Late Effects After Treatment in Patients With Previously Diagnosed High-Risk Neuroblastoma

Recurrent Neuroblastoma Clinical Trial

Official title:

LEAHRN (Late Effects After High-Risk Neuroblastoma) Study

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT03057626
• Other study ID #: ALTE15N2
• Secondary ID: NCI-2017-00170AL
• Status: Active, not recruiting
• Start date: June 5, 2017
• Est. completion date: September 30, 2024

Study information

• Verified date: October 2023
• Source: Children's Oncology Group
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

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 information / eligibility

• Status: Active, not recruiting
• Enrollment: 376
• Est. completion date: September 30, 2024
• Est. primary completion date: September 30, 2021
• Accepts healthy volunteers: No
• Gender: All
• Age group: 5 Years to 50 Years

Eligibility

Inclusion Criteria:

• 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

Exclusion Criteria:

• 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

Related Conditions & MeSH terms

• Neuroblastoma
• Recurrent Neuroblastoma
• Stage 2A Neuroblastoma
• Stage 2B Neuroblastoma
• Stage 3 Neuroblastoma
• Stage 4 Neuroblastoma
• Stage 4S Neuroblastoma

Intervention

Other:
• Cytology Specimen Collection Procedure
Undergo collection of blood and urine
• Laboratory Biomarker Analysis
Correlative studies
• Quality-of-Life Assessment
Ancillary studies

Locations

Country	Name	City	State
Australia	Perth Children's Hospital	Perth	Western Australia
Australia	Princess Margaret Hospital for Children	Perth	Western Australia
Canada	Alberta Children's Hospital	Calgary	Alberta
Canada	University of Alberta Hospital	Edmonton	Alberta
Canada	IWK Health Centre	Halifax	Nova Scotia
Canada	McMaster Children's Hospital at Hamilton Health Sciences	Hamilton	Ontario
Canada	Kingston Health Sciences Centre	Kingston	Ontario
Canada	Centre Hospitalier Universitaire Sainte-Justine	Montreal	Quebec
Canada	The Montreal Children's Hospital of the MUHC	Montreal	Quebec
Canada	Children's Hospital of Eastern Ontario	Ottawa	Ontario
Canada	Centre Hospitalier Universitaire de Quebec	Quebec
Canada	Hospital for Sick Children	Toronto	Ontario
Canada	CancerCare Manitoba	Winnipeg	Manitoba
New Zealand	Starship Children's Hospital	Grafton	Auckland
United States	Children's Hospital Medical Center of Akron	Akron	Ohio
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	Dell Children's Medical Center of Central Texas	Austin	Texas
United States	Sinai Hospital of Baltimore	Baltimore	Maryland
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	Roswell Park Cancer Institute	Buffalo	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	Carolinas Medical Center/Levine Cancer Institute	Charlotte	North Carolina
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	Rainbow Babies and Childrens Hospital	Cleveland	Ohio
United States	Nationwide Children's Hospital	Columbus	Ohio
United States	Medical City Dallas Hospital	Dallas	Texas
United States	UT Southwestern/Simmons Cancer Center-Dallas	Dallas	Texas
United States	Blank Children's Hospital	Des Moines	Iowa
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	Broward Health Medical Center	Fort Lauderdale	Florida
United States	Golisano Children's Hospital of Southwest Florida	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	Penn State Children's Hospital	Hershey	Pennsylvania
United States	Kapiolani Medical Center for Women and Children	Honolulu	Hawaii
United States	Baylor College of Medicine/Dan L Duncan Comprehensive Cancer Center	Houston	Texas
United States	University of Iowa/Holden Comprehensive Cancer Center	Iowa City	Iowa
United States	University of Mississippi Medical Center	Jackson	Mississippi
United States	Nemours Children's Clinic-Jacksonville	Jacksonville	Florida
United States	Children's Mercy Hospitals and Clinics	Kansas City	Missouri
United States	East Tennessee Childrens Hospital	Knoxville	Tennessee
United States	University of Kentucky/Markey Cancer Center	Lexington	Kentucky
United States	Arkansas Children's Hospital	Little Rock	Arkansas
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	Saint Jude Children's Research Hospital	Memphis	Tennessee
United States	Children's Hospitals and Clinics of Minnesota - Minneapolis	Minneapolis	Minnesota
United States	University of Minnesota/Masonic Cancer Center	Minneapolis	Minnesota
United States	Vanderbilt University/Ingram Cancer Center	Nashville	Tennessee
United States	Yale University	New Haven	Connecticut
United States	Children's Hospital New Orleans	New Orleans	Louisiana
United States	Advocate Children's Hospital-Oak Lawn	Oak Lawn	Illinois
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	AdventHealth Orlando	Orlando	Florida
United States	Arnold Palmer Hospital for Children	Orlando	Florida
United States	Nemours Children's Hospital	Orlando	Florida
United States	Lucile Packard Children's Hospital Stanford University	Palo Alto	California
United States	Advocate Children's Hospital-Park Ridge	Park Ridge	Illinois
United States	Saint Joseph's Regional Medical Center	Paterson	New Jersey
United States	Saint Jude Midwest Affiliate	Peoria	Illinois
United States	Children's Hospital of Philadelphia	Philadelphia	Pennsylvania
United States	Phoenix Childrens Hospital	Phoenix	Arizona
United States	Children's Hospital of Pittsburgh of UPMC	Pittsburgh	Pennsylvania
United States	Legacy Emanuel Children's Hospital	Portland	Oregon
United States	Oregon Health and Science University	Portland	Oregon
United States	Naval Medical Center - Portsmouth	Portsmouth	Virginia
United States	Virginia Commonwealth University/Massey Cancer Center	Richmond	Virginia
United States	Cardinal Glennon Children's Medical Center	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	UCSF Medical Center-Mission Bay	San Francisco	California
United States	Seattle Children's Hospital	Seattle	Washington
United States	Sanford USD Medical Center - Sioux Falls	Sioux Falls	South Dakota
United States	Providence Sacred Heart Medical Center and Children's Hospital	Spokane	Washington
United States	Baystate Medical Center	Springfield	Massachusetts
United States	Stony Brook University Medical Center	Stony Brook	New York
United States	Saint Joseph's Hospital/Children's Hospital-Tampa	Tampa	Florida
United States	Children's National Medical Center	Washington	District of Columbia
United States	Alfred I duPont Hospital for Children	Wilmington	Delaware
United States	Wake Forest University Health Sciences	Winston-Salem	North Carolina

Sponsors (2)

Lead Sponsor	Collaborator
Children's Oncology Group	National Cancer Institute (NCI)

Countries where clinical trial is conducted

United States,  Australia,  Canada,  New Zealand, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Prevalence of specific late effects	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.	Up to 3 years
Primary	Risk factors of late effects	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.	Up to 3 years
Primary	Pediatric Quality of Life (PedsQL) score	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.	Up to 3 years
Secondary	Collection and storage of blood samples	Proportion of survivors and their families that consent for future utilization of their banked sample for future research will be reported.	Up to 3 years