Late Effects After Treatment in Patients With Previously Diagnosed High-Risk Neuroblastoma
- Conditions
- Recurrent NeuroblastomaStage 2A NeuroblastomaStage 4S NeuroblastomaStage 2B NeuroblastomaStage 4 NeuroblastomaStage 3 Neuroblastoma
- Interventions
- Other: Cytology Specimen Collection ProcedureOther: Laboratory Biomarker AnalysisOther: Quality-of-Life Assessment
- Registration Number
- NCT03057626
- Lead Sponsor
- Children's Oncology Group
- Brief Summary
This research trial studies late effects after treatment in patients with previously diagnosed high-risk neuroblastoma. Studying late effects after treatment may help to decide which treatments for high-risk neuroblastoma are better tolerated with less side effects over time.
- Detailed Description
PRIMARY OBJECTIVES:
I. To estimate the prevalence of organ dysfunction, subsequent malignant neoplasm (SMN), growth impairment, abnormal pubertal development, and neurobehavioral dysfunction in a large cohort of representative 5-year survivors of high-risk neuroblastoma treated with modern therapy.
II. To identify the demographic, clinical and treatment-related risk factors associated with increased risk of organ dysfunction, SMN, growth impairment, abnormal pubertal development and neurobehavioral dysfunction in long-term survivors of high-risk neuroblastoma.
III. To explore the impact of new biologic therapies and diagnostics including immunotherapy, immunocytokines, isotretinoin (cis-retinoic acid) and iobenguane I-131 (131 I-MIBG) on the risk of late effects.
IV. To determine the impact of impaired organ function, physical growth, pubertal development, and neurobehavioral function on health-related quality of life (HRQOL) in long-term survivors of high-risk neuroblastoma.
SECONDARY OBJECTIVES:
I. To establish a cohort of high-risk neuroblastoma survivors, with stored peripheral blood samples, who were treated with multi-modal therapies since the year 2000 as a resource for future investigation.
OUTLINE:
Patients undergo collection of blood and urine samples on day 1. Patients also undergo clinical assessments, laboratory, radiographic, and other ancillary studies on day 1.
Recruitment & Eligibility
- Status
- ACTIVE_NOT_RECRUITING
- Sex
- All
- Target Recruitment
- 376
-
Patients must have been enrolled on COG neuroblastoma biology study ANBL00B1
-
Patient must have been diagnosed with high-risk neuroblastoma per ANBL00B1 definition
-
Patient must have been diagnosed on or after January 1, 2000
-
At least 5 years must have elapsed since diagnosis
-
Patients must have been treated for high-risk neuroblastoma
- Note: patients may have had any therapy for high-risk neuroblastoma, including second line or non-established therapies (for example in the setting of less than optimal initial response or concerns for high risk of relapse); patients may have received therapy for refractory or relapsed neuroblastoma, or treatment for an SMN; however all cytotoxic anti-neuroblastoma therapy should have been administered >= 2 years of the enrollment date; SMN therapy may be completed or ongoing at the time of enrollment
-
Patients must not be currently receiving active anti-neuroblastoma cytotoxic chemotherapy
-
Patients must not have received anti-neuroblastoma cytotoxic chemotherapy within the last two years
- Note: cytotoxic therapies include (but are not limited to) chemotherapy (platinum agents, alkylators, anthracyclines, topoisomerases, vinca alkaloids, other cytotoxic chemotherapy), any kind of transplant, MIBG therapy, and/or radiation therapy
- Non-cytotoxic (biologic/targeted/differentiating/other) therapies are permitted at the time of enrollment; for example, patients receiving oral differentiating agents, antiangiogenic therapy, immune modulators, holistic therapies, difluoromethylornithine (DMFO), other minimal residual disease (MRD) therapies/relapse-prevention therapies are eligible
-
Patients with current active neuroblastoma relapse are ineligible
Study & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Arm && Interventions
Group Intervention Description Observational (specimen collection) Laboratory Biomarker Analysis Patients undergo collection of blood and urine samples on day 1. Patients also undergo clinical assessments, laboratory, radiographic, and other ancillary studies on day 1. Observational (specimen collection) Cytology Specimen Collection Procedure Patients undergo collection of blood and urine samples on day 1. Patients also undergo clinical assessments, laboratory, radiographic, and other ancillary studies on day 1. Observational (specimen collection) Quality-of-Life Assessment Patients undergo collection of blood and urine samples on day 1. Patients also undergo clinical assessments, laboratory, radiographic, and other ancillary studies on day 1.
- Primary Outcome Measures
Name Time Method Pediatric Quality of Life (PedsQL) score Up to 3 years PedsQL score will be reported descriptively (mean, standard deviation). The proportion of patients with impaired physical growth, delayed pubertal development, chronic disease, impaired executive functioning, and impaired social functioning will also be reported.
Prevalence of specific late effects Up to 3 years Late effects of interest are organ dysfunction, subsequent malignant neoplasms (SMN), growth impairment, abnormal pubertal development, and neurobehavioral dysfunction. Prevalence will be calculated as the number of patients with late effects divided by the number with known status of that endpoint.
Risk factors of late effects Up to 3 years Risk factors of interest include sex, race, ethnicity, current age, length of follow up, MYCN status, stage, primary site, age at diagnosis, total anthracycline dose (doxorubicin equivalents), cyclophosphamide dose equivalent categories, total platinum exposure (dose), topotecan exposure (yes \[Y\]/no \[N\]), cis-retinoic acid exposure (Y/N), GD-2/cytokine exposure (Y/N), radiation (Y/N), abdominal RT (Y/N), radiation to metastatic sites (Y/N), number of transplants, number of meta-iodobenzylguanidine (MIBG) scans, and therapeutic MIBG (Y/N). Will be reported as the number of patients with the risk factor divided by the number with known status of that risk factor for categorical variables and descriptively (mean, standard deviation) for continuous variables.
- Secondary Outcome Measures
Name Time Method Collection and storage of blood samples Up to 3 years Proportion of survivors and their families that consent for future utilization of their banked sample for future research will be reported.
Trial Locations
- Locations (98)
Children's Hospital of Pittsburgh of UPMC
🇺🇸Pittsburgh, Pennsylvania, United States
Sinai Hospital of Baltimore
🇺🇸Baltimore, Maryland, United States
Medical City Dallas Hospital
🇺🇸Dallas, Texas, United States
Lurie Children's Hospital-Chicago
🇺🇸Chicago, Illinois, United States
University of Illinois
🇺🇸Chicago, Illinois, United States
University of Chicago Comprehensive Cancer Center
🇺🇸Chicago, Illinois, United States
Rainbow Babies and Childrens Hospital
🇺🇸Cleveland, Ohio, United States
Seattle Children's Hospital
🇺🇸Seattle, Washington, United States
Baylor College of Medicine/Dan L Duncan Comprehensive Cancer Center
🇺🇸Houston, Texas, United States
University of Texas Health Science Center at San Antonio
🇺🇸San Antonio, Texas, United States
University of Minnesota/Masonic Cancer Center
🇺🇸Minneapolis, Minnesota, United States
Duke University Medical Center
🇺🇸Durham, North Carolina, United States
Methodist Children's Hospital of South Texas
🇺🇸San Antonio, Texas, United States
Nemours Children's Clinic-Jacksonville
🇺🇸Jacksonville, Florida, United States
City of Hope Comprehensive Cancer Center
🇺🇸Duarte, California, United States
Children's National Medical Center
🇺🇸Washington, District of Columbia, United States
UCSF Benioff Children's Hospital Oakland
🇺🇸Oakland, California, United States
University of Kentucky/Markey Cancer Center
🇺🇸Lexington, Kentucky, United States
Connecticut Children's Medical Center
🇺🇸Hartford, Connecticut, United States
Stony Brook University Medical Center
🇺🇸Stony Brook, New York, United States
Kaiser Permanente Downey Medical Center
🇺🇸Downey, California, United States
Baystate Medical Center
🇺🇸Springfield, Massachusetts, United States
Children's Hospital Los Angeles
🇺🇸Los Angeles, California, United States
Johns Hopkins All Children's Hospital
🇺🇸Saint Petersburg, Florida, United States
Broward Health Medical Center
🇺🇸Fort Lauderdale, Florida, United States
Lucile Packard Children's Hospital Stanford University
🇺🇸Palo Alto, California, United States
Alfred I duPont Hospital for Children
🇺🇸Wilmington, Delaware, United States
Golisano Children's Hospital of Southwest Florida
🇺🇸Fort Myers, Florida, United States
Arkansas Children's Hospital
🇺🇸Little Rock, Arkansas, United States
Kaiser Permanente-Oakland
🇺🇸Oakland, California, United States
Children's Healthcare of Atlanta - Egleston
🇺🇸Atlanta, Georgia, United States
Hospital for Sick Children
🇨🇦Toronto, Ontario, Canada
IWK Health Centre
🇨🇦Halifax, Nova Scotia, Canada
Roswell Park Cancer Institute
🇺🇸Buffalo, New York, United States
Sanford USD Medical Center - Sioux Falls
🇺🇸Sioux Falls, South Dakota, United States
Blank Children's Hospital
🇺🇸Des Moines, Iowa, United States
The Montreal Children's Hospital of the MUHC
🇨🇦Montreal, Quebec, Canada
Starship Children's Hospital
🇳🇿Grafton, Auckland, New Zealand
University of Wisconsin Carbone Cancer Center
🇺🇸Madison, Wisconsin, United States
Alberta Children's Hospital
🇨🇦Calgary, Alberta, Canada
Advocate Children's Hospital-Oak Lawn
🇺🇸Oak Lawn, Illinois, United States
Advocate Children's Hospital-Park Ridge
🇺🇸Park Ridge, Illinois, United States
Saint Jude Midwest Affiliate
🇺🇸Peoria, Illinois, United States
Saint Joseph's Regional Medical Center
🇺🇸Paterson, New Jersey, United States
Washington University School of Medicine
🇺🇸Saint Louis, Missouri, United States
Nationwide Children's Hospital
🇺🇸Columbus, Ohio, United States
Kingston Health Sciences Centre
🇨🇦Kingston, Ontario, Canada
University of Iowa/Holden Comprehensive Cancer Center
🇺🇸Iowa City, Iowa, United States
Children's Hospital of Philadelphia
🇺🇸Philadelphia, Pennsylvania, United States
Carolinas Medical Center/Levine Cancer Institute
🇺🇸Charlotte, North Carolina, United States
Providence Sacred Heart Medical Center and Children's Hospital
🇺🇸Spokane, Washington, United States
Massachusetts General Hospital Cancer Center
🇺🇸Boston, Massachusetts, United States
Cincinnati Children's Hospital Medical Center
🇺🇸Cincinnati, Ohio, United States
Children's Hospitals and Clinics of Minnesota - Minneapolis
🇺🇸Minneapolis, Minnesota, United States
Centre Hospitalier Universitaire Sainte-Justine
🇨🇦Montreal, Quebec, Canada
Hackensack University Medical Center
🇺🇸Hackensack, New Jersey, United States
Saint Jude Children's Research Hospital
🇺🇸Memphis, Tennessee, United States
University of Mississippi Medical Center
🇺🇸Jackson, Mississippi, United States
McMaster Children's Hospital at Hamilton Health Sciences
🇨🇦Hamilton, Ontario, Canada
Cardinal Glennon Children's Medical Center
🇺🇸Saint Louis, Missouri, United States
Centre Hospitalier Universitaire de Quebec
🇨🇦Quebec, Canada
Cook Children's Medical Center
🇺🇸Fort Worth, Texas, United States
Children's Hospital Medical Center of Akron
🇺🇸Akron, Ohio, United States
UT Southwestern/Simmons Cancer Center-Dallas
🇺🇸Dallas, Texas, United States
BI-LO Charities Children's Cancer Center
🇺🇸Greenville, South Carolina, United States
University of Alberta Hospital
🇨🇦Edmonton, Alberta, Canada
Children's Hospital of Eastern Ontario
🇨🇦Ottawa, Ontario, Canada
Princess Margaret Hospital for Children
🇦🇺Perth, Western Australia, Australia
Perth Children's Hospital
🇦🇺Perth, Western Australia, Australia
University of Oklahoma Health Sciences Center
🇺🇸Oklahoma City, Oklahoma, United States
Legacy Emanuel Children's Hospital
🇺🇸Portland, Oregon, United States
Oregon Health and Science University
🇺🇸Portland, Oregon, United States
Phoenix Childrens Hospital
🇺🇸Phoenix, Arizona, United States
Children's Hospital of Alabama
🇺🇸Birmingham, Alabama, United States
UCSF Medical Center-Mission Bay
🇺🇸San Francisco, California, United States
Vanderbilt University/Ingram Cancer Center
🇺🇸Nashville, Tennessee, United States
Dana-Farber Cancer Institute
🇺🇸Boston, Massachusetts, United States
CancerCare Manitoba
🇨🇦Winnipeg, Manitoba, Canada
Naval Medical Center - Portsmouth
🇺🇸Portsmouth, Virginia, United States
Penn State Children's Hospital
🇺🇸Hershey, Pennsylvania, United States
East Tennessee Childrens Hospital
🇺🇸Knoxville, Tennessee, United States
University of Florida Health Science Center - Gainesville
🇺🇸Gainesville, Florida, United States
AdventHealth Orlando
🇺🇸Orlando, Florida, United States
Children's Hospital Colorado
🇺🇸Aurora, Colorado, United States
Kapiolani Medical Center for Women and Children
🇺🇸Honolulu, Hawaii, United States
Norton Children's Hospital
🇺🇸Louisville, Kentucky, United States
Yale University
🇺🇸New Haven, Connecticut, United States
Saint Joseph's Hospital/Children's Hospital-Tampa
🇺🇸Tampa, Florida, United States
UNC Lineberger Comprehensive Cancer Center
🇺🇸Chapel Hill, North Carolina, United States
Arnold Palmer Hospital for Children
🇺🇸Orlando, Florida, United States
Nemours Children's Hospital
🇺🇸Orlando, Florida, United States
Children's Hospital New Orleans
🇺🇸New Orleans, Louisiana, United States
C S Mott Children's Hospital
🇺🇸Ann Arbor, Michigan, United States
Children's Mercy Hospitals and Clinics
🇺🇸Kansas City, Missouri, United States
Wake Forest University Health Sciences
🇺🇸Winston-Salem, North Carolina, United States
Medical University of South Carolina
🇺🇸Charleston, South Carolina, United States
Virginia Commonwealth University/Massey Cancer Center
🇺🇸Richmond, Virginia, United States
Dell Children's Medical Center of Central Texas
🇺🇸Austin, Texas, United States