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

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ClinicalTrials.gov Identifier: NCT03057626
Recruitment Status : Recruiting
First Posted : February 20, 2017
Last Update Posted : August 7, 2018
Sponsor:
Collaborator:
National Cancer Institute (NCI)
Information provided by (Responsible Party):
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.

Condition or disease Intervention/treatment
Recurrent Neuroblastoma Stage 2A Neuroblastoma Stage 2B Neuroblastoma Stage 3 Neuroblastoma Stage 4 Neuroblastoma Stage 4S Neuroblastoma Other: Cytology Specimen Collection Procedure Other: Laboratory Biomarker Analysis Other: Quality-of-Life Assessment

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.


Study Type : Observational
Estimated Enrollment : 400 participants
Official Title: LEAHRN (Late Effects After High-Risk Neuroblastoma) Study
Actual Study Start Date : June 5, 2017
Estimated Primary Completion Date : August 1, 2020
Estimated Study Completion Date : August 1, 2020

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Neuroblastoma

Group/Cohort Intervention/treatment
Observational (specimen collection)
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.
Other: Cytology Specimen Collection Procedure
Undergo collection of blood and urine
Other Name: Cytologic Sampling

Other: Laboratory Biomarker Analysis
Correlative studies

Other: Quality-of-Life Assessment
Ancillary studies
Other Name: Quality of Life Assessment




Primary Outcome Measures :
  1. Prevalence of specific late effects [ Time Frame: Up to 3 years ]
    Will calculate as the number of patients with late effects divided by the number with known status of that endpoint. Will be analyzed using appropriate descriptive statistics: mean, standard deviation, quartiles for continuous endpoints, proportions and confidence intervals for binary, ordinal, and categorical endpoints. Graphical presentations will include scatterplots and histograms. Using data from ANBL00B1, a descriptive comparison will be performed of patients who did not enroll on the study versus those who did enroll, in terms of standard clinical and biological neuroblastoma risk factors from the time of diagnosis. We will characterize the patient cohort in terms of clinical and biological characteristics using the appropriate descriptive statistics. For each patient with one or more blood relatives with cancer, a pedigree will be generated.

  2. Prevalence of subsequent malignant neoplasm (SMN) [ Time Frame: Up to 3 years ]
    Will calculate as the number of patients with SMN divided by the number with known status of that endpoint. Will be analyzed using appropriate descriptive statistics: mean, standard deviation, quartiles for continuous endpoints, proportions and confidence intervals for binary, ordinal, and categorical endpoints. Graphical presentations will include scatterplots and histograms. Using data from ANBL00B1, a descriptive comparison will be performed of patients who did not enroll on the study versus those who did enroll, in terms of standard clinical and biological neuroblastoma risk factors from the time of diagnosis. We will characterize the patient cohort in terms of clinical and biological characteristics using the appropriate descriptive statistics. For each patient with one or more blood relatives with cancer, a pedigree will be generated.

  3. Risk factors [ Time Frame: Up to 3 years ]
    Will 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 MIBG scans, and therapeutic MIBG (Y/N). In univariate tests of association, we will perform one-sided two-sample t-tests between a risk factor (binary) and a late effect (continuous), chi-squared tests (binary or ordinal variables), or linear regression (continuous variables).

  4. Prognostic of decreased PedsQL score [ Time Frame: Up to 3 years ]
    Will perform univariate one-sided two-sample t-tests and analysis of variance using the independent variables: impaired physical growth, delayed pubertal development, chronic disease, impaired executive functioning, and impaired social functioning.


Secondary Outcome Measures :
  1. Collection and storage of blood samples [ Time Frame: Up to 3 years ]
    Survivors and their families will be consented for future utilization of their banked sample for future research. Samples will be linked to clinical information but results will not be returned to the treating physician.



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Ages Eligible for Study:   5 Years to 50 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Patients must have been enrolled on COG Neuroblastoma Biology Study ANBL00B1.
Criteria

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03057626


  Show 69 Study Locations
Sponsors and Collaborators
Children's Oncology Group
National Cancer Institute (NCI)
Investigators
Principal Investigator: Tara Henderson Children's Oncology Group

Responsible Party: Children's Oncology Group
ClinicalTrials.gov Identifier: NCT03057626     History of Changes
Other Study ID Numbers: ALTE15N2
NCI-2017-00170 ( Registry Identifier: CTRP (Clinical Trial Reporting Program) )
ALTE15N2 ( Other Identifier: Childrens Oncology Group )
COG-ALTE15N2 ( Other Identifier: DCP )
ALTE15N2 ( Other Identifier: CTEP )
UG1CA189955 ( U.S. NIH Grant/Contract )
First Posted: February 20, 2017    Key Record Dates
Last Update Posted: August 7, 2018
Last Verified: May 2018

Additional relevant MeSH terms:
Neuroblastoma
Neuroectodermal Tumors, Primitive, Peripheral
Neuroectodermal Tumors, Primitive
Neoplasms, Neuroepithelial
Neuroectodermal Tumors
Neoplasms, Germ Cell and Embryonal
Neoplasms by Histologic Type
Neoplasms
Neoplasms, Glandular and Epithelial
Neoplasms, Nerve Tissue