Combination Chemotherapy Plus Peripheral Stem Cell Transplantation in Treating Children With Newly Diagnosed Neuroblastoma

This study has been completed.
Sponsor:
Collaborator:
Information provided by (Responsible Party):
Children's Oncology Group
ClinicalTrials.gov Identifier:
NCT00017368
First received: June 6, 2001
Last updated: February 12, 2014
Last verified: February 2014
  Purpose

RATIONALE: Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die. Combining more than one drug may kill more tumor cells. Peripheral stem cell transplantation may allow the doctor to give higher doses of chemotherapy drugs and kill more tumor cells.

PURPOSE: Phase II trial to study the effectiveness of combination chemotherapy followed by peripheral stem cell transplantation in treating children who have newly diagnosed neuroblastoma.


Condition Intervention Phase
Neuroblastoma
Biological: filgrastim
Biological: sargramostim
Drug: carboplatin
Drug: cisplatin
Drug: cyclophosphamide
Drug: doxorubicin hydrochloride
Drug: etoposide
Drug: etoposide phosphate
Drug: ifosfamide
Drug: isotretinoin
Drug: melphalan
Drug: thiotepa
Drug: vincristine sulfate
Procedure: conventional surgery
Procedure: peripheral blood stem cell transplantation
Radiation: radiation therapy
Phase 2

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: A Pilot Study Of Tandem High Dose Chemotherapy With Stem Cell Rescue Following Induction Therapy In Children With High Risk Neuroblastoma

Resource links provided by NLM:


Further study details as provided by Children's Oncology Group:

Primary Outcome Measures:
  • Transplant-related mortality [ Designated as safety issue: Yes ]
    The endpoint used for early stopping rule 9.51 will be transplant-related mortality (TRM). A TRM is defined as any death occurring within 30 days after either the first or second HDC/SCR. The acceptable TRM rate is 7.5%. This rate is based on TRM rates previously observed in the prior CCG study 594, CCG study 3891, and POG study 9640 of 7%, 6%, and 0%, respectively.


Secondary Outcome Measures:
  • Incidence of symptomatic CMV, disseminated adenovirus infection, or EBV-LPD [ Designated as safety issue: Yes ]
    The endpoint to be used in assessing early stopping rule 9.52 will be the incidence of symptomatic CMV, disseminated adenovirus infection, or EBV-LPD. Patients with positive CMV antigenemia or CMV urine culture only (without symptoms of CMV infection), or patients who have a positive culture for adenovirus without evidence of dissemination, or patients with a positive EBV PCR who do not exhibit significant clinical signs (such as adenopathy) or symptoms will not trigger the stopping rule.

  • Event-free Survival [ Time Frame: 1 year ] [ Designated as safety issue: No ]
    Assessment of feasibility and toxicity at the end of the study is time to event. Time to event is defined as the time from registration on the study until the first of the following events occurs: relapse, progression, secondary malignancy, or death, including toxic death. If none of those events occurs, then the time of last contact with the patient is used. Time to event will be used to calculate the one-year Event-free Survival (EFS) rate.


Enrollment: 42
Study Start Date: April 2001
Study Completion Date: January 2012
Primary Completion Date: September 2005 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: All Patients Biological: filgrastim Biological: sargramostim Drug: carboplatin Drug: cisplatin Drug: cyclophosphamide Drug: doxorubicin hydrochloride Drug: etoposide Drug: etoposide phosphate Drug: ifosfamide Drug: isotretinoin Drug: melphalan Drug: thiotepa Drug: vincristine sulfate Procedure: conventional surgery Procedure: peripheral blood stem cell transplantation Radiation: radiation therapy

  Show Detailed Description

  Eligibility

Ages Eligible for Study:   up to 30 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

DISEASE CHARACTERISTICS:

  • Newly diagnosed high-risk neuroblastoma

    • Histologically proven AND/OR
    • Bone marrow specimen showing clumps of tumor cells accompanied by elevated urinary catecholamines
    • Age 1-30:

      • Must meet one of the following INSS staging criteria:

        • Stage IV regardless of biologic factors
        • Stage IIa/IIb with MYCN oncogene amplification (greater than 10) and unfavorable pathology
        • Stage III with MYCN oncogene amplification (greater than 10) or unfavorable pathology
        • Initially stage I, II, or IVS, that has progressed without interval chemotherapy
    • Under age 1:

      • INSS stage III, IV, or IVS with MYCN amplification (greater than 10)
  • Must enter neuroblastoma biology study COG-ANBL00B1 within 2 weeks of diagnosis and before entry on this study

PATIENT CHARACTERISTICS:

Age:

  • 30 and under at original diagnosis

Performance status:

  • Not specified

Life expectancy:

  • Not specified

Hematopoietic:

  • Not specified

Hepatic:

  • Not specified

Renal:

  • Not specified

Other:

  • Not pregnant or nursing
  • Fertile patients must use effective contraception

PRIOR CONCURRENT THERAPY:

Biologic therapy:

  • Not specified

Chemotherapy:

  • See Disease Characteristics
  • No more than 1 prior course of chemotherapy on the intergroup low- or intermediate-risk neuroblastoma studies prior to determination of MYCN status and Shimada histology

Endocrine therapy:

  • Not specified

Radiotherapy:

  • Prior emergent radiotherapy to sites of function- or life-threatening neuroblastoma allowed

Surgery:

  • Not specified

Other:

  • No other prior systemic therapy for neuroblastoma
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00017368

Locations
United States, Georgia
AFLAC Cancer Center and Blood Disorders Service of Children's Healthcare of Atlanta - Scottish RiteCampus
Atlanta, Georgia, United States, 30342
United States, Massachusetts
Dana-Farber/Harvard Cancer Center at Dana Farber Cancer Institute
Boston, Massachusetts, United States, 02115
Floating Hospital for Children
Boston, Massachusetts, United States, 02111
United States, Oregon
CCOP - Columbia River Oncology Program
Portland, Oregon, United States, 97225
United States, Pennsylvania
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104
United States, Texas
Baylor College of Medicine
Houston, Texas, United States, 77030
CCOP - Scott and White Hospital
Temple, Texas, United States, 76508
United States, Wisconsin
CCOP - Marshfield Clinic Research Foundation
Marshfield, Wisconsin, United States, 54449
Australia, Western Australia
Princess Margaret Hospital for Children
Perth, Western Australia, Australia, 6001
Sponsors and Collaborators
Children's Oncology Group
Investigators
Study Chair: Stephan A. Grupp, MD, PhD Children's Hospital of Philadelphia
  More Information

Additional Information:
Publications:
Responsible Party: Children's Oncology Group
ClinicalTrials.gov Identifier: NCT00017368     History of Changes
Other Study ID Numbers: ANBL00P1, COG-ANBL00P1, CDR0000068681
Study First Received: June 6, 2001
Last Updated: February 12, 2014
Health Authority: United States: Federal Government

Keywords provided by Children's Oncology Group:
regional neuroblastoma
disseminated neuroblastoma
localized unresectable neuroblastoma
stage 4S neuroblastoma

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
Liposomal doxorubicin
Etoposide phosphate
Isophosphamide mustard
Cisplatin
Cyclophosphamide
Doxorubicin
Etoposide
Ifosfamide
Melphalan
Thiotepa
Vincristine
Carboplatin
Lenograstim
Isotretinoin
Antineoplastic Agents
Therapeutic Uses
Pharmacologic Actions
Radiation-Sensitizing Agents
Immunosuppressive Agents
Immunologic Factors

ClinicalTrials.gov processed this record on August 19, 2014