NCT07161544

A Study of AAVB-039 in Participants With Stargardt Disease (STGD1)

An Open-label, Multicenter, Two Part, Ascending Dose Followed by a Controlled Trial to Assess the Safety and Efficacy of a Subretinal Administration of AAVB-039 in Participants With Stargardt Disease (STGD1) (CELESTE)

• Status: Recruiting
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 75 (estimated)
• Sponsor: AAVantgarde Bio Srl · Industry
• Sex: All
• Age: 8 Years – 55 Years
• Healthy volunteers: Not accepted

Summary

The purpose of the 039-101 study is to evaluate the safety and tolerability of a single subretinal injection of AAVB-039 in participants with Stargardt disease secondary to a biallelic mutation of the ABCA4 gene. The study will also assess initial efficacy following AAVB-039 administration.

Conditions

• Stargardt Disease

Interventions

Type	Name	Description
BIOLOGICAL	AAVB-039	Single subretinal administration

Timeline

• Start date: 2025-09-29
• Primary completion: 2032-07-01
• Completion: 2032-07-01
• First posted: 2025-09-08
• Last updated: 2026-03-09

Locations

6 sites across 2 countries: United States, United Kingdom

Regulatory

• FDA-regulated drug study