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

AAVantgarde Bio Srl

Status and phase: Enrolling; Phase 2; Phase 1

Conditions: Stargardt Disease

Treatments: Biological: AAVB-039

Study type: Interventional

Funder types: Industry; Other

Identifiers

NCT07161544

039-101

Details and patient eligibility

About

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.

Enrollment: 75 estimated patients

Sex: All

Ages: 8 to 55 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Sequential Assignment

Masking: None (Open label)

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75 participants in 4 patient groups

Cohort 1

Experimental group

Description:

AAVB-039 dose level 1 (low dose)

Treatment:

Biological: AAVB-039

Cohort 2

Experimental group

Description:

AAVB-039 dose level 2 (mid dose)

Treatment:

Biological: AAVB-039

Cohort 3

Experimental group

Description:

AAVB-039 dose level 3 (high dose)

Treatment:

Biological: AAVB-039

Cohorts 4-5

Experimental group

Description:

AAVB-039 dose level 1, 2 or 3

Treatment:

Biological: AAVB-039