Ganglioneuroblastoma Clinical Trial
Official title:
Pilot Study Using Myeloablative Busulfan/Melphalan (BuMel) Consolidation Following Induction Chemotherapy for Patients With Newly Diagnosed High-Risk Neuroblastoma
NCT number | NCT01798004 |
Other study ID # | ANBL12P1 |
Secondary ID | NCI-2012-02211AN |
Status | Active, not recruiting |
Phase | Phase 1 |
First received | |
Last updated | |
Start date | April 8, 2013 |
Verified date | March 2024 |
Source | Children's Oncology Group |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Interventional |
This pilot clinical trial studies busulfan, melphalan, and stem cell transplant after chemotherapy in treating patients with newly diagnosed neuroblastoma that is likely to come back or spread. Giving chemotherapy to the entire body before a stem cell transplant stops the growth of tumor cells by stopping them from dividing or killing them. After treatment, stem cells are collected from the patient's blood and stored. More chemotherapy or radiation therapy is given to prepare the bone marrow for the stem cell transplant. The stem cells are then returned to the patient to replace the blood-forming cells that were destroyed by the chemotherapy.
Status | Active, not recruiting |
Enrollment | 150 |
Est. completion date | |
Est. primary completion date | July 28, 2015 |
Accepts healthy volunteers | No |
Gender | All |
Age group | N/A to 30 Years |
Eligibility | Inclusion Criteria: - Patients must have a diagnosis of neuroblastoma (International Classification of Diseases for Oncology [ICD-O] morphology 9500/3) or ganglioneuroblastoma (nodular or intermixed) 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 avian myelocytomatosis viral oncogene neuroblastoma derived homolog (MYCN) amplification (> 4-fold increase in MYCN signals as compared to reference signals), regardless of age or additional biologic features or - Age > 18 months (> 547 days) regardless of biologic features or - 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 indeterminate/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), regardless of age or additional biologic features or - Age > 18 months (> 547 days) with unfavorable pathology, regardless of MYCN status - Patients with newly diagnosed neuroblastoma with INSS stage 2A/2B with MYCN amplification (> 4-fold increase in MYCN signals as compared to reference signals), regardless of age or additional biologic features - Patients with newly diagnosed neuroblastoma with INSS stage 4S with MYCN amplification (> 4-fold increase in MYCN expression signals as compared to reference signals), regardless of additional biologic features - Patients >= 365 days initially diagnosed with neuroblastoma INSS stage 1, 2, 4S who progressed to a stage 4 without interval chemotherapy; these patients must have been enrolled on ANBL00B1; study enrollment on ANBL12P1 must occur within 4 weeks of progression to stage 4 for INSS stage 1, 2, 4S - Patients must not 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 per a low or intermediate risk neuroblastoma regimen (as per P9641, A3961, ANBL0531, or similar) prior to determination of MYCN amplification status and histology - Creatinine clearance or radioisotope glomerular filtration rate (GFR) >= 70 mL/min/1.73 m^2 or a serum creatinine based on age/gender as follows: - Age 1 month to < 6 months: 0.4 mg/dL - Age 6 months to < 1 year: 0.5 mg/dL - Age 1 to < 2 years: 0.6 mg/dL - Age 2 to < 6 years: 0.8 mg/dL - Age 6 to < 10 years: 1 mg/dL - Age 10 to < 13 years: 1.2 mg/dL - Age 13 to < 16 years: 1.5 mg/dL (males), 1.4 mg/dL (females) - Age >= 16 years: 1.7 mg/dL (males), 1.4 mg/dL (females) - Total bilirubin =< 1.5 x upper limit of normal (ULN) for age, and - Serum glutamic oxaloacetic transaminase (SGOT) (aspartate aminotransferase [AST]) or serum glutamate pyruvate transaminase (SGPT) (alanine aminotransferase [ALT]) < 10 x ULN for age - Shortening fraction of >= 27% by echocardiogram, or - Ejection fraction of >= 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 that determined to be feasible at the collecting institution, 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 that are 12-18 months of age with INSS stage 4 and all 3 favorable biologic features (ie, nonamplified MYCN, favorable pathology, and DNA index > 1) are not eligible - Female patients who are pregnant are ineligible - Lactating females are not eligible unless they have agreed not to breastfeed their infants - Female patients of childbearing potential are not eligible unless a negative pregnancy test result has been obtained - Sexually active patients of reproductive potential are not eligible unless they have agreed to use an effective contraceptive method for the duration of their study participation |
Country | Name | City | State |
---|---|---|---|
Australia | Princess Margaret Hospital for Children | Perth | Western Australia |
Canada | IWK Health Centre | Halifax | Nova Scotia |
Canada | McMaster Children's Hospital at Hamilton Health Sciences | Hamilton | Ontario |
Canada | Centre Hospitalier Universitaire Sainte-Justine | Montreal | Quebec |
Canada | The Montreal Children's Hospital of the MUHC | Montreal | Quebec |
Canada | CHU de Quebec-Centre Hospitalier de l'Universite Laval (CHUL) | Quebec | |
Canada | Hospital for Sick Children | Toronto | Ontario |
Canada | British Columbia Children's Hospital | Vancouver | British Columbia |
Canada | CancerCare Manitoba | Winnipeg | Manitoba |
New Zealand | Starship Children's Hospital | Grafton | Auckland |
United States | University of New Mexico Cancer Center | Albuquerque | New Mexico |
United States | C S Mott Children's Hospital | Ann Arbor | Michigan |
United States | Mission Hospital | Asheville | North Carolina |
United States | Children's Healthcare of Atlanta - Egleston | Atlanta | Georgia |
United States | Augusta University Medical Center | Augusta | Georgia |
United States | Children's Hospital Colorado | Aurora | Colorado |
United States | Johns Hopkins University/Sidney Kimmel Cancer Center | Baltimore | Maryland |
United States | Sinai Hospital of Baltimore | Baltimore | Maryland |
United States | Walter Reed National Military Medical Center | Bethesda | Maryland |
United States | Lehigh Valley Hospital - Muhlenberg | Bethlehem | Pennsylvania |
United States | Children's Hospital of Alabama | Birmingham | Alabama |
United States | Dana-Farber Cancer Institute | Boston | Massachusetts |
United States | Massachusetts General Hospital Cancer Center | Boston | Massachusetts |
United States | Tufts Children's Hospital | Boston | Massachusetts |
United States | Montefiore Medical Center - Moses Campus | Bronx | New York |
United States | UNC Lineberger Comprehensive Cancer Center | Chapel Hill | North Carolina |
United States | Medical University of South Carolina | Charleston | South Carolina |
United States | T C Thompson Children's Hospital | Chattanooga | Tennessee |
United States | Lurie Children's Hospital-Chicago | Chicago | Illinois |
United States | University of Chicago Comprehensive Cancer Center | Chicago | Illinois |
United States | University of Illinois | Chicago | Illinois |
United States | Cincinnati Children's Hospital Medical Center | Cincinnati | Ohio |
United States | Cleveland Clinic Foundation | Cleveland | Ohio |
United States | Rainbow Babies and Childrens Hospital | Cleveland | Ohio |
United States | Columbia Regional | Columbia | Missouri |
United States | Prisma Health Richland Hospital | Columbia | South Carolina |
United States | Nationwide Children's Hospital | Columbus | Ohio |
United States | UT Southwestern/Simmons Cancer Center-Dallas | Dallas | Texas |
United States | Dayton Children's Hospital | Dayton | Ohio |
United States | Blank Children's Hospital | Des Moines | Iowa |
United States | Wayne State University/Karmanos Cancer Institute | Detroit | Michigan |
United States | Kaiser Permanente Downey Medical Center | Downey | California |
United States | City of Hope Comprehensive Cancer Center | Duarte | California |
United States | Duke University Medical Center | Durham | North Carolina |
United States | Golisano Children's Hospital of Southwest Florida | Fort Myers | Florida |
United States | Lee Memorial Health System | Fort Myers | Florida |
United States | Cook Children's Medical Center | Fort Worth | Texas |
United States | University of Florida Health Science Center - Gainesville | Gainesville | Florida |
United States | BI-LO Charities Children's Cancer Center | Greenville | South Carolina |
United States | Hackensack University Medical Center | Hackensack | New Jersey |
United States | Connecticut Children's Medical Center | Hartford | Connecticut |
United States | Memorial Regional Hospital/Joe DiMaggio Children's Hospital | Hollywood | Florida |
United States | Baylor College of Medicine/Dan L Duncan Comprehensive Cancer Center | Houston | Texas |
United States | Ascension Saint Vincent Indianapolis Hospital | Indianapolis | Indiana |
United States | Riley Hospital for Children | Indianapolis | Indiana |
United States | University of Mississippi Medical Center | Jackson | Mississippi |
United States | Nemours Children's Clinic-Jacksonville | Jacksonville | Florida |
United States | Bronson Methodist Hospital | Kalamazoo | Michigan |
United States | Children's Mercy Hospitals and Clinics | Kansas City | Missouri |
United States | East Tennessee Childrens Hospital | Knoxville | Tennessee |
United States | Alliance for Childhood Diseases/Cure 4 the Kids Foundation | Las Vegas | Nevada |
United States | Nevada Cancer Research Foundation NCORP | Las Vegas | Nevada |
United States | Summerlin Hospital Medical Center | Las Vegas | Nevada |
United States | Dartmouth Hitchcock Medical Center/Dartmouth Cancer Center | Lebanon | New Hampshire |
United States | University of Kentucky/Markey Cancer Center | Lexington | Kentucky |
United States | Saint Barnabas Medical Center | Livingston | New Jersey |
United States | Loma Linda University Medical Center | Loma Linda | California |
United States | Children's Hospital Los Angeles | Los Angeles | California |
United States | Norton Children's Hospital | Louisville | Kentucky |
United States | University of Wisconsin Carbone Cancer Center | Madison | Wisconsin |
United States | Marshfield Medical Center-Marshfield | Marshfield | Wisconsin |
United States | Loyola University Medical Center | Maywood | Illinois |
United States | Miami Cancer Institute | Miami | Florida |
United States | University of Miami Miller School of Medicine-Sylvester Cancer Center | Miami | Florida |
United States | NYU Winthrop Hospital | Mineola | New York |
United States | Children's Hospitals and Clinics of Minnesota - Minneapolis | Minneapolis | Minnesota |
United States | University of Minnesota/Masonic Cancer Center | Minneapolis | Minnesota |
United States | Morristown Medical Center | Morristown | New Jersey |
United States | Vanderbilt University/Ingram Cancer Center | Nashville | Tennessee |
United States | Rutgers Cancer Institute of New Jersey-Robert Wood Johnson University Hospital | New Brunswick | New Jersey |
United States | The Steven and Alexandra Cohen Children's Medical Center of New York | New Hyde Park | New York |
United States | Children's Hospital New Orleans | New Orleans | Louisiana |
United States | NYP/Columbia University Medical Center/Herbert Irving Comprehensive Cancer Center | New York | New York |
United States | Newark Beth Israel Medical Center | Newark | New Jersey |
United States | Kaiser Permanente-Oakland | Oakland | California |
United States | UCSF Benioff Children's Hospital Oakland | Oakland | California |
United States | University of Oklahoma Health Sciences Center | Oklahoma City | Oklahoma |
United States | Children's Hospital and Medical Center of Omaha | Omaha | Nebraska |
United States | University of Nebraska Medical Center | Omaha | Nebraska |
United States | Children's Hospital of Orange County | Orange | California |
United States | AdventHealth Orlando | Orlando | Florida |
United States | Nemours Children's Hospital | Orlando | Florida |
United States | Lucile Packard Children's Hospital Stanford University | Palo Alto | California |
United States | Saint Joseph's Regional Medical Center | Paterson | New Jersey |
United States | Nemours Children's Clinic - Pensacola | Pensacola | Florida |
United States | Saint Jude Midwest Affiliate | Peoria | Illinois |
United States | Children's Hospital of Philadelphia | Philadelphia | Pennsylvania |
United States | Children's Hospital of Pittsburgh of UPMC | Pittsburgh | Pennsylvania |
United States | Naval Medical Center - Portsmouth | Portsmouth | Virginia |
United States | Virginia Commonwealth University/Massey Cancer Center | Richmond | Virginia |
United States | Mayo Clinic in Rochester | Rochester | Minnesota |
United States | University of Rochester | Rochester | New York |
United States | University of California Davis Comprehensive Cancer Center | Sacramento | California |
United States | Mercy Hospital Saint Louis | Saint Louis | Missouri |
United States | Washington University School of Medicine | Saint Louis | Missouri |
United States | Johns Hopkins All Children's Hospital | Saint Petersburg | Florida |
United States | Methodist Children's Hospital of South Texas | San Antonio | Texas |
United States | University of Texas Health Science Center at San Antonio | San Antonio | Texas |
United States | Rady Children's Hospital - San Diego | San Diego | California |
United States | UCSF Medical Center-Mission Bay | San Francisco | California |
United States | UCSF Medical Center-Parnassus | San Francisco | California |
United States | Memorial Health University Medical Center | Savannah | Georgia |
United States | Maine Children's Cancer Program | Scarborough | Maine |
United States | Seattle Children's Hospital | Seattle | Washington |
United States | Providence Sacred Heart Medical Center and Children's Hospital | Spokane | Washington |
United States | Southern Illinois University School of Medicine | Springfield | Illinois |
United States | State University of New York Upstate Medical University | Syracuse | New York |
United States | Saint Joseph's Hospital/Children's Hospital-Tampa | Tampa | Florida |
United States | Mercy Children's Hospital | Toledo | Ohio |
United States | ProMedica Toledo Hospital/Russell J Ebeid Children's Hospital | Toledo | Ohio |
United States | Children's National Medical Center | Washington | District of Columbia |
United States | MedStar Georgetown University Hospital | Washington | District of Columbia |
United States | Alfred I duPont Hospital for Children | Wilmington | Delaware |
Lead Sponsor | Collaborator |
---|---|
Children's Oncology Group | National Cancer Institute (NCI) |
United States, Australia, Canada, New Zealand,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Other | Incidence of Non-hematologic Organ Toxicity (Grade 3 and Higher) and All Cause Mortality Graded According to CTC v4.0 | Assessed by a descriptive analysis of the incidence of grade 3-5 non-hematologic toxicities (CTC v4.0) and all-cause mortality during consolidation therapy. In addition, a descriptive analysis of "late" onset grade 4-5 pulmonary and hepatic complications that occur within 180 days of the start of consolidation therapy will be examined, regardless if the patient has proceeded to other therapy (including chimeric antibody) during that 180 day period. | Up to 180 days | |
Other | Response Rate Determined Using the International Response Criteria | Up to 5 years | ||
Other | EFS | Up to 5 years | ||
Other | Overall Survival | Up to 5 years | ||
Other | First Dose Area Under the Curve (AUC) and Average Daily AUC for Busulfan | Relationship with occurrence of non-hematologic toxicities assessed by a descriptive analysis. Association between busulfan exposure levels as measured by the area under the curve (AUC) and event-free survival and overall survival will be examined using Cox proportional hazards models. | Within 28 days following consolidation | |
Other | Percentage of Centrally Reviewed Post-course 4 MIBG Scans Reporting a Curie Score Considered to Have Been Determined in "Real Time" | Up to week 12 (course 4 of induction therapy) | ||
Other | Percentage of MIBG Scans Receiving Institutionally and Centrally Reviewed and Automated Advanced Assisted Scoring Platform Curie Scores Within 1 Unit of Each Other | Cohen's kappa will be calculated to evaluate the concordance in Curie scores between each of the scoring methods at each time point. Up to 160 MIBG scans are expected at diagnosis and up to 144 MIBG scans from the 90% of patients estimated to be MIBG avid are projected post-course 4 of induction therapy, for a total of up to 304 MIBG scans. | Up to week 12 (course 4 of induction therapy) | |
Other | Proportion of High-risk Neuroblastoma Patients for Whom ALK Status Can be Obtained | Within 6 weeks of diagnosis | ||
Other | Proportion of High-risk Neuroblastoma Patients With MYCN Non-amplified Tumors for Whom Molecular Profiling Results Can be Obtained | Within 8 weeks of diagnosis | ||
Other | Melphalan Pharmacokinetics and the Combination of Busulfan and Melphalan AUC (Optional) | A descriptive analysis of the relationship between melphalan pharmacokinetics and the combination of busulfan and melphalan AUC with the occurrence of non-hematologic toxicities within 28 days following completion of consolidation will be assessed. In addition, the association between melphalan exposure levels as measured by the AUC and event-free survival and overall survival will be examined using Cox proportional hazards models. | Within 28 days post-consolidation | |
Primary | The Tolerability of BuMel Regimen | Number of patients who experience one or more unacceptable toxicities (severe sinusoidal obstruction syndrome [SOS] or Grade 4-5 pulmonary toxicity per Common Toxicity Criteria [CTC] v.4.0) during the Consolidation phase of therapy. | Up to 28 days post-consolidation therapy, up to 1 year |
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