Clinical Trials Logo

Clinical Trial Summary

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.


Clinical Trial Description

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 Design


Related Conditions & MeSH terms


NCT number NCT04126005
Study type Observational
Source Aspa Therapeutics
Contact Michelle Nelken
Phone 833-764-2267 or 617-861-4617
Email CANinform@aspatx.com
Status Recruiting
Phase
Start date October 10, 2019
Completion date April 30, 2025

See also
  Status Clinical Trial Phase
Recruiting NCT04998396 - A Study of AAV9 Gene Therapy in Participants With Canavan Disease Phase 1/Phase 2
Withdrawn NCT00657748 - Lithium and Acetate for Canavan Disease Phase 2
Enrolling by invitation NCT03655223 - Early Check: Expanded Screening in Newborns
Completed NCT02851563 - A Natural History Study of Canavan Disease
Recruiting NCT00724802 - Oral Glyceryl Triacetate (GTA) in Newborns With Canavan N/A
Completed NCT01999257 - Efficacy Study of an Online Educational Module Before Carrier Genetic Screening in Persons of Ashkenazi Jewish Descent. N/A
Active, not recruiting NCT02699190 - LeukoSEQ: Whole Genome Sequencing as a First-Line Diagnostic Tool for Leukodystrophies
Recruiting NCT03047369 - The Myelin Disorders Biorepository Project
Recruiting NCT04833907 - rAAV-Olig001-ASPA Gene Therapy for Treatment of Children With Typical Canavan Disease Phase 1/Phase 2
No longer available NCT05317780 - Canavan-Single Patient IND