NCT04126005

Natural History Study of Patients With Canavan Disease (CANinform Study)

A Combination Retrospective Medical History and Prospective Observational Study of Patients With Canavan Disease for Assessment of Natural History of Canavan Disease

• Status: Completed
• Phase: —
• Study type: Observational
• Enrollment: 67 (actual)
• Sponsor: Aspa Therapeutics · Industry
• Sex: All
• Age: —
• Healthy volunteers: Not accepted

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.

Detailed 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.

Conditions

• Canavan Disease

Timeline

• Start date: 2019-10-10
• Primary completion: 2025-05-21
• Completion: 2025-08-31
• First posted: 2019-10-14
• Last updated: 2026-04-17

Locations

3 sites across 2 countries: United States, Germany