Modulation of Intensive Melphalan (L-PAM) by Buthionine Sulfoximine (BSO) with Autologous Stem Cell Support for Recurrent High-Risk Neuroblastoma (A Phase I Study)
What is the purpose of this study?
The goals of this research study are:
- to find the highest dose of melphalan that can be given with BSO without causing severe side effects
- to measure blood levels of melphalan and BSO during treatment
- to determine if the tumor responds (gets smaller) after receiving treatment
Who will be included in this study?
Patients with high risk neuroblastoma who are > 9 months and ≤ 30 years of age and whose disease has not responded to standard therapy are eligible to participate in this clinical trial.
What is involved?
Treatment on this study will last for at least 60 days or for as long as it takes for the stem cells to regrow and make enough normal blood cells to recover and for a recovery from any side effects of the treatment.
Medical tests before treatment:
- Physical exam
- Various scans such as CT / MRI scans and MIBG scans
- Blood tests
- Tests of kidney function
- Bone marrow tests
- Tests of heart and lung function
- Pregnancy test
- Tests of individual kidney function in patients with a history of getting radiation to their kidneys
- Urine tests
Medical tests during treatment:
- Physical exam
- Blood tests
- Urine tests
- Tests of individual kidney function in patients with a history of getting radiation to their kidneys
- Other optional laboratory studies
Medical tests after treatment:
- Physical exam
- Various scans
- Blood tests
- Tests of kidney function
- Bone marrow tests
- Tests of heart and lung function
- Urine tests
What are the benefits?
There may be no direct benefit for you, if you agree to take part in this research study. It is unlikely that you will experience significant benefit or cure from the drugs, Buthionine Sulfoximine (BSO) and melphalan. The potential benefit is that BSO and melphalan may cause your neuroblastoma to stop growing or go into partial remission for a period of time, which may allow you to receive other therapies. It may also lessen symptoms caused by your tumor. Because there is not much information about the effects of this treatment in humans, we do not know if you will benefit from taking part in this study. The knowledge gained from this study may benefit others. The disease may continue to become worse despite taking this drug.
Clinical studies are essential in finding new and improved treatments and diagnostic tools.
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. Your physician will review the consent form with you and will be sure that all of your 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?
Peggy Kaiser, RN, BSN
Cincinnati Children's Hospital Medical Center
Division of Hematology / Oncology
3333 Burnet Ave., Cincinnati, OH 45229-3039
Phone: 513-636-2799
cancer@cchmc.org
