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NCT Number: NCT06591806

An Observational Study in Children and Adults With Stargardt Disease

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.

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This study is active but is not currently recruiting participants.

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Key information

Conditions

Stargardt Disease Congenital, Hereditary, and Neonatal Diseases and Abnormalities Eye Diseases Eye Diseases, Hereditary Genetic Diseases, Inborn Macular Degeneration Retinal Degeneration Retinal Diseases

Age range

8 year–50 year

Sex eligibility

All sexes

Study type

Observational

Primary location

Oslo University hospital Ullevål, Oslo, Norway

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • 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.

Exclusion criteria

  • 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.

Treatment and study plan

Primary outcomes

  1. To measure the change from baseline in Fundus Autofluorescence

    Time frame: 24 months

Sponsors and collaborators

Lead sponsor

AAVantgarde Bio Srl

Industry

Registry information

Official study title

A Multicenter, Prospective, Longitudinal, Observational Study in Children and Adults With Stargardt Disease Related Atrophy Secondary to Biallelic Mutations in the ABCA4 Gene

Important dates

Study start
2024
Primary completion
2027
Study completion
2027
First posted
Sep 19, 2024
Registry last updated
Jun 16, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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