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Pivotal 2 Study of RGX-314 Gene Therapy in Participants With nAMD (ASCENT)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT05407636
Recruitment Status : Recruiting
First Posted : June 7, 2022
Last Update Posted : August 21, 2023
Sponsor:
Information provided by (Responsible Party):
AbbVie

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 WetAMD CNV Genetic: RGX-314 Dose 1 Genetic: RGX-314 Dose 2 Biological: Aflibercept (EYLEA®) Phase 3

Detailed Description:
This randomized, partially masked, controlled, Phase 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 gene therapy 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 aflibercept. Approximately 465 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 : 465 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Intervention Model Description: 2 RGX-314 treatment arms, 1 control arm (aflibercept)
Masking: Single (Participant)
Masking Description: Care Provider, Investigator, Outcomes Assessor
Primary Purpose: Treatment
Official Title: A Randomized, Partially Masked, Controlled, Phase 3 Clinical Study to Evaluate the Efficacy and Safety of RGX-314 Gene Therapy in Participants With nAMD
Actual Study Start Date : December 28, 2021
Estimated Primary Completion Date : February 2025
Estimated Study Completion Date : December 2025

Resource links provided by the National Library of Medicine

Drug Information available for: Aflibercept

Arm Intervention/treatment
Experimental: RGX-314 Dose 1
RGX-314 Dose 1 administered via subretinal delivery one time.
Genetic: RGX-314 Dose 1
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 Dose 2
AAV8 vector containing a transgene for anti-VEGF Fab (Dose 2)

Active Comparator: Control Arm
Aflibercept administered via intravitreal injection approximately every 8 weeks
Biological: Aflibercept (EYLEA®)

2.0 mg (0.05 mLsolution) administered by intravitreal injection approximately every 8 weeks after 3 monthly injections

Other Names:

• Eylea (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 Serious Adverse Events (SAEs) [ Time Frame: At Week 54 ]
    Incidences of ocular and overall Serious Adverse Events

  2. Mean change from baseline in BCVA [ Time Frame: At Week 54 (RGX-314 randomized participants only) ]
    BCVA measured by ETDRS

  3. Mean change from baseline in CRT and CPT as measured by SD-OCT [ Time Frame: At Week 54 and Week 90 ]
    CRT and CPT as measured by SD-OCT

  4. Mean reduction in supplemental anti VEGF injection annualized rate compared with the prior year of therapy [ Time Frame: Through Week 54 and Week 108 ]
    Supplemental anti-VEGF treatments required post therapy to the year prior

  5. Mean supplemental anti VEGF injection annualized rate in the RGX-314 arms [ Time Frame: Through Week 54 and Week 108 ]
    Supplemental anti-VEGF treatments required post therapy to the year prior

  6. Aqueous RGX-314 TP concentrations [ Time Frame: At Week 14, Week 38, Week 54, Week 74, Week 90 and Week 108 ]
    Observation of concentration of RGX-314 in the aqueous humor over time



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 or current retinal tear in the study eye
  5. Advanced glaucoma or history of secondary glaucoma in the study eye
  6. Myocardial infarction, cerebrovascular accident, or transient ischemic attach within the past 6 months.
  7. History of intraocular surgery in the study eye within 12 weeks prior to Screening Visit 1
  8. 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.
  9. Prior treatment with gene therapy.

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): NCT05407636


Contacts
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Contact: Patient Advocacy +(1) 866-860-0117 Patientadvocacy@regenxbio.com

Locations
Show Show 94 study locations
Sponsors and Collaborators
AbbVie
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Responsible Party: AbbVie
ClinicalTrials.gov Identifier: NCT05407636    
Other Study ID Numbers: RGX-314-3101
First Posted: June 7, 2022    Key Record Dates
Last Update Posted: August 21, 2023
Last Verified: August 2023
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 AbbVie:
Age-Related Macular Degeneration
Neovascular Age-Related Macular Degeneration
Macular Degeneration
Wet Macular Degeneration
Choroidal Neovascularization
Retinal Degeneration
Retinal Diseases
Eye Diseases
Ranibizumab
Aflibercept
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
Aflibercept
Angiogenesis Inhibitors
Angiogenesis Modulating Agents
Growth Substances
Physiological Effects of Drugs
Growth Inhibitors
Antineoplastic Agents