Spots Global Cancer Trial Database for Natural History Study for DNA Repair Disorders
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Trial Identification
Brief Title: Natural History Study for DNA Repair Disorders
Official Title: Natural History Study for DNA Repair Disorders
Study ID: NCT05484570
Study Description
Brief Summary: This will be a single-center, single-arm, non-interventional natural history study to evaluate the longitudinal clinical course, functional outcome measures, and candidate biomarkers for individuals with DNA repair disorders, including Cockayne syndrome (CS), xeroderma pigmentosum (XP), and trichothiodystrophy (TTD).
Detailed Description: This will be a single-center, single-arm, non-interventional natural history study to evaluate the longitudinal clinical course, functional outcome measures, and candidate biomarkers for individuals with DNA repair disorders, including Cockayne syndrome (CS), xeroderma pigmentosum (XP), and trichothiodystrophy (TTD). Our hypothesis is that a reliable and reproducible baseline natural history course can be established for DNA repair disorders using the Early Childhood Assessment of Balance (ECAB) as a primary endpoint and other measures as secondary and exploratory endpoints that may be used in future therapeutic clinical trials.
Keywords
Eligibility
Minimum Age: 6 Months
Eligible Ages: CHILD, ADULT, OLDER_ADULT
Sex: ALL
Healthy Volunteers: No
Locations
University of Minnesota- Twin Cities, Minneapolis, Minnesota, United States
Contact Details
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