A natural history study of patients with Canavan disease.

A natural history study of patients with Canavan disease.

A natural history study of patients with Canavan disease.

A natural history study of patients with Canavan disease.

What Is a Natural History Study?

  • A natural history study is used to understand the natural course of a disease, and can help doctors know what clinical improvements may look like in patients who receive a potential treatment.
  • A robust natural history study can be an alternative to a placebo arm in clinical trials.
  • Regulators, like the Food and Drug Administration (FDA), can use natural history data to understand more about disease progression as they evaluate potential therapies for approval.

Importance of a natural history study in patients with Canavan disease.

Why Should Families Consider Participating?

  • Patients can contribute to a greater knowledge of Canavan disease, since Aspa will share data from CANinform with the Canavan patient community, which may enable doctors to provide better care for those affected.
  • Patient data from a natural history study can help define meaningful changes that may show whether a treatment is working.
  • Greater family participation can provide valuable data that may help support an approval of a new therapy for Canavan Disease.

Hear from Canavan patient advocacy groups on enrolling in the CANinform natural history study.

CANinform is a natural history study of patients with Canavan disease that will help researchers better understand the disease and can provide a foundation for Aspa’s gene therapy program. The study is open to all families with a confirmed diagnosis of Canavan disease.

Medical records are a key component for gathering this data and are needed to participate in CANinform. Aspa is offering a record retrieval service to help families (outside of the EU) to obtain past medical records using three simple steps, detailed below.

TO ENROLL IN CANinform:

TO ENROLL IN CANinform:

1 Families can call 1-833-764-2267 (toll-free) or 1-617-861-4617 or email CanavanMedRec@veristat.com to use a free service to collect medical records.

2 Fill out a short online form to provide consent to share medical provider information and begin the process of obtaining the records.

3 Once records are retrieved, contribute to the Canavan community by enrolling in CANinform, the Canavan natural history study. Support other Canavan families by joining this important effort.

If you call any of our call centers to express interest in participating in Aspa’s clinical trials, we may request additional information from you. This information is necessary to confirm whether your child is eligible for screening, which is a step to determine potential enrollment in our clinical trials. The information you provide and any information obtained through the screening process would be used solely to determine your child’s eligibility to participate in Aspa’s programs.

TO ENROLL IN CANinform:

Families can call 1-833-764-2267 (toll-free) or 1-617-861-4617 or email CanavanMedRec@veristat.com to use a free service to collect medical records.

Fill out a short online form to provide consent to share medical provider information and begin the process of obtaining the records.

Once records are retrieved, contribute to the Canavan community by enrolling in CANinform, the Canavan natural history study. Support other Canavan families by joining this important effort.

If you call any of our call centers to express interest in participating in Aspa’s clinical trials, we may request additional information from you. This information is necessary to confirm whether your child is eligible for screening, which is a step to determine potential enrollment in our clinical trials. The information you provide and any information obtained through the screening process would be used solely to determine your child’s eligibility to participate in Aspa’s programs.

CANinform SITES

FREQUENTLY ASKED QUESTIONS

About CANinform

What is a natural history study?

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.

Why is Aspa conducting a natural history study?

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.

Why should we consider enrolling our loved one in the CANinform study??

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.

Will my child receive any benefit from participating in CANinform?

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.

Will my child be prescribed medicines as part of CANinform?

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.

If my child is on medications prescribed by their doctor, will they have to stop taking those medications?

CANinform does not require any changes in participants’ medications or other aspects of their care.

Will there be costs to families for participating in CANinform?

No, there will not be any cost to families who participate in CANinform.

Who can participate in the CANinform study?

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.

If my child participates in CANinform will that exclude them from enrolling in CANaspire, Aspa’s gene therapy trial for patients with Canavan disease?

Participating in CANinform will not impact a child’s ability to be enrolled in Aspa’s gene therapy trial for Canavan disease.

If my child participates in CANinform, will my child be automatically able to enroll in Aspa’s gene therapy trial for patients with 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 my child has passed away, could their data still be included in CANinform?

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.

What will happen if we decide to enroll our child in the CANinform study?

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?”

Will participation involve in-person visits with a CANinform study site?

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.

What will happen during a CANinform study visit?

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.

Do I have to go to every hospital to obtain my child’s medical records if we do not have them?

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.

How does CANinform relate to CANaspire, Aspa’s gene therapy trial?

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.