Clinical trial · Interventional
N99-02: Melphalan and Buthionine Sulfoximine
Modulation of Intensive Melphalan (L-PAM) by Buthionine Sulfoximine (BSO) Autologous Stem Cell Support for Resistant or Recurrent High-Risk Neuroblastoma (IND 69-112)
- Source
- ClinicalTrials.gov
- Retrieved
- Sep 8, 2026
- Layer
- normalized (units and labels harmonized; values unchanged)
- Run
- ING-CLINICALTRIALS-20260908-000001
Summary
Brief summary (as posted)
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.
Conditions
Conditions (1)
Free-text conditions as registered, with the CancerIndex entity they were reconciled to and the match type.
| Condition (as posted) | Mapped entity | Match | Confidence |
|---|---|---|---|
| Neuroblastoma | Neuroblastoma | ONTOLOGY_EXACT | 0.90 |
Interventions
Interventions (4)
| Intervention | Type | Mapped drug | Match |
|---|---|---|---|
| buthionine sulfoximine | Drug | — | UNRESOLVED |
| Filgrastim | Other | — | UNRESOLVED |
| melphalan | Drug | Melphalan | ALIAS |
| Peripheral blood stem cell infusion | Procedure | — | UNRESOLVED |
Design
Arms and outcomes
Arms (1)
- type
- EXPERIMENTAL
- label
- Single Group
- interventionNames
- Drug: buthionine sulfoximine
- Drug: melphalan
- Procedure: Peripheral blood stem cell infusion
- Other: Filgrastim
Primary outcomes (1)
- measure
- 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
Secondary outcomes (4)
- measure
- To determine the pharmacokinetics (PK) of BSO and L-PAM in pediatric patients.
- timeFrame
- For BSO: just before start of therapy to 8 hours post end of 72 infusion. For L-PAM : just before start of 2nd dose to 4 hours post.
Eligibility
Eligibility (as posted)
- Sex
- All
- Maximum age
- 30 Years
Show eligibility criteria text
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.
References
Publications (1)
- BACKGROUNDVillablanca JG, Volchenboum SL, Cho H, Kang MH, Cohn SL, Anderson CP, Marachelian A, Groshen S, Tsao-Wei D, Matthay KK, Maris JM, Hasenauer CE, Czarnecki S, Lai H, Goodarzian F, Shimada H, Reynolds CP. A Phase I New Approaches to Neuroblastoma Therapy Study of Buthionine Sulfoximine and Melphalan With Autologous Stem Cells for Recurrent/Refractory High-Risk Neuroblastoma. Pediatr Blood Cancer. 2016 Aug;63(8):1349-56. doi: 10.1002/pbc.25994. Epub 2016 Apr 19. PMID 27092812