Comparing 4 approaches for neuroblastoma

Inclusion

• +Age ≥ 547 days and INRG Stage M regardless of biologic features
• +Age: Less than 30 years old at initial diagnosis
• +Pathology All subjects must have a confirmed diagnosis of tumor type. Phase IPhase 1The earliest stage of human testing, in a small group, focused on safety.Read more →: Relapsed/refractory solid tumors: Neuroblastoma, Ewing Sarcoma, Osteosarcoma, Rhabdomyosarcoma, Liposarcoma
• +Relapsed/refractory Neuroblastoma
• +Relapsed/refractory Ewing sarcoma
• +Tumor assessment:
• +Disease assessment is required for eligibilityEligibility criteriaThe full list of requirements for taking part in a study.Read more → and must be done after last dose of previous therapy and prior to first dose of study drug.
• +Disease Status:
• +International Neuroblastoma Risk Group Staging System (INRG) High Risk NB defined as one of the following:
• +Any age with International Neuroblastoma Risk Group (INRG) Stage L2, MS, or M with MYCN amplification
• +Measurable or evaluable disease, including at least one of the following:
• +Measurable tumor by CT or MRI
• +MIBG or PET that is positive for disease
• +Bone Marrow biopsy/aspirate that is positive for disease
• +Timing from prior therapy:
• +Subjects must have fully recovered from the acute toxic effects of all prior anti- cancer therapy and be within the following timelines:
• +Small Molecule Inhibitors (anti-neoplastic agent): At least 2 weeks from the completion of therapy with a small molecule inhibitor.
• +Stem Cell Transplant:
• +Allogeneic: No evidence of active graft vs. host disease
• +Allogeneic/Autologous: ≥ 2 months must have elapsed since transplant.
• +MIBG Therapy: At least 6 weeks since treatment with MIBG therapy.
• +Subjects must have a Lansky or Karnofsky Performance Scale score of \>/= 50.
• +Subjects must have adequate organ function at the time of enrollmentEnrolmentThe number of participants a study plans to include, or has included.Read more →:
• +Cardiac: Subjects must have a QTcF ≤ 480 msc.
• +Hematological: Hematological recovery as defined by ANC ≥750/μL
• +Liver: Adequate liver function as defined by AST and ALT \<5x upper limit of normal
• +Renal: Subjects must have adequate renal function defined as:
• +estimated Glomerular Filtration rate (eGFR) as calculated from the Bedside Schwartz equation (for subjects \< 17 years old) (in units of mL/min/1.73 m2) or via radioisotope GFR of ≥ 70 mL/min/1.73 m2. The Bedside Schwartz equation is: \[(0.413) X (Height in cm)\] / SCr
• +estimated Glomerular Filtration rate (eGFR) as calculated from the Cockcroft and Gault formula (for subjects ≥17 years old (in units of mL/min/1.73 m2) or via radioisotope GFR of ≥ 70 mL/min/1.73 m2. The Cockcroft and Gault formula is: \[(140-age) x (Wt in kg) x (0.85 if female)\] / (72 x SCr)
• +OR a 24 hour urine Creatinine clearance ≥ 70 mL/min/1.73 m2
• +Written informed consentInformed consentThe process of being told what taking part involves, then choosing freely.Read more → in accordance with institutional and FDA guidelines must be obtained from all subjects (or subjects' legal representative).
• +Subjects of childbearing potential must have a negative serum pregnancy test. Subjects of childbearing potential must agree to use effective measures to avoid pregnancy.
• +Relapsed/Refractory Neuroblastoma Relapsed disease defined as neuroblastoma that was previously in remission after standard therapy (at least 4 cycles of aggressive multi-drug induction chemotherapy, with or without radiation and surgery, followed by immunotherapy, or according to a standard high-risk treatment/neuroblastoma protocolProtocolThe detailed plan a study must follow.Read more →) and has now relapsed and is in any number of relapses.
• +Refractory disease defined as High-risk neuroblastoma (as defined by INRG) that failed to achieve CR after at least 4 cycles of aggressive multi-drug induction chemotherapy, progression during upfront therapy or with disease remaining after standard immunotherapy.
• +Any age initially diagnosed with INRG Stage L1 MYCN amplified NBL who have progressed to Stage M without systemic chemotherapy
• +Age ≥ 547 days of age initially diagnosed with INRG Stage L1, L2, or MS who have progressed to Stage M without systemic chemotherapy
• +Relapsed/refractory Sarcoma Subjects that have relapsed following standard of careStandard of careThe treatment normally given for a condition outside a study.Read more → therapy or having progressed during standard of care therapy. Standard of care therapy for sarcoma includes multi-agent chemotherapy with local controlControl groupThe group a new treatment is measured against.Read more → consisting of either surgery or radiation therapy.
• +Myelosuppressive chemotherapy: Must not have received within 2 weeks of enrollment onto this study.
• +Immunotherapy: At least 4 weeks since the completion of any type of immunotherapy, e.g. tumor vaccines, CAR-T cells, anti-GD2 Monoclonal antibodies (ex. naxitamab, dinutuximab, etc.).
• +Radiotherapy: At least 30 days since the last treatment except for radiation delivered with palliative intent to a non-target siteTrial siteA hospital or clinic where a study is actually run.Read more →.