Ganglioneuroblastoma Clinical Trial
Official title:
A COG Pilot Study of Intensive Induction Chemotherapy and 131I-MIBG Followed by Myeloablative Busulfan/Melphalan (Bu/Mel) for Newly Diagnosed High-Risk Neuroblastoma
Verified date | December 2023 |
Source | Children's Oncology Group |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Interventional |
This pilot clinical trial studies induction therapy followed by iobenguane I 131 and chemotherapy in treating patients with newly diagnosed high-risk neuroblastoma undergoing stem cell transplant, radiation therapy, and maintenance therapy with isotretinoin. Radioisotope therapy, such as iobenguane I 131, releases radiation that kills tumor cells. Drugs used in chemotherapy, such as carboplatin, etoposide phosphate, busulfan, and melphalan, work in different ways to stop the growth of tumor cells, either by killing the cells or by stopping them from dividing. A peripheral stem cell transplant may be able to replace blood-forming cells that are destroyed by iobenguane I 131 and chemotherapy. Giving radioisotope therapy, chemotherapy, and peripheral stem cell transplant may kill more tumor cells.
Status | Active, not recruiting |
Enrollment | 99 |
Est. completion date | September 28, 2024 |
Est. primary completion date | September 28, 2016 |
Accepts healthy volunteers | No |
Gender | All |
Age group | 1 Year to 30 Years |
Eligibility | Inclusion Criteria: - Patients have a diagnosis of neuroblastoma (International Classification of Diseases for Oncology [ICD-O] morphology 9500/3) or ganglioneuroblastoma 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: - v-MYC myelocytomatosis viral related oncogene, neuroblastoma derived (avian) (MYCN) amplification (> 4-fold increase in MYCN signals as compared to reference signals) and age >= 365 days regardless of 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 deoxyribonucleic acid [DNA] index = 1) or any biologic feature that is indeterminant/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), and age >= 365 days, regardless of additional biologic features - Age > 18 months (> 547 days) with unfavorable pathology, regardless of MYCN status - Patients with newly diagnosed INSS stage 2a/2b with MYCN amplification (> 4-fold increase in MYCN signals as compared to reference signals) and age >= 365 days, regardless of additional biologic features - Patients >= 365 days initially diagnosed with: INSS stage 1, 2, 4S who progressed to a stage 4 without interval chemotherapy; these patients must have been enrolled on ANBL00B1; it is to be noted that study enrollment must occur within 4 weeks of progression to stage 4 for INSS stage 1, 2, 4S - Patients must have had no 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 low- or intermediate-risk neuroblastoma therapy (P9641, A3961, ANBL0531) prior to determination of MYCN amplification and histology - Creatinine clearance or radioisotope glomerular filtration rate (GFR) >= 70 mL/min/1.73 m^2 OR serum creatinine based on age and/or gender as follows: - =< 0.6 mg/dL (1 to < 2 years of age) - =< 0.8 mg/dL (2 to < 6 years of age) - =< 1.0 mg/dL (6 to < 10 years of age) - =< 1.2 mg/dL (10 to < 13 years of age) - =< 1.5 mg/dL (male) or 1.4 mg/dL (female) (13 to < 16 years of age) - =< 1.7 mg/dL (male) or 1.4 mg/dL (female) ( >= 16 years of age) - Total bilirubin =< 1.5 x upper limit of normal (ULN) for age - Serum glutamic oxaloacetic transaminase (SGOT) (aspartate aminotransferase [AST]) or serum glutamate pyruvate transaminase (SGPT) (alanine aminotransferase [ALT]) < 10 x ULN for age - Shortening fraction >= 27% by echocardiogram or - Ejection fraction >= 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 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: - Females of childbearing potential must have a negative pregnancy test; patients of childbearing potential must agree to use an effective birth control method - Female patients who are lactating must agree to stop breast-feeding - Patients that are 12-18 months of age with INSS stage 4 and all 3 favorable biologic features (i.e., non-amplified MYCN, favorable pathology, and DNA index > 1) are not eligible - Patients are not eligible if they have received local radiation which includes any of the following: 1200 centigray (cGy) to more than 33% of both kidneys (patient must have at least 1 kidney that has not exceeded the dose/volume of radiation listed) or 1800 cGy to more than 30% of liver and/or 900 cGy to more than 50% of liver; emergency local irradiation is allowed prior to study entry, provided the patient still meets eligibility criteria |
Country | Name | City | State |
---|---|---|---|
United States | C S Mott Children's Hospital | Ann Arbor | Michigan |
United States | Children's Healthcare of Atlanta - Egleston | Atlanta | Georgia |
United States | Children's Hospital Colorado | Aurora | Colorado |
United States | Children's Hospital of Alabama | Birmingham | Alabama |
United States | University of Alabama at Birmingham Cancer Center | Birmingham | Alabama |
United States | Dana-Farber Cancer Institute | Boston | Massachusetts |
United States | UNC Lineberger Comprehensive Cancer Center | Chapel Hill | North Carolina |
United States | Medical University of South Carolina | Charleston | South Carolina |
United States | University of Chicago Comprehensive Cancer Center | Chicago | Illinois |
United States | Cincinnati Children's Hospital Medical Center | Cincinnati | Ohio |
United States | UT Southwestern/Simmons Cancer Center-Dallas | Dallas | Texas |
United States | Duke University Medical Center | Durham | North Carolina |
United States | Cook Children's Medical Center | Fort Worth | Texas |
United States | Connecticut Children's Medical Center | Hartford | Connecticut |
United States | Children's Hospital Los Angeles | Los Angeles | California |
United States | University of Wisconsin Carbone Cancer Center | Madison | Wisconsin |
United States | Children's Hospital of Wisconsin | Milwaukee | Wisconsin |
United States | Children's Hospital of Philadelphia | Philadelphia | Pennsylvania |
United States | Phoenix Childrens Hospital | Phoenix | Arizona |
United States | Primary Children's Hospital | Salt Lake City | Utah |
United States | UCSF Medical Center-Mission Bay | San Francisco | California |
United States | UCSF Medical Center-Parnassus | San Francisco | California |
United States | Seattle Children's Hospital | Seattle | Washington |
United States | Providence Sacred Heart Medical Center and Children's Hospital | Spokane | Washington |
United States | Children's National Medical Center | Washington | District of Columbia |
Lead Sponsor | Collaborator |
---|---|
Children's Oncology Group | National Cancer Institute (NCI) |
United States,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Percentage of MIBG Avid Patients Treated With Meta-iodobenxylguanide (MIBG) Labeled With Iodine-131 | Number of MIBG avid patients who receive 131I-MIBG divided by the number of patients evaluable for the feasibility of MIBG endpoint x 100%. | Up to 6 weeks after course 5 of induction | |
Primary | Percentage of MIBG Avid Patients Treated With MIBG Labeled With Iodine-131 and Bu/Mel Chemotherapy | Number of MIBG avid patients who receive 131I-MIBG and Bu/Mel divided by the number of patients evaluable for the feasibility of MIBG and Bu/Mel consolidation endpoint x 100%. | Up to day -6 of conditioning | |
Secondary | Incidence of Unacceptable Toxicity and Sinusoidal Obstruction Syndrome (SOS), Assessed by Common Terminology Criteria (CTV)v.4.0 for Toxicity Assessment and Grading for I-MIBG | Number of patients who had an unacceptable toxicity or experienced SOS. Unacceptable toxicity was defined as CTC Grade 4-5 Pulmonary/Respiratory. | Up to 6 weeks after course 5 of induction |
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