Natural History Study of Patients With Canavan Disease

Canavan Disease Clinical Trial

Official title:

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT04126005
• Other study ID #: CVN-101
• Secondary ID: CANinform
• Status: Recruiting
• Start date: October 10, 2019
• Est. completion date: April 30, 2025

Study information

• Verified date: July 2023
• Source: Aspa Therapeutics
• Contact: Michelle Nelken
• Phone: 833-764-2267 or 617-861-4617
• Email: CANinform[@]aspatx.com
• Is FDA regulated: No
• Study type: Observational

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.

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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 40
• Est. completion date: April 30, 2025
• Est. primary completion date: December 31, 2024
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A and older

Eligibility

Inclusion Criteria:

1. Meet age criteria of a specific cohort. Note: In the case of a deceased patient whose parent(s) and/or legal guardian(s) have provided informed consent for study participation, the Investigator will review the patient's medical record(s) to determine study eligibility.

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.

Related Conditions & MeSH terms

• Canavan Disease

Locations

Country	Name	City	State
Germany	University Medical Center Hamburg-Eppendorf	Hamburg
United States	Massachusetts General Hospital	Boston	Massachusetts
United States	NYU Langone Medical Center	New York	New York

Sponsors (1)

Lead Sponsor	Collaborator
Aspa Therapeutics

Countries where clinical trial is conducted

United States,  Germany, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	To characterize the natural history of Canavan disease	To enhance the understanding of the natural history of Canavan disease through retrospective data collection from patient medical records and prospective data collection from living patients, including: phenotypic characteristics and variability, genotype characteristics and variability, and disease progression and natural history.	approximately 3 years

External Links

•   Aspa Therapeutics Company Website
•   CANinform Study Website