NCT05197270

4D-150 in Patients With Neovascular (Wet) Age-Related Macular Degeneration

A Phase 1/2 Dose-Escalation and Randomized, Controlled, Masked Expansion Trial of Intravitreal 4D-150 Gene Therapy in Adults With Neovascular (Wet) Age-Related Macular Degeneration

• Status: Recruiting
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 215 (estimated)
• Sponsor: 4D Molecular Therapeutics · Industry
• Sex: All
• Age: 50 Years
• Healthy volunteers: Not accepted

Summary

Phase 1/2 dose-escalation and randomized, controlled, masked expansion trial in adults with wet AMD undergoing active anti-VEGF treatment. Substudies will evaluate the safety and tolerability of 4D-150 contralateral eye dosing and characterize vector shedding.

Detailed description

This Phase 1/2 trial is a prospective, multicenter, Phase 1/2 dose-escalation and randomized, controlled, masked expansion trial in adults with wet AMD undergoing active anti-VEGF treatment who have demonstrated a clinical response consistent with anti-VEGF activity. The trial consists of Dose Escalation, Dose Expansion, Steroid Optimization, and\[DD1.1\]\[JA1.2\] Population Extension Cohorts. After receiving one time administration of 4D-150 by intravitreal injection (IVT), subjects will undergo assessments at monthly intervals for 24 months to assess safety and efficacy outcomes. Only subjects that received 4D-150 will then enter a long-term follow-up (LTFU) period to assess long-term safety of 4D-150 gene therapy and duration of clinical activity through year 5 (60 months). Eligible subjects who received 4D-150 in the study eye may participate in a sub-study to evaluate one-time IVT administration of 4D-150 to the contralateral ("fellow") eye. Additional subjects will be enrolled in a separate sub-study to characterize the vector shedding profile of one-time administration of IVT 4D-150. In both sub-studies, subjects will undergo regular assessments to assess safety and tolerability through Week 52 and then will enter long-term follow-up for safety evaluation through year 5 (60 months).

Conditions

• Neovascular (Wet) Age-Related Macular Degeneration

Interventions

Type	Name	Description
BIOLOGICAL	4D-150 IVT	4D-150: AAV-based gene therapy comprised of miRNA targeting VEGF-C and codon-optimized sequence encoding aflibercept
BIOLOGICAL	Aflibercept IVT	Commercially available Active Comparator Other Name: Eylea

Timeline

• Start date: 2021-12-09
• Primary completion: 2027-03-01
• Completion: 2031-01-01
• First posted: 2022-01-19
• Last updated: 2026-02-12

Locations

25 sites across 2 countries: United States, Puerto Rico

Regulatory

• FDA-regulated drug study