NCT06377150

Brief Summary

Retrospective chart review study to elucidate the phenotype and genotype of children with ABCA4-associated Stargardt disease.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
39

participants targeted

Target at P25-P50 for all trials

Timeline
Completed

Started Apr 2022

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

April 1, 2022

Completed
2 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

March 31, 2024

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

March 31, 2024

Completed
12 days until next milestone

First Submitted

Initial submission to the registry

April 12, 2024

Completed
10 days until next milestone

First Posted

Study publicly available on registry

April 22, 2024

Completed
Last Updated

June 29, 2025

Status Verified

April 1, 2024

Enrollment Period

2 years

First QC Date

April 12, 2024

Last Update Submit

June 25, 2025

Conditions

Stargardt Disease

Keywords

Stargardt DiseaseABCA4inherited retinal dystrophy

Outcome Measures

Primary Outcomes (6)

  • Best Corrected Visual Acuity (BCVA)

    best corrected visual acuity

    January 2014 - May 2021

  • Visual Field (VF)

    visual field as measured by semi-automated 90° kinetic visual field exam using target III4e

    January 2014 - May 2021

  • Fundus Photography (FP)

    characterization of the retina with fundus photography

    January 2014 - May 2021

  • Fundus Autofluorescence (AF)

    characterization of the retina with fundus autofluorescence

    January 2014 - May 2021

  • Optical Coherence Tomography (OCT)

    characterization of the retina with optical coherence tomography, e.g. foveal ellipsoid zone loss

    January 2014 - May 2021

  • Full-Field Electroretinography (ff-ERG)

    characterization of retinal function with full-field electroretinogram

    January 2014 - May 2021

Eligibility Criteria

Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)
Sampling MethodNon-Probability Sample
Study Population

Patients with genetically confirmed ABCA4-associated inherited retinal dystrophy.

You may qualify if:

  • genetically confirmed ABCA4-associated inherited retinal dystrophy

You may not qualify if:

  • years old and older

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

University Hospital Tuebingen

Tübingen, Baden-Wuerttemberg, Germany, 72076, Germany

Location

MeSH Terms

Conditions

Stargardt Disease

Condition Hierarchy (Ancestors)

Eye Diseases, HereditaryEye DiseasesMacular DegenerationRetinal DegenerationRetinal DiseasesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

  • Laura Kuehlewein, MD

    Centre for Ophthalmology, University of Tuebingen

    PRINCIPAL INVESTIGATOR

  • Susanne Kohl, PhD

    Molecular Genetics Laboratory, Centre for Ophthalmology, University of Tuebingen

    PRINCIPAL INVESTIGATOR

  • Jan-Philipp Bodenbender, MD

    Centre for Ophthalmology, University of Tuebingen

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
observational
Observational Model
COHORT
Time Perspective
CROSS SECTIONAL
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

April 12, 2024

First Posted

April 22, 2024

Study Start

April 1, 2022

Primary Completion

March 31, 2024

Study Completion

March 31, 2024

Last Updated

June 29, 2025

Record last verified: 2024-04

Data Sharing

IPD Sharing
Will not share

Locations

DE(1)