Neuroblastoma Clinical Trials & Research

Clinical trials evaluate the safety and effectiveness of new drugs or combinations of treatments. Many of the innovative therapies available at MSK Kids for children with neuroblastoma are investigational treatments being assessed in clinical trials. These include therapies that are not widely available elsewhere. We have more clinical trials of immunotherapy for neuroblastoma than any other hospital.

Can my child be in a clinical trial?

Your child’s care team will let you know if your child may be a candidate for any clinical trials. Our clinical research team is highly experienced in selecting the children who are most likely to benefit from a particular investigational therapy. We can guide you through the process of choosing the most appropriate clinical trial to meet your child’s needs. All aspects of a clinical trial are explained to you thoroughly, and your decision to enroll your child is entirely voluntary.

What kinds of therapies are being studied?

One of our approaches has been to take advantage of new drugs already in clinical trials for adults. Some of these drugs can enhance the effectiveness of chemotherapy without adding side effects, especially in people with disease that has become resistant to standard treatment. Other therapies are designed to block the ability of neuroblastoma cells to spread. You can find studies of such treatments as antibodies, cancer vaccines, and other immunotherapies. These studies often offer treatment options for people with neuroblastoma that has continued to grow or that came back despite prior therapies.

Translational Research for Neuroblastoma

In addition to our clinical researchers, MSK Kids has laboratory investigators who are exploring how neuroblastoma develops, spreads, and resists treatment. We work with clinical researchers to translate the findings of those studies into new options for young patients. The findings of clinical trials can inform new avenues of study.

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8 Clinical Trials found
Researchers want to find the best dose of avutometinib to treat children and young adults with advanced solid tumors. The people in this study have cancers that have spread or come back after treatment. In addition, their cancers have a mutation (change or variant) in a protein family called MAPK. These proteins tell cancer cells to grow.
Researchers are assessing a new type of therapy in young people with solid tumors that keep growing even after treatment. The therapy is called afamitresgene autoleucel. The people in this study have synovial sarcoma, malignant peripheral nerve sheath tumor (MPNST), neuroblastoma, or osteosarcoma. In addition, they have tested positive for the HLA-A*02 gene and their cancers make a protein called MAGE-A4. This protein plays a role in cancer growth.
Researchers want to find the best schedule for giving a new vaccine to people with neuroblastoma. The people in this study have neuroblastoma that is in remission (no signs of cancer). In addition, their cancers have a strong chance of coming back (high risk). The vaccine is given with a naturally occurring substance called beta-glucan.
Researchers are assessing a new combination therapy for neuroblastoma that has a high chance of coming back after treatment. This study is for children with high-risk neuroblastoma who have not gotten more than 1 chemotherapy cycle.
The purpose of this study is to assess the safety and effectiveness of combining a "bivalent" vaccine with two agents that stimulate the immune system: a sugar called beta-glucan and a medication called GM-CSF. The treatment is designed to prevent the relapse of patients with high-risk neuroblastoma that is in complete remission. This bivalent vaccine works by stimulating an immune response against two different antigens, which are markers on the surface of a cell.
The purpose of this study is to establish a registry of patients with a rare disease called pediatric-onset opsoclonus myoclonus ataxia syndrome (POOMAS). POOMAS affects the brain, spinal cord, and other nerves. Its exact cause is unknown, though a small number of patients with neuroblastoma may have this condition.
Metaiodobenzylguanidine (MIBG) is a substance taken up specifically by neuroblastoma, pheochromocytoma, or paraganglioma tumor cells. MIBG can be combined with radioactive iodine (<sup>131</sup>I) in the laboratory to form the radioactive compound <sup>131</sup>I-MIBG. The <sup>131</sup>I-MIBG compound concentrates more in cancer cells than in normal cells. It may therefore deliver more radiation directly to cancer cells while sparing normal organs.
The genetic and biochemical makeup of a tumor influences its aggressiveness and the choice of the most appropriate therapy. The purpose of this research study is to determine the genetic and biochemical makeup of neuroblastic tumors, including neuroblastoma, ganglioneuroblastoma, ganglioneuroma, and paraganglioma. These research studies will include analysis of substances called markers that exist on the surface of cancer cells; cytogenetic studies; the identification of genes related to the development of these cancers and to tumor growth; and studies of tumor growth in test tubes. Patients' blood and bone marrow will also be tested for the presence of tumor cells or leukemia cells.