Clinical trial · Interventional
Study of Systemic and Spinal Chemotherapy Followed by Radiation for Infants With Brain Tumors
Pilot Study of Systemic and Intrathecal Chemotherapy Followed by Conformal Radiation for Infants With Brain Tumors
NCT00042367CI-TRIAL-00043785BB'98completedN/AClinicalTrials.gov clinicaltrialsProvenance
- 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)
The purposes of this study are to find the highest dose of mafosfamide that can be given without causing severe side effects, to see how well the combination of these chemotherapy drugs and lower doses of radiation work to delay or stop the growth of the tumor, and to evaluate the pharmacokinetics (how the body handles) of Mafosfamide.
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 |
|---|---|---|---|
| Brain Tumors | Brain Neoplasm | ALIAS | 0.90 |
Interventions
Interventions (10)
| Intervention | Type | Mapped drug | Match |
|---|---|---|---|
| Cycle B | Drug | — | UNRESOLVED |
| Induction therapy (Regimen 1, Course 1, Cycle A1 | Drug | — | UNRESOLVED |
| Intrathecal Mafosfamide | Drug | — | UNRESOLVED |
| radiation therapy | Radiation | — | UNRESOLVED |
| Regimen 1, Course 1, Cycle A2 (Days 22 - 42) | Drug | — | UNRESOLVED |
| Regimen 1, Course 2 | Drug | — | UNRESOLVED |
| Regimen 1 Course 2, IT mafosfamide | Drug | — | UNRESOLVED |
| Regimen 2, Course 1 | Drug | — | UNRESOLVED |
Design
Arms and outcomes
Arms (0)
[]Primary outcomes (2)
- measure
- To evaluate the feasibility, including expected disease progression, of delivering 20 weeks of systemic chemotherapy plus (IT) mafosfamide.
- timeFrame
- 20 weeks
- description
- controlling the disease for at least 20 weeks.
- measure
- To evaluate the safety and feasibility of a limited dose escalation schedule of IT mafosfamide in children < 3 years of age.
- timeFrame
- 5 years
- description
- Toxicities observed during the last 18 weeks of IT therapy (prior to irradiation) together with the acute toxicities profiles observed during the initial 2 weeks of IT therapy will be used to derive the dose to be evaluated in the feasibility component of this study.
Eligibility
Eligibility (as posted)
- Sex
- All
- Maximum age
- 36 Months
Show eligibility criteria text
Inclusion/Exclusion Criteria: * Age: \< 3 years. * Histology: Patient must have a histologically confirmed primary intracranial CNS medulloblastoma/PNET or other embryonal tumor (medulloepithelioma, ependymoblastoma, neuroblastoma, pineoblastoma), atypical teratoid/rhabdoid tumor, intracranial germ cell tumor, or choroid plexus carcinoma. Patients with M+ ependymoma are also eligible. * Performance Status: Karnofsky or Lansky \>= 30% * Bone Marrow Function: All patients must have a Hgb \>= 10 g/dl, ANC \>= 1,500/mm3, and a platelet \>= 100,000/mm3. If the patient has a positive bone scan, then a pretreatment bone marrow aspirate and biopsy must be free of tumor. * Hepatic/Renal Function: All patients must have adequate hepatic (total bilirubin \< 1.5 mg/dl, SGPT \< 5x normal), and renal (normal serum creatinine for age or technetium clearance \> 40/ml/min/m2) function. * Prior Therapy: Patients may not have received prior radiotherapy or chemotherapy, with the exception of steroids. Patients must not be receiving any other investigational agents. (Patients may receive investigational agents for supportive care 30 days after completion of all mafosfamide therapy.) * Surgery: Patients must begin protocol therapy within 35 days of definitive surgery. * Central Line: Patients must be willing to have a central line. * CSF flow: Patients must be willing to have a CSF flow study to determine whether or not they will receive intrathecal chemotherapy. Patients without a VP or VA shunt must be willing to have an Ommaya reservoir if their CSF flow study does not show any evidence of obstruction to or compartmentalization of flow. Patients with obstruction to or compartmentalization of CSF flow on their initial flow study must be willing to have a repeat flow performed within the initial 10 weeks of induction therapy, ideally during weeks 8-10. If a repeat flow study shows resolution of obstruction or compartmentalization, patients are expected to begin intrathecal mafosfamide during Regimen 1 course 2 of therapy. Patients without a VP or VA shunt who have resolution of normal flow should additionally have an Ommaya reservoir placed.
References
Publications (1)
- DERIVEDMcLendon RE, Adekunle A, Rajaram V, Kocak M, Blaney SM. Embryonal central nervous system neoplasms arising in infants and young children: a pediatric brain tumor consortium study. Arch Pathol Lab Med. 2011 Aug;135(8):984-93. doi: 10.5858/2010-0515-OAR1. PMID 21809989