A Natural History Study of Canavan Disease
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT02851563|
Recruitment Status : Completed
First Posted : August 1, 2016
Last Update Posted : November 1, 2019
|Condition or disease|
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, please go to: www.neurobank.org.
The NCRI Data Management Team is trained and knowledgeable regarding confidentiality and integrity of data. They will be responsible for all aspects of data procedures. Alex Sherman is the ALD Connect network strategist and member of the NEALS ALS consortium. He is the director of Strategic Development and Systems of NCRI, serves on the Executive Committee of the ALS Research Group, and is one of the leaders of the NEALS ALS Consortium.
Data Quality Checks, Logic Checks and Queries:
The Data Manager (DM) at the Neurological Clinical Research Institute (NCRI) at the Massachusetts General Hospital will conduct monthly Data Quality Checks, Logic Checks, and internal data quality audits. Data field queries will be resolved in an established workflow according to the Standard Operating Procedures (SOPs). The queries may be created either at the point of entry during the data entry process, manually by the NCRI DM, or as the result of executing monthly Logic Checks.
Study-Specific Database Backup and Maintenance:
The NCRI personnel will be responsible for the database backups that will be conducted daily. Simultaneously, a separate copy of the backup in the encrypted compressed format will be maintained and saved by the System Analysts. The backups will be saved on a separate computer partition in a password-protected compressed format and also will be burnt into DVD medium and stored in a secure location.
|Study Type :||Observational|
|Actual Enrollment :||21 participants|
|Official Title:||A Natural History Study of Canavan Disease: MGH Site|
|Study Start Date :||May 2016|
|Actual Primary Completion Date :||April 2019|
|Actual Study Completion Date :||April 2019|
- Change in Gross Motor Function Measure over time [ Time Frame: baseline, 6 months, 1 year, 18 months, 2 years ]
- Change in magnetic resonance imaging findings over time [ Time Frame: baseline, 6 months, 1 year, 18 months, 2 years ]
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02851563
|United States, Massachusetts|
|Massachusetts General Hospital|
|Boston, Massachusetts, United States, 02114|
|United States, New York|
|NYU Langone Medical Center|
|New York, New York, United States, 10016|
|University Medical Center Hamburg-Eppendorf|
|Hamburg, Germany, 20246|
|Principal Investigator:||Florian Eichler, MD||Massachusetts General Hospital|