Skip to main content
OpenTrials
Completed

NCT Number: NCT01526603

High Dose Chemotherapy and Autologous Transplant for Neuroblastoma

This is a standard of care document, outlining the therapy for children with high risk neuroblastoma who are not eligible for Children's Oncology Group (COG) studies.

Completed

Looking for future studies?

Notify Me

Key information

Conditions

Neuroblastoma Neoplasms Neoplasms by Histologic Type Neoplasms, Germ Cell and Embryonal Neoplasms, Glandular and Epithelial Neoplasms, Nerve Tissue Neoplasms, Neuroepithelial Neuroectodermal Tumors Neuroectodermal Tumors, Primitive Neuroectodermal Tumors, Primitive, Peripheral

Age range

Up to 30 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Not applicable

Primary location

Masonic Cancer Center, University of Minnesota

Minneapolis, Minnesota, 55455, United States

About this study

This therapy involves the use of melphalan, etoposide, and carboplatin (consolidation chemotherapy); autologous stem cell rescue, post-transplant radiation therapy and a maintenance phase with Isotretinoin (Accutane, 13-cis-retinoic acid) therapy. If available, patients should also consider post-transplant therapy with cytokines and monoclonal antibody (ch14.18) on a COG or New Approaches to Neuroblastoma Therapy (NANT) trial.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Less than 30 years of age at diagnosis of neuroblastoma
  • No evidence of disease progression: defined as increase in tumor size of >25% or new lesions
  • Recovery from last induction course of chemotherapy (absolute neutrophil count > 500 and platelet > 20,000)
  • No uncontrolled infection
  • Minimum frozen peripheral blood stem cells (PBSCs) of 2 x 10^6 CD34 cells/kg for transplant are mandatory and 2 x 10^6 CD34 cells/kg for back-up are strongly recommended (thus, PBSC of 4 x 106 CD34 cells/kg is encouraged)
  • Adequate organ function defined as:
  • Hepatic: aspartate aminotransferase (AST) < 3 x upper limit of institutional normal 8 Cardiac: shortening fraction ≥ 27% or ejection fraction ≥ 50%, no clinical congestive heart failure 8 Renal: Creatinine clearance or glomerular filtration rate (GFR) > 60 mL/min/1.73m^2 If a creatinine clearance is performed at end induction and the result is < 100 ml/min/1.73m^2, a GFR must then be performed using a nuclear blood sampling method or iothalamate clearance method. Camera method is NOT allowed as measure of GFR prior to or during Consolidation therapy for patients with GFR or creatinine clearance of < 100 ml/min/1.73m^2

Exclusion criteria

  • Patients with progressive disease should consider participating in phase I studies since consolidation therapy using the regimen outlined in this document have not been determined to be useful.
  • Patients who are delayed in consolidation chemotherapy beyond 8 weeks, and don't meet organ function criteria.

Treatment and study plan

carboplatin

Drug

Carboplatin intravenously (IV), 425 mg/m2/dose (or if ≤ 12kg, 14.2 mg/kg/dose) once daily x 4 doses on days 7 through 4 pretransplant.

Other names: Paraplatin

Autologous stem cell infusion

Biological

On day 0 the stem cells will be infused immediately after thawing over 15-60 minutes per institutional guidelines.

Granulocyte colony stimulating factor

Biological

Beginning on day 0 after infusion of the PBSC, patients will receive G-CSF subcutaneously (SQ) or IV (SQ preferred) 5 micrograms/kg once daily and continuing once daily until post-nadir absolute neutrophil count (ANC) > 2000/μL for 3 consecutive days.

Other names: G-CSF

radiation therapy

Radiation

It is suggested that patients who have a complete surgical resection of the primary tumor receive 21.6 Gy external beam radiation therapy (EBRT) to the post-induction chemotherapy, pre-operative primary tumor volume. It is suggested that patients who have an incomplete surgical resection of the primary tumor (residual soft tissue mass measuring >1 cm3) will receive 21.6 Gy EBRT to the postinduction chemotherapy, pre-operative primary tumor volume and an additional boost of 14.4 Gy EBRT to the gross residual tumor (total dose 36 Gy to gross residual tumor volume). Radiation should be given after stem cell transplantation and should start no sooner than 28 days post transplant.

Isotretinoin (13-cis-retinoic acid)

Drug

Post-transplant maintenance therapy with cis-RA daily for 14 days every 28 days repeated for 6 months. This phase of the therapy can be initiated by the BMT team and continued by the referring physician. It is recommended to begin Isotretinoin at day 66 post-transplant and no later than day 100. For patients ≤12 kg, isotretinoin (accutane) should be administered at 5.33 mg/kg/dose divided twice daily. For patients >12 kg isotretinoin (accutane) should be administered at 160 mg/m^2/day divided twice a day. Patients should be considered for monoclonal antibody therapy against GD2, such as ch14.18 if such trials are available.

Other names: Accutane

melphalan

Drug

Melphalan Intravenously (IV), 70 mg/m2/dose (or if ≤ 12 kg, 2.3 mg/kg/dose) once daily x 3 doses on days 7 through 5 pretransplant

Other names: Alkeran

etoposide

Drug

Etoposide intravenously (IV), 338 mg/m2/dose (or if ≤ 12kg, 11.3 mg/kg/dose) once daily x 4 doses on days 7 through 4 pretransplant

Other names: Eposin, VP-16

Primary outcomes

  1. Number of Patients with Successful Engraftment

    Time frame: Day 42

    The time to neutrophil engraftment will be assessed by standard statistical approaches.

Secondary outcomes

  1. Number of Patients with Disease Free Survival

    Time frame: 2 Years

    The number of patients alive and disease free will be assessed using standard statistical approaches.

  2. Overall Survival

    Time frame: 2 Years

    The number of patients alive will be assessed by standard statistical approaches.

  3. Number of Patients with Treatment Related Death

    Time frame: 1 Year

    The rate of treatment related mortality will be assessed by cumulative incidence approach.

  4. Number of Patients with Disease Free Survival

    Time frame: 5 Years

    The number of patients alive and disease free will be assessed using standard statistical approaches.

  5. Overall Survival

    Time frame: 5 Years

    The number of patients alive will be assessed by standard statistical approaches.

Sponsors and collaborators

Lead sponsor

Masonic Cancer Center, University of Minnesota

Other

Registry information

Official study title

High Dose Chemotherapy and Autologous Peripheral Blood Stem Cell (PBSC) Rescue for Neuroblastoma: Standard of Care Considerations

Important dates

Study start
2012
Primary completion
2023
Study completion
2023
First posted
Feb 6, 2012
Registry last updated
Feb 26, 2024

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Related clinical trials

Published trials that share one or more normalized conditions with this study.