A Study of AAVB-039 in Participants With Stargardt Disease (STGD1)
CELESTE
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)
1 other identifier
interventional
75
2 countries
6
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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P75+ for phase_1
Started Sep 2025
Longer than P75 for phase_1
6 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
August 8, 2025
CompletedFirst Posted
Study publicly available on registry
September 8, 2025
CompletedStudy Start
First participant enrolled
September 29, 2025
CompletedPrimary Completion
Last participant's last visit for primary outcome
July 1, 2032
ExpectedStudy Completion
Last participant's last visit for all outcomes
July 1, 2032
March 9, 2026
March 1, 2026
6.8 years
August 8, 2025
March 6, 2026
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
To measure the number and severity of treatment related adverse events following treatment with AAVB-039
0-60 months
Secondary Outcomes (4)
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
Study Arms (4)
Cohort 1
EXPERIMENTALAAVB-039 dose level 1 (low dose)
Cohort 2
EXPERIMENTALAAVB-039 dose level 2 (mid dose)
Cohort 3
EXPERIMENTALAAVB-039 dose level 3 (high dose)
Cohorts 4-5
EXPERIMENTALAAVB-039 dose level 1, 2 or 3
Interventions
Eligibility Criteria
You may qualify if:
- Molecular diagnosis of Stargardt disease due to ABCA4 mutation
- Willingness to adhere to protocol per informed consent
You may not qualify if:
- 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
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- AAVantgarde Bio UK Ltdcollaborator
- AAVantgarde Bio Srllead
Study Sites (6)
Retina Vitreous Associates Medical Group
Los Angeles, California, 90211, United States
Mayo Clinic
Rochester, Minnesota, 55905, United States
Retina Consultants of Texas
Bellaire, Texas, 77401, United States
Retina Foundation of the Southwest
Dallas, Texas, 75231, United States
Moorfields Eye Hospital NHS Foundation Trust
London, England, EC1V 2PD, United Kingdom
The Retina Clinic
London, England, W1G 9AX, United Kingdom
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Central Study Contacts
Study Design
- Study Type
- interventional
- Phase
- phase 1
- Allocation
- NON RANDOMIZED
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SEQUENTIAL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
August 8, 2025
First Posted
September 8, 2025
Study Start
September 29, 2025
Primary Completion (Estimated)
July 1, 2032
Study Completion (Estimated)
July 1, 2032
Last Updated
March 9, 2026
Record last verified: 2026-03
Data Sharing
- IPD Sharing
- Will not share