Introduction

This family protocol summary is intended to provide a general overview of the Children’s Oncology Group study #ACCL1031. It will provide information about children who 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 healthcare team.

ACCL1031 is a called a Cancer Control Study. Cancer Control studies investigate the broad area called supportive care. These studies involve measurement, treatment, and prevention of the complications of cancer and cancer treatment. To reduce the effects of these complications, Cancer Control studies investigate traditional medical treatments, non-traditional medications classified as complementary and alternative medicines, and neuropsychological approaches. Cancer control studies may focus on complications that have a major impact on the quality of life or on life expectancy, or they may involve measurement and evaluation of treatment associated behaviors. This study will help to improve/expand what is known about the treatment of mucositis during stem cell transplants (SCT).

If you consent to being a part of this study, you or your child will receive one of two different treatment plans. The treatment plan 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. The randomization process is described in the COG Family Handbook for Children with Cancer. Some patients will be randomized to receive Caphosol; others will be randomized to receive saline (salt water) rinse used as a placebo. A placebo is an inactive substance or treatment that looks the same as, and is given in the same way as an active drug or treatment being tested. The effects of the active drug or treatment are compared to the effects of the placebo. However, even the placebo saline treatment used 4 times each day is likely to be beneficial to patients.

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 at www.childrensoncologygroup.org Always discuss any questions that you may have with your health care team.

Study Number

ACCL1031

Official Title

Phase III Study for patients undergoing myeloablative autologous or allogeneic Hematopoietic Stem Cell Transplantation. Limited to COG-Accredited HSCT Centers Registered with the Center for International Blood and Marrow Transplant Research Registry (CIBMTR).

Trial Opening Date

The trial opened in March 2011 and will enroll 200 patients over a 3 year period.

General Patient Eligibility

Age: 4 - 21 Treatment: Most patients who are going through stem cell transplant (SCT) can be on the study. However, patients cannot be in the study if they:

• are having a reduced intensity transplant
• Have received a drug called palifermin to prevent mouth sores in the past 30 days
• have ever received the drug Caphosol
• Please consult your doctor to determine whether your child may participate in this study.

General Background and Study Goal

Oral mucositis is another name for painful sores in the mouth. Oral mucositis is important for the following reasons: It is painful for patients and the pain may prevent patients from eating or drinking (and therefore require feeding/hydration through a tube in the vein in severe cases); Infections may occur as a result of mucositis. There is no standard treatment to prevent oral mucositis in children, adolescents, and young adults. Treatments used in the past to try to reduce mucositis or make patients more comfortable include ice chips, mouthwashes, and a variety of different drugs. Many different treatments are used and work to some extent, but none have prevented mucositis from occurring in the first place or have been shown to make it heal faster.

Caphosol is a mouth rinse, and some studies have shown it improves and/or prevents mucositis in adults undergoing SCT. It is approved for use in adults. Caphosol has not been used in children, adolescents, or young adults undergoing SCT. Study doctors would like to know how well Caphosol works to prevent and treat mucositis in these patients. If Caphosol is able to prevent severe mucositis, patients may be able to be in less pain, continue eating on their own, and have fewer (or no) infections. Now we would like to see if the same benefits can be found in children.

The overall goal of this study is to compare the effects of Caphosol versus saline rinse for the treatment of mucositis in children, adolescents, and young adults undergoing SCT. In this study, patients get either Caphosol or saline rinse, but do not get both.

Summary of the Treatment

In this study patients get one of two treatment plans. The two treatment plans are the same except for the different mouth rinse you will use. The treatment on this study will take about one month.

The treatment involves the use of Caphosol or a saline rinse (salt water rinse) starting on the first day of the conditioning regimen (the first day of chemotherapy and/or radiation therapy) for SCT. You will use the study rinse 4 times each day. However, if you develop severe mucositis, you have the choice to use the study rinse up to 6 times each day. Treatment with Caphosol or the saline rinse will continue until Day +20 after your stem cell transplant unless you are discharged sooner, whichever happens first. The study doctors will continue to examine your mouth until Day +20 unless you are discharged sooner.

Special Considerations

Detailed assessments of the mouth will be done daily during the study and will include the ratings from the child or parent about the child’s pain, eating, drinking, and swallowing.

During the study certain other medicines to manage mouth sores are allowed, including: lidocaine, “magic mouthwash” (lidocaine, diphenhydramine and Maalox) and pain medication. However, the following medicines are not allowed: Palifermin, topical glutamine and Gelclair.

Risks and Side Effects

Medications 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.

Contact Information

Your child’s oncologist is the best source for further information.

Study Chair

Nathaniel S. Treister, DMD, DMSc

FAMILY PROTOCOL SUMMARY REVIEW/APPROVALS

Initial development    Name    Date
Written by (protocol nurse)    Ellen Olson    February 2012
Reviewed/approved by (PI)    Nathaniel Treister    February 2012

Ongoing review       
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