Observing the natural history of Canavan disease in patients
A Combination Retrospective Medical History and Prospective Observational Study of Patients With Canavan Disease for Assessment of Natural History of Canavan Disease
Aspa Therapeutics · NCT04126005
This study is looking to learn more about Canavan disease by tracking the health and development of patients over time, including gathering information from their medical records and family visits.
Quick facts
| Study type | Observational |
|---|---|
| Enrollment | 70 (estimated) |
| Sex | All |
| Sponsor | Aspa Therapeutics (industry) |
| Locations | 4 sites (Oakland, California and 3 other locations) |
| Trial ID | NCT04126005 on ClinicalTrials.gov |
What this trial studies
The CANinform study is a multinational observational effort aimed at gathering both retrospective and prospective data on Canavan disease. It involves extracting medical records from both living and deceased patients to document disease characteristics and developmental milestones. Additionally, the study collects longitudinal data through motor function assessments conducted remotely or in-clinic over a period of approximately three years. Families are invited to participate in clinic visits and follow-ups to enhance data collection and understanding of the disease's progression.
Who should consider this trial
Good fit: Ideal candidates include children diagnosed with Canavan disease who have accessible medical records documenting their disease history.
Not a fit: Patients who do not have a confirmed diagnosis of Canavan disease or lack the necessary medical records will not benefit from this study.
Why it matters
Potential benefit: If successful, this study could provide valuable insights into the natural progression of Canavan disease, potentially informing future treatment strategies.
How similar studies have performed: While this study represents a novel multinational effort, similar observational studies have successfully gathered critical data on rare diseases, suggesting potential for meaningful insights.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: 1. Meet age criteria of a specific cohort. 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.
Where this trial is running
Oakland, California and 3 other locations
- UCSF Benioff Children's Hospital Oakland — Oakland, California, United States (RECRUITING)
- Massachusetts General Hospital — Boston, Massachusetts, United States (ACTIVE_NOT_RECRUITING)
- NYU Langone Medical Center — New York, New York, United States (WITHDRAWN)
- University Medical Center Hamburg-Eppendorf — Hamburg, Germany (RECRUITING)
Study contacts
- Study coordinator: Mary Rohrer, RN, BSN
- Email: CANinform@aspatx.com
- Phone: 833-764-2267 or 617-861-4617
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
Conditions: Canavan Disease, AAV, AAV9, Gene therapy, Aspartoacylase, ASPA, ASPA gene, rAAV9