High Dose Chemotherapy and Autologous Transplant for Neuroblastoma

NCT01526603 | Completed

• 2 other identifiers: 2011OC072MT2011-11C
• Study Type: interventional
• Target: 13
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Neuroblastoma

Keywords

peripheral blood stem cell transplantation; autologous stem cell transplant

Outcome Measures

Primary Outcomes (1)

• Number of Patients with Successful Engraftment

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

  Day 42

Secondary Outcomes (5)

• Number of Patients with Disease Free Survival

  2 Years

• Overall Survival

  2 Years

• Number of Patients with Treatment Related Death

  1 Year

• Number of Patients with Disease Free Survival

  5 Years

• Overall Survival

  5 Years

Study Arms (1)

Patients Treated for Neuroblastoma

OTHER

According to patient weight and renal function, consolidation chemotherapy using various doses of Melphalan, Etoposide, and Carboplatin followed by autologous stem cell infusion and serial post-transplant Granulocyte Colony Stimulating Factor, radiation therapy and Isotretinoin maintenance therapy.

Drug: Carboplatin; Biological: Autologous stem cell infusion; Biological: Granulocyte colony stimulating factor; Radiation: Radiation therapy; Drug: Isotretinoin (13-cis-retinoic acid); Drug: Melphalan; Drug: Etoposide

Interventions

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.

Also known as: Paraplatin

Patients Treated for Neuroblastoma

Autologous stem cell infusion BIOLOGICAL

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

Patients Treated for Neuroblastoma

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.

Also known as: G-CSF

Patients Treated for Neuroblastoma

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.

Patients Treated for Neuroblastoma

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.

Also known as: Accutane

Patients Treated for Neuroblastoma

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

Also known as: Alkeran

Patients Treated for Neuroblastoma

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

Also known as: Eposin, VP-16

Patients Treated for Neuroblastoma

Eligibility Criteria

• Age: Up to 30 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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

You may not qualify if:

• 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.

Sponsors & Collaborators

• Masonic Cancer Center, University of Minnesota: lead

Study Sites (1)

Masonic Cancer Center, University of Minnesota

Minneapolis, Minnesota, 55455, United States

Location

MeSH Terms

Conditions

Neuroblastoma

Interventions

Carboplatin; Granulocyte Colony-Stimulating Factor; Radiotherapy; Isotretinoin; Melphalan; Etoposide

Condition Hierarchy (Ancestors)

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

Intervention Hierarchy (Ancestors)

Coordination Complexes; Organic Chemicals; Colony-Stimulating Factors; Glycoproteins; Glycoconjugates; Carbohydrates; Hematopoietic Cell Growth Factors; Cytokines; Intercellular Signaling Peptides and Proteins; Peptides; Amino Acids, Peptides, and Proteins; Proteins; Biological Factors; Therapeutics; Retinoids; Carotenoids; Polyenes; Alkenes; Hydrocarbons, Acyclic; Hydrocarbons; Cyclohexenes; Cyclohexanes; Cycloparaffins; Hydrocarbons, Alicyclic; Hydrocarbons, Cyclic; Terpenes; Pigments, Biological; Nitrogen Mustard Compounds; Mustard Compounds; Hydrocarbons, Halogenated; Phenylalanine; Amino Acids, Aromatic; Amino Acids, Cyclic; Amino Acids; Podophyllotoxin; Tetrahydronaphthalenes; Naphthalenes; Polycyclic Aromatic Hydrocarbons; Hydrocarbons, Aromatic; Polycyclic Compounds; Glucosides; Glycosides

Study Officials

• Ashish Gupta, MBBS, MPH

  Masonic Cancer Center, University of Minnesota

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Purpose: OTHER
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 31, 2012
• First Posted: February 6, 2012
• Study Start: March 28, 2012
• Primary Completion: May 30, 2023
• Study Completion: May 30, 2023
• Last Updated: February 26, 2024

Record last verified: 2024-02

Locations

US (1)