Trial Information

Summary: A Phase II Study of Sulindac and Tamoxifen in Patients with Desmoid Tumors that are Recurrent or Not Amenable to Standard Therapy

What is the purpose of this study?
Sulindac is a drug approved to treat inflammation in patients with arthritis. Several published reports suggest that sulindac may have anti-tumor activity in adults with desmoid tumor. Tamoxifen is an estrogen-blocking drug approved for the treatment of breast cancer. Researchers have also reported that tamoxifen may have anti-tumor activity against desmoid tumors. Sulindac and tamoxifen have been investigated, separately and together, in recent clinical studies of adults and children with different types of tumor, including desmoid tumor. Pediatric researchers would like to determine whether this combination of sulindac and tamoxifen, taken by mouth, can be tolerated by children and whether this combination has anti-tumor activity against desmoid tumor in children whose tumor cannot be treated with surgery or radiation therapy or whose desmoid tumor has returned.

This study is being done:

• To determine the effects (good and bad) of the combination of sulindac and tamoxifen
• To determine if sulindac and tamoxifen are effective for treating desmoid tumors
• To study tumor samples in a laboratory and find out if there is a way to tell if tumor cells will respond to therapy with sulindac and tamoxifen

Who will be included in this study?
Patients who are 18 years old or younger at initial diagnosis who have a rare soft tissue tumor called a desmoid tumor (desmoid tumors are also called fibromatosis or aggressive fibromatosis) that cannot be treated with surgery and/or radiation, or because the tumor is recurrent, may be eligible to participate in this clinical trial.

What is involved?
Patients will be treated on this study for up to one year. However, patients will continue to have physical exams and blood tests for a few years after treatment so that researchers can continue to observe any effects of treatment and to monitor for tumor recurrence. Children’s Oncology Group would like to collect information about how patients are doing for about five years after the study closes to new patients. Keeping in touch with patients and checking on how their health is every year for awhile after the study closes helps us understand the long-term effects of the study.

Standard Medical Tests
Before treatment on this study begins, and while receiving treatment, patients will receive a series of standard medical tests:

• Physical exam
• Blood tests (tests to determine if the bone marrow, liver, and kidneys are functioning properly)
• Endocrine tests (tests to determine if normal levels of thyroid and reproductive hormones are present)
• MRI scan of the tumor
• Chest X-ray

Participants in this study will have the following tests performed more often:

• Endocrine tests (tests to determine if normal levels of thyroid and reproductive hormones are present)
• Tests for finding out about bone age (usually x-rays of the wrist) and density (DEXA x-ray scan which measures bone loss)
• Tests to find out how patients are maturing (measurement of height, weight, and assessment of age-appropriate sexual development)
• Optional research studies

What are the benefits?
Participants who agree to take part in this research study may or may not experience a direct medical benefit. Participants may benefit from the use of the two experimental drugs. If the tumor shrinks as a result of taking these drugs, patients may then be able to have surgery to completely remove the tumor without having cosmetic problems or loss of function. The information learned from this research study may benefit other patients with desmoid tumors 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.