Last updated on April 2019

Response and Biology-Based Risk Factor-Guided Therapy in Treating Younger Patients With Non-high Risk Neuroblastoma


Brief description of study

This phase III trial studies how well response and biology-based risk factor-guided therapy works in treating younger patients with non-high risk neuroblastoma. Sometimes a tumor may not need treatment until it progresses. In this case, observation may be sufficient. Measuring biomarkers in tumor cells may help plan when effective treatment is necessary and what the best treatment is. Response and biology-based risk factor-guided therapy may be effective in treating patients with non-high risk neuroblastoma and may help to avoid some of the risks and side effects related to standard treatment.

Detailed Study 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 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.

SECONDARY 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 3 years.

Clinical Study Identifier: NCT02176967

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Ochsner Medical Center Jefferson

New Orleans, LA United States
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Mount Sinai Hospital

New York, NY United States
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Kaiser Permanente-Oakland

Oakland, CA United States
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Inova Fairfax Hospital

Falls Church, VA United States
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Florida Hospital Orlando

Orlando, FL United States
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Baystate Medical Center

Springfield, MA United States
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Tampa General Hospital

Tampa, FL United States
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University of Virginia Cancer Center

Charlottesville, VA United States
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Cleveland Clinic Foundation

Cleveland, OH United States
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Saint Mary's Hospital

West Palm Beach, FL United States
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Sinai Hospital of Baltimore

Baltimore, MD United States
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Children's Hospital of Alabama

Birmingham, AL United States
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Arkansas Children's Hospital

Little Rock, AR United States
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Children's Hospital Los Angeles

Los Angeles, CA United States
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Nemours Children's Hospital

Orlando, FL United States
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University of Illinois

Chicago, IL United States
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Riley Hospital for Children

Indianapolis, IN United States
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Blank Children's Hospital

Des Moines, IA United States
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Children's Hospital New Orleans

New Orleans, LA United States
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C S Mott Children's Hospital

Ann Arbor, MI United States
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Mayo Clinic

Rochester, MN United States
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Columbia Regional

Columbia, MO United States
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The Childrens Mercy Hospital

Kansas City, MO United States
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University of Rochester

Rochester, NY United States
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Rhode Island Hospital

Providence, RI United States
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Primary Children's Hospital

Salt Lake City, UT United States
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Seattle Children's Hospital

Seattle, WA United States
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Phoenix Childrens Hospital

Phoenix, AZ United States
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Cedars Sinai Medical Center

Los Angeles, CA United States
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UCSF Medical Center-Mission Bay

San Francisco, CA United States
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Yale University

New Haven, CT United States
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Broward Health Medical Center

Fort Lauderdale, FL United States
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Miami Cancer Institute

Miami, FL United States
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Johns Hopkins All Children's Hospital

Saint Petersburg, FL United States
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Tripler Army Medical Center

Honolulu, HI United States
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Norton Children's Hospital

Louisville, KY United States
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Maine Children's Cancer Program

Scarborough, ME United States
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Hurley Medical Center

Flint, MI United States
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Bronson Methodist Hospital

Kalamazoo, MI United States
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West Michigan Cancer Center

Kalamazoo, MI United States
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Mercy Hospital Saint Louis

Saint Louis, MO United States
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Morristown Medical Center

Morristown, NJ United States
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Saint Peter's University Hospital

New Brunswick, NJ United States
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Albany Medical Center

Albany, NY United States
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NYU Winthrop Hospital

Mineola, NY United States
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New York Medical College

Valhalla, NY United States
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East Carolina University

Greenville, NC United States
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Geisinger Medical Center

Danville, PA United States
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Palmetto Health Richland

Columbia, SC United States
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T C Thompson Children's Hospital

Chattanooga, TN United States
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Driscoll Children's Hospital

Corpus Christi, TX United States
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El Paso Children's Hospital

El Paso, TX United States
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Cook Children's Medical Center

Fort Worth, TX United States
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Marshfield Clinic

Marshfield, WI United States
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John Hunter Children's Hospital

Hunter Regional Mail Centre, Australia
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Christchurch Hospital

Christchurch, New Zealand
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Recruitment Status: Open


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