Clinical Trials

Study : NANT 2001-03

CEP-701 in Refractory Neuroblastoma Patients

Protocol Title: A Phase I Study Of CEP-701 In Patients With Refractory Neuroblastoma

Study Chair: John Maris, MD. Children's Hospital of Philadelphia

What is this study about:

This clinical research study uses the experimental drug CEP-701. CEP-701 is an anti-cancer drug that works differently than standard chemotherapy medicines. It blocks signals that help neuroblastoma cancer cells to live and grow. These signals are taken into the cell through proteins called "Trks" found on surface of most neuroblastoma cancer cells. CEP-701 has been found to block these signals in laboratory studies. CEP-701 has been given safely to adults with different cancers. This is first study of CEP-701 in children.

Why is this study being done:

• To test the safety of giving CEP-701 to children and see what effects (both good and bad) this treatment has on children and the neuroblastoma tumor.
• To find the highest dose of CEP-701 that can be given without causing severe side effects. This is done by dose escalation. Each patient in the study will only get one dose of the drug. Each dose level is tested in 3 to 6 patients at a time. The dose of CEP-701 will be increased ("dose escalation") from one group of patients to the next until side effects are seen. At this point researchers will have found the highest dose of CEP-701 that can be given safely to children.
• Other reasons this study is being done are to measure levels of CEP-701 in the blood and to see if CEP-701 is killing neuroblastoma tumor cells in the blood and bone marrow, and to see if the tumor gets smaller with CEP-701 treatment.

Criteria that need to be met to participate in this study:

• Patients must have been no greater than 21 years of age when they were first diagnosed with neuroblastoma.
• Patients must have relapsed neuroblastoma or persistent neuroblastoma that is not responding well to treatment. Patients who are in first response with tumor still present on CT/MRI or MIBG/Bone scans after treatment with stem cell transplant or those patients who are medically ineligible to have gotten treatment with stem cell transplant MUST have a surgical biopsy done of the tumor site seen on scan to verify that it is active neuroblastoma in order to enter on study. Patients who are in second response (patients who have already relapsed) do not need to have a biopsy done to enter on study.
• Patients must have adequate kidney, liver and bone marrow function. Patients who have bone marrow disease are eligible if they can meet the other organ function requirements.
• Patients must have normal heart and lung function.

Patients cannot participate in this study if:

• They have been treated with CEP-701 before.
• They are pregnant or breast feeding.
• They have an uncontrolled infection.
• They have a history of prior allogeneic stem cell transplantation AND active graft versus host disease of > Grade 2.

Study procedures:

CEP-701 is taken by mouth twice a day with or after breakfast and dinner for 5 days (Monday through Friday) followed by 2 days of rest (Saturday and Sunday) when no drug is taken. This is repeated 4 times for a total of 28 days for one "cycle" of treatment. The drug comes as a liquid and should be mixed with juice. You may continue to take Cep-701 for as long as your doctor feels it is helping you and is not causing severe side effects.

If you would like to download or print a sample consent form for this clinical trial, please click here : N01-03con.doc

If you would like to download this page in Microsoft Word format, please click here : N2001-03

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