Clinical Trials / Research Studies

Clinical Trials / Research Studies

Who can participate?

Inclusion Criteria:

• Patients must have a diagnosis of neuroblastoma either by histologic verification of neuroblastoma and/or demonstration of tumor cells in the bone marrow with increased urinary catecholamines.
• Patients must have a history of high-risk neuroblastoma according to COG risk classification at the time of study registration. Patients who were initially considered low or intermediate-risk, but then reclassified as high-risk are also eligible.
• Patients must have at least ONE of the following:
  
  • Recurrent/progressive disease at any time prior to study enrollment
  • Refractory disease
  • Persistent disease
• Patients must have at least ONE of the following (lesions may have received prior radiation therapy as long as they meet the other criteria listed below):
  
  • For recurrent/progressive or refractory disease, at least one MIBG avid bone site.
  • For persistent disease, If a patient has only 1 or 2 MIBG avid lesions AND a Curie Score of 1-2, then biopsy confirmation of neuroblastoma and/or ganglioneuroblastoma in at least one site present at the time of enrollment (bone marrow, bone, or soft tissue) is required to be obtained at any time point prior to enrollment.
  • FDG-PET avid tumors: A biopsy confirmation of neuroblastoma and/or ganglioneuroblastoma is required at any time prior to enrollment OR anatomical imaging is required at the time of enrollment consistent with a bone metastasis for at least one FDG-avid bone site.
• Any amount of neuroblastoma tumor cells in the bone marrow done at the time of study enrollment based on routine morphology with or without immunocytochemistry in at least one sample from bilateral aspirates and biopsies.
• At least one soft tissue lesion that meets criteria for a TARGET lesion as defined by:
  
  • SIZE: Lesion can be accurately measured in at least one dimension with a longest diameter ≥ 10 mm, or for lymph nodes ≥ 15 mm on short axis.
  • In addition to measurable size, a lesion needs to meet the following criteria:
    
    • MIBG avid. For patients with persistent disease only: If a patient has only 1 or 2 MIBG avid lesions ANDa Curie Score of 1 - 2, then biopsy confirmation of neuroblastoma and/or ganglioneuroblastoma in at least one site present at time of enrollment is required to be obtained.
    • MIBG non avid tumors: These patients require a biopsy done at any time prior to enrollment confirming neuroblastoma and/or ganglioneuroblastoma in at least one soft tissue site (even if FDG-PET avid) present at the time of enrollment.
• At least one non-target soft tissue lesion that is not measurable, but had a biopsy positive for neuroblastoma and/or ganglioneuroblastom or is MIBG avid at any time prior to enrollment.
• Patients must have a life expectancy of at least 12 weeks and a Lansky (≤16 years) or Karnofsky (>16 years) score of at least 50.
• Prior Therapy
  
  • Patients must have fully recovered from the acute toxic effects of all prior chemotherapy, immunotherapy, or radiotherapy prior to study registration.
  • Patients must not have received the therapies indicated below after disease evaluation or within the specified time period prior to registration on this study as follows:
    
    • Myelosuppressive chemotherapy: must not have received within 2 weeks prior to registration.
    • Biologic anti-neoplastics- agents not known to be associated with reduced platelet or ANC counts (including retinoids): must not have received within 7 days prior to registration.
    • Monoclonal antibodies: must have received last dose at least 7 days or 3 half-lives whichever is longer, but no longer than 30 days (with recovery of any associated toxicities), prior to protocol therapy.
    • Cellular Therapy (e.g. modified T cells, NK cells, dendritic cells cells etc.): must not have received within 3 weeks and resolution of all toxicities.
    • Radiation: must not have received small port radiation within 7 days prior to registration.
    • Hematopoietic Stem Cell Transplant:
    • IVIG
• All patients must have adequate organ function defined as:
• Hematological Function:
  
  • Absolute Phagocyte count (APC= neutrophils and monocytes): ≥ 1000/µL
  • Absolute Neutrophil count: ≥750/µL
  • Absolute Lymphocyte count ≥ 500/µL
  • Platelet count: ≥ 50,000/µL, transfusion independent (no platelet transfusions within 1 week)
  • Hemoglobin ≥ 10 g/dL (may transfuse)
  • Patients with known bone marrow metastatic disease will be eligible for study as long as they meet hematologic function criteria above.
• Renal Function: Age-adjusted serum creatinine ≤ to 1.5 x normal for age/gender OR creatinine clearance or GFR greater than or equal to 60 cc/min/1.73m2
• Liver Function: Total bilirubin ≤ 1.5 x normal for age, AND SGPT (ALT) 135 and SGOT (AST) ≤ 3 x upper limit of normal. Sinusoidal obstruction syndrome (SOS) if present, must be stable or improving clinically
• Cardiac Function: Normal ejection fraction documented by either echocardiogram or radionuclide MUGA evaluation OR Normal fractional shortening documented by echocardiogram
• Pulmonary Function: No dyspnea at rest, no oxygen requirement.
• Reproductive Status: All post-menarchal females must have a negative beta-HCG. Males and females of reproductive age and childbearing potential must use effective contraception for the duration of their participation.
• Patients with other ongoing serious medical issues must be approved by the study chair prior to registration.
• Patients may not receive any other anti-cancer agents or radiotherapy while on protocol therapy.
• Ability to Swallow Pills

Exclusion Criteria:

• Pregnancy: Quantitative Serum B-HCG must be negative in girls who are post-menarchal.
• Breast feeding women are not eligible.
• Active or uncontrolled infection
• CNS metastasis.
• Hypersensitivity to thalidomide, including history of erythema nodosum if characterized by a desquamating rash while taking thalidomide or similar drugs (dose level 4 only).
• Patient declines participation in NANT 2004-05; unless the institution has been granted special exemption from mandatory registration on NANT 2004-05 by the NANT Operations Center.