NeuroblastomaNCT01526603
High Dose Chemotherapy and Autologous Transplant for Neuroblastoma
A study testing Carboplatin, Autologous stem cell infusion and Granulocyte colony stimulating factor in people with Neuroblastoma, run by Masonic Cancer Center, University of Minnesota at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages Up to 30 years
- Conditions the study is for
- Neuroblastoma
- What is being tested?
- Carboplatin, Autologous stem cell infusion and Granulocyte colony stimulating factor
- Type of treatment
- Injection or infusion, Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 13 participants
- Where
- 1 siteUnited States
- Study dates
- 28 Mar 2012to 30 May 2023
- Phase
- Interventional
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Up to 30 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- 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
Who cannot join
- 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.
Trial locations and contacts
1 site in 1 country.
United States1 site
Masonic Cancer Center, University of Minnesota
Minneapolis, Minnesota, 55455
About this study
Official title: High Dose Chemotherapy and Autologous Peripheral Blood Stem Cell (PBSC) Rescue for Neuroblastoma: Standard of Care Considerations
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.
Read the detailed description
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.
- Conditions
- Neuroblastoma
- Being tested
- Carboplatin, Autologous stem cell infusion, Granulocyte colony stimulating factor, Radiation therapy, Isotretinoin (13-cis-retinoic acid)
- Keywords
- peripheral blood stem cell transplantation, autologous stem cell transplant
- Medical subject headings
- Neuroblastoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CarboplatinDrug
Also called Paraplatin
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.
Autologous stem cell infusionBiological
On day 0 the stem cells will be infused immediately after thawing over 15-60 minutes per institutional guidelines.
Granulocyte colony stimulating factorBiological
Also called G-CSF
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.
Radiation therapyRadiation
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
Also called Accutane
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.
MelphalanDrug
Also called Alkeran
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
EtoposideDrug
Also called Eposin, VP-16
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
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: 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 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Patients with Successful Engraftment
The time to neutrophil engraftment will be assessed by standard statistical approaches.
Measured: Day 42
Secondary outcomes (5)
Number of Patients with Disease Free Survival
The number of patients alive and disease free will be assessed using standard statistical approaches.
Measured: 2 Years
Overall Survival
The number of patients alive will be assessed by standard statistical approaches.
Measured: 2 Years
Number of Patients with Treatment Related Death
The rate of treatment related mortality will be assessed by cumulative incidence approach.
Measured: 1 Year
Number of Patients with Disease Free Survival
The number of patients alive and disease free will be assessed using standard statistical approaches.
Measured: 5 Years
Overall Survival
The number of patients alive will be assessed by standard statistical approaches.
Measured: 5 Years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 Feb 2012 (estimated)
- Study start28 Mar 2012
- Primary completion30 May 2023
- Study completion30 May 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for NeuroblastomaStudies of the same condition, open and under way.
- More trials run by Masonic Cancer Center, University of MinnesotaThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Other
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 13 participants (actual)
Sponsor and investigators
- Lead sponsor
- Masonic Cancer Center, University of Minnesota
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Ashish Gupta, MBBS, MPH, Principal investigator, Masonic Cancer Center, University of Minnesota
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01526603
- Sponsor's protocol ID
- 2011OC072
- Other IDs
- MT2011-11C
- Status last verified
- Feb 2024
- First submitted
- 31 Jan 2012
- Last updated
- 26 Feb 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 26 Feb 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- High Dose Chemotherapy and Autologous Peripheral Blood Stem Cell (PBSC) Rescue for Neuroblastoma: Standard of Care Considerations
- ClinicalTrials.gov ID
- NCT01526603
- Acronym / sponsor ID
- 2011OC072
- Conditions
- Neuroblastoma
- Keywords
- peripheral blood stem cell transplantation, autologous stem cell transplant
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 13 participants (actual)
- Lead sponsor
- Masonic Cancer Center, University of MinnesotaUniversity, hospital or other
- Study start
- 28 Mar 2012 (actual)
- Primary completion
- 30 May 2023 (actual)
- Study completion
- 30 May 2023 (actual)
- First posted
- 6 Feb 2012 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
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.
Study design
- Primary purpose
- Other
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Patients Treated for Neuroblastoma | Other | Drug: Carboplatin, Biological: Autologous stem cell infusion, Biological: Granulocyte colony stimulating factor, Radiation: Radiation therapy, Drug: Isotretinoin (13-cis-retinoic acid), Drug: Melphalan, Drug: EtoposideAccording 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. |
Who may qualify
- Ages
- Up to 30 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (7 listed)
- 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)
Key exclusion criteria (2 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| CarboplatinAlso: ParaplatinCarboplatin 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. | Drug | Patients Treated for Neuroblastoma |
| Autologous stem cell infusionOn day 0 the stem cells will be infused immediately after thawing over 15-60 minutes per institutional guidelines. | Biological | Patients Treated for Neuroblastoma |
| Granulocyte colony stimulating factorAlso: G-CSFBeginning 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. | Biological | Patients Treated for Neuroblastoma |
| Radiation therapyIt 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. | Radiation | Patients Treated for Neuroblastoma |
| Isotretinoin (13-cis-retinoic acid)Also: AccutanePost-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. | Drug | Patients Treated for Neuroblastoma |
| MelphalanAlso: AlkeranMelphalan 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 | Drug | Patients Treated for Neuroblastoma |
| EtoposideAlso: Eposin, VP-16Etoposide 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 | Drug | Patients Treated for Neuroblastoma |
Primary outcome
Number of Patients with Successful Engraftment
Time frame: Day 42
The time to neutrophil engraftment will be assessed by standard statistical approaches.
5 secondary outcomes
Number of Patients with Disease Free Survival
Time frame: 2 Years
Overall Survival
Time frame: 2 Years
Number of Patients with Treatment Related Death
Time frame: 1 Year
Number of Patients with Disease Free Survival
Time frame: 5 Years
Overall Survival
Time frame: 5 Years
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 26 Feb 2024.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Ashish Gupta, MBBS, MPH principal investigator
Masonic Cancer Center, University of Minnesota
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Carboplatin, Autologous stem cell infusion and Granulocyte colony stimulating factor in people with Neuroblastoma, run by Masonic Cancer Center, University of Minnesota at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 26 Feb 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Patients with Successful Engraftment (time frame: Day 42). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 26 Feb 2024.