Frequently Asked Questions
General Questions
What is the purpose of the registry?
The purpose of the registry is to collect important clinical information from patients diagnosed with ETMR to increase our overall knowledge about the tumor. The registry is highly focused on improving treatment outcomes and accelerating ETMR research. We hope to accomplish these aims through the use of our consensus therapy protocol, the collection of ETMR tissue specimens, and the development of new clinical trials.
Who is eligible for the registry?
All patients of any age who currently have or have ever been diagnosed with ETMR are eligible to join in the registry. This includes both patients who are living and those who have passed away.
How was the consensus therapy developed?
Drug screens testing a large number of chemotherapy drugs have been performed in ETMR cell lines and animal models. It was noted that the chemotherapy drugs found to be most effective were rarely used to treat ETMR. In order to improve outcomes for patients, we developed a consensus protocol using the drugs found to be most effective in the ETMR drug screens. This consensus therapy has been successfully used in a small number of patients with good results. We want to expand access to the consensus regimen and evaluate its effectiveness on a larger scale using the data from our registry.
What is being done with the tumor tissue that is collected?
All tumor specimens from the registry will be sent to the Children’s Brain Tumor Network (CBTN) repository located at the Children’s Hospital of Philadelphia. There, the tumor specimens will undergo processing, which will include full genetic sequencing. We also aim to develop cell lines and transplantable tumor models from the registry tumor specimens. The data, biospecimens, cell lines, and tumor models derived from registry participants will be made available to researchers around the world.
Prior to accessing any data or tissue, researchers will be required to submit a research proposal which will need to be approved by the CBTN Scientific Committee. This process ensures that these precious resources are only released to committed researchers with a valid scientific plan.
FAQs For Parents
How will my child benefit from participating in the registry?
As the registry only involves the collection of clinical data and tumor tissue, there is no direct benefit to patients for participating in the registry. The goal of the registry is to eventually benefit all children with ETMR through the development of improved treatments.
The registry does provide a consensus protocol derived from the ETMR medical literature and expert experience, as well as access to case reviews by our Medical Advisory Board. Both of these may potentially provide benefit to patients, but are not required for participation in the registry.
What are the risks of participating in the registry?
The main risk to participants is the breach of confidentiality. Every reasonable effort will be made to maintain the privacy and confidentiality of registry participants. As the registry only involves the collection of clinical data and tumor specimens and biologic samples previously collected for clinical purposes, there are no physical risks to participating in the registry.
The consensus protocol is a chemotherapy regimen and, by nature, does carry the potential for significant risks and side effects. Treatment on the consensus therapy is not required for registry participation and the decision to follow the protocol is to be made by the treating physician and patient family. The risks, benefits, and side effects of consensus protocol therapy should be discussed with your child’s doctor.
Any treatment recommendations made by the Medical Advisory Board may carry risks. The purpose of these recommendations is to provide helpful guidance. The decision to follow any recommendations will be made by the treating physician and patient family. The risks, benefits, and side effects of any treatment should be discussed with your child’s doctor.
Does my child need to be treated following the consensus therapy?
Treatment on the consensus therapy is not required for registry participation and the decision to follow the protocol is to be made by the treating physician and patient family.
Do I need to participate in the registry to receive the consensus therapy?
If you do not wish to have your child participate in the registry but are interested in receiving treatment following the consensus protocol, you should ask your child’s doctor to contact the registry to obtain the consensus therapy protocol.
Do I need to participate in the registry to have access to the Medical Advisory Board?
The Medical Advisory Board will only formally review the cases of registry participants. However, our board members are willing and available to provide individual consultations. Participating in the registry is not required for access to our physician team.
Who do I contact for a second opinion?
Please fill out the contact form or call the number on our website and we will help to arrange a consultation with Dr. Hanson.
FAQs for Physicians
Why is ETMR One a registry study and not a clinical trial?
Due to the rarity of ETMR, conducting a formal clinical trial involving up-front treatment of the tumor is logistically challenging. In order to enroll the required number of participants, the trial would need to be run at a very large scale, for a long period of time, and require a tremendous amount resources. Timing, location, and eligibility issues would exclude many patients, preventing them from accessing the treatment regimen.
Alternatively, a registry study with a consensus protocol removes many of the hurdles associated with a clinical trial. This format requires IRB approval at a single site, with all informed consent discussions being performed by registry staff. Through this design, all patients have the opportunity to receive consensus protocol therapy regardless of their treatment site location and without meeting rigid parameters.
What is my involvement in enrolling patients into the registry?
It is our goal to have the registry onboarding process run seamlessly and to minimize any time burden for collaborating physicians.
Interested patients/families will contact the registry directly to find out more information and ask any questions. If they express a desire to participate, a member of our registry team will contact the patient/family to obtain written informed consent/assent. Once a participant is enrolled, one of our registry coordinators will work with your staff to obtain all necessary source documentation including medical records, radiographic imaging, and pathology specimens, if available.
Do I need to obtain IRB approval in order for my patient to participate in the registry?
The ETMR One registry is based at Hackensack University Medical Center where IRB approval for the study is maintained. As this is a registry protocol, we only ask that collaborating centers assist in the transfer of patient information and specimens once informed consent is obtained from the registry participants. Since the informed consent process is to be solely performed by Hackensack University Medical Center staff, institutions should not need to obtain full IRB approval.
What guidelines do I need to follow for my patient enrolled in the registry?
You should continue to deliver what is, in your opinion, the best care possible for your patient. The consensus protocol is available as a guide should you choose to use this treatment regimen. We recommend following the protocol closely, but you are not required to strictly adhere to the protocol. The Medical Advisory Board will review your patient’s case on a periodic basis and provide management recommendations. As the treating physician, you will make the choice of whether or not to follow those recommendations.
How do I obtain the consensus protocol?
Once your patient is enrolled in the registry, our team will contact you and provide a copy of the consensus protocol for you to review.
Am I required to follow the consensus regimen if my patient is enrolled in the registry?
As the treating physician, it is up to you to decide the best treatment regimen for your patient. We encourage physicians to follow the consensus protocol as it will allow the registry to prospectively evaluate a uniformly-treated group of patients. However, adhering to the consensus protocol is not required for registry participation.
How do I participate in the Medical Advisory Board?
Medical Advisory Board meetings take place via a secure internet conference. You will receive an invitation link to participate in your patient’s case reviews. We value your opinion and insight into your patient’s case and encourage you to participate in these reviews.