RECRUITING

An Observational Study in Children and Adults With Stargardt Disease

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

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.

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

Quick Facts

Study Start:2024-05-01

Study Completion:2027-06-30

Study Type:Not specified

Phase:Not Applicable

Enrollment:Not specified

Status:RECRUITING

Study ID

NCT06591806

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.

Ages Eligible for Study:8 Years to 50 Years

Sexes Eligible for Study:ALL

Accepts Healthy Volunteers:No

Standard Ages:CHILD, ADULT

Inclusion Criteria	Exclusion 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.

Inclusion Criteria

Exclusion 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.

Contacts and Locations

Study Contact

Clinical Operations Manager

CONTACT

+44 (0) 800 046 5680

clinicaltrials@aavantgarde.com

Study Locations (Sites)

Retina Foundation of the Southwest

Dallas, Texas, 75231

United States

Collaborators and Investigators

Sponsor: AAVantgarde Bio Srl

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2024-05-01

Study Completion Date2027-06-30

Study Record Updates

Study Start Date2024-05-01

Study Completion Date2027-06-30

Terms related to this study

Keywords Provided by Researchers

ABCA4

Additional Relevant MeSH Terms

Stargardt Disease