Long-term Follow-Up Study of RGX-314 and Fellow Eye Substudy (RGX-314 SRLTFU)

• ClinicalTrials.gov Identifier: NCT03999801
• Recruitment Status: Enrolling by invitation
• First Posted: June 27, 2019
• Last Update Posted: May 22, 2023
• Sponsor: AbbVie
• Information provided by (Responsible Party): AbbVie

Tracking Information

• First Submitted Date: June 18, 2019
• First Posted Date: June 27, 2019
• Last Update Posted Date: May 22, 2023
• Actual Study Start Date: May 31, 2019
• Estimated Primary Completion Date: December 2028 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: March 29, 2023)

• Incidence of ocular adverse events and any serious adverse events in the study eye [ Time Frame: 5 years inclusive of parent study ]
• Fellow eye substudy: Incidence of ocular adverse events and any serious adverse events in the fellow eye [ Time Frame: 54 weeks ]

• Original Primary Outcome Measures (submitted: June 24, 2019): Incidence of ocular adverse events, any serious adverse events, and any anti-VEGF adverse events of Special Interest [ Time Frame: 5 years inclusive of parent study ]

Current Secondary Outcome Measures (submitted: March 29, 2023)

• Change from baseline in best corrected visual acuity (BCVA) in the study eye [ Time Frame: 5 years inclusive of parent study ]
  Mean change and proportion of participants losing greater than or equal to 5, 10, and 15 letters
• Change from baseline in Central Retinal Thickness (CRT) in the study eye [ Time Frame: 5 years inclusive of parent study ]
  Mean change from baseline in CRT as measured by spectral domain optical coherence tomography (SD-OCT)
• Number of supplemental anti-VEGF injections in the study eye [ Time Frame: 5 years inclusive of parent study ]
  Mean number of supplemental anti-VEGF injections based on chart review
• Number of retinal specialist visits for the study eye [ Time Frame: 5 years inclusive of parent study ]
  Mean number of retinal specialist visits attended for nAMD based on chart review
• Fellow eye substudy: Change from baseline in best corrected visual acuity [ Time Frame: 54 weeks ]
  Mean change from baseline in BCVA in the fellow eye
• Fellow eye substudy: Change from baseline in Central Retinal Thickness in the fellow eye [ Time Frame: 54 weeks ]
  Mean change from baseline in CRT in the fellow eye as measured by SD-OCT
• Fellow eye substudy: Number of supplemental anti-VEGF injections in the fellow eye [ Time Frame: 54 weeks ]
  Mean number of supplemental anti-VEGF injections in the fellow eye
• Fellow eye substudy: Aqueous and Serum RGX-314 transgene product (TP) concentrations in the fellow eye [ Time Frame: 54 weeks ]
  Fellow eye aqueous and serum RGX-314 TP concentrations at assessed time points
• Fellow eye substudy: Immunogenicity measurements in the fellow eye [ Time Frame: 54 weeks ]
  Fellow eye immunogenicity measurements at assessed timepoints

Original Secondary Outcome Measures (submitted: June 24, 2019)

• Mean change from baseline in best corrected visual acuity [ Time Frame: 5 years inclusive of parent study ]
  Baseline is defined as the latest assessment prior to RGX-314 administration in the parent study
• Proportion of participants gaining or losing ≥5, 10, and 15 letters in best corrected visual acuity compared with baseline [ Time Frame: 5 years inclusive of parent study ]
  Baseline is defined as the latest assessment prior to RGX-314 administration in the parent study
• Mean change from baseline in Central Retinal Thickness [ Time Frame: 5 years inclusive of parent study ]
  Measured by spectral domain optical coherence tomography; baseline is defined as the latest assessment prior to RGX-314 administration in the parent study
• Mean number of supplemental anti-VEGF injections [ Time Frame: 5 years inclusive of parent study ]
  Based on chart review
• Mean number of retinal specialist visits [ Time Frame: 5 years inclusive of parent study ]
  Based on chart review

• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: Long-term Follow-Up Study of RGX-314 and Fellow Eye Substudy
• Official Title: A Long-term Follow-Up Study to Evaluate the Safety and Efficacy of RGX-314 Following Subretinal Administration in Participants With Neovascular Age-related Macular Degeneration and Fellow Eye Treatment Substudy

Brief Summary

This is a prospective, observational study designed to evaluate the long-term safety and efficacy of RGX-314. Eligible participants are those who were previously enrolled in a clinical study in which they received a single subretinal administration of RGX-314 in their study eye. Enrollment of each participant in the current study should occur after the participant has completed either the end of study or early termination visit in the previous (parent) clinical study. Participants will be followed for up to 5 years post-RGX-314 administration (inclusive of the parent study). After enrollment and a 6-month follow-up visit, participants will attend at least annual study visits through the end of the 5-year post-RGX-314 administration follow-up period.

Additionally, an interventional fellow eye treatment substudy will evaluate the safety, efficacy, and immunogenicity of subretinal RGX-314 administration in the fellow eye of participants having bilateral disease who previously received a subretinal injection of RGX-314 in their study eye. Participants who qualify for the substudy will receive subretinal administration of RGX-314 in their fellow eye and complete 13 study visits in a 54-week period. Following completion of the substudy participants will continue in the observational portion of the study for up to 5 years post RGX-314 administration in their fellow eye.

• Detailed Description: Not Provided
• Study Type: Interventional
• Study Phase: Phase 2

Study Design

Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Intervention Model Description:

1 main Observational study arm, 1 RGX-314 Fellow Eye Treatment substudy

Masking: None (Open Label)
Primary Purpose: Treatment

Condition

• Neovascular Age-related Macular Degeneration
• Wet Macular Degeneration

Intervention

Genetic: RGX-314

AAV8 vector containing a transgene for anti-VEGF Fab

Study Arms

• No Intervention: Main Observational Study
  All subjects that previously received RGX-314 in a subretinal administration parent study are enrolled into this arm.
• Experimental: RGX-314 Fellow Eye Treatment Substudy
  RGX-314 Fellow Eye Treatment
  Intervention: Genetic: RGX-314

• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Enrolling by invitation
• Estimated Enrollment (submitted: March 29, 2023): 865
• Original Estimated Enrollment (submitted: June 24, 2019): 42
• Estimated Study Completion Date: December 2028
• Estimated Primary Completion Date: December 2028 (Final data collection date for primary outcome measure)

Eligibility Criteria

Main Observational Study:

Inclusion Criteria:

1. Able and willing to provide written consent
2. Previously enrolled in a clinical study of RGX-314 and received a single subretinal administration of RGX-314

Exclusions Criteria:

1. None

Fellow Eye Substudy:

Inclusion Criteria

1. Age ≤ 93 years
2. Currently or previously enrolled in the main observational study
3. Diagnosis of bilateral choroidal neovascularization secondary to age-related macular degeneration
4. Active nAMD in the fellow eye
5. BCVA between ≤ 80 and ≥ 20 letters in the fellow eye

Exclusion Criteria:

1. CNV or macular edema in the fellow eye secondary to causes other than nAMD
2. Subfoveal fibrosis or atrophy in the fellow eye
3. Advanced glaucoma or history of secondary glaucoma in the fellow eye or any invasive procedure to treat glaucoma
4. Myocardial infarction, cerebrovascular accident, or transient ischemic attack within the past 6 months
5. History of intraocular surgery in the fellow eye within 12 weeks of screening
6. History of intravitreal therapy, such as intravitreal steroid injection or investigational product, other than an intravitreal therapy for nAMD, in the fellow eye within 6 months of screening

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 50 Years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: United States

Administrative Information

• NCT Number: NCT03999801
• Other Study ID Numbers: RGX-314-5101
• 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

• Plan to Share IPD: No

• Current Responsible Party: AbbVie
• Original Responsible Party: REGENXBIO Inc.
• Current Study Sponsor: AbbVie
• Original Study Sponsor: REGENXBIO Inc.
• Collaborators: Not Provided
• Investigators: Not Provided
• PRS Account: AbbVie
• Verification Date: May 2023