Trial Information

Summary: A Feasibility Pilot and Phase 2 Study of Chemoimmunotherapy with Epratuzumab for Children with Relapsed CD22-Positive Acute Lymphoblastic Leukemia

What is the purpose of this study?
There are two parts of this study. This first part is a Phase 1 study of a drug called epratuzumab, used alone and in combination with chemotherapy drugs used to treat acute lymphoblastic leukemia that has come beck (recurred). Epratuzumab is considered experimental because it has not been approved by the Food and Drug Administration (FDA). This study is being done to determine if epratuzumab that can be given safely, both alone and with other chemotherapy drugs. A safe medication is one that does not cause unacceptable side effects. After the feasibility part of this study has been completed, and found that epratuzumab could be given to children with acute lymphoblastic leukemia, another type of study, called a Phase 2 study will be done to find out if epratuzumab, when given along with chemotherapy, is a helpful and an effective therapy for treating children with acute lymphoblastic leukemia that has come back after treatment.

The goals of this study are:

• To find out if epratuzumab, given both alone and with chemotherapy drugs, can be given without causing severe side effects;
• To learn what kind of side effects epratuzumab, given with chemotherapy drugs, can cause;
• To learn more about the pharmacology (how the body handles the drug) of epratuzumab;
• To determine whether epratuzumab with chemotherapy is a beneficial treatment for this type of cancer;
• To learn more about this type of leukemia.

Patients who are 21 years old and younger who have experienced a relapse of acute lymphoblastic leukemia may be eligible to participate in this clinical trial.

What is involved?
Before beginning the study...
Participants will need to have the following exams, tests, or procedures to find out if they can be in the study. These exams, tests, or procedures are part of regular cancer care and may be done even if they do not join the study. If participants have had some of these tests recently, they may not need to be repeated. This will be up to the study doctor.

• Medical history
• Physical exam
• Vital signs such as blood pressure, pulse, and temperature
• Blood tests
• Urine tests
• Pregnancy test for women who could have children
• Lumbar puncture to test the fluid in the spinal cord
• Bone marrow test to check the leukemia

If the exams, tests, and procedures show that participants can be in this study, and they choose to take part in the study, several tests will need to be done to see how the leukemia is responding to this treatment, and to monitor for any side effects. These tests are a part of standard care for leukemia and may be done even if participants do not join this study:

• Physical exam; at least weekly
• Vital signs, such as blood pressure, pulse, temperature; weekly
• Blood tests; at least weekly
• Urine tests
• Bone marrow tests to find out if the disease is getting better
• Chest x-ray
• Echocardiogram to check heart function
• Lumbar puncture with every dose of intrathecal chemotherapy

Part A of the study is approximately 17 weeks, and Part B of the study is approximately 15 weeks.

We would like to continue to find out about the health of the participant for about 5 years after the study closes to new patients. If they do not visit our clinic at least once a year after treatment ends, we would like to do this by calling on the telephone once a year ( for 5 years) to check how the participant is doing. Keeping in touch and checking on how the participant’s health every year for a while after the study closes helps us understand the long-term effects of the study.

What are the benefits?
There may be no direct benefit to participants for taking part in this research study. It is unlikely that they will experience significant benefit or cure from this drug, epratuzumab. Because there is not much information about the effects of epratuzumab in children, we do not know if participants will benefit from taking part in this study. While doctors hope epratuzumab with chemotherapy will be more useful against leukemia compared to the usual treatment, there is no proof of this yet. The disease may continue to become worse despite taking this drug. The potential benefit is that epratuzumab may stop the relapsed acute lymphoblastic leukemia from growing or go into partial remission for a period of time. It may lessen the symptoms, such as pain, that are caused by the cancer. The knowledge gained from this study may benefit others in the future.

Will I get all the facts about the study?
Parents interested in having their child participate 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/patient 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 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.

Who should I contact for more information?

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