Trial Information

Summary: Phase I Study of Fenretinide (4-HPR, NSC 374551) Lym-X-Sorb™ (LXS) Oral Powder In Patients with Recurrent or Resistant Neuroblastoma IND# 68,254

What is the purpose of this study?
Fenretinide is an anticancer agent that may work differently than standard chemotherapy medicines. It may cause the build up of a wax-like substance in neuroblastoma cancer cells, called "ceramide". In laboratory studies it was found that if too much ceramide builds up in neuroblastoma cells, they die.

Fenretinide has been given by mouth as a capsule to many people, including children. When fenretinide is given in capsules, very little of the drug is absorbed through the intestines into the body. That meant that patients had to take many capsules of fenretinide by mouth several times a day. In this study, a new oral preparation of fenretinide (called 4-HPR/LXS oral powder) is being tested to see if more fenretinide can be absorbed into the body. This is the first study of 4-HPR/LXS oral powder in either children or adults.

The purpose of this research study is to:

• Find the highest dose of fenretinide (also called 4-HPR) prepared in a special mixture of fats, sugar, and wheat flour (called Lym-X-Sorb? [LXS] oral powder) that can be given by mouth (orally), twice a day, for 7 days, every 3 weeks, without causing severe side effects
• Find out the side effects seen by giving 4-HPR/LXS oral powder by mouth on this schedule at different dose levels
• Measure blood levels of fenretinide during treatment
• Measure levels of fenretinide in normal white blood cells during treatment
• Determine if the tumor gets smaller after treatment with 4-HPR/LXS oral powder

Who will be included in this study?
Patients who are 30 years old or younger who have neuroblastoma may be eligible to participate in this clinical trial.

What is involved?
Patients can continue to take 4-HPR/LXS oral powder every 21 days for a total of six courses as long as the neuroblastoma responds to the treatment or the patient does not have bad side effects from taking 4-HPR/LXS oral powder. If the tumor is responding, and there is enough drug available, patients may be able to receive more than six courses of therapy with the agreement of the treating doctor. After the treatment with 4-HPR/LXS oral powder is stopped, patients will continue to have tests and scans done to measure how much tumor is left. The patients doctor will determine how often these tests will be done. Evaluations for this study will continue unless the tumor gets worse or the patient starts another treatment for neuroblastoma.

Required Medical Tests
You will have a series of standard medical tests before, during, and following treatment with 4-HPR/LXS oral powder. Researchers will use these tests to measure the effectiveness of the treatment and what it is doing to your body.

Medical Tests Before and During Treatment

• Physical exam
• Blood tests
• Pregnancy test
• Urine tests
• Tests of heart function
• Tests of vision
• Chest x-rays (only before treatment starts)
• Bone marrow tests
• Various scans
• Optional research tests

Medical Tests Following Treatment

• Physical exam
• Blood tests
• Urine tests
• Tests of heart function
• Bone marrow tests
• Various scans

What are the benefits?
Patients may experience no direct benefit for taking part in this research study. It is unlikely that patients will experience significant benefit or cure from this drug, fenretinide. The potential benefit is that fenretinide may cause the neuroblastoma to stop growing or go into partial remission for a period of time or it may lessen symptoms. Because there is not much information about the effects of fenretinide taken with 4-HPR/LXS oral powder in humans, we do not know if patients will benefit from taking part in this study. The knowledge gained from this study may benefit others with solid cancers. The disease may continue to become worse despite taking this drug.

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.

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