The VWM Registery is officially open! The Center for Childhood White Matter Disorders at the VU University Medical Center (VUmc) in Amsterdam, The Netherlands, has developed an online Registry for patients with Vanishing White Matter (VWM). The aim of the Registry is to collect as much insight in the disease course as possible, regarding both clinical features and MRI. This database will play an important role in developing treatment strategies and making decisions regarding therapeutic trials. Information is needed on as many patients as possible, even if patients are no longer alive. Register via: registerVWM@vumc.nl
Aim
The aim of the Registry is to collect as much insight in the disease course as possible, regarding both clinical features and MRI. This database will play an important role in developing treatment strategies and making decisions regarding therapeutic trials.
Privacy
We use Castor EDC, an online, cloud-based Electronic Data Capture platform, approved by external auditors, to collect longitudinal data of VWM patients. Castor is secured according to the most recent standards in order to protect the data and complies with all applicable laws and regulations (Good Clinical Practice (GCP), 21 CFR Part 11, EU Annex 11, General Data Protection Regulation (GDPR), HIPAA (US) ISO 9001 and ISO 27001).
Who can take part in the Registry?
Patients of all ages with genetically proven Vanishing White Matter.
What does taking part in the Registry mean?
When a patient is signed up for the Registry, we will first ask to sign informed consent. Subsequently, we will collect information on the clinical situation, MRI(s) and genetic testing results. In addition, we will send an email to provide access to the Registry, where questionnaires and a score form are available to be filled in. The frequency of the invites to fill out questionnaires and score forms depends on the age, and will be: 4 times a year (age 0-4 years), 2 times a year (4-8 years) or once a year (8 years and older). Per invite we will send a maximum of 3 questionnaires and 3 score forms, which will take 30 minutes in total to fill out. No additional hospital visits are necessary for the Registry. When an MRI is obtained for clinical purposes, we appreciate a copy.
Register
You can sign up for the online Registry per email. After receiving your message, we will send more detailed information about the Registry, so that you can decide to participate in the Registry. We will provide consent forms that need to be signed and returned to us. Register via: registerVWM@vumc.nl
Investigators
- Dr. Marjo S. van der Knaap, professor of Pediatric Neurology, Director of the Center for Childhood White Matter Disorders
- Dr. Renate J. Verbeek, pediatric neurologist
- Menno D. Stellingwerff, research assistant