Ignite Creation Date:
2025-12-25 @ 3:20 AM
Ignite Modification Date:
2026-02-25 @ 5:51 PM
Study NCT ID:
NCT04126005
Status:
ACTIVE_NOT_RECRUITING
Last Update Posted:
2025-06-22
First Post:
2019-10-10
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Natural History Study of Patients With Canavan Disease (CANinform Study)
Sponsor:
Aspa Therapeutics
Organization:
Study Overview
Official Title:
A Combination Retrospective Medical History and Prospective Observational Study of Patients With Canavan Disease for Assessment of Natural History of Canavan Disease
Status:
ACTIVE_NOT_RECRUITING
Status Verified Date:
2024-10
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief 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.
Study Oversight
Has Oversight DMC:
False
Is a FDA Regulated Drug?:
False
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
False
Is an FDA AA801 Violation?:
Secondary ID Infos
| Secondary ID | Type | Domain | Link | View |
|---|---|---|---|---|
| CANinform | OTHER | Aspa Therapeutics | View |