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Clinical Trial COGANBL12P1

Title

Pilot Study Using Myeloablative Busulfan/Melphalan (BuMel) Consolidation Following Induction Chemotherapy for Patients with Newly Diagnosed High-Risk Neuroblastoma

Principal Investigator(s)

Haydar Frangoul

Details

  • Protocol No. COGANBL12P1
  • Open Date: 06/05/2013
  • Staging: Pilot
  • Age Group: Both Child and Adult
  • Scope: National
  • Objective: To determine if the acute toxicity of an autologous stem cell transplant with a busulfan/melphalan (BuMel) based regimen is tolerable when given as Consolidation therapy for high-risk neuroblastoma.
  • Disease Sites: Neuroblastoma (Pediatrics)
  • Therapies: Molecular Targeted Agents / Immunotherapy / Biologics
  • Drugs: Cisplatin; Cyclophosphamide (CTX); Doxorubicin; Etoposide; Topotecan (parenteral) IV; Vincristine
  • Participating Institutions: Vanderbilt University
  • National Clinical Trial ID: NCT01798004
  • Secondary Protocol No: ANBL12P1

Description

None Provided.

Eligibility

Ages Eligible for Study:N/A to 30 Years
Genders Eligible for Study:Both
Accepts Healthy Volunteers:No

Criteria

Inclusion Criteria:
• Patients must have a diagnosis of neuroblastoma (International Classification of Diseases for Oncology [ICD-O] morphology 9500/3) or ganglioneuroblastoma (nodular or intermixed) verified by histology or demonstration of clumps of tumor cells in bone marrow with elevated urinary catecholamine metabolites; patients with the following disease stages at diagnosis are eligible, if they meet the other specified criteria
• Patients with newly diagnosed neuroblastoma with International Neuroblastoma Staging System (INSS) stage 4 are eligible with the following:
• MYCN amplification (> 4-fold increase in MYCN signals as compared to reference signals), regardless of age or additional biologic features
• Age > 18 months (> 547 days) regardless of biologic features
• Age 12-18 months (365-547 days) with any of the following 3 unfavorable biologic features (MYCN amplification, unfavorable pathology and/or DNA index = 1) or any biologic feature that is indeterminate/unsatisfactory/unknown
• Patients with newly diagnosed neuroblastoma with INSS stage 3 are eligible with the following:
• MYCN amplification (> 4-fold increase in MYCN signals as compared to reference signals), regardless of age or additional biologic features
• Age > 18 months (> 547 days) with unfavorable pathology, regardless of MYCN status
• Patients with newly diagnosed neuroblastoma with INSS stage 2A/2B with MYCN amplification (> 4-fold increase in MYCN signals as compared to reference signals), regardless of age or additional biologic features
• Patients with newly diagnosed neuroblastoma with INSS Stage 4S with MYCN amplification (> 4-fold increase in MYCN expression signals as compared to reference signals), regardless of additional biologic features
• Patients >= 365 days initially diagnosed with neuroblastoma INSS stage 1, 2, 4S who progressed to a stage 4 without interval chemotherapy; these patients must have been enrolled on ANBL00B1; study enrollment on ANBL12P1 must occur within 4 weeks of progression to stage 4 for INSS stage 1, 2, 4S
• Patients must not have had prior systemic therapy except for localized emergency radiation to sites of life-threatening or function-threatening disease and/or no more than 1 cycle of chemotherapy per a low or intermediate risk neuroblastoma regimen (as per P9641, A3961, ANBL0531, or similar) prior to determination of MYCN amplification status and histology
• Creatinine clearance or radioisotope glomerular filtration rate (GFR) >= 70 mL/min/1.73 m^2 or a serum creatinine based on age/gender as follows:
• Age 1 month to < 6 months: 0.4 mg/dL
• Age 6 months to < 1 year: 0.5 mg/dL
• Age 1 to < 2 years: 0.6 mg/dL
• Age 2 to < 6 years: 0.8 mg/dL
• Age 6 to < 10 years: 1 mg/dL
• Age 10 to < 13 years: 1.2 mg/dL
• Age 13 to < 16 years: 1.5 mg/dL (males), 1.4 mg/dL (females)
• Age >= 16 years: 1.7 mg/dL (males), 1.4 mg/dL (females)
• Total bilirubin =< 1.5 x upper limit of normal (ULN) for age, and
• Serum glutamic oxaloacetic transaminase (SGOT) (aspartate aminotransferase [AST]) or serum glutamate pyruvate transaminase (SGPT) (alanine aminotransferase [ALT]) < 10 x ULN for age
• Shortening fraction of >= 27% by echocardiogram, or ejection fraction of >= 50% by radionuclide evaluation
• No known contraindication to peripheral blood stem cell (PBSC) collection; examples of contraindications might be a weight or size less than that determined to be feasible at the collecting institution, or a physical condition that would limit the ability of the child to undergo apheresis catheter placement (if necessary) and/or the apheresis procedure.
Exclusion Criteria:
• Patients that are 12-18 months of age with INSS stage 4 and all 3 favorable biologic features (ie, nonamplified MYCN, favorable pathology, and DNA index > 1) are not eligible
• Female patients who are pregnant are ineligible due to risks of fetal and teratogenic adverse events
• Lactating females are not eligible unless they have agreed not to breastfeed their infants
• Female patients of childbearing potential are not eligible unless a negative pregnancy test result has been obtained
• Sexually active patients of reproductive potential are not eligible unless they have agreed to use an effective contraceptive method for the duration of their study participation.