This is the classic website, which will be retired eventually. Please visit the modernized ClinicalTrials.gov instead.
Working…
ClinicalTrials.gov
ClinicalTrials.gov Menu

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

Tracking Information
First Submitted Date  ICMJE January 28, 2022
First Posted Date  ICMJE June 7, 2022
Last Update Posted Date August 21, 2023
Actual Study Start Date  ICMJE December 28, 2021
Estimated Primary Completion Date February 2025   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: June 2, 2022)
Mean change from baseline in Best Corrected Visual Acuity (BCVA) [ Time Frame: At Week 54 ]
BCVA measured by Early Treatment Diabetic Retinopathy Study (ETDRS)
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: May 18, 2023)
  • Incidences of ocular and overall Serious Adverse Events (SAEs) [ Time Frame: At Week 54 ]
    Incidences of ocular and overall Serious Adverse Events
  • Mean change from baseline in BCVA [ Time Frame: At Week 54 (RGX-314 randomized participants only) ]
    BCVA measured by ETDRS
  • 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
  • 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
  • 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
  • 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
Original Secondary Outcome Measures  ICMJE
 (submitted: June 2, 2022)
  • Incidences of ocular and overall Serious Adverse Events (SAEs) [ Time Frame: At Week 54 ]
    Incidences of ocular and overall Serious Adverse Events
  • Mean change from baseline in BCVA [ Time Frame: At Week 54 (RGX-314 randomized participants only) ]
    BCVA measured by ETDRS
  • 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
  • Mean reduction in supplemental anti VEGF injection annualized rate compared with the prior year of therapy [ Time Frame: Through Week 54 and Week 90 ]
    Supplemental anti-VEGF treatments required post therapy to the year prior
  • Mean supplemental anti VEGF injection annualized rate in the RGX-314 arms [ Time Frame: Through Week 54 and Week 90 ]
    Supplemental anti-VEGF treatments required post therapy to the year prior
  • Aqueous RGX-314 TP concentrations [ Time Frame: At Week 14, Week 38, Week 54, Week 74 and Week 90 ]
    Observation of concentration of RGX-314 in the aqueous humor over time
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Pivotal 2 Study of RGX-314 Gene Therapy in Participants With nAMD
Official Title  ICMJE A Randomized, Partially Masked, Controlled, Phase 3 Clinical Study to Evaluate the Efficacy and Safety of RGX-314 Gene Therapy in Participants With nAMD
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.
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.
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE 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
Condition  ICMJE
  • AMD
  • nAMD
  • Wet Age-related Macular Degeneration
  • wAMD
  • WetAMD
  • CNV
Intervention  ICMJE
  • Genetic: RGX-314 Dose 1
    AAV8 vector containing a transgene for anti-VEGF Fab (Dose 1)
  • Genetic: RGX-314 Dose 2
    AAV8 vector containing a transgene for anti-VEGF Fab (Dose 2)
  • 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)

Study Arms  ICMJE
  • Experimental: RGX-314 Dose 1
    RGX-314 Dose 1 administered via subretinal delivery one time.
    Intervention: Genetic: RGX-314 Dose 1
  • Experimental: RGX-314 Dose 2
    RGX-314 Dose 2 administered via subretinal delivery one time.
    Intervention: Genetic: RGX-314 Dose 2
  • Active Comparator: Control Arm
    Aflibercept administered via intravitreal injection approximately every 8 weeks
    Intervention: Biological: Aflibercept (EYLEA®)
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: June 2, 2022)
465
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE December 2025
Estimated Primary Completion Date February 2025   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 50 Years to 89 Years   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Patient Advocacy +(1) 866-860-0117 Patientadvocacy@regenxbio.com
Listed Location Countries  ICMJE Canada,   Puerto Rico,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT05407636
Other Study ID Numbers  ICMJE RGX-314-3101
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Current Responsible Party AbbVie
Original Responsible Party REGENXBIO Inc.
Current Study Sponsor  ICMJE AbbVie
Original Study Sponsor  ICMJE REGENXBIO Inc.
Collaborators  ICMJE Not Provided
Investigators  ICMJE Not Provided
PRS Account AbbVie
Verification Date August 2023

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP