Yükleniyor... / Loading...
Dinutuximab, Sargramostim, and Combination Chemotherapy in Treating Patients With Newly Diagnosed High-Risk Neuroblastoma
Bu çalışma, yeni tanı almış yüksek riskli nöroblastom (high-risk neuroblastoma; çocukluk çağında görülen bir kanser türü) olan hastalarda Dinutuximab, Sargramostim ve çeşitli kemoterapi ilaçlarının birlikte kullanımını değerlendirmektedir. Çalışmaya, önceki tarama protokollerine kayıtlı, tümör patolojisiyle nöroblastom veya ganglionöroblastom tanısı doğrulanmış, yüksek riskli hastalık ölçütlerini karşılayan çocuklar katılabilmiştir. Çalışma Faz 2 aşamasında yürütülmüş ve tamamlanmıştır.
Yukarıdaki özet, resmî kaydın sade dile aktarılmış halidir. Orijinal İngilizce başlık referans için gösterilir.
Kriterler ClinicalTrials.gov kaydından orijinal İngilizce haliyle alınmıştır. Uygunluk kararını yalnızca deneyi yürüten ekip verebilir.
Inclusion Criteria:
* Patients must be enrolled on ANBL00B1 or APEC14B1 prior to enrollment on ANBL17P1.
* Patients must have a diagnosis of neuroblastoma or ganglioneuroblastoma (nodular) verified by tumor pathology analysis or demonstration of clumps of tumor cells in bone marrow with elevated urinary catecholamine metabolites. The following disease groups are eligible:
* Patients with International Neuroblastoma Risk Group (INRG) stage M disease are eligible if found to have either of the following features:
* MYCN amplification (\> 4-fold increase in MYCN signals as compared to reference signals), regardless of age or additional biologic features; OR
* Age \> 547 days regardless of biologic features;
* Patients with INRG stage MS disease with MYCN amplification
* Patients with INRG stage L2 disease with MYCN amplification
* Patients \> 547 days of age initially diagnosed with INRG stage L1, L2 or MS disease who progress to stage M without prior chemotherapy may enroll within 4 weeks of progression to stage M.
* Patients \>= 365 days of age initially diagnosed with MYCN amplified INRG stage L1 disease who progress to stage M without systemic therapy may enroll within 4 weeks of progression to stage M.
* Patients initially recognized to have high-risk disease must have had no prior systemic therapy (other than topotecan/cyclophosphamide initiated on an emergent basis and within allowed timing as described).
* Patients observed or treated with a single cycle of chemotherapy per a low or intermediate risk neuroblastoma regimen (e.g., as per ANBL0531, ANBL1232 or similar) for what initially appeared to be non-high risk disease but subsequently found to meet the criteria will also be eligible.
* Patients who receive localized emergency radiation to sites of life-threatening or function-threatening disease prior to or immediately after establishment of the definitive diagnosis will be eligible.
* Creatinine clearance (CrCl) or radioisotope glomerular filtration rate (GFR) \>= 70 mL/min/1.73 m\^2 or a serum creatinine based on age/sex as follows:
* Age 1 month to \< 6 months (male 0.4 mg/dL, female 0.4 mg/dL)
* Age 6 months to \< 1 year (male 0.5 mg/dL, female 0.5 mg/dL)
* Age 1 to \< 2 years (male 0.6 mg/dL, female 0.6 mg/dL)
* Age 2 to \< 6 years (male 0.8 mg/dL, female 0.8 mg/dL)
* Age 6 to \< 10 years (male 1 mg/dL, female 1 mg/dL)
* Age 10 to \< 13 years (male 1.2 mg/dL, female 1.2 mg/dL)
* Age 13 to \< 16 years (male 1.5 mg/dL, female 1.4 mg/dL)
* Age \>= 16 years (male 1.7 mg/dL, female 1.4 mg/dL) (within 7 days prior to enrollment).
* Total bilirubin =\< 1.5 x upper limit of normal (ULN) for age (within 7 days prior to enrollment).
* Serum glutamate pyruvate transaminase (SGPT) (alanine aminotransferase \[ALT\]) \< 10 x ULN. For the purposes of this study, ULN for ALT is 45 IU/L (within 7 days prior to enrollment).
* Shortening fraction of \>= 27% by echocardiogram (within 7 days prior to enrollment).
* Ejection fraction of \>= 50% by echocardiogram or radionuclide angiogram (within 7 days prior to enrollment).
* No known contraindication to peripheral blood stem cell (PBSC) collection. Examples of contraindications might be a weight or size less than the collecting institution finds feasible, or a physical condition that would limit the ability of the child to undergo apheresis catheter placement (if necessary) and/or the apheresis procedure.
* All patients and/or their parents or legal guardians must sign a written informed consent.
* All institutional, Food and Drug Administration (FDA), and National Cancer Institute (NCI) requirements for human studies must be met.
Exclusion Criteria:
* Patients \>18 months of age with INRG stage L2, MYCN non-amplified, regardless of additional biologic features.
* Patients with bone marrow failure syndromes.
* Patients that are \>= 12 and =\< 18 months of age with INRG stage M and all 3 favorable biologic features (i.e., non-amplified MYCN, favorable pathology, and deoxyribonucleic acid \[DNA\] index \> 1) are not eligible.
* Patients on immunosuppressive medications (e.g. tacrolimus, cyclosporine, corticosteroids for reasons other than prevention/treatment of allergic reactions, adrenal replacement therapy, etc.) are not eligible.
* Female patients who are pregnant are ineligible since fetal toxicities and teratogenic effects have been noted for several of the study drugs. A pregnancy test is required for female patients of childbearing potential.
* Lactating females who plan to breastfeed their infants.
* Sexually active patients of reproductive potential who have not agreed to use an effective contraceptive method during study therapy and for two months after the last dose of ch14.18 (dinutuximab) are not eligible.