Patients Between the Ages of 12 Months to 21 Years With Newly-Diagnosed High-Risk Neuroblastoma Will Receive Children's Oncology Group (COG) Type Recommended Therapy With the Addition of Naxitamab and Granulocyte-Macrophage Colony Stimulating Factor (GM-CSF) to Induction Cycles 1-5

A vizsgálat adatai

Ki végzi

Shaare Zedek Medical Center

Azonosító

NCT07537400

A regiszteradat frissítve

2026. szeptember 17.

Eredeti leírás

This clinical trial will evaluate the safety of chemoimmunotherapy with Naxitamab and COG-type induction chemotherapy in newly-diagnosed patients with high-risk neuroblastoma. We aim to recruit 10 patients over the next 2 years.A teljes eredeti leírásKevesebb

Phase II Trial of Naxitamab, Granulocyte-Macrophage Colony Stimulating Factor (GM-CSF) in Combination With Induction Chemotherapy for Patients With Newly-Diagnosed High-Risk Neuroblastoma

This clinical trial will evaluate the safety of chemoimmunotherapy with Naxitamab and COG-type induction chemotherapy in newly-diagnosed patients with high-risk neuroblastoma. We aim to recruit 10 patients over the next 2 years.

Bevonási feltételek: Age - 12 months to 21 years at protocol enrollment; Clinical eligibility criteria:; Newly diagnosed high risk neuroblastoma; BOTH stage M (INRG - International Neuroblastoma Risk Group) and age ≥547 days; Patients ≥ 547 days of age who were initially diagnosed with INRG L1 or L2 disease but progress to Stage M without chemotherapy; Patients < 547 days of age with INRG Stage M or MS disease and patients of any age with INRG L2 with MYCN amplification (v-myc avian myelocytomatosis viral related oncogene); Pathology:; Neuroblastoma (NBL) or ganglioneuroblastoma (nodular) verified by tumor pathology analysis, or; demonstration of clumps of tumor cells in bone marrow with elevated urinary catecholamines; Molecular testing:; MYCN testing will be done by FISH (Fluorescence In Situ Hybridization) assessment of the FFPE (Formalin-Fixed Paraffin-Embedded) material submitted from the tumor mass. > 4-fold increase in MYCN signals as compared to reference signals will qualify as MYCN amplified; ONCOMINE testing will evaluate ALK (Anaplastic Lymphoma Kinase) mutation; Timing of patient enrollment; Patients will be enrolled up to 6 weeks from primary diagnosis; Pre-study imaging tests are acceptable up to 3 weeks prior to study enrollment and only if done after any pre-protocol chemotherapy; Pre-treatment functional status:; No active bacterial infection without adequate antibiotic therapy; No uncontrolled viral infection - decision will be made after infectious disease consultation; No uncontrolled organ dysfunction:; Renal function based on the Schwartz formula (Schwartz et al. J. Peds, 106:522, 1985). If creatinine level is abnormal chemotherapy treatment will be planned in consultation with Nephrology service. Naxitamab will be initiated if creatinine level is up to 1.2 of normal; Adequate liver function defined as: -; Total bilirubin ≤ 1.5 x upper limit of normal (ULN) for age,; and SGPT (Serum Glutamic Pyruvic Transaminase - ALT) ≤ 10 x ULN* iii. Adequate cardiac function defined as: -; Shortening fraction of ≥ 27% by echocardiogram, 2. or Ejection fraction of ≥ 50% by echocardiogram or radionuclide angiogram.

Kizáró feltételek: Subjects who 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; Female patients who are pregnant since fetal toxicities and teratogenic effects have been noted for several of the study drugs. A pregnancy test is required prior to enrollment for female patients of childbearing potential; Patients with known bone marrow failure syndromes; Patients on chronic immunosuppressive medications (e.g., tacrolimus, cyclosporine, corticosteroids) for reasons other than prevention/treatment of allergic reactions and adrenal replacement therapy are not eligible. Topical and inhaled corticosteroids are acceptable; Patients with a primary immunodeficiency syndrome who require ongoing immune globulin replacement therapy; Patients who are 365-546 days of age with INRG Stage M and MYCN non-amplified NBL, irrespective of additional biologic features; Patients ≥547 days of age with INRG Stage L2, MYCN non-amplified NBL, regardless of additional biologic features; This will not restrict the emergency regimen at initial diagnosis; Lactating females who plan to breastfeed their infants; Sexually active patients of reproductive potential who have not agreed to use an effective contraceptive method for the duration of their study participation.

ClinicalTrials.gov NCT07537400