Natural History Study of Patients With Canavan Disease (CANinform Study)
NCT ID: NCT04126005
Last Updated: 2025-06-22
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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ACTIVE_NOT_RECRUITING
70 participants
OBSERVATIONAL
2019-10-10
2025-07-31
Brief Summary
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Detailed Description
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Conditions
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Study Design
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COHORT
OTHER
Study Groups
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Cohort 1 (Age < 18 Months)
* Motor function assessments (remote or in-clinic) every 2 months
* Clinic assessments every 6 months
No interventions assigned to this group
Cohort 2 (Age ≥ 18 Months - 3 Years)
* Motor function assessments (remote or in-clinic) every 4 months
* Clinic assessments every 6 months
No interventions assigned to this group
Cohort 3 (Age > 3 - 5 Years)
* Motor function assessments (remote or in-clinic) every 6 months
* Clinic assessments every 6 months
No interventions assigned to this group
Cohort 4 (Age > 5 Years)
* Motor function assessments (remote or in-clinic) 12 months
* Clinic assessments every 12 months
No interventions assigned to this group
Cohort 5 (Deceased)
• The patient's medical history records will be reviewed. In addition, a parent interview will be performed.
No interventions assigned to this group
Eligibility Criteria
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Inclusion Criteria
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.
ALL
No
Sponsors
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Aspa Therapeutics
INDUSTRY
Responsible Party
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Locations
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UCSF Benioff Children's Hospital Oakland
Oakland, California, United States
Massachusetts General Hospital
Boston, Massachusetts, United States
University Medical Center Hamburg-Eppendorf
Hamburg, , Germany
Countries
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Related Links
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Aspa Therapeutics Company Website
CANinform Study Website
Other Identifiers
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CANinform
Identifier Type: OTHER
Identifier Source: secondary_id
CVN-101
Identifier Type: -
Identifier Source: org_study_id
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