Clinical trial · Interventional
Phase II Study of Intraventricular Methotrexate in Children With Recurrent or Progressive Malignant Brain Tumors
A Phase II Study of Intraventricular Methotrexate With Systemic Topotecan and Cyclophosphamide in Children With Recurrent or Progressive Malignant Brain Tumors
NCT02684071CI-TRIAL-00042693terminatedPhase 2ClinicalTrials.gov clinicaltrialsProvenance
- Source
- ClinicalTrials.gov
- Retrieved
- Sep 8, 2026
- Layer
- normalized (units and labels harmonized; values unchanged)
- Run
- ING-CLINICALTRIALS-20260908-000001
Why stopped (as posted): Lack of additional funding; patients (3) no longer receiving intervention.
Summary
Brief summary (as posted)
The purpose of this research study is to test an experimental treatment method for recurrent or progressive brain tumors in children aged from 0-22 years. The use of methotrexate and chemotherapy (topotecan and cyclophosphamide) is experimental in this study. This means that their use by themselves or together has not been approved by the U.S. Food and Drug Administration for this usage.
Conditions
Conditions (5)
Free-text conditions as registered, with the CancerIndex entity they were reconciled to and the match type.
| Condition (as posted) | Mapped entity | Match | Confidence |
|---|---|---|---|
| Childhood Atypical Teratoid/Rhabdoid Tumor | Childhood Atypical Teratoid/Rhabdoid Tumor | ONTOLOGY_EXACT | 0.98 |
| Embryonal Tumor With Abundant Neuropil and True Rosettes | Embryonal Tumor with Multilayered Rosettes, C19MC-Altered | ALIAS | 0.90 |
| Metastatic Malignant Neoplasm to the Leptomeninges | — | UNRESOLVED | — |
| Recurrent Childhood Ependymoma | Childhood Ependymoma | CURATED_BROADER | 0.78 |
| Recurrent Childhood Medulloblastoma | Childhood Medulloblastoma | CURATED_BROADER | 0.78 |
Interventions
Interventions (3)
| Intervention | Type | Mapped drug | Match |
|---|---|---|---|
| cyclophosphamide | Drug | Cyclophosphamide | ALIAS |
| Intra thecal methotrexate | Drug | — | UNRESOLVED |
| topotecan | Drug | Topotecan | ALIAS |
Design
Arms and outcomes
Arms (1)
- type
- EXPERIMENTAL
- label
- Intra thecal methotrexate
- description
- IT methotrexate via Ommaya reservoir with concomitant systemic topotecan and cyclophosphamide
- interventionNames
- Drug: Intra thecal methotrexate
- Drug: topotecan
- Drug: cyclophosphamide
Primary outcomes (1)
- measure
- Tumor response
- timeFrame
- 6 months
- description
- MRI response
Secondary outcomes (1)
- measure
- Toxicity of chemotherapy regimen (topotecan and cyclophosphamide)
- timeFrame
- 6 months
Eligibility
Eligibility (as posted)
- Sex
- All
- Maximum age
- 22 Years
Show eligibility criteria text
Inclusion Criteria: * Recurrent or progressive supratentorial or posterior fossa tumors with measurable disease on imaging studies of the brain and spine or CSF cytology. * Tumor histology, including: Medulloblastoma , PNET, or other embryonal tumor, Atypical teratoid/rhaboid tumor (AT/RT), and Ependymoma. * Leptomeningeal dissemination of a previously diagnosed CNS tumor. * Diagnosis of tumor pathology will be based upon pathology diagnosis from previous surgeries for patients with recurrent tumors. * Patients must have received prior radiation before current recurrence, unless the patient is less than 36 months old at diagnosis and has progressed after at least one upfront chemotherapy regimen in which case no prior radiation is required. * Patients must have a life expectancy of at least 12 weeks as indicated by the patient's oncologist and/or neurosurgeon. * Lansky or Karnofsky Performance status of at least 50. * Negative pregnancy test. * Specific organ function requirements for: Central Nervous System, Bone Marrow, renal and liver. Exclusion Criteria: * Patients that do not meet the inclusion criteria above. * Pregnant or lactating female patients. * Patients currently enrolled in another experimental treatment protocol. * Patients with documented allergies to any of the chemotherapy agents used in this study. * Patient/Parent refuses study participation. * Patient is severely somnolent or comatose. * Unable or unwilling to commit to return or to follow-up visits.
References
Publications (6)
- BACKGROUNDGururangan 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
- BACKGROUNDSlavc I, Schuller E, Czech T, Hainfellner JA, Seidl R, Dieckmann K. Intrathecal mafosfamide therapy for pediatric brain tumors with meningeal dissemination. J Neurooncol. 1998 Jun-Jul;38(2-3):213-8. doi: 10.1023/a:1005940405165. PMID 9696374
- BACKGROUNDYoshimura J, Nishiyama K, Mori H, Takahashi H, Fujii Y. Intrathecal chemotherapy for refractory disseminated medulloblastoma. Childs Nerv Syst. 2008 May;24(5):581-5. doi: 10.1007/s00381-007-0538-8. Epub 2007 Dec 5. PMID 18057943
- BACKGROUNDSandberg DI, Solano J, Petito CK, Mian A, Mou C, Koru-Sengul T, Gonzalez-Brito M, Padgett KR, Luqman A, Buitrago JC, Alam F, Wilkerson JR, Crandall KM, Kuluz JW. Safety and pharmacokinetic analysis of methotrexate administered directly into the fourth ventricle in a piglet model. J Neurooncol. 2010 Dec;100(3):397-406. doi: 10.1007/s11060-010-0210-0. Epub 2010 May 4. PMID 20440538
- BACKGROUNDSandberg DI, Crandall KM, Koru-Sengul T, Padgett KR, Landrum J, Babino D, Petito CK, Solano J, Gonzalez-Brito M, Kuluz JW. Pharmacokinetic analysis of etoposide distribution after administration directly into the fourth ventricle in a piglet model. J Neurooncol. 2010 Mar;97(1):25-32. doi: 10.1007/s11060-009-9998-x. Epub 2009 Aug 18. PMID 19688296
- RESULTSandberg DI, Crandall KM, Petito CK, Padgett KR, Landrum J, Babino D, He D, Solano J, Gonzalez-Brito M, Kuluz JW. Chemotherapy administration directly into the fourth ventricle in a new piglet model. Laboratory Investigation. J Neurosurg Pediatr. 2008 May;1(5):373-80. doi: 10.3171/PED/2008/1/5/373.