Stargardt DiseaseNCT06591806
An Observational Study in Children and Adults With Stargardt Disease
An observational study of Stargardt Disease, run by AAVantgarde Bio Srl at 2 sites in 2 countries.
Trial summary
This multicenter, prospective, longitudinal, observational study in approximately 80 subjects with Stargardt disease secondary to biallelic mutations in the ABCA4 gene (STGD1) aims to evaluate prognostic factors of disease progression, and to further characterize the patient population for future clinical studies. Read more about this study
- Who is it for?
- Ages 8 to 50 years
- Conditions the study is for
- Stargardt Disease
- How many people
- 80 participants
- Where
- 2 sitesin 2 countries
- Study dates
- 1 May 2024to 30 Jun 2027
- Phase
- Observational
- 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 50 years
- All sexes
- No healthy volunteers
- Child, Adult
Study population: Patients with Stargardt disease secondary to mutation in the ABCA4 gene
Who may be able to join
- Male and female subjects between 8 and 50 years of age at the time of enrolment.
- Willingness to adhere to the protocol as evidenced by written informed consent if the subject is 18 years or older. If the subject is under 18 years of age, written assent must be obtained from the subject and written informed consent must be obtained from the subject's legally authorized representative (parent or legal guardian).
- Confirmed mutation in the ABCA4 gene.
Who cannot join
- History of uveitis.
- Any ocular disease in either eye that may confound assessment of the retina morphologically and functionally.
- Any pathology of the posterior segment other than ABCA4 retinopathy.
- Presence of any other genetic mutation(s) that have been associated with retinal or macular dystrophy.
Trial locations and contacts
2 sites in 2 countries.
2 sites in 2 countries
United States1 site
Retina Foundation of the Southwest
Dallas, Texas, 75231
About this study
Official title: A Multicenter, Prospective, Longitudinal, Observational Study in Children and Adults With Stargardt Disease Related Atrophy Secondary to Biallelic Mutations in the ABCA4 Gene
This multicenter, prospective, longitudinal, observational study in approximately 80 subjects with Stargardt disease secondary to biallelic mutations in the ABCA4 gene (STGD1) aims to evaluate prognostic factors of disease progression, and to further characterize the patient population for future clinical studies.
- Conditions
- Stargardt Disease
- Keywords
- ABCA4
- Medical subject headings
- Stargardt Disease
What is being measured
The primary outcome is the main question the study is designed to answer.
To measure the change from baseline in Fundus Autofluorescence
Measured: 24 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Sep 2024
- Study start1 May 2024
- Primary completion30 Jun 2027 (estimated)
- Study completion30 Jun 2027 (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
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 80 participants (estimated)
Sponsor and investigators
- Lead sponsor
- AAVantgarde Bio Srl
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06591806
- Sponsor's protocol ID
- NHS-OBS-01
- Status last verified
- Jun 2026
- First submitted
- 29 Jul 2024
- Last updated
- 16 Jun 2026
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 16 Jun 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multicenter, Prospective, Longitudinal, Observational Study in Children and Adults With Stargardt Disease Related Atrophy Secondary to Biallelic Mutations in the ABCA4 Gene
- ClinicalTrials.gov ID
- NCT06591806
- Acronym / sponsor ID
- NHS-OBS-01
- Conditions
- Stargardt Disease
- Keywords
- ABCA4
- Study type
- Observational
- Recruitment status
- Active, not recruiting
- Enrolment
- 80 participants (estimated)
- Lead sponsor
- AAVantgarde Bio SrlIndustry (pharma, biotech, devices)
- Study start
- 1 May 2024 (actual)
- Primary completion
- 30 Jun 2027 (estimated)
- Study completion
- 30 Jun 2027 (estimated)
- First posted
- 19 Sep 2024 (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.
This multicenter, prospective, longitudinal, observational study in approximately 80 subjects with Stargardt disease secondary to biallelic mutations in the ABCA4 gene (STGD1) aims to evaluate prognostic factors of disease progression, and to further characterize the patient population for future clinical studies.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
Who may qualify
- Ages
- 8 to 50 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
- Study population
- Patients with Stargardt disease secondary to mutation in the ABCA4 gene
Key inclusion criteria (3 listed)
- Male and female subjects between 8 and 50 years of age at the time of enrolment.
- Willingness to adhere to the protocol as evidenced by written informed consent if the subject is 18 years or older. If the subject is under 18 years of age, written assent must be obtained from the subject and written informed consent must be obtained from the subject's legally authorized representative (parent or legal guardian).
- Confirmed mutation in the ABCA4 gene.
Key exclusion criteria (4 listed)
- History of uveitis.
- Any ocular disease in either eye that may confound assessment of the retina morphologically and functionally.
- Any pathology of the posterior segment other than ABCA4 retinopathy.
- Presence of any other genetic mutation(s) that have been associated with retinal or macular dystrophy.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
To measure the change from baseline in Fundus Autofluorescence
Time frame: 24 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 16 Jun 2026.
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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. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
2 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Stargardt Disease, run by AAVantgarde Bio Srl at 2 sites in 2 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 3 inclusion and 4 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: under way, not looking for participants, as of the sponsor's last update on 16 Jun 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in United States, 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.
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?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
To measure the change from baseline in Fundus Autofluorescence (time frame: 24 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 16 Jun 2026.