Stargardt DiseasePhase 1/2CELESTENCT07161544
A Study of AAVB-039 in Participants With Stargardt Disease (STGD1)
A Phase 1/2 study testing AAVB-039 in people with Stargardt Disease, run by AAVantgarde Bio Srl at 11 sites in 3 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 8 to 55 years
- Conditions the study is for
- Stargardt Disease
- What is being tested?
- AAVB-039
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 75 participants
- Where
- 11 sitesin 3 countries
- Study dates
- 29 Sep 2025to Jul 2032
- Phase
- Phase 1/2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 8 to 55 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Molecular diagnosis of Stargardt disease due to ABCA4 mutation
- Willingness to adhere to protocol per informed consent
Who cannot join
- 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 locations and contacts
11 sites in 3 countries. Contact the study team or a site near you to ask about taking part.
11 sites in 3 countries
United States8 sites, 8 recruiting
- Recruiting
Retina Consultants of Texas
Bellaire, Texas, 77401
Dr. Kenneth Fan
- Recruiting
Retina Foundation of the Southwest
Dallas, Texas, 75231
Senior Clinical Research Coordinator, jclark@retinafoundation.org
Dr. Karl Csaky
- Recruiting
University of Florida Health Science Center
Jacksonville, Florida, 32209
G. Shabbir Hamdani, Shabbir.hamdani@ufhealth.org
Dr. Sandeep Grover
- Recruiting
Children's Hospital Los Angeles
Los Angeles, California, 90027
Hillary Schwartz-Gale, hschwartz@chla.usc.edu
Dr. Aaron Nagiel
- Recruiting
Retina Vitreous Associates Medical Group
Los Angeles, California, 90211
Kristina Passoian, Trials Director, Kpassoian@laretina.com
Study Referrals, Studyreferrals@laretina.com
- Recruiting
Associated Retina Consultants
Phoenix, Arizona, 85020
Study Team, hello@doctrials.com
Dr. Benjamin Bakall
- Recruiting
Mayo Clinic
Rochester, Minnesota, 55905
Suzy Wernimont, 507-538-8119, Wernimont.Suzanne@mayo.edu
Dr. Brittni Scruggs
- Recruiting
University of Utah
Salt Lake City, Utah, 84112
Kimberley Wegner Lead Coordinator, Kimberley.wegner@hsc.utah.edu
Dr. Paul Bernstein
United Kingdom2 sites, 2 recruiting
- Recruiting
Moorfields Eye Hospital NHS Foundation Trust
London, England, EC1V 2PD
Mithusa Pirabakaran, m.pirabakaran@nhs.net
Prof. Michel Michaelides
- Recruiting
The Retina Clinic
London, England, W1G 9AY
ctenquiries@theretinacliniclondon.com
Prof. Paulo-Eduardo Stanga
Norway1 site, 1 recruiting
- Recruiting
Oslo University Hospital
Oslo, Kirkeveien 166
Rachel Eilertsen, (+47) 23034387, racei1@ous-hf.no
Prof. Josephine Prener Holtan
About this study
Official title: 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)
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
- Being tested
- AAVB-039
- Keywords
- Stargardt Disease, Biallelic Mutation, Gene Mutation, ABCA4
- Medical subject headings
- Stargardt Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AAVB-039Biological
Single subretinal administration
| Study group | Role | What they receive |
|---|---|---|
| Cohort 1 | Experimental | AAVB-039 dose level 1 (low dose) Receives: Biological: AAVB-039 |
| Cohort 2 | Experimental | AAVB-039 dose level 2 (mid dose) Receives: Biological: AAVB-039 |
| Cohort 3 | Experimental | AAVB-039 dose level 3 (high dose) Receives: Biological: AAVB-039 |
| Cohorts 4-5 | Experimental | AAVB-039 dose level 1, 2 or 3 Receives: Biological: AAVB-039 |
What is being measured
The primary outcome is the main question the study is designed to answer.
To measure the number and severity of treatment related adverse events following treatment with AAVB-039
Measured: 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
Measured: 0-60 months
Change in ellipsoid zone (EZ) loss following treatment with AAVB-039
Measured: 0-60 months
Change in macular sensitivity assessed via microperimetry following treatment with AAVB-039
Measured: 0-60 months
Change in visual acuity following treatment with AAVB-039
Measured: 0-60 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Sep 2025
- Study start29 Sep 2025
- Primary completionJul 2032 (estimated)
- Study completionJul 2032 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Stargardt DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 75 participants (estimated)
Sponsor and investigators
- Lead sponsor
- AAVantgarde Bio Srl
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- AAVantgarde Bio UK Ltd
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07161544
- Sponsor's protocol ID
- 039-101
- Acronym
- CELESTE
- Status last verified
- Sep 2026
- First submitted
- 8 Aug 2025
- Last updated
- 30 Sep 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 30 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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)
- ClinicalTrials.gov ID
- NCT07161544
- Acronym / sponsor ID
- CELESTE / 039-101
- Conditions
- Stargardt Disease
- Keywords
- Stargardt Disease, Biallelic Mutation, Gene Mutation, ABCA4
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 75 participants (estimated)
- Lead sponsor
- AAVantgarde Bio SrlIndustry (pharma, biotech, devices)
- Collaborators
- AAVantgarde Bio UK Ltd
- Study start
- 29 Sep 2025 (actual)
- Primary completion
- Jul 2032 (estimated)
- Study completion
- Jul 2032 (estimated)
- First posted
- 8 Sep 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| Cohort 1 | Experimental | Biological: AAVB-039AAVB-039 dose level 1 (low dose) |
| Cohort 2 | Experimental | Biological: AAVB-039AAVB-039 dose level 2 (mid dose) |
| Cohort 3 | Experimental | Biological: AAVB-039AAVB-039 dose level 3 (high dose) |
| Cohorts 4-5 | Experimental | Biological: AAVB-039AAVB-039 dose level 1, 2 or 3 |
Who may qualify
- Ages
- 8 to 55 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (2 listed)
- Molecular diagnosis of Stargardt disease due to ABCA4 mutation
- Willingness to adhere to protocol per informed consent
Key exclusion criteria (5 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| AAVB-039Single subretinal administration | Biological | Cohort 1, Cohort 2, Cohort 3, Cohorts 4-5 |
Primary outcome
To measure the number and severity of treatment related adverse events following treatment with AAVB-039
Time frame: 0-60 months
4 secondary outcomes
Change in the area of atrophy (assessed via short wavelength fundus autofluorescence (SW-FAF), following treatment with AAVB-039
Time frame: 0-60 months
Change in ellipsoid zone (EZ) loss following treatment with AAVB-039
Time frame: 0-60 months
Change in macular sensitivity assessed via microperimetry following treatment with AAVB-039
Time frame: 0-60 months
Change in visual acuity following treatment with AAVB-039
Time frame: 0-60 months
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 30 Sep 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
11 sites in 3 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing AAVB-039 in people with Stargardt Disease, run by AAVantgarde Bio Srl at 11 sites in 3 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 2 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 30 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 11 sites in United States, United Kingdom, Norway. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact AAVantgarde Clinical Operations Lead (+448000465680, clinicaltrials@aavantgarde.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
To measure the number and severity of treatment related adverse events following treatment with AAVB-039 (time frame: 0-60 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 30 Sep 2026.