Late Effects After Tx for High-Risk Neuroblastoma

September 18, 2018

LEAHRN (Late Effects After High-Risk Neuroblastoma) Study

  • Clinical Trial Information

    Trial Contact: El-Shami, Jessica; Francois, Laetitia Claire; Leffin, Melissa; Spinelli, Jennifer

  • IRB No: ALTE15N2

    Protocol Abbrev: ALTE15N2

    Principal Investigator: Vincent F. Giusti, MD

    Age Group: Pediatric

    Secondary Protocol No: ALTE15N2

    Treatment: Cytology Specimen Collection Procedure, Laboratory Biomarker Analysis, Quality-of-Life Assessment

    ClinicalTrials.gov ID: NCT03057626

  • Objective

    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.

  • Key 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
    •  The patient or a parent/guardian must have receptive and expressive language skills in English, French, or Spanish since the assessment instruments are available in these languages only. If the patient is over the age of 18 and does not have expressive language skills it is permitted for a parent/guardian to complete the forms.