NCT04832724

RGX-314 Gene Therapy Pharmacodynamic Study for Neovascular Age-related Macular Degeneration (nAMD)

Completed Phase 2 Last updated 5 September 2024

What this trial tests

Phase 2 trial testing RGX-314 in Neovascular Age-related Macular Degeneration in 60 participants. Completed in 18 March 2024.

Timeline

22 February 2021

Primary endpoint
2 October 2023

18 March 2024

Quick facts

Lead sponsor	AbbVie
Phase	Phase 2
Status	Completed
Study type	INTERVENTIONAL
Allocation	non randomized
Design	sequential
Masking	none
Primary purpose	treatment
Enrollment	60
Start date	22 February 2021
Primary completion	2 October 2023
Estimated completion	18 March 2024
Sites	13 locations across United States

Drugs / interventions tested

• RGX-314

Conditions studied

• Neovascular Age-related Macular Degeneration — all drugs for Neovascular Age-related Macular Degeneration →
• Wet Macular Degeneration — all drugs for Wet Macular Degeneration →
• Wet Age-related Macular Degeneration — all drugs for Wet Age-related Macular Degeneration →

Sponsor

AbbVie — full company profile →

Who can join

Adults 50 to 89, any sex, with Neovascular Age-related Macular Degeneration or Wet Macular Degeneration. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

RGX-314 is being developed as a novel one-time gene therapy for the treatment of neovascular (wet) age-related macular degeneration (nAMD) also referred to as Wet AMD. Wet AMD is characterized by loss of vision due to new, leaky blood vessel formation in the retina. The purpose of this phase 2, open label study is to evaluate whether different doses of RGX-314 from two different formulations (clinical versus eventual commercial formulation) perform the same in humans when delivered by subretinal administration

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

1. Gene Therapy for Retinal Degenerative Diseases: Progress, Challenges, and Future Directions.
   Drag S, Dotiwala F, Upadhyay AK. · · 2023 · cited 88× · PMID 37389545 · DOI 10.1167/iovs.64.7.39
2. Emerging therapeutic strategies for unmet need in neovascular age-related macular degeneration.
   Khachigian LM, Liew G, Teo KYC, Wong TY, et al · · 2023 · cited 61× · PMID 36810060 · DOI 10.1186/s12967-023-03937-7
3. Exudative versus Nonexudative Age-Related Macular Degeneration: Physiopathology and Treatment Options.
   Fernandes AR, Zielińska A, Sanchez-Lopez E, Dos Santos T, et al · · 2022 · cited 50× · PMID 35269743 · DOI 10.3390/ijms23052592
4. Update on Viral Gene Therapy Clinical Trials for Retinal Diseases.
   Cheng SY, Punzo C. · · 2022 · cited 42× · PMID 36074935 · DOI 10.1089/hum.2022.159
5. AAV for Gene Therapy in Ocular Diseases: Progress and Prospects.
   He X, Fu Y, Ma L, Yao Y, et al · · 2023 · cited 37× · PMID 38188726 · DOI 10.34133/research.0291
6. Twenty Years of Anti-Vascular Endothelial Growth Factor Therapeutics in Neovascular Age-Related Macular Degeneration Treatment.
   Moon BH, Kim Y, Kim SY. · · 2023 · cited 35× · PMID 37629185 · DOI 10.3390/ijms241613004
7. Ocular Drug Delivery: Advancements and Innovations.
   Tian B, Bilsbury E, Doherty S, Teebagy S, et al · · 2022 · cited 28× · PMID 36145679 · DOI 10.3390/pharmaceutics14091931
8. Gene-Based Therapeutics for Acquired Retinal Disease: Opportunities and Progress.
   Tan TE, Fenner BJ, Barathi VA, Tun SBB, et al · · 2021 · cited 20× · PMID 34950193 · DOI 10.3389/fgene.2021.795010