Introduction:

This family protocol summary is intended to provide a general overview of the Children’s Oncology Group study # AAML0531. It will provide information about the children that are eligible for this study and basic information regarding the specifics of the study. The informed consent and protocol contain the full details of the study. Those documents are available to you from your oncologist.

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 healthcare team in any way. You can find additional information about participation on clinical trials on CureSearch.org. Always discuss any questions that you may have with your healthcare team.

Brief Title:COG AAML0531 for newly diagnosed Acute Myeloid Leukemia in Children, Adolescents and Young Adults, A Phase III Groupwide Study

Official Title:A Phase III Randomized Trial of Gemtuzumab Ozogamicin (Mylotarg®) Combined with Conventional Chemotherapy for De Novo Acute Myeloid Leukemia (AML) in Children, Adolescents, and Young Adults

Trial Opening Date:Opened August 26, 2006. 1000 patients are to be enrolled over 3 ½ years.

General Patient Eligibility:

Patient Age: Patients between ages 1 month and age 30 are eligible for this study. If the patient also has Down syndrome, they must be age 4 or older.

For patients with: Newly diagnosed Acute Myeloid Leukemia (AML) that has not been treated with chemotherapy.

General Background and Study Goal

Nearly 500 children are diagnosed with AML every year and half are cured with standard therapy. In other words, half of the children diagnosed with AML and treated as described above remain with no signs of cancer (remission) for five years. The overall goal of this study is to see if we can increase this cure rate without causing more serious side effects of therapy. Side effects are unintended and unwanted results of treatment.

Researchers want to know if they can improve the cure rate for AML by adding a new chemotherapy drug, called gemtuzumab, to the standard chemotherapy treatments. gemtuzumab has been studied in adults with AML in combination with standard chemotherapy drugs. It has also been studied in small groups of pediatric patients. These studies have determined what dose of gemtuzumab can be given safely with other chemotherapy drugs.

Subjects (people participating in the study) will receive one of two different treatment plans. The treatment plan they receive is decided by a process called randomization. Randomization means that the treatment is assigned based on chance. It is a lot like flipping a coin, except that it is done by computer to make sure that there are about the same number of people on each treatment plan of the study.

In this study, randomization takes place at the beginning of the study. Subjects will be assigned to either Arm A, the current standard therapy, or Arm B, which is considered the experimental arm. Arm B uses the current standard therapy in combination with gemtuzumab. Gemtuzumab has been given to children with AML before but it has not yet been compared to the current therapy to see if it improves outcome.

Another goal of this study is to determine which children with AML need a stem cell transplant and which children do not. During intensification treatment, the study doctors will assign subjects to either receive more chemotherapy, or a stem cell transplant. This is decided based on two factors: 1) the subject’s risk of AML coming back, and 2) if they have a matched stem cell donor available.

We will also be studying how well children with Down syndrome do on the standard therapy. Down syndrome patients may respond differently to chemotherapy drugs than patients without Down syndrome. This study will give study doctors information about how Down syndrome patients respond to standard therapy.

Subjects with Down syndrome enrolled on this study will not be randomized, and will be assigned to Arm A, the standard therapy. Prior studies of children with Down syndrome and AML show that they are often more responsive to treatment but have more complications than children without Down syndrome. Therefore, there needs to be more experience gained in treating children with Down syndrome and they will not receive the additional experimental drug in this study

Summary of the Treatment

The treatment plan involves cancer fighting medicine called chemotherapy.The treatment on this clinical trial takes about 6 to 8 months. Multiple chemotherapy medicines are given. This is called combination chemotherapy. It is divided into 5 stages: Induction 1, Induction 2, Intensification 1, Intensification 2, and Intensification 3. Instead of Intensification 2 and 3 some subjects will receive a stem cell transplant. Details about the individual drugs and the schedule they are given on are listed in the informed consent document.

Risks and Side Effects

Chemotherapy can cause short- and long-term side effects. All patients will be closely monitored to reduce the likelihood of negative side effects. All risks and side effects will be explained in detail by your healthcare team during the consent process. They can answer any questions that you may have regarding participation on the clinical trial or other aspects of care. Please refer to the consent document for a detailed explanation of the side effects associated with the treatment on this study.

For subjects who are randomized to experimental Arm B, the use of gemtuzumab may cause more complications when combined with the use of standard chemotherapy treatment for AML. The combination of standard therapy with gemtuzumab may result in possible side effects, including a serious liver ailment called Veno-occlusive Disease (VOD). VOD is characterized by an increased bilirubin in the blood and possible jaundice (temporary yellowing of the skin), an enlarged and painful liver, and fluid retention with associated weight gain. In severe cases, VOD can be fatal. The risk of VOD may be greater if you get gemtuzumab and a stem cell transplant

Contact Information

Your child’s oncologist is the best source for further information.Physicians with eligible patients should refer questions to the principal investigator:

Principal Investigator Alan S Gamis, M.D., M.P.H.
Children’s Mercy Hospitals & Clinics
Division of Hematology/Oncology
2401 Gillham Road
Kansas City, MO 64108
Phone: (816) 234-3265
Fax: (816) 855-1700
E-mail:This email address is being protected from spambots. You need JavaScript enabled to view it.