CLINICAL TRIAL / NCT03057626
Late Effects After Treatment in Patients With Previously Diagnosed High-Risk Neuroblastoma
- Observational
- Recruiting
- NCT03057626
Contact Information
LEAHRN (Late Effects After High-Risk Neuroblastoma) Study
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.
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.
Gender
All
Age Group
5 Years to 50 Years
Accepting Healthy Volunteers
No
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
- Neuroblastoma