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

Phase 2

• Conditions: Neovascular Age-related Macular Degeneration; Wet Macular Degeneration
• Interventions: Genetic: RGX-314

• Registration Number: NCT03999801
• Lead Sponsor: AbbVie

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 available

Basic Information
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Recruitment & Eligibility
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Study & Design
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Trial Locations
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Related News

Study Timeline

• Study Start: 2019-05-31
• Study First Submitted: 2019-06-18
• Study First Submitted QC: 2019-06-24
• Study First Posted: 2019-06-27
• Status Verified: 2024-06
• Last Update Submitted: 2024-06-14
• Last Update Posted: 2024-06-17
• Primary Completion: 2028-12
• Study Completion: 2028-12

Recruitment & Eligibility

• Status: ENROLLING_BY_INVITATION
• Sex: All
• Target Recruitment: 865

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

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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

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
RGX-314 Fellow Eye Treatment Substudy	RGX-314	RGX-314 Fellow Eye Treatment

Primary Outcome Measures

Name	Time	Method
Fellow eye substudy: Incidence of ocular adverse events and any serious adverse events in the fellow eye	54 weeks
Incidence of ocular adverse events and any serious adverse events in the study eye	5 years inclusive of parent study

Secondary Outcome Measures

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

Trial Locations

Locations (9)

Retinal Research Institute, LCC; 🇺🇸 Phoenix, Arizona, United States

Sierra Eye Associates; 🇺🇸 Reno, Nevada, United States

MidAtlantic Retina; 🇺🇸 Philadelphia, Pennsylvania, United States

California Retina Consultants CRC; 🇺🇸 Santa Barbara, California, United States

Johns Hopkins University; 🇺🇸 Baltimore, Maryland, United States

Ophthalmic Consultants of Boston; 🇺🇸 Boston, Massachusetts, United States

University of Pennsylvania Department of Ophthalmology; 🇺🇸 Philadelphia, Pennsylvania, United States

Retina Consultants of Texas Research Center; 🇺🇸 The Woodlands, Texas, United States

Charles Retina Institute; 🇺🇸 Germantown, Tennessee, United States