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

AbbVie

Status and phase: Invitation-only; Phase 2

Conditions: Wet Macular Degeneration; Neovascular Age-related Macular Degeneration

Treatments: Genetic: RGX-314

Study type: Interventional

Funder types: Industry

Identifiers

NCT03999801

RGX-314-5101

Details and patient eligibility

About

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.

Enrollment: 865 estimated patients

Sex: All

Ages: 50+ years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion 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

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Single Group Assignment

Masking: None (Open label)

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865 participants in 2 patient groups

Main Observational Study

No Intervention group

Description:

All subjects that previously received RGX-314 in a subretinal administration parent study are enrolled into this arm.

RGX-314 Fellow Eye Treatment Substudy

Experimental group

Description:

RGX-314 Fellow Eye Treatment

Treatment:

Genetic: RGX-314