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Combination Chemotherapy With or Without Filgrastim Before Surgery, High-Dose Chemotherapy, and Radiation Therapy Followed by Isotretinoin With or Without Monoclonal Antibody in Treating Patients With Neuroblastoma

Phase 3
Conditions
Neuroblastoma
Registration Number
NCT00030719
Lead Sponsor
University of Leicester
Brief Summary

RATIONALE: Colony-stimulating factors such as filgrastim may increase the number of immune cells found in bone marrow or peripheral blood and may help a person's immune system recover from the side effects of chemotherapy. Combining chemotherapy with peripheral stem cell transplant may allow the doctor to give higher doses of chemotherapy drugs and kill more tumor cells. Monoclonal antibodies can locate tumor cells and either kill them or deliver tumor-killing substances to them without harming normal cells. Combining isotretinoin and monoclonal antibodies may kill any remaining tumor cells following surgery. It is not yet known which treatment regimen is more effective in treating neuroblastoma.

PURPOSE: This randomized phase III trial is studying how well combination chemotherapy with or without filgrastim before surgery, high-dose chemotherapy, and radiation therapy followed by isotretinoin with or without monoclonal antibody work in treating patients with neuroblastoma.

Detailed Description

OBJECTIVES:

* Compare the efficacy of myeloablative therapy with busulfan and melphalan vs carboplatin, etoposide, and melphalan, in terms of 3- and 5-year event-free survival (EFS), progression-free survival (PFS), and overall survival (OS), in patients with high-risk neuroblastoma.

* Compare the 3-year EFS in these patients treated with isotretinoin with or without monoclonal antibody Ch14.18 after myeloablative therapy.

* Determine the response at metastatic sites after induction chemotherapy in these patients.

* Determine the effect of metastatic disease response after induction chemotherapy on EFS, PFS, and OS in these patients.

* Compare the toxicity and episodes of febrile neutropenia in patients treated with induction chemotherapy with or without filgrastim (G-CSF).

* Determine the effect of elective hematopoietic support with G-CSF during induction chemotherapy on peripheral blood stem cell collection in these patients.

* Compare the acute and long-term toxic effects of the 2 myeloablative therapy regimens in these patients.

* Determine the effect of radiotherapy on pre-surgical tumor volume at the primary site on local control, EFS, PFS, and OS in these patients.

* Determine the tolerability of isotretinoin with or without monoclonal antibody Ch14.18 after myeloablative therapy in these patients.

OUTLINE: This is a randomized, multicenter study. Patients are stratified according to disease stage (2 or 3 with MycN amplification vs 4). Patients are randomized to 1 of 8 treatment arms:

Arm I:

* Patients receive induction chemotherapy comprising vincristine IV, carboplatin IV over 1 hour, and etoposide IV over 4 hours on days 1 and 41; vincristine IV and cisplatin IV over 24 hours on days 11, 31, 51, and 71; and vincristine IV on days 21 and 61 and cyclophosphamide IV and etoposide over 4 hours on days 21, 22, 61, and 62. Patients receive filgrastim (G-CSF) subcutaneously on days 3-8, 12-18, 23-28, 32-38, 43-48, 52-58, 63-68, and 72 until peripheral blood stem cell (PBSC) collection.

* Patients undergo PBSC collection beginning on day 80. Patients then undergo surgery on day 95.

* Patients receive myeloablative therapy comprising oral busulfan 4 times daily on days -6 to -3 and melphalan IV over 15 minutes on day -2. Patients undergo PBSC infusion on day 0.

* Patients undergo radiotherapy in 14 fractions over 21 days.

* Beginning within 30 days after radiotherapy, patients receive oral isotretinoin twice daily on days 1-14. Treatment repeats every 28 days for 6 courses.

Arm II:

* Patients receive induction chemotherapy as in arm I, but with no G-CSF. Patients then undergo PBSC collection and surgery as in arm I. Patients receive myeloablative therapy and undergo PBSC infusion as in arm I. Patients undergo radiotherapy as in arm I.

* Patients receive oral isotretinoin twice daily on days 1-14 and monoclonal antibody Ch14.18 IV over 8 hours on days 1-5. Treatment repeats every 28 days for 6 courses for isotretinoin and every 28 days for 5 courses for monoclonal antibody Ch14.18.

Arm III:

* Patients receive induction chemotherapy and G-CSF as in arm I. Patients then undergo PBSC collection and surgery as in arm I. Patients receive myeloablative therapy and undergo PBSC infusion as in arm I. Patients undergo radiotherapy as in arm I. Patients receive isotretinoin as in arm I.

Arm IV:

* Patients receive induction chemotherapy as in arm II. Patients then undergo PBSC collection and surgery as in arm I. Patients receive myeloablative therapy and undergo PBSC infusion as in arm I. Patients undergo radiotherapy as in arm I. Patients receive isotretinoin and monoclonal antibody Ch14.18 as in arm II.

Arm V:

* Patients receive induction chemotherapy and G-CSF as in arm I.

* Patients receive myeloablative therapy comprising carboplatin IV continuously and etoposide IV continuously on days -7 to -4 and melphalan IV over 15 minutes on days -7 to -5. Patients undergo PBSC infusion on day 0.

* Patients undergo radiotherapy as in arm I. Patients receive isotretinoin as in arm I.

Arm VI:

* Patients receive induction chemotherapy as in arm II. Patients receive myeloablative therapy and undergo PBSC infusion as in arm V. Patients undergo radiotherapy as in arm I. Patients receive isotretinoin and monoclonal antibody Ch14.18 as in arm II.

Arm VII:

* Patients receive induction chemotherapy and G-CSF as in arm I. Patients receive myeloablative therapy and undergo PBSC infusion as in arm V. Patients undergo radiotherapy as in arm I. Patients receive isotretinoin as in arm I.

Arm VIII:

* Patients receive induction chemotherapy as in arm II. Patients receive myeloablative therapy and undergo PBSC infusion as in arm V. Patients undergo radiotherapy as in arm I. Patients receive isotretinoin and monoclonal antibody Ch14.18 as in arm II.

Patients on all treatment arms are followed every 6 months for 3 years and then annually for 2 years.

Peer Reviewed and Funded or Endorsed by Cancer Research UK

PROJECTED ACCRUAL: Approximately 175 patients per year will be accrued for this study.

Recruitment & Eligibility

Status
UNKNOWN
Sex
All
Target Recruitment
175
Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

Study Type
INTERVENTIONAL
Study Design
Not specified
Primary Outcome Measures
NameTimeMethod
Event-free survival at 3 years
Mean number of febrile events during induction
Secondary Outcome Measures
NameTimeMethod
Event-free survival at 5 years
Overall survival
Toxicity
Response rate assessed by the International Neuroblastoma Response Criteria after 4 and 8 induction chemotherapy courses
Biological factors (i.e., MycNM amplification, 1p deletion, ploidy, 17 q+, CD44, and Trk-A)
Serum concentrations of lactic dehydrogenase, ferritin, neurone specific enolase
Urinary catecholamines at diagnosis

Trial Locations

Locations (31)

Universitair Ziekenhuis Gent

🇧🇪

Ghent, Belgium

Rikshospitalet University Hospital

🇳🇴

Oslo, Norway

Birmingham Children's Hospital

🇬🇧

Birmingham, England, United Kingdom

Our Lady's Hospital for Sick Children Crumlin

🇮🇪

Dublin, Ireland

Schneider Children's Medical Center of Israel

🇮🇱

Petah-Tikva, Israel

Southampton General Hospital

🇬🇧

Southampton, England, United Kingdom

Royal Aberdeen Children's Hospital

🇬🇧

Aberdeen, Scotland, United Kingdom

Instituto Portugues de Oncologia de Francisco Gentil - Centro Regional de Oncologia de Lisboa, SA

🇵🇹

Lisbon, Portugal

Centre Hospitalier Universitaire Vaudois

🇨🇭

Lausanne, Switzerland

Leeds Cancer Centre at St. James's University Hospital

🇬🇧

Leeds, England, United Kingdom

Royal Liverpool Children's Hospital, Alder Hey

🇬🇧

Liverpool, England, United Kingdom

Queen's Medical Centre

🇬🇧

Nottingham, England, United Kingdom

Children's Hospital - Sheffield

🇬🇧

Sheffield, England, United Kingdom

Sir James Spence Institute of Child Health at Royal Victoria Infirmary

🇬🇧

Newcastle-Upon-Tyne, England, United Kingdom

Royal Manchester Children's Hospital

🇬🇧

Manchester, England, United Kingdom

Leicester Royal Infirmary

🇬🇧

Leicester, England, United Kingdom

Middlesex Hospital

🇬🇧

London, England, United Kingdom

Oxford Radcliffe Hospital

🇬🇧

Oxford, England, United Kingdom

Royal Marsden - Surrey

🇬🇧

Sutton, England, United Kingdom

Fondazione Istituto Nazionale dei Tumori

🇮🇹

Milan, Italy

Aarhus Universitetshospital - Aarhus Sygehus

🇩🇰

Aarhus, Denmark

Karolinska University Hospital - Solna

🇸🇪

Stockholm, Sweden

St. Anna Children's Hospital

🇦🇹

Vienna, Austria

Institut Gustave Roussy

🇫🇷

Villejuif, France

Hospital Universitario La Fe

🇪🇸

Valencia, Spain

Addenbrooke's Hospital

🇬🇧

Cambridge, England, United Kingdom

Institute of Child Health at University of Bristol

🇬🇧

Bristol, England, United Kingdom

Childrens Hospital for Wales

🇬🇧

Cardiff, Wales, United Kingdom

Royal Belfast Hospital for Sick Children

🇬🇧

Belfast, Northern Ireland, United Kingdom

Royal Hospital for Sick Children

🇬🇧

Glasgow, Scotland, United Kingdom

Great Ormond Street Hospital for Children

🇬🇧

London, England, United Kingdom

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