Stargardt DiseaseNCT06377150
ABCA4-associated Disease in Childhood and Adolescence - a Phenotype Study
An observational study of Stargardt Disease, run by University Hospital Tuebingen at 1 site in Germany.
Trial summary
Retrospective chart review study to elucidate the phenotype and genotype of children with ABCA4-associated Stargardt disease. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Stargardt Disease
- How many people
- 39 participants
- Where
- 1 siteGermany
- Study dates
- 1 Apr 2022to 31 Mar 2024
- Phase
- Observational
- Are there results?
- None posted in the registry
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 Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Patients with genetically confirmed ABCA4-associated inherited retinal dystrophy.
Who may be able to join
- genetically confirmed ABCA4-associated inherited retinal dystrophy
Who cannot join
- 18 years old and older
Trial locations and contacts
1 site in 1 country.
Germany1 site
University Hospital Tuebingen
Tübingen, Baden-Wuerttemberg, Germany, 72076
About this study
Retrospective chart review study to elucidate the phenotype and genotype of children with ABCA4-associated Stargardt disease.
- Conditions
- Stargardt Disease
- Keywords
- Stargardt Disease, ABCA4, inherited retinal dystrophy
- Medical subject headings
- Stargardt Disease
What is being measured
The primary outcome is the main question the study is designed to answer.
Best Corrected Visual Acuity (BCVA)
best corrected visual acuity
Measured: January 2014 - May 2021
Visual Field (VF)
visual field as measured by semi-automated 90° kinetic visual field exam using target III4e
Measured: January 2014 - May 2021
Fundus Photography (FP)
characterization of the retina with fundus photography
Measured: January 2014 - May 2021
Fundus Autofluorescence (AF)
characterization of the retina with fundus autofluorescence
Measured: January 2014 - May 2021
Optical Coherence Tomography (OCT)
characterization of the retina with optical coherence tomography, e.g. foveal ellipsoid zone loss
Measured: January 2014 - May 2021
Full-Field Electroretinography (ff-ERG)
characterization of retinal function with full-field electroretinogram
Measured: January 2014 - May 2021
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Apr 2024
- Study start1 Apr 2022
- Primary completion31 Mar 2024
- Study completion31 Mar 2024
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.
- More trials run by University Hospital TuebingenThe sponsor's other registered studies.
- 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
- Cross-sectional
- Enrolment
- 39 participants (actual)
Sponsor and investigators
- Lead sponsor
- University Hospital Tuebingen
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Laura Kuehlewein, MD, Principal investigator, Centre for Ophthalmology, University of TuebingenSusanne Kohl, PhD, Principal investigator, Molecular Genetics Laboratory, Centre for Ophthalmology, University of TuebingenJan-Philipp Bodenbender, MD, Principal investigator, Centre for Ophthalmology, University of Tuebingen
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06377150
- Sponsor's protocol ID
- ABCA4-1382022BO2
- Status last verified
- Apr 2024
- First submitted
- 12 Apr 2024
- Last updated
- 29 Jun 2025
- Results posted
- No
- Data monitoring committee
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 29 Jun 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- ABCA4-associated Disease in Childhood and Adolescence - a Phenotype Study
- ClinicalTrials.gov ID
- NCT06377150
- Acronym / sponsor ID
- ABCA4-1382022BO2
- Conditions
- Stargardt Disease
- Keywords
- Stargardt Disease, ABCA4, inherited retinal dystrophy
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 39 participants (actual)
- Lead sponsor
- University Hospital TuebingenUniversity, hospital or other
- Study start
- 1 Apr 2022 (actual)
- Primary completion
- 31 Mar 2024 (actual)
- Study completion
- 31 Mar 2024 (actual)
- First posted
- 22 Apr 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.
Retrospective chart review study to elucidate the phenotype and genotype of children with ABCA4-associated Stargardt disease.
Study design
- Observational model
- Cohort
- Time perspective
- Cross-sectional
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Patients with genetically confirmed ABCA4-associated inherited retinal dystrophy.
Key inclusion criteria (1 listed)
- genetically confirmed ABCA4-associated inherited retinal dystrophy
Key exclusion criteria (1 listed)
- 18 years old and older
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
Best Corrected Visual Acuity (BCVA)
Time frame: January 2014 - May 2021
best corrected visual acuity
Visual Field (VF)
Time frame: January 2014 - May 2021
visual field as measured by semi-automated 90° kinetic visual field exam using target III4e
Fundus Photography (FP)
Time frame: January 2014 - May 2021
characterization of the retina with fundus photography
Fundus Autofluorescence (AF)
Time frame: January 2014 - May 2021
characterization of the retina with fundus autofluorescence
Optical Coherence Tomography (OCT)
Time frame: January 2014 - May 2021
characterization of the retina with optical coherence tomography, e.g. foveal ellipsoid zone loss
Full-Field Electroretinography (ff-ERG)
Time frame: January 2014 - May 2021
characterization of retinal function with full-field electroretinogram
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 29 Jun 2025.
- 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. This study is not recruiting at the moment.
Laura Kuehlewein, MD principal investigator
Centre for Ophthalmology, University of Tuebingen
Susanne Kohl, PhD principal investigator
Molecular Genetics Laboratory, Centre for Ophthalmology, University of Tuebingen
Jan-Philipp Bodenbender, MD principal investigator
Centre for Ophthalmology, University of Tuebingen
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Stargardt Disease, run by University Hospital Tuebingen at 1 site in Germany.
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 1 inclusion and 1 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: finished, as of the sponsor's last update on 29 Jun 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Germany. 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?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Best Corrected Visual Acuity (BCVA) (time frame: January 2014 - May 2021). The study lists 6 primary outcomes in all. 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 29 Jun 2025.