High Dose Chemotherapy and Autologous Transplant for Neuroblastoma
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|ClinicalTrials.gov Identifier: NCT01526603|
Recruitment Status : Recruiting
First Posted : February 6, 2012
Last Update Posted : March 16, 2023
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|Condition or disease||Intervention/treatment||Phase|
|Neuroblastoma||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||Not Applicable|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||20 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||High Dose Chemotherapy and Autologous Peripheral Blood Stem Cell (PBSC) Rescue for Neuroblastoma: Standard of Care Considerations|
|Actual Study Start Date :||March 28, 2012|
|Estimated Primary Completion Date :||February 2024|
|Estimated Study Completion Date :||February 2024|
Patients Treated for Neuroblastoma
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.
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 Name: Paraplatin
Biological: Autologous stem cell infusion
On day 0 the stem cells will be infused immediately after thawing over 15-60 minutes per institutional guidelines.
Biological: Granulocyte colony stimulating factor
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 Name: G-CSF
Radiation: Radiation therapy
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.
Drug: Isotretinoin (13-cis-retinoic acid)
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 Name: Accutane
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 Name: Alkeran
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
- Number of Patients with Successful Engraftment [ Time Frame: Day 42 ]The time to neutrophil engraftment will be assessed by standard statistical approaches.
- 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.
- Overall Survival [ Time Frame: 2 Years ]The number of patients alive will be assessed by standard statistical approaches.
- Number of Patients with Treatment Related Death [ Time Frame: 1 Year ]The rate of treatment related mortality will be assessed by cumulative incidence approach.
- 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.
- Overall Survival [ Time Frame: 5 Years ]The number of patients alive will be assessed by standard statistical approaches.
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|Ages Eligible for Study:||up to 30 Years (Child, Adult)|
|Sexes Eligible for Study:||All|
|Accepts Healthy Volunteers:||No|
- 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
- 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.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01526603
|Contact: Lisa Burke||612-273-8482||lburke3@Fairview.org|
|United States, Minnesota|
|Masonic Cancer Center, University of Minnesota||Recruiting|
|Minneapolis, Minnesota, United States, 55455|
|Contact: Lisa Burke 612-273-8482 lburke3@Fairview.org|
|Principal Investigator:||Ashish Gupta, MBBS, MPH||Masonic Cancer Center, University of Minnesota|
|Responsible Party:||Masonic Cancer Center, University of Minnesota|
|Other Study ID Numbers:||
MT2011-11C ( Other Identifier: Blood and Marrow Transplantation Program )
|First Posted:||February 6, 2012 Key Record Dates|
|Last Update Posted:||March 16, 2023|
|Last Verified:||March 2023|
|Studies a U.S. FDA-regulated Drug Product:||No|
|Studies a U.S. FDA-regulated Device Product:||No|
peripheral blood stem cell transplantation
autologous stem cell transplant
Neuroectodermal Tumors, Primitive, Peripheral
Neuroectodermal Tumors, Primitive
Neoplasms, Germ Cell and Embryonal
Neoplasms by Histologic Type
Neoplasms, Glandular and Epithelial
Neoplasms, Nerve Tissue
Antineoplastic Agents, Phytogenic
Topoisomerase II Inhibitors
Molecular Mechanisms of Pharmacological Action
Antineoplastic Agents, Alkylating
Physiological Effects of Drugs