Not currently recruiting at UC Davis

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

a study on Macular Degeneration WetAMD Copy Number Variation

Summary

Eligibility
for people ages 50-89 (full criteria)
Location
at Sacramento, California and other locations
Dates
study started
study ends around

Description

Summary

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.

Official Title

A Randomized, Partially Masked, Controlled, Phase 3 Clinical Study to Evaluate the Efficacy and Safety of RGX-314 Gene Therapy in Participants With nAMD

Details

This randomized, partially masked, controlled, Phase 3 clinical study will evaluate the efficacy and safety of ABBV-RGX-314 gene therapy in participants with nAMD. The study will evaluate 2 dose levels of RGX-314 gene therapy relative to an active comparator. The primary endpoint of this study is mean change in best-corrected visual acuity (BCVA) of ABBV-RGX-314 relative to aflibercept. Approximately 714 participants who meet the inclusion/exclusion criteria, will be enrolled into one of 3 arms.

A bilateral treatment substudy conducted at US sites is an open-label, partially randomized, parallel arm study to evaluate the safety and efficacy of subretinal ABBV-RGX-314 administered bilaterally in participants who have bilateral nAMD. Previously treated crossover participants from the control arm of the main study who crossed over and received ABBV-RGX-314 in the study eye will receive the same ABBV-RGX-314 dose in the contralateral eye (ie, same dose as in the study eye), while newcomers (participants who have not been randomized in an ABBV-RGX-314 study) and untreated crossover participants (ongoing control participants in the main study who have completed Week 54 but have not crossed over to receive ABBV-RGX-314 in the main study) will be randomized in a 2:1 ratio to receive ABBV-RGX-314 Dose 1 or ABBV-RGX-314 Dose 2 in both eyes. Up to 15 participants who qualify for the substudy will be enrolled and followed for a minimum of 50 weeks.

Keywords

AMD, nAMD, Wet Age-related Macular Degeneration, wAMD, WetAMD, CNV, Age-Related Macular Degeneration, Neovascular Age-Related Macular Degeneration, Macular Degeneration, Wet Macular Degeneration, Choroidal Neovascularization, Retinal Degeneration, Retinal Diseases, Eye Diseases, Ranibizumab, Aflibercept, Angiogenesis Inhibitors, Angiogenesis Modulating Agents, Growth Substances, Physiological Effects of Drugs, Growth Inhibitors, Antineoplastic Agents, Gene Therapy, Anti-vascular endothelial grown factory therapy, Anti-VEGF therapy, wet AMD, ABBV-RGX-314 Dose 1, ABBV-RGX-314 Dose 2, Aflibercept (EYLEA®)

Eligibility

You can join if…

Open to people ages 50-89

  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 choroidal neovascularization (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

You CAN'T join if...

  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. Advanced glaucoma or history of secondary glaucoma in the study eye
  5. Myocardial infarction, cerebrovascular accident, or transient ischemic attack within the past 6 months
  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 medicinal product, other than an intravitreal therapy for AMD, in the 6 months prior to Week -6
  8. Prior treatment with gene therapy

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. Advanced glaucoma or history of secondary glaucoma in either eye
  5. Myocardial infarction, cerebrovascular accident, or transient ischemic attack within the past 6 months
  6. History of intraocular surgery in either eye within 12 weeks prior to randomization
  7. History of intravitreal therapy in the study eye, such as intravitreal steroid injection or investigational medicinal product, other than an intravitreal therapy for AMD, in the 6 months prior to Week -6.
  8. Prior treatment with gene therapy (*) For previously treated crossover participants, criteria apply to the eye not treated in the main study only.

Note: Other inclusion/exclusion criteria apply

Locations

  • UC Davis Health Eye Center in progress, not accepting new patients
    Sacramento California 95817 United States
  • Retinal Consultants Medical Group, Inc. - Greenback /ID# 256353 in progress, not accepting new patients
    Sacramento California 95841 United States
  • Bay Area Retina Associates - Walnut Creek - Lennon Lane /ID# 268513 accepting new patients
    Walnut Creek California 94598 United States
  • Byers Eye Institute Stanford /ID# 262853 accepting new patients
    Palo Alto California 94303 United States

Details

Status
accepting new patients at some sites,
but this study is not currently recruiting here
Start Date
Completion Date
(estimated)
Sponsor
AbbVie
ID
NCT05407636
Phase
Phase 3 research study
Study Type
Interventional
Participants
Expecting 714 study participants
Last Updated