An Observational Study in Children and Adults With Stargardt Disease

Description

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

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Study Overview

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Study Details

Study overview

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.

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

An Observational Study in Children and Adults With Stargardt Disease

Condition
Stargardt Disease
Intervention / Treatment

-

Contacts and Locations

Dallas

Retina Foundation of the Southwest, Dallas, Texas, United States, 75231

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

For general information about clinical research, read Learn About Studies.

Eligibility Criteria

  • 1. Male and female subjects between 8 and 50 years of age at the time of enrolment.
  • 2. 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).
  • 3. Confirmed mutation in the ABCA4 gene.
  • 1. History of uveitis.
  • 2. Any ocular disease in either eye that may confound assessment of the retina morphologically and functionally.
  • 3. Any pathology of the posterior segment other than ABCA4 retinopathy.
  • 4. Presence of any other genetic mutation(s) that have been associated with retinal or macular dystrophy.

Ages Eligible for Study

8 Years to 50 Years

Sexes Eligible for Study

ALL

Accepts Healthy Volunteers

No

Collaborators and Investigators

AAVantgarde Bio Srl,

Study Record Dates

2027-06-30