CompletedPhase 1

N99-02: Melphalan and Buthionine Sulfoximine

United States, Canada31 participantsStarted 2001-08

Plain-language summary

RATIONALE: Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die. Combining chemotherapy with bone marrow or peripheral stem cell transplantation may allow the doctor to give higher doses of chemotherapy drugs and kill more tumor cells. PURPOSE: Phase I trial to study the effectiveness of melphalan and buthionine sulfoximine followed by bone marrow or peripheral stem cell transplantation in treating children who have resistant or recurrent neuroblastoma.

Who can participate

Age range

30 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion Criteria: * Patients have relapsed neuroblastoma and must have exhausted all other options for treatment before they can be considered for treatment on this study. * Relapsed patients who are greater than 6 months since having a stem cell transplant can enter on this study. * Patients must have stem cells collected and stored before starting treatment. * Patients must have a double lumen central venous line in place. * Patients must have adequate kidney and liver function measured by blood tests and test of renal function (creatinine clearance or glomerular filtration rate (GFR)). * Patients must have normal heart and lung function measured by lack of physical evidence or clinical history of difficulties breathing and tests of cardiac function (Echocardiogram or MUGA evaluation). * Patients must have an essentially normal neurological exam. * Patients must have one entire kidney that has not had any radiation at treatment doses. (Xrays and scans are ok). * Patients must have recovered from the effects of any prior treatment for their tumor. Exclusion Criteria: * They have had any radiation therapy to the brain. * They have known history of or current tumor found in the brain or surrounding tissues. * They have a history of seizures. * They have a history of changes in a test of kidney function with antibiotic use in the 6 months immediately before entering on this study.

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

To determine the maximum tolerated dose(MTD) and the toxicities of Melphalan (L-PAM) escalated in the presence of Buthionine sulphoxamine (BSO) and followed by autologous stem cells rescue for pediatric patients with high-risk neuroblastoma.

Timeframe: Within 4 weeks of completion of BSO/L-PAM therapy

Trial details

NCT IDNCT00005835

SponsorNew Approaches to Neuroblastoma Therapy Consortium

Sponsor typeOTHER

Study typeINTERVENTIONAL

Primary completion2014-12

View on ClinicalTrials.gov More Neuroblastoma trials