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Pivotal 1 Study of RGX-314 Gene Therapy in Participants With nAMD (ATMOSPHERE)

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ClinicalTrials.gov Identifier: NCT04704921
Recruitment Status : Recruiting
First Posted : January 12, 2021
Last Update Posted : May 6, 2021
Sponsor:
Information provided by (Responsible Party):
Regenxbio Inc.

Brief Summary:
RGX-314 is being developed as a novel one-time gene therapy 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 12 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
AMD nAMD Wet Age-related Macular Degeneration wAMD Wet AMD CNV Genetic: RGX-314 Biological: Ranibizumab (LUCENTIS®) Phase 2 Phase 3

Detailed Description:
This randomized, partially masked, controlled, Phase 2b/3 clinical study will evaluate the efficacy and safety of RGX-314 gene therapy in participants with nAMD. The study will evaluate 2 dose levels of RGX-314 relative to an active comparator. The primary endpoint of this study is mean change in best-corrected visual acuity (BCVA) of RGX-314 relative to ranibizumab. Approximately 300 participants who meet the inclusion/exclusion criteria, will be enrolled into one of 3 arms.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 300 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Intervention Model Description: 2 RGX-314 treatment arms, 1 control arm (ranibizumab)
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Masking Description: The administration of RGX-314 requires an outpatient surgical procedure performed in an operating room, while the active control, ranibizumab, is administered via intravitreal injection in an office setting. This study will be partially masked which will include masking of key study assessors and study drug dose.
Primary Purpose: Treatment
Official Title: A Randomized, Partially Masked, Controlled, Phase 2b/3 Clinical Study to Evaluate the Efficacy and Safety of RGX-314 Gene Therapy in Participants With nAMD (ATMOSPHERE)
Actual Study Start Date : December 29, 2020
Estimated Primary Completion Date : March 2023
Estimated Study Completion Date : March 2024

Resource links provided by the National Library of Medicine

Drug Information available for: Ranibizumab

Arm Intervention/treatment
Experimental: RGX-314 Dose 1
RGX-314 Dose 1 administered via subretinal delivery one time.
Genetic: RGX-314
AAV8 vector containing a transgene for anti-VEGF Fab (Dose 1)

Experimental: RGX-314 Dose 2
RGX-314 Dose 2 administered via subretinal delivery one time.
Genetic: RGX-314
AAV8 vector containing a transgene for anti-VEGF Fab (Dose 2)

Active Comparator: Control Arm
Ranibizumab administered via intravitreal injection approximately every 28 days
Biological: Ranibizumab (LUCENTIS®)
0.5 mg (0.05 mL of 10 mg/mL solution) administered by intravitreal injection approximately every 28 days
Other Name: Ranibizumab (anti-VEGF agent)




Primary Outcome Measures :
  1. Mean change from baseline in Best Corrected Visual Acuity (BCVA) [ Time Frame: At Week 54 ]
    BCVA measured by Early Treatment Diabetic Retinopathy Study (ETDRS)


Secondary Outcome Measures :
  1. Incidences of ocular and overall adverse events (AEs) [ Time Frame: Through Week 98 ]
    Evaluate the safety and tolerability of RGX-314

  2. Mean change from baseline in BCVA [ Time Frame: At Week 98 (RGX-314 randomized participants only) ]
    BCVA measured by ETDRS

  3. Mean change from baseline in Central Retinal Thickness (CRT) and Center Point Thickness (CPT) as measured by Spectral Domain Optical Coherence Tomography (SD-OCT) [ Time Frame: At Week 54 and Week 98 ]
  4. Mean reduction in supplemental anti VEGF injection annualized rate compared with the prior 52 weeks preceding the first ranibizumab injection received as part of the Active Run-in Period (RGX 314 randomized participants) [ Time Frame: Through Week 54 and Week 98 ]
  5. Mean supplemental anti-VEGF injection annualized rate in the RGX-314 arms [ Time Frame: Through Week 54 and Week 98 ]
  6. Aqueous RGX-314 target protein (TP) concentrations [ Time Frame: At Week 14, Week 26, Week 38, Week 54, and Week 98 ]


Information from the National Library of Medicine

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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:

  1. Age ≥ 50 years and ≤ 89 years
  2. An ETDRS BCVA letter score between ≤ 78 and ≥ 40 in the study eye
  3. Diagnosis of subfoveal CNV secondary to AMD in the study eye previously treated with anti-VEGF
  4. Must be pseudophakic (at least 12 weeks postcataract surgery) in the study eye.
  5. Willing and able to provide written, signed informed consent for this study
  6. Participants must have demonstrated a meaningful response to anti-VEGF therapy at study entry

Exclusion Criteria:

  1. CNV or macular edema in the study eye secondary to any causes other than AMD
  2. Subfoveal fibrosis or atrophy in the study eye
  3. Any condition in the investigator's opinion that could limit VA improvement in the study eye
  4. Active or history of retinal detachment in the study eye
  5. Uncontrolled glaucoma in the study eye
  6. History of intraocular surgery in the study eye within 12 weeks prior to Screening Visit 1
  7. History of intravitreal therapy in the study eye, such as intravitreal steroid injection or investigational product, other than anti-VEGF therapy, in the 6 months prior to Screening Visit 1.
  8. Prior treatment with gene therapy.
  9. Recent myocardial infarction, cerebrovascular accident, or transient ischemic attack within the past 6 months.

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): NCT04704921


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
United States, California
Beverly Hills Location Recruiting
Beverly Hills, California, United States, 90211
United States, Massachusetts
Boston Location Recruiting
Boston, Massachusetts, United States, 02114
United States, Nevada
Reno Location Recruiting
Reno, Nevada, United States, 89502
United States, New Mexico
Albuquerque Location Recruiting
Albuquerque, New Mexico, United States, 87109
United States, Ohio
Cincinnati Location Recruiting
Cincinnati, Ohio, United States, 45242
United States, Pennsylvania
Philadelphia Location Recruiting
Philadelphia, Pennsylvania, United States, 19107
United States, Texas
Dallas Location Recruiting
Dallas, Texas, United States, 75231
Sponsors and Collaborators
Regenxbio Inc.
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Responsible Party: Regenxbio Inc.
ClinicalTrials.gov Identifier: NCT04704921    
Other Study ID Numbers: RGX-314-2104
First Posted: January 12, 2021    Key Record Dates
Last Update Posted: May 6, 2021
Last Verified: May 2021
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.:
Age-Related Macular Degeneration
Neovascular Age-Related Macular Degeneration
Macular Degeneration
Wet Macular Degeneration
Choroidal Neovascularization
Retinal Degeneration
Retinal Diseases
Eye Diseases
Ranibizumab
Angiogenesis Inhibitors
Angiogenesis Modulating Agents
Growth Substances
Physiological Effects of Drugs
Growth Inhibitors
Antineoplastic Agents
Gene therapy
Anti-vascular endothelial grown factory therapy
Anti-VEGF therapy
Additional relevant MeSH terms:
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Macular Degeneration
Retinal Degeneration
Retinal Diseases
Eye Diseases
Ranibizumab
Angiogenesis Inhibitors
Angiogenesis Modulating Agents
Growth Substances
Physiological Effects of Drugs
Growth Inhibitors
Antineoplastic Agents