Clinical Trials

Study : NANT 2004-04

Oral Fenretinide LXS Powder in Recurrent or Resistant Neuroblastoma Patients

Protocol Title: A Phase I Study of Fenretinide Lym-X-Sorb^TM (LXS) Oral Powder in Patients with Recurrent or Resistant Neuroblastoma (IND # 68,254)

Study Chair: Barry Maurer MD, PhD. (Childrens Hospital Los Angeles)

What is this study about:

Fenretinide is an agent that works differently from standard chemotherapy drugs in treating cancer. Fenretinide may cause the build up of a wax-like substance in neuroblastoma cancer cells, called "ceramide". In laboratory studies, it has been found that if too much ceramide builds up in neuroblastoma cells, they die.

In the past, Fenretinide has been given to people (including children) with cancer. It has only been able to be given as a large capsule – this meant it was difficult for many children (especially ones who are small) to swallow the number of pills they needed to. When Fenretinide is given in capsules, very little of the drug is absorbed through the intestines into the body. In this study, an investigational oral preparation of fenretinide (called 4-HPR/LXS powder) is being tested to see if more fenretinide can be absorbed into the body than was seen with the large capsules. This is the first study of 4-HPR/LXS powder in either children or adults. This investigational oral preparation of fenretinide has not been approved by the Food and Drug Administration (FDA). This clinical research study will help find out the highest dose of oral fenretinide LXS powder that can be safely given to patients with resistant or recurrent neuroblastoma.

Why is this study being done:

• To test the safety of giving fenretinide prepared in a special mixture of fats, sugar and wheat flour (called Lym-X-Sorb^TM (LXS) or powder) in children and see what effects (good and bad) this treatment has on children and the neuroblastoma tumor.
• To find the highest dose of oral fenretinide LXS powder that can be given by mouth twice a day for 7 days every 3 weeks without causing severe (bad) side effects. This is done by dose escalation. Each dose level of oral fenretinide LXS powder is tested in groups of 3 to 6 patients at a time. Each patient will only get one dose level of oral fenretinide LXS powder for the study. The dose of oral fenretinide LXS powder will be increased (dose escalation) from one group of patients to the next until side effects are seen. At this point researchers will have found the highest dose of oral fenretinide LXS powder that can be given safely to children.
• To measure the levels of oral fenretinide LXS powder in the blood, and to see if the tumor gets smaller with treatment.
• To measure levels of fenretinide in normal white blood cells during treatment.

Criteria that need to be met to participate in this study:

• Patients must be no more than 30 years of age when registerd on this study.
• Patients must have relapsed neuroblastoma, refractory neuroblastoma that had less than a partial response to standard treatment or persistent neuroblastoma that had at least a partial response to standard treatment.

  Patients who have at least a partial response to standard treatment who still have neuroblastoma that can be seen on CT/MRI or MIBG scans must have a surgical biopsy done of the tumor to confirm that it is neuroblastoma. Patients with relapsed or refractory neuroblastoma do not need to have a biopsy done to enter on study.

• Patients must have adequate heart, kidney, liver and bone marrow function. Patients who have bone marrow disease must meet the bone marrow function criteria to enter the study.
• Patients must consent to participate in pharmacokinetic studies (measure the level of fenretinide in the blood).

Patients cannot participate in the study if:

• They have been treated before with Fenretinide. They can enter on study if they had treatment before with other retinoids such as 13-cis-Retinoic Acid.
• Neuroblastoma is present in the brain on a CT or MRI scan done at study entry. Patients with neuroblastoma found in the bones of the skull are eligible if there is no tumor mass associated with them pressing on the brain.
• They are pregnant or breast feeding.
• They have an allergy to soy products or allergy or sensitivity to wheat gluten.
• They need medications to maintain a stable heart rhythm.
• They have had an organ transplant (like kidney or liver) or an allogeneic stem cell transplant in the past. (Using stem cells from a donor other than yourself).

Study procedures:

Oral Fenretinide LXS powder is mixed in SlimFast Meal^TM or Slim Fast Optima^TM or Optima High Protein^TM to drink by mouth. It is taken twice a day for seven days every three weeks (or 21 days). The reason we are using SlimFast products is because the fenretinide powder cannot be mixed with any product that has milk fats in it. You will have to buy SlimFast Meal^TM or Slim Fast Optima^TM or Optima High Protein^TM from a store. One “course” of treatment with Oral Fenretinide LXS powder is 21 days. You may continue with this treatment for 6 courses. If your tumor is responding and there is enough drug available, you may be able to receive more than 6 courses of treatment as long as your doctor feels it is helping you and it is not causing severe side effects.

If you would like to download or print a sample consent form for this clinical trial, please click here : N04-04 consent form

If you would like to download or watch a video describing this clinical trial, please click here: N04-04 video
The Childrens Hospital Los Angeles, Barry Maurer, M.D., PhD, and C. Patrick Reynolds, M.D., PhD., of Childrens Hospital Los Angeles hold patents and/or patent applications on how fenretinide (the study drug) was made into a Lym-X-Sorb oral powder and they may benefit financially from the future use of the study drug.

If you would like to download or print information about getting the correct Slim-Fast^TM product for this clinical trial, please click here: Getting the correct Slim-Fast

If you would like to download this page in Microsoft Word format, please click here : N2004-04

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