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

Phases

Phase 1Phase 2

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT07161544

Acronym

CELESTE

Enrollment

Registered

2025-09-08

Start date

2025-09-29

Completion date

2032-07-01

Last updated

2026-07-30

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Stargardt Disease

Keywords

Stargardt Disease, Biallelic Mutation, Gene Mutation, ABCA4

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.

Interventions

BIOLOGICALAAVB-039

Single subretinal administration

Study design

Allocation

NON_RANDOMIZED

Intervention model

SEQUENTIAL

Primary purpose

TREATMENT

Masking

NONE

Eligibility

Sex/Gender

ALL

Age

8 Years to 55 Years

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

Design outcomes

Primary

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

Secondary

Measure	Time frame
Change in the area of atrophy (assessed via short wavelength fundus autofluorescence (SW-FAF), following treatment with AAVB-039	0-60 months
Change in ellipsoid zone (EZ) loss 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

Countries

United Kingdom, United States

Contacts

CONTACTAAVantgarde Clinical Operations Lead

clinicaltrials@aavantgarde.com+448000465680

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Jul 31, 2026