Clinical Trial: A Natural History Study of Canavan Disease
Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Observational
Official Title: A Natural History Study of Canavan Disease: MGH Site
Brief Summary: The purpose of this study is to learn more about the natural history of Canavan disease
Detailed Summary:
For this study, families of subjects will fill out a Canavan Patient Survey which asks questions about disease development, symptoms and progression. Patients will be seen in Dr. Eichler's Leukodystrophy clinic for standard of care appointments. These appointments will be scheduled every 6 months.
The investigators will also collect MRI images and reports that have already been done or which will be done during participation in the study on a clinical basis.
GLOBAL UNIQUE IDENTIFIERS (GUID):
A patient Global Unique Identifier (GUID) will be used as the identifier for individuals participating in the study in NeuroBANK™. The GUID is an 11-character string that is generated using encryption technology and algorithms licensed by the NCRI from the National Institutes of Health (NIH).
The GUID is generated on a secure website that utilizes 128-bit Secure Socket Layer (SSL). Of note, this website is not linked to NeuroBANK™. The GUID is generated using an irreversible encryption algorithm - it accepts twelve identifying data elements, (e.g. last name at birth, first name at birth, gender at birth, day, month and year of birth, city and country of birth, etc.), and produces a unique random-generated character string, or GUID. No identifying information is stored in the system; it is simply used to generate the GUID. If the same information is entered again, the same GUID will be returned.
The GUID is entered into NeuroBANK™ when the patient is being created in the system. As the same patient may participate in multiple studies, NeuroBANK™ will also allow capturing a study-specific ID for the patient. For more information about NeuroBANK™ or the GUID, pl
Sponsor: Massachusetts General Hospital
Current Primary Outcome: Change in Gross Motor Function Measure over time [ Time Frame: baseline, 6 months, 1 year, 18 months, 2 years ]
Original Primary Outcome: Same as current
Current Secondary Outcome: Change in magnetic resonance imaging findings over time [ Time Frame: baseline, 6 months, 1 year, 18 months, 2 years ]
Original Secondary Outcome: Same as current
Information By: Massachusetts General Hospital
Dates:
Date Received: July 28, 2016
Date Started: May 2016
Date Completion: May 2018
Last Updated: May 12, 2017
Last Verified: September 2016
