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Enrolling by Invitation

NCT Number: NCT03999801

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

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.

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Key information

Conditions

Neovascular Age-related Macular Degeneration Eye Diseases Macular Degeneration Retinal Degeneration Retinal Diseases Wet Macular Degeneration

Age range

50 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Retinal Research Institute, LCC, Phoenix, Arizona, United States

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Main Observational Study:

Inclusion criteria

  • 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

Exclusions Criteria:

  • None

Fellow Eye Substudy:

Inclusion criteria

  • 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

Exclusion criteria

  • 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

Treatment and study plan

RGX-314

Genetic

AAV8 vector containing a transgene for anti-VEGF Fab

Primary outcomes

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

    Time frame: 5 years inclusive of parent study

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

    Time frame: 54 weeks

Secondary outcomes

  1. 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

  2. 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)

  3. 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

  4. 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

  5. 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

  6. 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

  7. 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

  8. 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

  9. Fellow eye substudy: Immunogenicity measurements in the fellow eye

    Time frame: 54 weeks

    Fellow eye immunogenicity measurements at assessed timepoints

Sponsors and collaborators

Lead sponsor

AbbVie

Industry

Registry information

Official study 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

Acronym: RGX-314 SRLTFU

Important dates

Study start
2019
Primary completion
2028
Study completion
2028
First posted
Jun 27, 2019
Registry last updated
Jun 17, 2024

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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