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

Trial statusRecruiting
Trial phasePhase 3
Trial typeInterventional
Biological sexAll
Age50-89
SponsorAbbVie

About this trial

ABBV-RGX-314 (also known as RGX-314 and surabgene lomparvovec (sura-vec)) is being developed as a novel one-time gene therapy for the treatment of neovascular (wet) age-related macular degeneration (wet AMD). Wet AMD is characterized by loss of vision due to new, leaky blood vessel formation in the retina. Wet AMD is a significant cause of vision loss in the United States, Europe and Japan, with up to 2 million people living with wet AMD in these geographies alone. Current anti-vascular endothelial growth factor (VEGF) therapies have significantly changed the landscape for treatment of wet AMD, becoming the standard of care due to their ability to prevent progression of vision loss in the majority of patients. These therapies, however, require life-long intraocular injections, typically repeated every four to 12 weeks in frequency, to maintain efficacy. Due to the burden of treatment, patients often experience a decline in vision with reduced frequency of treatment over time. ABBV-RGX-314 is being developed as a potential one-time treatment for wet AMD.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

Age ≥ 50 years and ≤ 89 years

An ETDRS BCVA letter score between ≤ 78 and ≥ 40 in the study eye

Diagnosis of subfoveal choroidal neovascularization (CNV) secondary to AMD in the study eye previously treated with anti-VEGF

Must be pseudophakic (at least 12 weeks postcataract surgery) in the study eye

Disqualifiers

CNV or macular edema in the study eye secondary to any causes other than AMD

Subfoveal fibrosis or atrophy in the study eye

Any condition in the investigator's opinion that could limit VA improvement in the study eye

Advanced glaucoma or history of secondary glaucoma in the study eye

Trial design

Design model

Parallel

Treatments tested in this trial

  • ABBV-RGX-314 Dose 1

    Genetic

    AAV8 vector containing a transgene for anti-VEGF Fab (Dose 1)

  • ABBV-RGX-314 Dose 2

    Genetic

    AAV8 vector containing a transgene for anti-VEGF Fab (Dose 2)

  • Aflibercept (EYLEA®)

    Biological/Vaccine

    2.0 mg (0.05 mLsolution) administered by intravitreal injection approximately every 8 weeks after 3 monthly injections

Treatment groups

714 Participants
are divided into 3 treatment groups
Group A: ABBV-RGX-314 Dose 1Experimental treatment 1 intervention
Group B: ABBV-RGX-314 Dose 2Experimental treatment 1 intervention
Group C: Control ArmActive comparator 1 intervention

Trial outcomes

Primary outcomes

1

Mean change from baseline in Best Corrected Visual Acuity (BCVA)

To evaluate the noninferiority of ABBV-RGX-314 relative to aflibercept in mean change from Baseline BCVA at Week 54

Time frame
At Week 54
2

Bilateral Treatment Substudy: Incidence of ocular AEs and any SAEs

AEs and SAEs through Week 50

Time frame
Week 50

Secondary outcomes

1

Incidences of ocular and overall AEs

To evaluate the safety and tolerability of ABBV-RGX-314 through Week 54

Time frame
Through Week 54
2

Proportion of participants with improved BCVA

To evaluate the effect of ABBV-RGX-314 relative to aflibercept on BCVA

Time frame
Week 54
3

Proportion of participants with worsened BCVA

To evaluate the effect of ABBV-RGX-314 relative to aflibercept on BCVA

Time frame
Week 54
4

Proportion of participants (1) gaining or losing greater than 0 letters; (2) maintaining vision compared with baseline as per BCVA

To evaluate the effect of ABBV-RGX-314 relative to aflibercept on BCVA

Time frame
Week 54

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

AbbVie

Lead sponsor

REGENXBIO Inc.

Collaborator