This study uses medical records that allow retrospective data extraction of critical milestone and motor function data. In addition, prospective assessments collect data relevant to the natural history of Canavan disease in children.
The CANinform natural history study is the first multinational effort to rigorously gather both retrospective and prospective data from this patient population. Data collection includes extraction of retrospective data from medical records of living and deceased patients, and collection of prospective, longitudinal data from living patients and their parent(s)/caregiver(s). Motor function assessments are performed remotely in the home via video or in the clinic by qualified study team members. Families will be invited to attend clinic visits and/or will be followed remotely by the clinical site for approximately 3 years.
Study Type
OBSERVATIONAL
Enrollment
70
UCSF Benioff Children's Hospital Oakland
Oakland, California, United States
Massachusetts General Hospital
Boston, Massachusetts, United States
University Medical Center Hamburg-Eppendorf
Hamburg, Germany
To characterize the natural history of Canavan disease
To enhance the understanding of the natural history of Canavan disease through retrospective data collection from patient medical records and prospective data collection from living patients, including: phenotypic characteristics and variability, genotype characteristics and variability, and disease progression and natural history.
Time frame: approximately 3 years
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