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

Not Applicable

Not yet recruiting

• Conditions: Stargardt Disease
• Interventions: Biological: AAVB-039

• Registration Number: NCT07161544
• Lead Sponsor: AAVantgarde Bio Srl

Brief 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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2025-08-08
• Status Verified: 2025-09
• Study Start: 2025-09-01
• Study First Submitted QC: 2025-09-04
• Last Update Submitted: 2025-09-04
• Study First Posted: 2025-09-08
• Last Update Posted: 2025-09-08
• Primary Completion: 2032-07
• Study Completion: 2032-07-01

Recruitment & Eligibility

• Status: NOT_YET_RECRUITING
• Sex: All
• Target Recruitment: 75

Inclusion Criteria

• Molecular diagnosis of Stargardt disease due to ABCA4 mutation
• Willingness to adhere to protocol per informed consent

Exclusion Criteria

• Unwillingness to meet the requirements of the study
• Participation in a clinical study with another Investigation Medicinal Product
• Previous participation in another gene or cell therapy trial
• Any condition that would preclude subretinal surgery
• Complicating ocular and/or systemic diseases

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SEQUENTIAL

Arm && Interventions

Group	Intervention	Description
Cohort 1	AAVB-039	AAVB-039 dose level 1 (low dose)
Cohort 2	AAVB-039	AAVB-039 dose level 2 (mid dose)
Cohort 3	AAVB-039	AAVB-039 dose level 3 (high dose)
Cohorts 4-6	AAVB-039	AAVB-039 dose level 1, 2 or 3

Primary Outcome Measures

Name	Time	Method
To measure the number and severity of treatment related adverse events following treatment with AAVB-039	0-60 months

Secondary Outcome Measures

Name	Time	Method
Change in ellipsoid zone (EZ) loss following treatment with AAVB-039	0-60 months
Change in the area of atrophy (assessed via short wavelength fundus autofluorescence (SW-FAF), following treatment with AAVB-039	0-60 months
Change in macular sensitivity assessed via microperimetry following treatment with AAVB-039	0-60 months
Change in visual acuity following treatment with AAVB-039	0-60 months

Trial Locations

Locations (2)

Retina Consultants of Texas; 🇺🇸 Bellaire, Texas, United States

Retina Foundation of the Southwest; 🇺🇸 Dallas, Texas, United States