Introduction

This family protocol summary provides a general overview of the Children’s Oncology Group (COG) study ANBL09P1. It tells who is eligible and gives basic information about the study. More details about the study are in the consent form and protocol. You can get these from your oncologist.

ANBL09P1 is a pilot study. The purpose of this pilot study is to learn if a new treatment, when added to the standard therapy, is practical and can be successfully completed without unacceptable toxicity in many hospitals around the country. This study (clinical trial) is a therapeutic clinical trial. That means it is done to learn about treatment – its safety and how well it works. In this study, patients receive standard therapy plus a new treatment that was found to be safe and effective in other therapeutic trials. We are determining which dose of the new treatment is best when combined with standard therapy and whether the additional treatment creates any additional costs or hardships for families if they have to travel to another hospital for the additional therapy.

It is important to understand that participating in a clinical trial is entirely voluntary. The decision about whether or not to participate will not affect the care provided by the health care team in any way. You can find additional information about participation in clinical trials at www.childrensoncologygroup.org. Always discuss any questions that you may have with your health care team.

Study Number

ANBL09P1

Official Title

A COG Pilot Study of Intensive Induction Chemotherapy and ¹³¹I-MIBG Followed by Myeloablative Busulfan/Melphalan (Bu/Mel) for Newly Diagnosed High-Risk Neuroblastoma

Study Opening Date

ANBL09P1 opened on October 4, 2010. The ANBL09P1 committee hopes to enroll 105 patients. The study closed on 12/24/2015.

General Patient Eligibility

• Age: 1 - 30 years old
• Diagnosis: neuroblastoma or ganglioneuroblastoma that is “High-Risk”

Please consult your doctor to determine whether your child may participate in this study.

General Background and Study Goal

High-Risk Neuroblastoma is a childhood cancer that is often hard to cure; therefore additional therapies are needed. The main goal of this study is to determine if a new therapy, called ¹³¹I-Metaiodobenzylguanidine (¹³¹I-MIBG) is feasible (reasonable) to add to the standard therapy for High-Risk neuroblastoma. MIBG is a chemical agent taken up by neuroblastoma tumor cells. MIBG can be combined together with radioactive iodine (¹³¹I) in the laboratory to make the radioactive drug ¹³¹I-MIBG. The ¹³¹I-MIBG delivers radiation directly to the neuroblastoma cancer cells and causes them to die.

Summary of the Treatment

The current standard therapy for High-Risk neuroblastoma includes 3 phases of therapy: Induction, Consolidation, and Maintenance. Each phase of therapy has multiple components. In this study, the last cycle of chemotherapy during the induction phase will be replaced with ¹³¹I-MIBG therapy.

Induction Phase

• Six cycles of chemotherapy and surgery are used to kill as much tumor as possible. The chemotherapy used in the first 5 cycles is the same as the standard therapy for neuroblastoma patients. The sixth cycle is replaced by ¹³¹I-MIBG therapy.
• Blood stem cells are collected from the patient early in the induction phase and stored to use later in the therapy to help the bone marrow recover.
• ¹³¹I-MIBG therapy is a combination of MIBG, a chemical agent that can enter neuroblastoma tumor cells and radioactive iodine (¹³¹I). The ¹³¹I-MIBG delivers radiation to the neuroblastoma cells and causes them to die.
• Blood stem cells that were collected are given back to the patient to help the bone marrow recover.

Consolidation Phase

• Approximately 10-12 weeks after the patients receives ¹³¹I-MIBG therapy, the patient will begin the Consolidation phase. The Consolidation phase in this study is the same as the standard therapy for patients with neuroblastoma.
• One round of very high dose chemotherapy is given to kill any remaining tumor cells, followed by
• A second infusion of the blood stem cells that were collected in induction phase to help the bone marrow recover.
• After recovery, radiation therapy is given to any areas where the neuroblastoma tumors were found.

ANBL09P1 therapy is complete following radiation therapy.

A maintenance phase of therapy is typically given following consolidation therapy. The goal is to kill any remaining neuroblastoma cells. Your doctor will discuss further treatment plans with you.

Special Considerations

• Not all hospitals are able to give this special type of radiation so patients must be willing to travel to a second hospital (which could be several hours away or in another state) if the hospital where the patient starts treatment is not able to provide ¹³¹I-MIBG therapy. The study doctors hope to learn what effect traveling has on the family of the patient on this study.
  
  ¹³¹I-MIBG therapy requires some specific safety precautions:
  • A person who receives ¹³¹I-MIBG is considered to be “hot” or radioactive for a period of time after the injection, usually 3 to 5 days. Special care will be taken during that time to keep family and staff safe from the radiation.
  • The length of time family can visit inside the room (outside of the protective lead shield around the bed) will depend on how much radiation is measured in the room each day by the radiation specialist and depends on hospital policies. The amount of time will be limited, but may get longer as the radioactivity decreases.
  • The patient receiving this therapy also will have radioactive urine, so a catheter will be placed in the bladder (foley catheter) to drain the urine into a lead box.
  • It is possible that a family member may not be able to spend the night in the special room with the patient during this therapy. This will depend on the rules of the institution giving the MIBG.

Risks and Side Effects

Chemotherapy can cause side effects during and after treatment. All patients will be closely monitored for possible side effects of the medicines. All risks and side effects will be explained by your treatment team during the consent process. They can answer any questions that you may have about giving permission for your child to be in the clinical trial or other aspects of care. Please refer to the consent form for a detailed explanation of the side effects associated with the treatment on this study.

Contact Information

Your child’s oncologist and nurses are the best sources for further information.

Study Chairs

Brian Weiss, MD
Cincinnati Children’s Hospital Medical Center

FAMILY PROTOCOL SUMMARY REVIEW/APPROVALS