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

Официальное название

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

Обзор

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.

Вмешательства

• Генная терапия RGX-314
  AAV8 vector containing a transgene for anti-VEGF Fab

Первичные конечные точки

• Incidence of ocular adverse events and any serious adverse events in the study eye [Срок оценки: 5 years inclusive of parent study]
• Fellow eye substudy: Incidence of ocular adverse events and any serious adverse events in the fellow eye [Срок оценки: 54 weeks]

Вторичные конечные точки (9)

• Change from baseline in best corrected visual acuity (BCVA) in the study eye [Срок оценки: 5 years inclusive of parent study]
• Change from baseline in Central Retinal Thickness (CRT) in the study eye [Срок оценки: 5 years inclusive of parent study]
• Number of supplemental anti-VEGF injections in the study eye [Срок оценки: 5 years inclusive of parent study]
• Number of retinal specialist visits for the study eye [Срок оценки: 5 years inclusive of parent study]
• Fellow eye substudy: Change from baseline in best corrected visual acuity [Срок оценки: 54 weeks]
• Fellow eye substudy: Change from baseline in Central Retinal Thickness in the fellow eye [Срок оценки: 54 weeks]
• Fellow eye substudy: Number of supplemental anti-VEGF injections in the fellow eye [Срок оценки: 54 weeks]
• Fellow eye substudy: Aqueous and Serum RGX-314 transgene product (TP) concentrations in the fellow eye [Срок оценки: 54 weeks]
• Fellow eye substudy: Immunogenicity measurements in the fellow eye [Срок оценки: 54 weeks]

Критерии участия

Main Observational Study:

Критерии включения

• 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:

1\. None

Fellow Eye Substudy:

Критерии включения

• 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

Критерии исключения

• 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

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Распределение

Нерандомизированное

Модель

Одна группа

Маскирование

Открытое

Основная цель

Лечение

Центры проведения

США · 9 центров

• Retinal Research Institute, LCC — Phoenix
• California Retina Consultants CRC — Santa Barbara
• Johns Hopkins University — Baltimore
• Ophthalmic Consultants of Boston — Boston
• Sierra Eye Associates — Reno
• University of Pennsylvania Department of Ophthalmology — Philadelphia
• MidAtlantic Retina — Philadelphia
• Charles Retina Institute — Germantown
• … и ещё 1 центр

Идентификаторы

NCT: NCT03999801 · RGX-314-5101

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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