Pivotal 2 Study of RGX-314 Gene Therapy in Participants With nAMD (ASCENT)

JRCT ID: jRCT2073240017

Registered date:27/05/2024

Basic Information

• Recruitment status: Pending
• Health condition(s) or Problem(s) studied: Neovascular age-related macular degeneration
• Date of first enrollment: 01/06/2024
• Target sample size: 14
• Countries of recruitment: United States,Japan,Puerto Rico,Japan,Canada,Japan,France,Japan,Germany,Japan,Hungary,Japan,Israel,Japan,Italy,Japan,Spain,Japan,United Kingdom,Japan
• Study type: Interventional

Intervention(s)

RGX-314 Dose 1 AAV8 vector containing a transgene for anti-VEGF Fab (Dose1)
RGX-314 Dose 2 AAV8 vector containing a transgene for anti-VEGF Fab (Dose2)
Aflibercept (EYLEA) 2.0 mg (0.05 mLsolution) administered by intravitreal injection approximately every 8 weeks after 3 monthly injections

Outcome(s)

• Primary Outcome: Mean change from baseline in Best Corrected Visual Acuity (BCVA) [Time Frame: At Week 54]

Key inclusion & exclusion criteria

• Age minimum: >= 50age old
• Age maximum: <= 89age old
• Gender: Both

Include criteria

1. Age >= 50 years and <= 89 years
2. An ETDRS BCVA letter score between 78 and 40 in the study eye
3. Diagnosis of subfoveal CNV secondary to AMD in the study eye previously treated with anti-VEGF
4. Must be pseudophakic (at least 12 weeks postcataract surgery) in the study eye.
5. Willing and able to provide written, signed informed consent for this study
6. Participants must have demonstrated a meaningful response to anti-VEGF therapy at study entry

Exclude criteria

1. CNV or macular edema in the study eye secondary to any causes other than AMD
2. Subfoveal fibrosis or atrophy in the study eye
3. Any condition in the investigator's opinion that could limit VA improvement in the study eye
4. Active or history of retinal detachment or current retinal tear in the study eye
5. Advanced glaucoma or history of secondary glaucoma in the study eye
6. Myocardial infarction, cerebrovascular accident, or transient ischemic attach within the past 6 months.
7. History of intraocular surgery in the study eye within 12 weeks prior to Screening Visit 1
8. History of intravitreal therapy in the study eye, such as intravitreal steroid injection or investigational product, other than anti-VEGF therapy, in the 6 months prior to Screening Visit 1.
9. Prior treatment with gene therapy.

Related Information

• Primary Sponsor: Tetsuya Otani
• Secondary Sponsor: REGENXBIO Inc.
• Secondary ID(s): NCT05407636