The Amsterdam Leukodystrophy Center has developed an online registry for patients with Vanishing White Matter (VWM). The purpose of the registry is to gain as much insight as possible into the course of the disease, both in terms of clinical features and MRI. This database will play an important role in developing treatment strategies and making decisions about therapeutic trials. Information is needed about as many patients as possible, even if patients are no longer alive.
The database uses Castor EDC, an online, cloud-based electronic data collection platform, approved by third-party auditors, to collect longitudinal data from VWM patients. Castor is secured to the latest standards 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 participate in the registry?
Patients of all ages with genetically proven Vanishing White Matter, from all over the world.
What does participation in the register mean?
When a patient signs up for the Registry, we will first ask to sign informed consent. We then collect information about the clinical situation, MRI (s), and genetic test results. In addition, we will send an email to provide access to the register, where questionnaires and a score form are available for you to complete. The frequency of the invitations to fill in 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). We send a maximum of 3 questionnaires and 3 score forms per invitation, which take a total of 30 minutes. No additional hospital visits are required for the registry. When an MRI is obtained for clinical purposes, we appreciate a copy.
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
The local registry will be transformed into an international registry, which offers information to many different partners. The VWM consortium of experts will form the board of the Registry and patient advocates, the Industry Alliance Office, and other experts, as needed, will form the advisory board.
You can register for the online registration by e-mail. Upon receipt of your message, we will send you more detailed information about the Registry so that you can decide to participate in the Registry. We will provide consent forms to be signed and returned to us.