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

Primary Purpose

Neovascular Age-Related Macular Degeneration (nAMD)

Status
Recruiting
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
RGX-314 Dose 1
RGX-314 Dose 2
Ranibizumab
RGX-314 Dose 3
Local steroid
Topical steroid
Sponsored by
AbbVie
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Neovascular Age-Related Macular Degeneration (nAMD) focused on measuring AMD, wet AMD, wAMD, nAMD

Eligibility Criteria

50 Years - 89 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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.
  • Active or history of retinal detachment in the study eye.
  • History of intravitreal therapy in the study eye, such as intravitreal steroid injection or investigational product (IP), other than anti-VEGF therapy, in the 6 months prior to study entry.
  • Received any gene therapy.
  • Any condition preventing visualization of the fundus or VA improvement in the study eye, eg, cataract.
  • History of intraocular surgery in the study eye within 12 weeks of study entry.
  • Receipt of any IP within 30 days of study entry or 5 half-lives of the IP.
  • Myocardial infarction, cerebrovascular accident, or transient ischemic attacks within 6 months of study entry.
  • Cohorts 1 - 5 only: Uncontrolled glaucoma in the study eye.

  • COHORT 6 ONLY:

    • Active or history of glaucoma or ocular hypertension in the study eye.
    • Certain OCT characteristics including: Large Pigment Epithelial Detachments (PED), clinically significant Epiretinal Membrane (ERM) in the study eye at Visit 1.

Note: Other inclusion/exclusion criteria apply.

Sites / Locations

  • Phoenix LocationRecruiting
  • Bakersfield Location
  • Beverly Hills LocationRecruiting
  • Mountain View LocationRecruiting
  • Poway LocationRecruiting
  • Santa Barbara Location
  • Augusta LocationRecruiting
  • Baltimore LocationRecruiting
  • Boston LocationRecruiting
  • Reno LocationRecruiting
  • Albuquerque LocationRecruiting
  • Philadelphia LocationRecruiting
  • Germantown Location
  • Nashville Location
  • Woodlands Location

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm 4

Arm 5

Arm 6

Arm Type

Active Comparator

Experimental

Experimental

Experimental

Experimental

Experimental

Arm Label

Ranibizumab control

RGX-314 Treatment Arm (Dose 1)

RGX-314 Treatment Arm (Dose 2)

RGX-314 Treatment Arm (Dose 3)

RGX-314 Treatment Arm (Dose 3) and Local Steroid

RGX-314 Treatment Arm (Dose 3) and Topical Steroid

Arm Description

Control treatment arm

RGX-314 Dose 1

RGX-314 Dose 2

RGX-314 Dose 3

RGX-314 Dose 3 and Local Steroid

RGX-314 Dose 3 and Topical Steroid

Outcomes

Primary Outcome Measures

To evaluate the mean change in Best Corrected Visual Acuity (BCVA) for RGX-314 compared with ranibizumab monthly.
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

Evaluate the safety and tolerability of RGX-314
Incidence of overall and ocular adverse events (AEs) and serious adverse events (SAEs)
Evaluate the incidence of ocular inflammation following administration of RGX-314
Proportion of participants who experience ocular inflammation following SCS RGX-314 administration
Evaluate the effect of RGX-314 on choroidal neovascularization (CNV) lesion growth and leakage
Mean change from baseline in CNV lesion size and leakage area based on fluorescein angiography (FA) at Week 52.
Evaluate the effect of RGX-314 on BCVA
Mean change from baseline in BCVA to Week 52
Evaluate the effect of RGX-314 on central retinal thickness (CRT)
Mean change from baseline in CRT as measured by spectral domain-optical coherence tomography (SD-OCT) to Week 40 and Week 52
Assess the need for supplemental anti-vascular endothelial growth factor (VEGF) therapy in participants who receive RGX-314 treatment
Mean supplemental anti-VEGF injection annualized rate in the RGX-314 treatment arm through Week 40 and Week 52
Evaluate the concentration of RGX-314 transgene product (TP) in aqueous humor and serum
Mean change from baseline and Week 54 in serum RGX-314 TP concentration over time

Full Information

First Posted
August 4, 2020
Last Updated
May 18, 2023
Sponsor
AbbVie
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1. Study Identification

Unique Protocol Identification Number
NCT04514653
Brief Title
RGX-314 Gene Therapy Administered in the Suprachoroidal Space for Participants With Neovascular Age-Related Macular Degeneration (nAMD)
Acronym
AAVIATE
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)
Study Type
Interventional

2. Study Status

Record Verification Date
May 2023
Overall Recruitment Status
Recruiting
Study Start Date
August 25, 2020 (Actual)
Primary Completion Date
October 2023 (Anticipated)
Study Completion Date
January 2024 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
AbbVie

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
Yes
Device Product Not Approved or Cleared by U.S. FDA
Yes
Data Monitoring Committee
Yes

5. Study Description

Brief Summary
RGX-314 is being developed as a potential 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.
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 115 participants who meet the inclusion/exclusion criteria will be enrolled into one of 6 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. Participants enrolled in Cohort 6 will receive RGX-314 and will be randomized to one of two different post-procedural steroid regimens. Cohort 1 will evaluate RGX-314 Dose 1, Cohorts 2 and 3 will evaluate RGX-314 Dose 2, and Cohorts 4, 5, and 6 will evaluate RGX-314 Dose 3. Participants will be evaluated for efficacy, safety and tolerability of RGX-314 throughout the study.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Neovascular Age-Related Macular Degeneration (nAMD)
Keywords
AMD, wet AMD, wAMD, nAMD

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Sequential Assignment
Masking
Outcomes Assessor
Masking Description
The vision examiners and central reading center (CRC) graders will be masked, meaning they will be unaware of the participants' treatment assignment. All other individuals affiliated with the study (investigators, all study center personnel apart from the vision examiners, the participants, all Sponsor staff, all staff affiliated with the contract research organization, and all CRC staff apart from the CRC graders) will have knowledge of the treatment assignment.
Allocation
Randomized
Enrollment
115 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
Ranibizumab control
Arm Type
Active Comparator
Arm Description
Control treatment arm
Arm Title
RGX-314 Treatment Arm (Dose 1)
Arm Type
Experimental
Arm Description
RGX-314 Dose 1
Arm Title
RGX-314 Treatment Arm (Dose 2)
Arm Type
Experimental
Arm Description
RGX-314 Dose 2
Arm Title
RGX-314 Treatment Arm (Dose 3)
Arm Type
Experimental
Arm Description
RGX-314 Dose 3
Arm Title
RGX-314 Treatment Arm (Dose 3) and Local Steroid
Arm Type
Experimental
Arm Description
RGX-314 Dose 3 and Local Steroid
Arm Title
RGX-314 Treatment Arm (Dose 3) and Topical Steroid
Arm Type
Experimental
Arm Description
RGX-314 Dose 3 and Topical Steroid
Intervention Type
Genetic
Intervention Name(s)
RGX-314 Dose 1
Other Intervention Name(s)
Combination Product
Intervention Description
AAV8 vector containing a transgene for anti-VEGF fab (Dose 1)
Intervention Type
Genetic
Intervention Name(s)
RGX-314 Dose 2
Other Intervention Name(s)
Combination Product
Intervention Description
AAV8 vector containing a transgene for anti-VEGF fab (Dose 2)
Intervention Type
Biological
Intervention Name(s)
Ranibizumab
Intervention Description
Ranibizumab (anti-VEGF agent)
Intervention Type
Genetic
Intervention Name(s)
RGX-314 Dose 3
Other Intervention Name(s)
Combination Product
Intervention Description
AAV8 vector containing a transgene for anti-VEGF fab (Dose 3)
Intervention Type
Drug
Intervention Name(s)
Local steroid
Intervention Description
Local Steroid
Intervention Type
Drug
Intervention Name(s)
Topical steroid
Intervention Description
Topical steroid
Primary Outcome Measure Information:
Title
To evaluate the mean change in Best Corrected Visual Acuity (BCVA) for RGX-314 compared with ranibizumab monthly.
Description
The scale used is the early treatment diabetic retinopathy study (ETDRS) letter score from 0-100 and higher score being better vision.
Time Frame
40 weeks
Secondary Outcome Measure Information:
Title
Evaluate the safety and tolerability of RGX-314
Description
Incidence of overall and ocular adverse events (AEs) and serious adverse events (SAEs)
Time Frame
52 weeks
Title
Evaluate the incidence of ocular inflammation following administration of RGX-314
Description
Proportion of participants who experience ocular inflammation following SCS RGX-314 administration
Time Frame
52 weeks
Title
Evaluate the effect of RGX-314 on choroidal neovascularization (CNV) lesion growth and leakage
Description
Mean change from baseline in CNV lesion size and leakage area based on fluorescein angiography (FA) at Week 52.
Time Frame
52 weeks
Title
Evaluate the effect of RGX-314 on BCVA
Description
Mean change from baseline in BCVA to Week 52
Time Frame
52 weeks
Title
Evaluate the effect of RGX-314 on central retinal thickness (CRT)
Description
Mean change from baseline in CRT as measured by spectral domain-optical coherence tomography (SD-OCT) to Week 40 and Week 52
Time Frame
52 weeks
Title
Assess the need for supplemental anti-vascular endothelial growth factor (VEGF) therapy in participants who receive RGX-314 treatment
Description
Mean supplemental anti-VEGF injection annualized rate in the RGX-314 treatment arm through Week 40 and Week 52
Time Frame
52 weeks
Title
Evaluate the concentration of RGX-314 transgene product (TP) in aqueous humor and serum
Description
Mean change from baseline and Week 54 in serum RGX-314 TP concentration over time
Time Frame
52 weeks

10. Eligibility

Sex
All
Minimum Age & Unit of Time
50 Years
Maximum Age & Unit of Time
89 Years
Accepts Healthy Volunteers
No
Eligibility 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. Active or history of retinal detachment in the study eye. History of intravitreal therapy in the study eye, such as intravitreal steroid injection or investigational product (IP), other than anti-VEGF therapy, in the 6 months prior to study entry. Received any gene therapy. Any condition preventing visualization of the fundus or VA improvement in the study eye, eg, cataract. History of intraocular surgery in the study eye within 12 weeks of study entry. Receipt of any IP within 30 days of study entry or 5 half-lives of the IP. Myocardial infarction, cerebrovascular accident, or transient ischemic attacks within 6 months of study entry. Cohorts 1 - 5 only: Uncontrolled glaucoma in the study eye. COHORT 6 ONLY: Active or history of glaucoma or ocular hypertension in the study eye. Certain OCT characteristics including: Large Pigment Epithelial Detachments (PED), clinically significant Epiretinal Membrane (ERM) in the study eye at Visit 1. Note: Other inclusion/exclusion criteria apply.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Patient Advocacy
Phone
1-866-860-0117
Email
patientadvocacy@regenxbio.com
Facility Information:
Facility Name
Phoenix Location
City
Phoenix
State/Province
Arizona
ZIP/Postal Code
85014
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Principal Investigator
Facility Name
Bakersfield Location
City
Bakersfield
State/Province
California
ZIP/Postal Code
93309
Country
United States
Individual Site Status
Active, not recruiting
Facility Name
Beverly Hills Location
City
Beverly Hills
State/Province
California
ZIP/Postal Code
90211
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Principal Investigator
Facility Name
Mountain View Location
City
Mountain View
State/Province
California
ZIP/Postal Code
94040
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Principal Investigator
Facility Name
Poway Location
City
Poway
State/Province
California
ZIP/Postal Code
92064
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Principal Investigator
Facility Name
Santa Barbara Location
City
Santa Barbara
State/Province
California
ZIP/Postal Code
93103
Country
United States
Individual Site Status
Active, not recruiting
Facility Name
Augusta Location
City
Augusta
State/Province
Georgia
ZIP/Postal Code
30909
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Principal Investigator
Facility Name
Baltimore Location
City
Baltimore
State/Province
Maryland
ZIP/Postal Code
21287
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Principal Investigator
Facility Name
Boston Location
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02114
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Principal Investigator
Facility Name
Reno Location
City
Reno
State/Province
Nevada
ZIP/Postal Code
89502
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Principal Investigator
Facility Name
Albuquerque Location
City
Albuquerque
State/Province
New Mexico
ZIP/Postal Code
87109
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Principal Investigator
Facility Name
Philadelphia Location
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19107
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Principal Investigator
Facility Name
Germantown Location
City
Germantown
State/Province
Tennessee
ZIP/Postal Code
38138
Country
United States
Individual Site Status
Active, not recruiting
Facility Name
Nashville Location
City
Nashville
State/Province
Tennessee
ZIP/Postal Code
37203
Country
United States
Individual Site Status
Active, not recruiting
Facility Name
Woodlands Location
City
The Woodlands
State/Province
Texas
ZIP/Postal Code
77384
Country
United States
Individual Site Status
Active, not recruiting

12. IPD Sharing Statement

Plan to Share IPD
No

Learn more about this trial

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

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