Contact SCGE




Gene Therapy Trial Report

Summary

Pivotal 1 Study of ABBV-RGX-314 (Also Known as RGX-314) Gene Therapy Administered Via Subretinal Delivery One Time in Participants With nAMD


NCTID NCT04704921 (View at clinicaltrials.gov)
Description
Development Status Active
Indication Neovascular (Wet) Age-Related Macular Degeneration (nAMD)
Disease Ontology Term DOID:10871
Compound Name ABBV-RGX-314
Compound Description antibody similar to ranibizumab, VEGF antagonist
Sponsor AbbVie
Funder Type Industry
Recruitment Status
Enrollment Count 630 (ESTIMATED)
Results Posted Not Available

Therapy Information


Target Gene/Variant Anti-VEGF
Therapy Type Gene transfer
Therapy Route In-vivo
Mechanism of Action Genetic delivery of therapeutic protein
Route of Administration Subretinal
Drug Product Type Viral vector
Delivery System Viral transduction
Vector Type AAV8
Editor Type none
Dose 1 2.5E11 GC/eye
Dose 2 5.0E11 GC/eye
Dose 3 1.0E12 GC/eye
Dose 4 1.5E12 GC/eye (suprachoroidal delivery)

Study Record Dates


Current Stage Phase2, Phase3
Submit Date 2021-01-08
Completion Date 2027-11
Last Update 2026-02-17

Participation Criteria


Eligible Age 50 Years - 89 Years
Standard Ages Adult, Older adult
Sexes Eligible for Study ALL
Eligibility Criteria
Inclusion 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 Inclusion Criteria (Bilateral Treatment Substudy)\*: 1. An ETDRS BCVA letter score between ≤ 83 and ≥ 40 in both eyes 2. Diagnosis of subfoveal choroidal neovascularization (CNV) secondary to AMD in both eyes 3. Must be pseudophakic (at least 12 weeks postcataract surgery) in both eyes 4. Willing and able to provide written, signed informed consent for this study 5. Newcomers must have active disease in the study eye; crossover participants must have active disease in the eye not treated in the main study Exclusion 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, as determined by CRC 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 that cannot be treated, in the study eye 5. Advanced glaucoma or history of secondary glaucoma in the study eye 6. History of intraocular surgery in the study eye within 12 weeks prior to randomization 7. 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 8. Prior treatment with gene therapy 9. Recent myocardial infarction, cerebrovascular accident, or transient ischemic attack within the past 6 months Exclusion Criteria (Bilateral Treatment Substudy)\*: 1. CNV or macular edema in either eye secondary to any causes other than AMD 2. Subfoveal fibrosis or atrophy in either eye 3. Any condition in the investigator's opinion that could limit VA improvement in either eye 4. Active or history of retinal detachment, or current retinal tear that cannot be treated in either eye 5. Advanced glaucoma or history of secondary glaucoma in either eye 6. Myocardial infarction, cerebrovascular accident, or transient ischemic attack within the past 6 months 7. History of intraocular surgery in either eye within 12 weeks prior to randomization (Week -2) 8. History of intravitreal therapy in either eye, such as intravitreal steroid injection or investigational product, other than anti-VEGF therapy, in the 6 months prior to screening 9. Prior treatment with gene therapy (\*) For previously treated crossover participants, criteria apply to the eye not treated in the main study only.
View Inclusion and Exclusion Criteria at ClinicalTrials.gov

Locations


No.of Trial Sites 89
Locations United States

Regulatory Information


Has US IND True
FDA Designations
Recent Updates Pivotal data expected 2026

Resources/Links