Clinical trial · Interventional
Busulfan in Treating Children and Adolescents With Refractory CNS Cancer
Phase I Study of Intrathecal Spartaject-Busulfan in Children With Neoplastic Meningitis
- 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. PURPOSE: Phase I trial to study the safety of delivering intrathecal busulfan in children and adolescents who have refractory CNS cancer and to estimate the maximum tolerated dose of this treatment regimen.
Conditions
Conditions (7)
Free-text conditions as registered, with the CancerIndex entity they were reconciled to and the match type.
| Condition (as posted) | Mapped entity | Match | Confidence |
|---|---|---|---|
| Brain and Central Nervous System Tumors | — | UNRESOLVED | — |
| Childhood Germ Cell Tumor | Childhood Germ Cell Tumor | ONTOLOGY_EXACT | 0.98 |
| Leukemia | Leukemia | ONTOLOGY_EXACT | 0.90 |
| Lymphoma | Lymphoma | ONTOLOGY_EXACT | 0.90 |
| Metastatic Cancer | Malignant Neoplasm | CURATED_BROADER | 0.78 |
| Retinoblastoma | Retinoblastoma | ONTOLOGY_EXACT | 0.98 |
| Sarcoma | Sarcoma | ONTOLOGY_EXACT | 0.98 |
Interventions
Interventions (1)
| Intervention | Type | Mapped drug | Match |
|---|---|---|---|
| busulfan | Drug | Busulfan | ALIAS |
Design
Arms and outcomes
Arms (0)
[]Primary outcomes (3)
- measure
- Toxicities of IT administered busulfan in children and adolescents with refractory CNS malignancies
- measure
- Maximum tolerated dose of IT administered busulfan
- measure
- Serum and CSF pharmacokinetics of IT administered busulfan
Eligibility
Eligibility (as posted)
- Sex
- All
- Minimum age
- 3 Years
- Maximum age
- 21 Years
Show eligibility criteria text
DISEASE CHARACTERISTICS:
* Histologically confirmed CNS malignancy, including any of the following:
* Primary malignant brain tumor refractory to standard therapy and metastatic to the cerebrospinal fluid (CSF) or leptomeningeal subarachnoid space
* Recurrent or persistent leptomeningeal leukemia, lymphoma, or germ cell tumor refractory to conventional therapy
* In second or greater relapse
* CSF white blood count greater than 5 cells/mm3 with blasts on cytospin OR
* Evidence of leptomeningeal tumor by MRI
* No concurrent bone marrow disease
* No obstruction or compartmentalization of CSF flow on CSF flow study
PATIENT CHARACTERISTICS:
Age:
* 3 to 21
Performance status:
* Lansky 50-100% (under 10 years)
* Karnofsky 50-100% (10 to 21 years)
Life expectancy:
* Greater than 8 weeks
Hematopoietic:
* Absolute neutrophil count greater than 1,000/mm\^3
* Platelet count greater than 75,000/mm\^3
Hepatic:
* Bilirubin normal for age
* ALT and AST less than 5 times upper limit of normal (ULN)
* No hepatic disease
Renal:
* Creatinine no greater than 1.5 times ULN OR
* Glomerular filtration rate greater than 70 mL/min
* No renal disease
Cardiovascular:
* No cardiac disease
Pulmonary:
* No pulmonary disease
Other:
* No uncontrolled infection
* Not pregnant or nursing
* Negative pregnancy test
* Fertile patients must use effective contraception
PRIOR CONCURRENT THERAPY:
Biologic therapy:
* Not specified
Chemotherapy:
* At least 3 weeks since prior myelosuppressive chemotherapy (6 weeks for nitrosoureas)
* At least 1 week since prior intrathecal chemotherapy (2 weeks for cytarabine) and recovered
* Evidence of subsequent disease progression
* Concurrent systemic chemotherapy allowed for recurrent disease after first course of treatment except for the following:
* Chemotherapy targeted at leptomeningeal disease
* Other phase I agent
* Any agent that significantly penetrates the CSF (e.g., high dose methotrexate greater than 1 g/m2, thiotepa, high dose cytarabine, fluorouracil, IV mercaptopurine, nitrosoureas, or topotecan)
* Any agent that causes serious unpredictable CNS side effects
Endocrine therapy:
* Prior dexamethasone allowed with decreasing or stable dose at least one week before study
* Concurrent dexamethasone or prednisone with chemotherapy regimen allowed
Radiotherapy:
* At least 1 week since prior focal irradiation to the brain or spine
* At least 8 weeks since prior craniospinal irradiation
* No concurrent cranial or craniospinal irradiation
Surgery:
* Not specified
Other:
* No other concurrent intrathecal or systemic therapy for leptomeningeal diseaseReferences
Publications (1)
- RESULTGururangan S, Petros WP, Poussaint TY, Hancock ML, Phillips PC, Friedman HS, Bomgaars L, Blaney SM, Kun LE, Boyett JM. Phase I trial of intrathecal spartaject busulfan in children with neoplastic meningitis: a Pediatric Brain Tumor Consortium Study (PBTC-004). Clin Cancer Res. 2006 Mar 1;12(5):1540-6. doi: 10.1158/1078-0432.CCR-05-2094. PMID 16533779