Recruitment

Recruitment Status
Recruiting
Estimated Enrollment
803

Summary

Conditions
  • Ganglioneuroblastoma
  • Localized Resectable Neuroblastoma
  • Localized Unresectable Neuroblastoma
  • Neuroblastoma
Type
Interventional
Phase
Phase 3
Design
Allocation: Non-RandomizedIntervention Model: Parallel AssignmentMasking: None (Open Label)Primary Purpose: Treatment

Participation Requirements

Age
Younger than 18 years
Gender
Both males and females

Description

PRIMARY OBJECTIVES: I. To eliminate therapy as the initial approach for infants < 12 months of age with small International Neuroblastoma Risk Group (INRG) stage L1 neuroblastoma while maintaining an overall survival (OS) of 99%. II. To eliminate therapy as the initial approach for non-high-risk pat...

PRIMARY OBJECTIVES: I. To eliminate therapy as the initial approach for infants < 12 months of age with small International Neuroblastoma Risk Group (INRG) stage L1 neuroblastoma while maintaining an overall survival (OS) of 99%. II. To eliminate therapy as the initial approach for non-high-risk patients < 18 months of age with localized neuroblastoma and favorable biology (histologic and genomic features) while maintaining an OS of 99%. III. To achieve a 3-year OS of > 81% for infants < 18 months of age with INRG stage Ms neuroblastoma using objective criteria for early initiation of a response-based treatment algorithm. EXPLORATORY OBJECTIVES: I. To describe the time to intervention or tumor progression, type of intervention and site of progression for patients with localized neuroblastoma who experience progression after an initial period of observation. II. To characterize the pharmacokinetic profile of the chemotherapeutic agents carboplatin and etoposide in patients with stage Ms disease. III. To define the genomic profile of tumors from patients with non-high-risk neuroblastoma both at initial biopsy and at the time of subsequent biopsy or surgical resection. IV. To describe the histology of tumor specimens obtained at the time of subsequent biopsy or surgical resection. V. To determine the salvage rate (OS) of patients with tumor relapse or disease progression. VI. To determine the procedural complication rate (initial biopsy, resection [intraoperative and postoperative], subsequent biopsy) and correlate with the degree of surgical resection. VII. To determine the rate of reduction in image defined risk factors (IDRF) in L2 tumors following observation or chemotherapy. OUTLINE: Patients are assigned to 1 of 3 treatment groups. GROUP A: Patients undergo clinical observation for 96 weeks in the absence disease progression. GROUP B: Patients undergo clinical observation for 3 years in the absence of disease progression. Upon disease progression, patients undergo surgery or receive first-line chemotherapy comprising carboplatin intravenously (IV) over 1 hour on day 1 (courses 1, 2, 4, 6, and 7), etoposide IV over 1 hour on days 1-3 (courses 1, 3, 4, 5, and 7), cyclophosphamide IV over 1 hour on day 1 (courses 2, 3, 5, 6, and 8), and doxorubicin hydrochloride IV over 15 minutes on day 1 (courses 2, 4, 6 and 8). Treatment with chemotherapy repeats every 21 days for 2-8 courses in the absence of disease progression or unacceptable toxicity. Once a partial response (PR) or better is achieved, patients undergo clinical observation for 3 years. GROUP C: Patients at high risk for deterioration and a poor outcome immediately receive first-line chemotherapy as in Group B. All other patients undergo clinical observation for 3 years in the absence of disease progression. Upon disease progression, patients receive first-line chemotherapy as in Group B. Once a PR or better is achieved, patients undergo clinical observation for 3 years. After completion of study treatment, patients are followed up annually for up to 10 years post-enrollment.

Tracking Information

NCT #
NCT02176967
Collaborators
National Cancer Institute (NCI)
Investigators
Principal Investigator: Holly J Meany Children's Oncology Group