ELIGIBILITY FOR STRATUM S PATIENTS TO START MAINTENANCE CHEMOTHERAPY
Participants must be Stratum S (SHH)
ELIGIBILITY FOR STRATUM S PATIENTS TO START MAINTENANCE CHEMOTHERAPY:
INCLUSION CRITERIA - STRATUM A:
INCLUSION CRITERIA - STRATUM B
Patient must have unresectable primary tumor or metastases (including tumors with an intralesional resection)\r\n* For Stratum 1, patients must have a confirmed histologic diagnosis of osteosarcoma\r\n* For Stratum 2, any histologically confirmed solid tumor diagnosis is eligible
PHASE II: Tumor tissue for correlative studies is required for all patients except those with NF1 and LGG (stratum 2) unless surgery was performed prior to enrollment or any patient with optic pathway glioma (stratum 2 or 3), for whom tumor tissue is optional
PHASE II: Patients for whom tumor biopsy and/or resection is clinically indicated and who are eligible for and enrolled on the phase II component (any stratum) will also be eligible for the optional target validation stratum
Stratum D: Patients must have a histologically confirmed diagnosis of ependymoma that is recurrent, progressive or refractory following therapy which included radiotherapy
Stratum E: Patients must have a histologically confirmed diagnosis of medulloblastoma that is recurrent, progressive or refractory following therapy which included radiotherapy
INCLUSION CRITERIA FOR STRATUM C: Patient must have completed immunotherapy (e.g. tumor vaccines, oncolytic viruses, etc.) at least 42 days prior to enrollment
INCLUSION CRITERIA FOR STRATUM C: Patients must be fully recovered from all acute effects of prior surgical intervention
INCLUSION CRITERIA FOR STRATUM C: Albumin >= 2 g/dl
INCLUSION CRITERIA FOR STRATUM C: Patients must be off all colony-forming growth factor(s) for at least 1 week prior to registration (i.e. filgrastim; sargramostim; erythropoietin); 2 weeks must have elapsed for long-acting formulations
INCLUSION CRITERIA FOR STRATUM C: Patients must be willing to use brief courses (at least 72 hours) of steroids as directed for potential inflammatory side effects of the therapy if recommended by their treating physician
EXCLUSION CRITERIA FOR STRATUM C: Patients who are receiving any other anti-cancer or investigational drug therapy are ineligible
Stratum I
Stratum II
Patients of Stratum I who have:
Active disease at the end of Stratum I treatment
Disease reactivation in non-risk organs at any time after completion of Stratum I treatment
Stratum III
Patients from Stratum I who fulfill the following criteria:
Stratum IV
Patients from Stratum I or Stratum III who fulfill the following criteria:
Stratum V
Stratum VII -- All patients registered in LCH IV (regardless of treatment) as long as consent for longterm follow-up has not been withheld.
Stratum I
Stratum II
Stratum III
Stratum IV
Stratum VI
Patients with SS-LCH who have an isolated tumorous CNS lesion (they are eligible for Stratum V),
Patients with isolated \CNS-risk\ or multifocal bone lesions (they are eligible for Stratum I, Group 2)
STRATUM A: Participants with subependymoma or myxopapillary ependymoma
STRATUM A: Female participants who are breastfeeding a child
STRATUM B: Female participants who are breastfeeding a child
STRATUM C: Female participants who are breastfeeding a child
In stratum A, patients must have failed one prior systemic chemotherapy regimen; in stratum B, patients must have failed one prior systemic chemotherapy regimen, which must include cladribine/cytarabine or are not considered to be eligible for such treatment; in stratum C, Rosai Dorfman disease (RDD) patients must have failed treatment with corticosteroid; Erdheim Chester disease (ECD) patients who have confirmed BRAF V600E mutation must have failed treatment with a BRAF inhibitor or are not considered to be eligible for such treatment
PHASE II: Patients with recurrent or progressive disease, as defined in the following three strata below, will be eligible; for eligibility determination, tumor imaging from at least two time-points must be available to document radiographic progression or recurrence; patients with non-progressive refractory tumors will not be eligible \r\n* Stratum 1: patients with LGG with a BRAF truncated fusion that is measurable in at least two dimensions on imaging\r\n* Stratum 2: patients with NF1 and LGG that is measurable in at least two dimensions on imaging\r\n* Stratum 3: pediatric patients with a recurrent or progressive tumor thought to involve the Ras/Raf/ERK pathway but not included in strata 1 or 2 that is measurable in at least two dimensions on imaging; this includes any LGG not included in strata 1 or 2 (i.e., any LGG without a BRAF truncated fusion in a patient without NF1), any tumor other than LGG in a patient with NF1, and any other tumor with a documented activating BRAF, NRAS, or KRAS mutation\r\n* Stratum 4 (surgical arm, target validation): patients who meet criteria for stratum 1, 2, or 3 for whom tumor biopsy and/or resection is clinically indicated
PHASE II: Tumor tissue for correlative studies is required for all patients except those with NF1 and LGG (stratum 2) or any patient with optic pathway glioma (stratum 2 or 3), for whom tumor tissue is optional
Patients with clinical contraindication against lumbar puncture or Ommaya placement are NOT eligible for either Stratum
At least 28 days must have elapsed (at time of starting protocol therapy) since completion of focal radiation therapy for current recurrence for Stratum 2; patients who have already undergone radiation therapy for current recurrence are NOT eligible for Stratum 1
Patients receiving systemic corticosteroids are NOT eligible for either Stratum
INCLUSION CRITERIA FOR PATIENTS ON ALL STRATA EXCEPT STRATUM P
EXCLUSION CRITERIA FOR ALL STRATA EXCEPT STRATUM P
INCLUSION CRITERIA FOR PATIENTS TO BE TREATED ON STRATUM A
Patients with progressive synchronous/metachronous AT/RT and MRT will be eligible for stratum A3
INCLUSION CRITERIA FOR PATIENTS ON STRATUM D
INCLUSION CRITERIA FOR PATIENTS ON STRATUM P
Biological parent of patient enrolling on SJATRT will be assigned to Stratum P
Patients with other malignancies will not be eligible for stratum I or II; patients with disseminated disease including to the spine will not be eligible for stratum 1 but will be eligible for stratum II
Patients with newly diagnosed DIPG who have received vorinostat previously will not be eligible for stratum I; patients with progressive DIPG will be eligible if they have received either one of the two drugs vorinostat or temsirolimus but will not be eligible for stratum II if have received both the drugs before
Patients with previous history of irradiation at any dose to thoracic-spine, ribs or >= 20% of bone marrow cannot undergo TMI and will be eligible for bortezomib, fludarabine, and melphalan regimen (Stratum II); patients can be enrolled on Stratum II at their physician's discretion or if patients decline radiation therapy
Patients in Stratum F must have received craniospinal radiation
Presence of metastatic disease for patients in Stratum A, B, D and E; patients with metastatic high grade gliomas diagnosed at < 1 year of age are eligible for Stratum D at the time of recurrence; patients with low grade gliomas (Stratum C) may have tumor spread within the CNS; patients in Stratum F must have tumor spread within the CNS
STRATUM II:\r\n* MDD >= 1% and MRD negative (< 0.01%) on day 8 in T-lymphoblastic lymphoma \r\n* Bone marrow involvement microscopically present at diagnosis in B-lymphoblastic lymphoma \r\n* Any CNS involvement: CNS-3 status (i.e., >= 5 WBC/uL of CSF with blasts or cranial nerve palsy), CNS-2 status (< 5 WBC/uL of CSF with blasts) or traumatic LP (> 10 RBC/uL of CSF with blasts) but does not fulfill the criteria of stratum 3 \r\n* Overt testicular involvement (evidenced by ultrasonogram) but does not fulfill the criteria of stratum 3
Patient must have a histologically verified diagnosis of craniopharyngioma\r\n* Stratum 1: patients with progressive unresectable or recurrent craniopharyngiomas treated with surgery alone, who have not received radiation therapy; patients with unresectable craniopharyngiomas, (i.e. residual measurable disease following surgical resection) will be enrolled at the time of progression\r\n* Stratum 2: patients with progressive or recurrent craniopharyngiomas following radiation therapy; the patient must be at least 6 months post irradiation to be eligible
Stratum 1: patients must not have received radiation therapy
Stratum 1 patients: must not have had > 3 surgical debulking procedures/resections
STRATUM A INCLUSION CRITERIA:
STRATUM B INCLUSION CRITERIA:
Metastatic disease for stratum B only
Specific eligibility criteria stratum 1:\r\n* Disease status: \r\n** Radiographic disease progression as defined for stratum 3 (below) is not required for trial entry\r\n** Patient does not have clinical symptoms from the plexiform neurofibroma
Specific eligibility criteria stratum 2:\r\n* Disease status: \r\n** Radiographic disease progression as defined for stratum 3 (below) is not required for trial entry\r\n** Patient has clinical symptoms from the plexiform neurofibroma
RECURRENT/ PROGRESSIVE DIPG (STRATUM 1): Absolute neutrophil count >= 1,000/mm^3
RECURRENT/ PROGRESSIVE DIPG (STRATUM 1): Albumin >= 3 g/dl
RECURRENT/ PROGRESSIVE DIPG (STRATUM 1): Patients who have had > 60 Gy total radiation to the pons (e.g. patients who have received re-irradiation)
RECURRENT/ PROGRESSIVE DIPG (STRATUM 1): Patients have had prior histone deacetylase (HDAC), deacetylase (DAC), heat shock protein 90 (HSP90) inhibitors for the treatment of their DIPG
RECURRENT/ PROGRESSIVE DIPG (STRATUM 1): Patients have had prior bone marrow transplant
RECURRENT/ PROGRESSIVE DIPG (STRATUM 1): Patients have significant acute deterioration in neurologic status in 72 hours prior to enrollment, in the opinion of the treating physician
RECURRENT/ PROGRESSIVE DIPG (STRATUM 1): Patients have any other significant concurrent illness
RECURRENT/ PROGRESSIVE DIPG (STRATUM 1): Patients have a history of any other malignancy
RECURRENT/ PROGRESSIVE DIPG (STRATUM 1): Patients who are receiving any other anticancer or investigational drug therapy
RECURRENT/ PROGRESSIVE DIPG (STRATUM 1): Patients who are required to receive any medication which can prolong the QTc interval
RECURRENT/ PROGRESSIVE DIPG (STRATUM 1): Female patient is breastfeeding
NON-PROGRESSED DIPG (STRATUM 2): Patients with DIPG who have not yet progressed by clinical or radiographic criteria\r\n* Please note: patients with a radiographically typical DIPG, defined as a tumor with a pontine epicenter and diffuse involvement of more than 2/3 of the pons, are eligible without histologic confirmation\r\n* Patients with pontine lesions that do not meet these radiographic criteria will be eligible if there is histologic confirmation of malignant glioma World Health Organization (WHO) II-IV
NON-PROGRESSED DIPG (STRATUM 2): Absolute neutrophil count >= 1,000/mm^3
NON-PROGRESSED DIPG (STRATUM 2): ALT(SGPT) =< 3 x institutional upper limit of normal
NON-PROGRESSED DIPG (STRATUM 2): Albumin >= 3 g/dl
NON-PROGRESSED DIPG (STRATUM 2): Potassium >= LLN
NON-PROGRESSED DIPG (STRATUM 2): Patient has no ventricular arrhythmias except for benign premature ventricular contractions
NON-PROGRESSED DIPG (STRATUM 2): Patient has a QTc interval < 450 ms
NON-PROGRESSED DIPG (STRATUM 2): Patients must be off all colony-forming growth factor(s) for at least 7 days prior to enrollment (i.e. filgrastim, sargramostim or erythropoietin); 14 days must have elapsed if patients received PEG formulations
NON-PROGRESSED DIPG (STRATUM 2): Patients who have had > 60 Gy total radiation to the pons (e.g. patients who have received re-irradiation
NON-PROGRESSED DIPG (STRATUM 2): Patients have significant acute deterioration in neurologic status in 72 hours prior to enrollment, in the opinion of the treating physician
NON-PROGRESSED DIPG (STRATUM 2): Patients have diarrhea > CTCAE grade 2
NON-PROGRESSED DIPG (STRATUM 2): Patients have a history of any other malignancy
NON-PROGRESSED DIPG (STRATUM 2): Patients who are receiving any other anticancer or investigational drug therapy
NON-PROGRESSED DIPG (STRATUM 2): Patients who are required to receive any medication which can prolong the QTc interval
NON-PROGRESSED DIPG (STRATUM 2): Female patient is breastfeeding
Skin lesions on the breast that disrupt the stratum corneum (e.g. eczema, ulceration)
Specific Criteria by Stratum: Stratum 1: All patients must have completed standard upfront therapy that replicates treatment which patients who were enrolled on ANBL0032 received, including: intensive induction chemotherapy and (if feasible) resection of primary tumor, followed by: consolidation with high-dose chemotherapy with stem cell transplant and radiotherapy, followed by: immunotherapy with Ch14.18/IL-2/GM-CSF (dinutuximab) and retinoic acid;. All subjects on Stratum 1 must have also met the following criteria: • A pre-transplant disease status evaluation that met International Neuroblastoma Response Criteria (INRC) for CR (complete response), VGPR (very good partial response), or PR (partial response) for primary site, soft tissue metastases and bone metastases. Patients who meet those criteria must also meet the protocol-specified criteria for bone marrow response prior to transplant as outlined below: No more than 10% tumor involvement (based on total nucleated cellular content) seen on any specimen from a bilateral bone marrow aspirate/biopsy. Stratum 2: Neuroblastoma that is in first complete remission following standard upfront therapy different from that described for Stratum 1. Stratum 3: Neuroblastoma that failed to have a response of at least PR following induction chemotherapy and surgical resection of the primary tumor, but that has achieved CR following additional therapy. Stratum 4: Patients who have achieved a second or subsequent CR following relapse(s).
Skin lesions on the breast that disrupt the stratum corneum (e.g., eczema, ulceration)
Skin lesions that disrupt the stratum corneum (eg., eczema, ulceration) or any breakdown of the skin