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RGX-314 Gene Therapy Administered in the Suprachoroidal Space for Participants With Neovascular Age-Related Macular Degeneration (nAMD) (AAVIATE)

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ClinicalTrials.gov Identifier: NCT04514653
Recruitment Status : Recruiting
First Posted : August 17, 2020
Last Update Posted : April 7, 2022
Sponsor:
Information provided by (Responsible Party):
Regenxbio Inc.

Brief Summary:
RGX-314 is being developed as a novel one-time gene therapy treatment for the treatment of neovascular (wet) age related macular degeneration (wet AMD). Wet AMD is characterized by loss of vision due to new, leaky blood vessel formation in the retina. Wet AMD is a significant cause of vision loss in the United States, Europe and Japan, with up to 2 million people living with wet AMD in these geographies alone. Current anti-VEGF therapies have significantly changed the landscape for treatment of wet AMD, becoming the standard of care due to their ability to prevent progression of vision loss in the majority of patients. These therapies, however, require life-long intraocular injections, typically repeated every four to twelve weeks in frequency, to maintain efficacy. Due to the burden of treatment, patients often experience a decline in vision with reduced frequency of treatment over time. RGX-314 is being developed as a potential one-time treatment for wet AMD.

Condition or disease Intervention/treatment Phase
Neovascular Age-Related Macular Degeneration (nAMD) Biological: RGX-314 Dose 1 Biological: RGX-314 Dose 2 Biological: Ranibizumab (LUCENTIS®) Biological: RGX-314 Dose 3 Phase 2

Detailed Description:
This phase 2, randomized, dose-escalation study is designed to evaluate the efficacy, safety and tolerability of RGX-314 gene therapy in subjects with nAMD. Approximately 95 participants who meet the inclusion/exclusion criteria, will be enrolled into one of 5 cohorts. Participants will be randomized in Cohorts 1 and 2 to receive RGX-314 or Ranibizumab control, and participants enrolled in Cohorts 3 through 5 will receive RGX-314. Cohort 1 will evaluate RGX-314 Dose 1, Cohorts 2 and 3 will evaluate RGX-314 Dose 2, and Cohorts 4 and 5 will evaluate RGX-314 Dose 3. Participants in the ranibizumab control arm will be offered, if eligible, an opportunity to receive RGX-314 treatment after completion of the study at Week 52. Control arm participants who cross over to RGX-314 following Week 52 will receive dose level 2 and follow similar postsurgical visits as was outlined for participants who were originally randomized to receive RGX-314, for 26 weeks through Week80. Efficacy will be the primary focus of the study. Participants will be evaluated for safety and tolerability of RGX-314 throughout the study.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 95 participants
Allocation: Randomized
Intervention Model: Sequential Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase 2, Randomized, Dose-escalation, Ranibizumab-controlled Study to Evaluate the Efficacy, Safety, and Tolerability of RGX-314 Gene Therapy Delivered Via One or Two Suprachoroidal Space (SCS) Injections in Participants With Neovascular Age-Related Macular Degeneration (nAMD) (AAVIATE)
Actual Study Start Date : August 25, 2020
Estimated Primary Completion Date : March 2023
Estimated Study Completion Date : June 2023


Arm Intervention/treatment
Active Comparator: Ranibizumab control
Control treatment arm
Biological: Ranibizumab (LUCENTIS®)
Ranibizumab (anti-VEGF agent)

Experimental: RGX-314 Treatment Arm (Dose 1)
RGX-314 Dose 1
Biological: RGX-314 Dose 1
AAV8 vector containing a transgene for anti-VEGF fab (Dose 1)

Experimental: RGX-314 Treatment Arm (Dose 2)
RGX-314 Dose 2
Biological: RGX-314 Dose 2
AAV8 vector containing a transgene for anti-VEGF fab (Dose 2)

Experimental: RGX-314 Treatment Arm (Dose 3)
RGX-314 Dose 3
Biological: RGX-314 Dose 3
AAV8 vector containing a transgene for anti-VEGF fab (Dose 3)




Primary Outcome Measures :
  1. To evaluate the mean change in Best Corrected Visual Acuity (BCVA) for RGX-314 compared with ranibizumab monthly. [ Time Frame: 40 weeks ]
    The scale used is the early treatment diabetic retinopathy study (ETDRS) letter score from 0-100 and higher score being better vision.


Secondary Outcome Measures :
  1. Incidences of overall and ocular AEs and SAEs [ Time Frame: 52 weeks ]
    Evaluate the safety and tolerability of RGX

  2. Evaluate the effect of RGX-314 on choroidal neovascularization (CNV) lesion growth and leakage [ Time Frame: 52 weeks ]
    Mean change from baseline in CNV lesion size and leakage area based on FA at Week 40 and Week 52.

  3. Evaluate the effect of RGX-314 on Best Corrected Visual Acuity (BCVA) [ Time Frame: 52 weeks ]
    Mean change from baseline in Best Corrected Visual Acuity (BCVA) to Week 52

  4. Evaluate the effect of RGX-314 on central retinal thickness (CRT) [ Time Frame: 52 weeks ]
    Mean change from baseline in CRT as measured by SD-OCT to Week 40 and Week 52

  5. Assess the need for supplemental anti-vascular endothelial growth factor (VEGF) therapy in participants who receive RGX-314 treatment [ Time Frame: 52 weeks ]
    Mean supplemental anti-VEGF injection annualized rate in the RGX-314 treatment arm through Week 40 and Week 52



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Ages Eligible for Study:   50 Years to 89 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Age >/= 50 and </= 89
  • Diagnosis of CNV secondary to age-related macular degeneration in the study eye.
  • Participants must have demonstrated a meaningful response to anti-VEGF therapy.
  • Willing and able to provide written, signed informed consent for this study.

Exclusion Criteria:

  • CNV or macular edema in the study eye secondary to any causes other than AMD.
  • Subfoveal fibrosis or atrophy in study eye.
  • Participants who have had a prior vitrectomy.
  • Any condition in the investigator's opinion that could limit VA improvement in the study eye.
  • Active or history of retinal detachment in the study eye.
  • Uncontrolled glaucoma in the study eye.
  • Received any gene therapy.
  • Any condition preventing visualization of the fundus or VA improvement in the study eye, eg, cataract, vitreous opacity, fibrosis, atrophy, or retinal epithelial tear in the center of the fovea.
  • History of intraocular surgery in the study eye.
  • Receipt of any investigational product within 30 days of Visit 2.
  • Myocardial infarction, cerebrovascular accident, or transient ischemic attacks within 6 months of study entry.

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): NCT04514653


Contacts
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Contact: Patient Advocacy 1-866-860-0117 patientadvocacy@regenxbio.com

Locations
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United States, Arizona
Phoenix Location Recruiting
Phoenix, Arizona, United States, 85014
Contact: Principal Investigator         
United States, California
Bakersfield Location Recruiting
Bakersfield, California, United States, 93309
Contact: Principal Investigator         
Beverly Hills Location Recruiting
Beverly Hills, California, United States, 90211
Contact: Principal Investigator         
Mountain View Location Recruiting
Mountain View, California, United States, 94040
Contact: Principal Investigator         
Poway Location Recruiting
Poway, California, United States, 92064
Contact: Principal Investigator         
Santa Barbara Location Recruiting
Santa Barbara, California, United States, 93103
Contact: Principal Investigator         
United States, Georgia
Augusta Location Recruiting
Augusta, Georgia, United States, 30909
Contact: Principal Investigator         
United States, Maryland
Baltimore Location Recruiting
Baltimore, Maryland, United States, 21287
Contact: Principal Investigator         
United States, Massachusetts
Boston Location Recruiting
Boston, Massachusetts, United States, 02114
Contact: Principal Investigator         
United States, Nevada
Reno Location Recruiting
Reno, Nevada, United States, 89502
Contact: Principal Investigator         
United States, New Mexico
Albuquerque Location Recruiting
Albuquerque, New Mexico, United States, 87109
Contact: Principal Investigator         
United States, Pennsylvania
Philadelphia Location Recruiting
Philadelphia, Pennsylvania, United States, 19107
Contact: Principal Investigator         
United States, Tennessee
Germantown Location Recruiting
Germantown, Tennessee, United States, 38138
Contact: Principal Investigator         
Nashville Location Recruiting
Nashville, Tennessee, United States, 37203
Contact: Principal Investigator         
United States, Texas
Woodlands Location Recruiting
The Woodlands, Texas, United States, 77384
Contact: Principal Investigator         
Sponsors and Collaborators
Regenxbio Inc.
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Responsible Party: Regenxbio Inc.
ClinicalTrials.gov Identifier: NCT04514653    
Other Study ID Numbers: RGX-314-2102
First Posted: August 17, 2020    Key Record Dates
Last Update Posted: April 7, 2022
Last Verified: March 2022
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
Keywords provided by Regenxbio Inc.:
AMD
wet AMD
wAMD
nAMD
Additional relevant MeSH terms:
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Macular Degeneration
Wet Macular Degeneration
Choroidal Effusions
Retinal Degeneration
Retinal Diseases
Eye Diseases
Eye Abnormalities
Choroid Diseases
Uveal Diseases
Ranibizumab
Angiogenesis Inhibitors
Angiogenesis Modulating Agents
Growth Substances
Physiological Effects of Drugs
Growth Inhibitors
Antineoplastic Agents