean threshold sensitivity of all points will be determined to assess the macular functional response and determine GA progression.

  4. Change from Baseline in drusen volume using SD-OCT [ Time Frame: 12 months (Screening to 12 months post OCU410 administration) ]
    Measurement of change in drusen volume will be determined using Spectral Domain OCT measurements.


Eligibility Criteria
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Ages Eligible for Study:   50 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. Subjects 50 years of age or older.
  2. BCVA of approximately 24 letters or more using Early Treatment Diabetic Retinopathy Study (ETDRS) chart (20/320 Snellen equivalent).

  3. Fundus autofluorescence (FAF) imaging shows:

    1. Total GA area ≥2.5 and ≤17.5 mm2 (1 and 7 disk areas [DA], respectively)
    2. If GA is multifocal, at least one focal lesion must be ≥1.25 mm2 (0.5 DA), with the overall aggregate area of GA as specified above in 3.a
    3. 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
    4. Presence of any pattern of hyper-autofluorescence in the junctional zone of GA
  4. Subjects who had prior treatment with an approved drug for AMD, e.g. Izerway® (Avacincaptad pegol) or Syfovre® (Pegcetacoplan injection) can be included, after a washout period of at least 3 months in study eye or fellow eye

Exclusion Criteria:

  1. Previous treatment with a gene-therapy or cell therapy product
  2. 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).
  3. Spherical equivalent of the refractive error demonstrating > 6 diopters of myopia or an axial length >26 mm, inability to fixate, uncontrolled glaucoma, advanced cataract, corneal abnormalities, medium haze, and other retinal pathologies.
  4. Any history or current evidence of exudative ("wet") AMD including any evidence of retinal pigment epithelium rips, branch retinal artery or vein occlusion, corneal transplant, or evidence of neovascularization anywhere in the retina based on fluorescein angiogram.
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT06018558


Contacts
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Contact: Umair Qazi, MD, MPH 202 817 0787 umair.qazi@ocugen.com
Contact: Roshan George, MD, MPH (845) 664-1505 roshan.george@ocugen.com

Locations
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United States, Arizona
Associated Retina Consultants Recruiting
Phoenix, Arizona, United States, 85020
Contact: Erin Fox    480-999-5458    erin.fox@doctrials.com   
Contact: Mallory Mintert    480-999-5458    mallory.mintert@doctrials.com   
Principal Investigator: Benjamin Bakall, M.D; Ph.D         
United States, Mississippi
Mississippi Retina Associates Recruiting
Jackson, Mississippi, United States, 39202
Contact: Anne Britt, MHA, CCRC    601-981-4091 ext 646    Anne Britt <abritt@msretina.com>   
Contact: Mallie Taylor    601-981-4091 ext 646    mtaylor@retinaconsultantsofamerica.com   
Principal Investigator: Michael J Borne, M.D         
United States, North Carolina
Duke Eye Center Recruiting
Durham, North Carolina, United States, 27710
Contact: Lyndsay Lawter    919-681-9459    lyndsay.lawter@duke.edu   
Contact: Sarah Jones, MS, CRCC    919-681-6584    sarah.jones1@duke.edu   
Principal Investigator: Majda Hadziahmetovic, M.D         
United States, Texas
B) Retina Consultants of Texas Recruiting
Bellaire, Texas, United States, 77401
Contact: Rebbecca Taing    800-833-5921    rebbecca.taing@retinaconsultantstexas.com   
Principal Investigator: Charles Wykoff, M.D; Ph.D         
A) Retina Foundation of the Southwest Recruiting
Dallas, Texas, United States, 75231
Contact: Kimberly Cummings, CCRC    214-363-3911 ext 128    evasquez@retinafoundation.org   
Principal Investigator: Karl Csaky, M.D; Ph.D         
United States, Wisconsin
Gundersen Health System Recruiting
La Crosse, Wisconsin, United States, 54601
Contact: Erin Baumgartner    608-775-7119    erin.baumgartner@gundersenhealth.org   
Contact: Sandy Wee    608-775-7119    SKWee@gundersenhealth.org   
Principal Investigator: Syed M Shah, MD         
Sponsors and Collaborators
Ocugen
Investigators
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Study Director: Murthy Chavali, Ph.D Ocugen
More Information
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Responsible Party: Ocugen
ClinicalTrials.gov Identifier: NCT06018558    
Other Study ID Numbers: OCU410-101
First Posted: August 30, 2023    Key Record Dates
Last Update Posted: March 12, 2024
Last Verified: March 2024
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Geographic Atrophy
Atrophy
Pathological Conditions, Anatomical
Macular Degeneration
 Retinal Degeneration
Retinal Diseases
Eye Diseases