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

NCT03999801 | Enrolling By Invitation Phase 2 DMC FDA Drug

• 1 other identifier: RGX-314-5101
• Study Type: interventional
• Target: 865
• Locations: 1 country
• Sites: 9

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.

Conditions

Neovascular Age-related Macular Degeneration; Wet Macular Degeneration

Keywords

gene therapy; wet AMD; nAMD

Outcome Measures

Primary Outcomes (2)

• Incidence of ocular adverse events and any serious adverse events in the study eye

  5 years inclusive of parent study

• Fellow eye substudy: Incidence of ocular adverse events and any serious adverse events in the fellow eye

  54 weeks

Secondary Outcomes (9)

• Change from baseline in best corrected visual acuity (BCVA) in the study eye

  5 years inclusive of parent study

• Change from baseline in Central Retinal Thickness (CRT) in the study eye

  5 years inclusive of parent study

• Number of supplemental anti-VEGF injections in the study eye

  5 years inclusive of parent study

• Number of retinal specialist visits for the study eye

  5 years inclusive of parent study

• Fellow eye substudy: Change from baseline in best corrected visual acuity

  54 weeks

Study Arms (2)

Main Observational Study

NO INTERVENTION

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

RGX-314 Fellow Eye Treatment

Genetic: RGX-314

Interventions

RGX-314 GENETIC

AAV8 vector containing a transgene for anti-VEGF Fab

RGX-314 Fellow Eye Treatment Substudy

Eligibility Criteria

• Age: 50 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Able and willing to provide written consent
• Previously enrolled in a clinical study of RGX-314 and received a single subretinal administration of RGX-314
• \. None
• Fellow Eye Substudy:
• Age ≤ 93 years
• Currently or previously enrolled in the main observational study
• Diagnosis of bilateral choroidal neovascularization secondary to age-related macular degeneration
• Active nAMD in the fellow eye
• BCVA between ≤ 80 and ≥ 20 letters in the fellow eye

You may not qualify if:

• CNV or macular edema in the fellow eye secondary to causes other than nAMD
• Subfoveal fibrosis or atrophy in the fellow eye
• Advanced glaucoma or history of secondary glaucoma in the fellow eye or any invasive procedure to treat glaucoma
• Myocardial infarction, cerebrovascular accident, or transient ischemic attack within the past 6 months
• History of intraocular surgery in the fellow eye within 12 weeks of screening
• 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

Sponsors & Collaborators

• AbbVie: lead

Study Sites (9)

Retinal Research Institute, LCC

Phoenix, Arizona, 85053, United States

Location

California Retina Consultants CRC

Santa Barbara, California, 93103, United States

Location

Johns Hopkins University

Baltimore, Maryland, 21287, United States

Location

Ophthalmic Consultants of Boston

Boston, Massachusetts, 02114, United States

Location

Sierra Eye Associates

Reno, Nevada, 89502, United States

Location

University of Pennsylvania Department of Ophthalmology

Philadelphia, Pennsylvania, 19104, United States

Location

MidAtlantic Retina

Philadelphia, Pennsylvania, 19107, United States

Location

Charles Retina Institute

Germantown, Tennessee, 38138, United States

Location

Retina Consultants of Texas Research Center

The Woodlands, Texas, 77384, United States

Location

MeSH Terms

Conditions

Wet Macular Degeneration

Condition Hierarchy (Ancestors)

Macular Degeneration; Retinal Degeneration; Retinal Diseases; Eye Diseases

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

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

Study Record Dates

• First Submitted: June 18, 2019
• First Posted: June 27, 2019
• Study Start: May 31, 2019
• Primary Completion (Estimated): December 1, 2028
• Study Completion (Estimated): December 1, 2028
• Last Updated: June 17, 2024

Record last verified: 2024-06

Data Sharing

• IPD Sharing: Will not share

Locations

US (9)