Clinical trial NCT03999801: 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
Study type: INTERVENTIONAL
Overall status: ENROLLING_BY_INVITATION
Phase: PHASE2
Conditions: Neovascular Age-related Macular Degeneration
Wet Macular Degeneration
Keywords: gene therapy
wet AMD
nAMD
Lead sponsor: AbbVie
Sponsor class: INDUSTRY
First posted: 2019-06-27
Enrollment: 865
Sex: ALL
Minimum age (years): 50
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SINGLE_GROUP
Intervention model description: 1 main Observational study arm, 1 RGX-314 Fellow Eye Treatment substudy
Primary purpose: TREATMENT

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.


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

Primary outcomes:
measure: Incidence of ocular adverse events and any serious adverse events in the study eye
timeFrame: 5 years inclusive of parent study
measure: Fellow eye substudy: Incidence of ocular adverse events and any serious adverse events in the fellow eye
timeFrame: 54 weeks
Secondary outcomes:
measure: Change from baseline in best corrected visual acuity (BCVA) in the study eye
timeFrame: 5 years inclusive of parent study
measure: Change from baseline in Central Retinal Thickness (CRT) in the study eye
timeFrame: 5 years inclusive of parent study
measure: Number of supplemental anti-VEGF injections in the study eye
timeFrame: 5 years inclusive of parent study
measure: Number of retinal specialist visits for the study eye
timeFrame: 5 years inclusive of parent study
measure: Fellow eye substudy: Change from baseline in best corrected visual acuity
timeFrame: 54 weeks
measure: Fellow eye substudy: Change from baseline in Central Retinal Thickness in the fellow eye
timeFrame: 54 weeks
measure: Fellow eye substudy: Number of supplemental anti-VEGF injections in the fellow eye
timeFrame: 54 weeks
measure: Fellow eye substudy: Aqueous and Serum RGX-314 transgene product (TP) concentrations in the fellow eye
timeFrame: 54 weeks
measure: Fellow eye substudy: Immunogenicity measurements in the fellow eye
timeFrame: 54 weeks
Other outcomes:
Locations:
city: Phoenix
country: United States
facility: Retinal Research Institute, LCC
state: Arizona
city: Santa Barbara
country: United States
facility: California Retina Consultants CRC
state: California
city: Baltimore
country: United States
facility: Johns Hopkins University
state: Maryland
city: Boston
country: United States
facility: Ophthalmic Consultants of Boston
state: Massachusetts
city: Reno
country: United States
facility: Sierra Eye Associates
state: Nevada
city: Philadelphia
country: United States
facility: University of Pennsylvania Department of Ophthalmology
state: Pennsylvania
city: Philadelphia
country: United States
facility: MidAtlantic Retina
state: Pennsylvania
city: Germantown
country: United States
facility: Charles Retina Institute
state: Tennessee
city: The Woodlands
country: United States
facility: Retina Consultants of Texas Research Center
state: Texas
Study officials:
References: