Canavan Disease Clinical Trial
Official title:
A Combination Retrospective Medical History and Prospective Observational Study of Patients With Canavan Disease for Assessment of Natural History of Canavan Disease
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.
Status | Recruiting |
Enrollment | 40 |
Est. completion date | April 30, 2025 |
Est. primary completion date | December 31, 2024 |
Accepts healthy volunteers | No |
Gender | All |
Age group | N/A and older |
Eligibility | Inclusion Criteria: 1. Meet age criteria of a specific cohort. Note: In the case of a deceased patient whose parent(s) and/or legal guardian(s) have provided informed consent for study participation, the Investigator will review the patient's medical record(s) to determine study eligibility. 2. Confirmed clinical and biochemical diagnosis of Canavan disease. 3. Available medical records since birth that permit documentation of disease characteristics and developmental milestones. 4. Parent and/or legal guardian is able to read, understand, and sign the informed consent. Exclusion Criteria: 1. Patient does not meet the Inclusion Criteria. |
Country | Name | City | State |
---|---|---|---|
Germany | University Medical Center Hamburg-Eppendorf | Hamburg | |
United States | Massachusetts General Hospital | Boston | Massachusetts |
United States | NYU Langone Medical Center | New York | New York |
Lead Sponsor | Collaborator |
---|---|
Aspa Therapeutics |
United States, Germany,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | 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. | approximately 3 years |
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