Trial Information

Summary: A Phase I Study Of Zoledronic Acid (Zometa) with Cyclophosphamide in Children with Recurrent or Refractory Neuroblastoma and Cortical Bone Involvement

What is the purpose of this study?
The purposes of this study are:

• To find the highest dose of monthly intravenous Zometa that can be given with daily low doses of cyclophosphamide by mouth without causing severe side effects,
• To find out the side effects seen by giving Zometa and cyclophosphamide on this schedule at different dose levels,
• To measure blood and urine levels of Zometa during treatment,
• To determine if the tumor gets smaller after treatment with Zometa and cyclophosphamide,
• To measure the effects of Zometa on markers of bone breakdown found in urine, blood, and bone marrow, and
• To measure the effects of Zometa on the immune system.

Who will be included in this study?
Patients who are up to 30 years old at initial diagnosis who have neuroblastoma may be eligible to participate in this clinical trial.

What is involved?
Patients can continue to get Zometa every 28 days and cyclophosphamide every day as long as the neuroblastoma responds to the treatment or patients do not have bad side effects from taking either drug. After patients stop treatment with Zometa and cyclophosphamide, they will continue to have tests and scans done to measure how much tumor is left. Evaluations for this study will continue unless the tumor gets worse or the patient starts treatment on another study. Researchers will continue to collect information for a lifetime.

Before patients begin the study, they will need to have the following exams, tests, or procedures done to find out if they can be in the study.

• Physical exam
• Bone marrow tests
• Blood tests
• Various scans
• Pregnancy test
• Urine tests
• CT and/or MRI scans
• MIBG scans or bone scans
• Plain x-rays

The following exams, tests or procedures will be performed during the study.

• Physical exam
• Bone marrow tests
• Blood tests
• Various scans
• Urine tests
• Optional research studies

What are the benefits?
Participants who agree to take part in this research study may experience no direct benefit. It is unlikely that patients will experience significant benefit or cure from using Zometa and cyclophosphamide together as a treatment for their disease. The potential benefit is that using Zometa and cyclophosphamide together may cause the tumor to stop growing or go into partial remission for a period of time or it may lessen symptoms. Because there is not much information about the effects of using Zometa and cyclophosphamide together in children, we do not know if patients will benefit from taking part in this study. The knowledge gained from this study may benefit others. The disease may continue to become worse despite taking this drug.

Will I get all the facts about the study?
Parents interested in having their child participate, as well as adult patients/subjects, will be scheduled with a physician who will thoroughly explain all of the details of the study. The physician will review the consent form with the parent/guardian/adult subject and will be sure that all questions are answered. The consent form goes over all of the procedures, the risks, the benefits, the compensation, who to contact with questions or concerns and more. Study procedures will not begin until a parent/guardian or adult subject has signed this form and, if of age, the child has given at minimum his/her verbal agreement.

What are the risks?
A detailed list of side effects will be provided to those patients interested in knowing more about the study.

If you would like to learn more about participating in this study, please send an e-mail message using the form below.