Neuroblastoma

Clinical Trials

Mayo Clinic is a member of the Children's Oncology Group (COG), the national cooperative group for clinical trials and basic science investigation of childhood cancers. Mayo participates actively in COG trials to provide state-of-the-art clinical trials to children with cancer.

The Division of Pediatric Hematology/Oncology at Mayo Clinic is a member of a consortium of cancer centers across the United States established by the National Cancer Institute (NCI) as Phase I investigational centers. These institutions study and develop cutting-edge technologies and protocols for the successful treatment of neuroblastoma and other childhood cancers. Mayo specialists have access to experimental agents and new, investigational drugs as soon as these treatments are available. Phase I trials are utilized for patients whose tumors have recurred after standard therapy has failed.

Below is a list of Neuroblastoma clinical trials from the clinical trials database at Mayo Clinic.

This list includes only trials about which Mayo researchers choose to publish information. Mayo Clinic may be conducting other trials which are not in this database. Mayo's clinical trials include experimental treatments, often unavailable elsewhere, which frequently lead to improved patient care for people worldwide. Patients should ask their doctor at Mayo about clinical trials appropriate for their situation.

A Phase 2 Study of Irinotecan + Temozolomide (Temodar) in Children with Recurrent Neuroblastoma
In the past, children with neuroblastoma that has not responded to previous therapy or neuroblastoma that has come back after therapy has ended have not responded to chemotherapy very well. This study is being done to find out whether neuroblastoma in children who have recurrent or refractory (resistant) disease responds to treatment when the children are given the chemotherapy drugs irinotecan and temozolomide. It is also being done to find out what effects good and bad irinotecan and temozolomide have on the patient and their neuroblastoma. The drugs irinotecan and temozolomide can be given to patients who are not enrolled on this study. The experimental parts of this treatment are the drug doses and the schedule with which they are given.
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ANBL0532, Phase III Study of Single vs. Tandem Myeloablative Consolidation Therapy for High-Risk Neuroblastoma
Patients are being asked to take part in this study because they have high risk neuroblastoma. Neuroblastoma is a type of cancer. Neuroblastoma shows up as a lump or mass in the belly or around the spinal cord in the chest, neck, or pelvis. Neuroblastoma is a cancer of nerve cells. It develops in nerve cells that are outside of the brain. It often spreads to bone, liver, lymph nodes and bone marrow, which is the soft tissue in the center of bones where blood cells are made. Patients in this study have the type of neuroblastoma that is called High Risk because their tumor has spread from where it started or because their type of tumor is harder to treat.

What Is The Current Standard Of Treatment For This Disease?
The treatment for neuroblastoma includes three parts (phases) of therapy called Induction, Consolidation and Maintenance. During Induction therapy anti-cancer drugs (chemotherapy) and surgery are used to kill and remove as much tumor as possible. Blood stem cells are collected during the Induction phase of therapy. After collection the blood stem cells are frozen and stored to be used during the Consolidation phase of treatment. Blood stem cells are the cells that create new blood cells, such as red blood cells, white blood cells, and platelets. Blood stem cells can be collected from the blood by using a machine that can separate out the part of blood that has stem cells and then return the remaining blood back to the patient.

During the Consolidation phase of treatment extremely high doses of chemotherapy are given to better kill any remaining neuroblastoma cells. The extremely high doses of chemotherapy destroy healthy bone marrow. Bone marrow is the soft tissue in the hollow of flat bones of the body that produces new blood cells. The peripheral blood stem cells that were stored during the Induction phase of treatment are given back to the patient after the high dose Consolidation chemotherapy. These stems cells allow the bone marrow to return to normal so that new blood cells can be made. This type of therapy is called a hematopoietic stem cell transplant. Once the patient has healed from the effects of high doses of chemotherapy, radiation therapy is given to the first place the tumor was found and to any additional places where the tumor was found after Induction therapy.

During the third phase of therapy, Maintenance, an oral drug, called cis-retinoic acid (Accutane) is given.
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Perinatal Neuroblastoma: Expectant Observation
This protocol is designed to answer the question: Is it safe to avoid surgical removal of a small adrenal tumor mass in a young infant while closely watching the behavior of the tumor? Parents and guardians are being asked to allow their child to take part in this study because their child has a cystic or small mass in the adrenal gland. An evaluation of their child has not shown any other changes. This mass is most likely a tumor called neuroblastoma, although it could also represent a lung malformation called pulmonary sequestration, or a blood clot in the adrenal gland, referred to as adrenal hemorrhage. The current standard treatment for neuroblastoma is to remove the tumor through surgery.

The researchers have learned a great deal about this tumor. One of the key findings is that it behaves quite differently in infants compared to older children. Several recent studies have suggested that anywhere between 33 percent and 100 percent of these small adrenal tumors in young infants go away on their own with no treatment.

The benign (not cancer) behavior of these small adrenal tumors in young infants, including the reasonable chance of the tumor going away on its own, with no treatment, has led to the question of whether it would be better not to remove these tumors through surgery. The tumor will be closely watched using a combination of ultrasound imaging and urine chemistry tests to follow the growth and activity of the tumor. Because major abdominal surgery in infants has a problem rate of approximately 10 percent and mortality (death rate) of approximately 2 percent, any approach that safely reduces the rate of surgery in this age group, without changing the outcome related to the tumor, will be an improvement in care. In addition, because the adrenal gland does not regenerate, removal of one of the two adrenal glands in infancy would increase the individual's risk of adrenal insufficiency (a condition requiring life-long hormone replacement) if the other gland were damaged or removed later in life.

There are 2 Options for Parents/ Guardians:

Observational Group:
By agreeing to take part, the child will not undergo initial removal of the tumor but will be watched closely with a series of urine tests and ultrasound studies and other imaging (X-rays). If there is any significant growth of the mass, or change in function, as found by the urine tests, removing the tumor through surgery will be advised.

If the mass stays the same or decreases in size, continued watching studies will be done.
If the tumor grows, the child will be taken off this study and the tumor will be taken out surgically. Studies have shown that neuroblastoma tumors in infants behave in a very favorable manner. Even if growth is seen, surgery will probably be the only treatment required. In the unusual infant cases with spread of the tumor to other parts of the body, chemotherapy and radiotherapy are rarely necessary because most of the tumors go away without interference. Special studies can be done on the tumor when it is taken out to see if it is one of the rare aggressive tumors in infants that may require chemotherapy and radiation therapy.

Non-Observational Group:
By agreeing to take part, the child will have standard surgical removal of the mass in the adrenal gland. The research team will record the details of the child's pre-operative imaging studies, surgery and the pathologist's evaluation of the adrenal mass.
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Phase 3 Study of Chimeric Antibody 14.18 (Ch14.18) in High Risk Neuroblastoma Following Myeloablative Therapy and Autologous Stem Cell Rescue
Neuroblastoma is a solid, cancerous tumor that shows up as a lump or mass in the belly or around the spinal cord in the chest, neck, or pelvis. The standard treatment for neuroblastoma consists of anti-cancer drugs (chemotherapy), surgery, and radiation therapy. Children with high-risk neuroblastoma often respond to standard treatment at first, but there is a high risk that the cancer will come back. This study is being done to try to raise the number of children with high-risk neuroblastoma who can avoid having the cancer come back.

Prior to taking part in this study patients would have already had chemotherapy, surgery, radiation therapy, and stem cell transplant treatment. Patients were helped by this treatment.
The reason for this companion study is to compare two different treatments aimed at keeping or improving their response to previous treatments. This study involves the use of an investigational biologic therapy, ch14.18, a monoclonal antibody. Monoclonal antibodies are proteins made in the laboratory, designed to bind to specific cancer cells. ch14.18 was designed to bind to neuroblastoma cells and other cancer cells that express the GD-2 antigen. When ch14.18 binds to the neuroblastoma cells, the body's immune system is excited to attack and kill the neuroblastoma cells. Ch14.18 represents a new kind of cancer therapy that, unlike chemotherapy and radiation, targets the death of cancer cells without killing nearby healthy cells. There is laboratory proof to suggest that ch14.18 can target the body's own immune cells to get rid of cancer cells. These immune cells armed by ch14.18 include cells that are activated by GM-CSF and cells turned on by aldesleukin (IL-2). Prior studies had shown the safest doses of ch14.18 and IL-2 to be given after stem cell transplant.

GM-CSF and IL-2 are drugs that are like a fluid made by the body in everybody. Usually the body makes small amounts of GM-CSF that helps it to produce normal infection fighting white blood cells. The body also makes small amounts of IL-2 that helps white blood cells fight infection. It is now possible to make GM-CSF and IL-2 outside of the body and give humans much higher doses than their own body makes. There is some proof that, in the laboratory and in animals, GM-CSF and IL-2 raises the anti-cancer effect of monoclonal antibodies like ch14.18. The research staff wish to see if aldesleukin (IL-2) can help improve the helpfulness of ch14.18 in humans.
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