A natural history study is a non-interventional (observational) study where doctors follow patients to see how a disease progresses over time. This may also include review of past medical records. These studies are often conducted in rare diseases where there are limited data available on how a disease progresses.
In order to tell whether a new, investigational therapy (such as Aspa’s investigational gene therapy) is having an impact on progression of the disease, a natural history study is needed to better understand how Canavan disease affects patients over time. Information about the natural history of Canavan disease can be used to define the effects of treatment that are most meaningful for patients and their families, doctors, and regulators who approve new therapies. A robust natural history study can also be an alternative to a placebo arm in clinical trials.
The study will provide information that will greatly increase understanding of Canavan disease. Once the study is complete, Aspa will make the data publicly available to doctors, researchers and the Canavan community to share knowledge about the disease that may help advance the development of new treatments.
No, your child will not directly benefit from participating in the CANinform study. However, though your child will gain no medical benefit from participating in CANinform, you will have the opportunity to have access to leading Canavan disease experts at no cost to you, with regularly scheduled visits or check-ins to monitor your child’s health. In addition, by contributing to this important effort, you also can increase scientific knowledge about Canavan disease – knowledge that may help advance future therapies for this condition.
No, there will be no medicines prescribed to patients in CANinform. The medications your child is currently taking will be recorded during the specified study visits but will not be changed because of participation in the study.
CANinform does not require any changes in participants’ medications or other aspects of their care.
No, there will not be any cost to families who participate in CANinform.
Any child with Canavan disease is welcome to participate in this study. For families who have lost a child to Canavan disease, Aspa has established a process whereby the family can still participate in CANinform, as data in their child’s medical record can be very informative and could contribute to progress in overall Canavan research.
Participating in CANinform will not impact a child’s ability to be enrolled in Aspa’s gene therapy trial for Canavan disease.
No, participation in the CANinform study does not mean that your child will automatically be able to enroll in Aspa’s gene therapy trial.
If your child has passed away, their data can still be very helpful in contributing to the overall understanding of Canavan disease. Aspa has set up a medical record retrieval service to help families of Canavan patients obtain records so they can participate in CANinform.
All participants in CANinform will be assigned to a clinical site. Prior to enrollment, families will be asked to review and sign an appropriate consent form. The family will be asked to provide all available medical records to the clinical site in advance, to allow the study doctor to become familiar with the patient’s medical history.
For patients living in the US and certain other countries, Aspa is offering a free medical record retrieval service to help families obtain their child’s records.
Please see FAQ “What will happen during a CANinform study visit?”
Aspa understands that the COVID-19 pandemic has limited the ability of families to travel or enter research sites. Therefore, to permit the study to continue, participation will not require travel or in-person visits to the study site at this time.
In response to the COVID-19 pandemic, assessments may be conducted virtually, as appropriate. The study doctor and study staff may use video calling to perform study assessments of growth and development. Parents or caregivers may be asked to help collect some urine and saliva samples. When possible and with permission, a licensed professional may be sent to the participant’s home to collect blood samples. Standardized assessments that measure the effects of the disease on the patient’s and family’s quality of life and the emotional and social impact of Canavan disease may be given as well. If certain assessments become too much for the family, the family can choose to decline those assessments.
Not if you live in the United States or some other countries, outside of the EU. Aspa has hired a company that can obtain the medical records on your behalf. Families living in the US or outside of the EU can call 1-833-764-2267 or 1-617-861-4617 or email CanavanMedRec@veristat.com and provide consent, and the company will handle all the details and provide you with all the necessary records necessary for participation in CANinform. This service is free of charge to families.
CANinform is a natural history study that gathers information from Canavan patients’ medical records as well as from patients themselves to better understand the characteristic features of Canavan disease and how the disease progresses over time. CANinform will provide critical data to allow Aspa to measure whether treatment with its investigational gene therapy is beneficial. Participation in CANinform does not impact eligibility for participating in Aspa’s gene therapy trial for Canavan disease.
NAA is found mostly in neurons. If NAA is not broken down it accumulates in a child’s brain and may prevent the proper formation of myelin, the protective covering that helps neurons communicate with each other.
The deficits in myelin are thought to cause Canaan’s effects on movement, lanquage and vision.
Aspa’s investigational gene therapy is given in a single intravenous dose and aims to provide working copies of the
ASPA gene throughout a child’s body.
The treatment uses an AAV9 viral vector designed to deliver the functional ASPA gene throughout the body and the brain.
Once the functional ASPA gene is expressed, it may help restore myelin in the brain. This can potentially improve the course of Canavan disease by addressing the underlying genetic cause.