NeuroblastomaPhase 2NCT00165139
Intensive Chemo-Radiotherapy With Peripheral Blood Progenitor Cell Rescue for Children With Advanced Neuroblastoma and Sarcomas
A Phase 2 study testing Vincristine, Cyclophosphamide and Adriamycin in people with Neuroblastoma and Ewings Sarcoma, run by Dana-Farber Cancer Institute at 1 site in United States.
Trial summary
The main purpose of this study is to determine the short and long term side effects of a very intensive treatment, which includes combinations of chemotherapy drugs followed by radiation therapy and two transplants supported by peripheral blood progenitor cells (stem cells), for children with advanced stage… Read more about this study
- Who is it for?
- Ages Up to 19 years
- Conditions the study is for
- Neuroblastoma, Ewings Sarcoma, Non-rhabdomyosarcoma Soft Tissue Sarcoma
- What is being tested?
- Vincristine, Cyclophosphamide and Adriamycin
- How many people
- 20 participants
- Where
- 1 siteUnited States
- Study dates
- Jan 1996to Feb 2009
- Phase
- Phase 2
- 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 19 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Previously untreated neuroblastoma Stage D > 365 days
- Previously untreated neuroblastoma Stage C at age > 365 days with n-myc amplification
- Previously untreated metastatic Ewings sarcoma/PNET
- Previously untreated non-rhabdomyosarcoma soft tissue sarcoma
- Confirmation of neuroblastoma at time of diagnosis by histologic specimen or with bone marrow specimen showing solid tumor. Confirmation of sarcoma by histologic specimen with evidence of metastatic disease be imaging study
- Patient 19 years of age or younger
Show all 10Show fewer
- ANC > 1000
- Platelet count > 75,000
- SGOT < 2.5 x ULN
- Normal serum creatinine levels for age
Who cannot join
- Previous malignancy requiring nonsurgical treatment or bone marrow transplant.
- Relapsed disease
Trial locations and contacts
1 site in 1 country.
United States1 site
Dana-Farber Cancer Institute
Boston, Massachusetts, 02115
About this study
Official title: Double Dose Intensive Chemo-Radiotherapy With Peripheral Blood Progenitor Cell Rescue for Children With Advanced Stage Neuroblastoma and Sarcomas
The main purpose of this study is to determine the short and long term side effects of a very intensive treatment, which includes combinations of chemotherapy drugs followed by radiation therapy and two transplants supported by peripheral blood progenitor cells (stem cells), for children with advanced stage neuroblastoma and sarcomas.
Read the detailed description
- The treatment is separated into three stages: 1) Initial treatment-chemotherapy and stem cell collection; 2) Treatment targeted directly to the main tumor-surgery and radiation therapy; 3) Intensified treatment: two stem cell transplants.
- Patients will receive intensive treatment with a combination of seven drugs which will be given every 3 weeks, or as soon as the patient's blood counts are within safe limits. A total of 5 or 6 courses of chemotherapy will be given. The third and fourth course will be followed by stem cell collections and the last course will be followed by bone marrow harvest in preparation for transplant.
- The first treatment involves a high-dose of Cisplatin intravenously once a day for 5 days and VP-16 intravenously on days 2,3 and 4 of the treatment.
- The second course of treatment involves cyclophosphamide intravenously on day 1 and day 2, adriamycin intravenously continuously over 48 hours, and vincristine intravenously on day 1.
- The third course of treatment involves ifosfamide daily on days 1-5 and VP-16 on days 1-3. Mesna will also be given to prevent bladder irritation.
- During the fourth course patients will receive, carboplatin intravenously on days 1 and 2 and VP-16 on days 1,2 and 3 of treatment.
- The fifth course of treatment will be identical to the second course.
- G-CSF (granulocyte colony-stimulating factor) stimulates bone marrow to produce more white blood cells and will be given subcutaneously until the patients' white blood cell counts are at an acceptable level. This may require 10-14 days of G-CSF treatment.
- After the first two treatments, and after the last course of treatment, patients will be evaluated for response. If significant tumor is still present in the bone marrow before the third treatment, stem cell collection will be delayed. If the bone marrow still shows disease after the next treatment, this plan of treatment will be stopped.
- Patients may undergo surgery after the last treatment to remove the tumor and to evaluate the remaining disease. After recovery, radiation therapy may be performed to the main tumor area.
- Patients will receive transplantations of stem cells twice during this study. After each preparative treatment, half of the patient's stored stem cells will be given intravenously. Until the stem cells restore safe levels of blood cells, the patient will be hospitalized (3-4 weeks or longer).
- The first treatment will begin with high doses of carboplatin and VP-16 daily for 3 consecutive days followed by cyclophosphamide on the fourth and fifth days.
- Between 4-6 weeks after the start of the first course of high dose chemotherapy a second course consisting of melphalan and total body irradiation (TBI) will be given. Melphalan will be given daily for 3 consecutive days followed by TBI twice a day for 3 days.
- After each of the two high dose treatments outlined above, the stem cells that were previously harvested from the patient will be given back to them through a central line.
- Stem cells will be collected on 3 separate occasions following each of two cycles of chemotherapy. They will then be processed using a technique that attempts to separate stem cells from other cells, including any cancer cells that may be present.
- Bone marrow will also be harvested prior to the first transplant and will be used if blood counts do not recover after the stem cells are given.
- Being tested
- Vincristine, Cyclophosphamide, Adriamycin, Etoposide (VP-16), Cisplatin
- Keywords
- Stem cell transplant, High-risk pediatric tumors
- Medical subject headings
- Neuroblastoma, Sarcoma, Ewing, Sarcoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
VincristineDrug
Described under detailed description
CyclophosphamideDrug
Described under detailed description
AdriamycinDrug
Described under detailed description
Etoposide (VP-16)Drug
Described under detailed description
CisplatinDrug
Described under detailed description
CarboplatinDrug
Described under detailed description
MelphalanDrug
Described under detailed description
IfosfamideDrug
Described under detailed description
G-CSF (granulocyte-colony stimulating factor)Drug
Described under detailed description
MesnaDrug
Described under detailed description
What is being measured
The primary outcome is the main question the study is designed to answer.
To determine the toxicity and feasibility of double dose chemo-radiotherapy with blood progenitor cell rescue in this patient population.
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 posted14 Sep 2005 (estimated)
- Study startJan 1996
- Primary completionNov 2000
- Study completionFeb 2009
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 Dana-Farber Cancer InstituteThe 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 2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 20 participants (actual)
Sponsor and investigators
- Lead sponsor
- Dana-Farber Cancer Institute
- Sponsor type
- University, hospital or other
- Investigators
- Lisa Diller, MD, Principal investigator, Dana-Farber Cancer Institute
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00165139
- Sponsor's protocol ID
- 94-131
- Status last verified
- Oct 2009
- First submitted
- 9 Sep 2005
- Last updated
- 2 Nov 2009
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 2 Nov 2009. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Double Dose Intensive Chemo-Radiotherapy With Peripheral Blood Progenitor Cell Rescue for Children With Advanced Stage Neuroblastoma and Sarcomas
- ClinicalTrials.gov ID
- NCT00165139
- Acronym / sponsor ID
- 94-131
- Keywords
- Stem cell transplant, High-risk pediatric tumors
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 20 participants (actual)
- Lead sponsor
- Dana-Farber Cancer InstituteUniversity, hospital or other
- Collaborators
- Boston Children's Hospital, Children's Hospital of Philadelphia
- Study start
- Jan 1996
- Primary completion
- Nov 2000 (actual)
- Study completion
- Feb 2009 (actual)
- First posted
- 14 Sep 2005 (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.
The main purpose of this study is to determine the short and long term side effects of a very intensive treatment, which includes combinations of chemotherapy drugs followed by radiation therapy and two transplants supported by peripheral blood progenitor cells (stem cells), for children with advanced stage neuroblastoma and sarcomas.
Detailed description
- The treatment is separated into three stages: 1) Initial treatment-chemotherapy and stem cell collection; 2) Treatment targeted directly to the main tumor-surgery and radiation therapy; 3) Intensified treatment: two stem cell transplants.
- Patients will receive intensive treatment with a combination of seven drugs which will be given every 3 weeks, or as soon as the patient's blood counts are within safe limits. A total of 5 or 6 courses of chemotherapy will be given. The third and fourth course will be followed by stem cell collections and the last course will be followed by bone marrow harvest in preparation for transplant.
- The first treatment involves a high-dose of Cisplatin intravenously once a day for 5 days and VP-16 intravenously on days 2,3 and 4 of the treatment.
- The second course of treatment involves cyclophosphamide intravenously on day 1 and day 2, adriamycin intravenously continuously over 48 hours, and vincristine intravenously on day 1.
- The third course of treatment involves ifosfamide daily on days 1-5 and VP-16 on days 1-3. Mesna will also be given to prevent bladder irritation.
- During the fourth course patients will receive, carboplatin intravenously on days 1 and 2 and VP-16 on days 1,2 and 3 of treatment.
- The fifth course of treatment will be identical to the second course.
- G-CSF (granulocyte colony-stimulating factor) stimulates bone marrow to produce more white blood cells and will be given subcutaneously until the patients' white blood cell counts are at an acceptable level. This may require 10-14 days of G-CSF treatment.
- After the first two treatments, and after the last course of treatment, patients will be evaluated for response. If significant tumor is still present in the bone marrow before the third treatment, stem cell collection will be delayed. If the bone marrow still shows disease after the next treatment, this plan of treatment will be stopped.
- Patients may undergo surgery after the last treatment to remove the tumor and to evaluate the remaining disease. After recovery, radiation therapy may be performed to the main tumor area.
- Patients will receive transplantations of stem cells twice during this study. After each preparative treatment, half of the patient's stored stem cells will be given intravenously. Until the stem cells restore safe levels of blood cells, the patient will be hospitalized (3-4 weeks or longer).
- The first treatment will begin with high doses of carboplatin and VP-16 daily for 3 consecutive days followed by cyclophosphamide on the fourth and fifth days.
- Between 4-6 weeks after the start of the first course of high dose chemotherapy a second course consisting of melphalan and total body irradiation (TBI) will be given. Melphalan will be given daily for 3 consecutive days followed by TBI twice a day for 3 days.
- After each of the two high dose treatments outlined above, the stem cells that were previously harvested from the patient will be given back to them through a central line.
- Stem cells will be collected on 3 separate occasions following each of two cycles of chemotherapy. They will then be processed using a technique that attempts to separate stem cells from other cells, including any cancer cells that may be present.
- Bone marrow will also be harvested prior to the first transplant and will be used if blood counts do not recover after the stem cells are given.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
Who may qualify
- Ages
- Up to 19 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (10 listed)
- Previously untreated neuroblastoma Stage D > 365 days
- Previously untreated neuroblastoma Stage C at age > 365 days with n-myc amplification
- Previously untreated metastatic Ewings sarcoma/PNET
- Previously untreated non-rhabdomyosarcoma soft tissue sarcoma
- Confirmation of neuroblastoma at time of diagnosis by histologic specimen or with bone marrow specimen showing solid tumor. Confirmation of sarcoma by histologic specimen with evidence of metastatic disease be imaging study
Key exclusion criteria (2 listed)
- Previous malignancy requiring nonsurgical treatment or bone marrow transplant.
- Relapsed disease
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 |
|---|---|---|
| VincristineDescribed under detailed description | Drug | – |
| CyclophosphamideDescribed under detailed description | Drug | – |
| AdriamycinDescribed under detailed description | Drug | – |
| Etoposide (VP-16)Described under detailed description | Drug | – |
| CisplatinDescribed under detailed description | Drug | – |
| CarboplatinDescribed under detailed description | Drug | – |
| MelphalanDescribed under detailed description | Drug | – |
| IfosfamideDescribed under detailed description | Drug | – |
| G-CSF (granulocyte-colony stimulating factor)Described under detailed description | Drug | – |
| MesnaDescribed under detailed description | Drug | – |
Primary outcome
To determine the toxicity and feasibility of double dose chemo-radiotherapy with blood progenitor cell rescue in this patient population.
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 2 Nov 2009.
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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.
Lisa Diller, MD principal investigator
Dana-Farber Cancer Institute
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Vincristine, Cyclophosphamide and Adriamycin in people with Neuroblastoma and Ewings Sarcoma, run by Dana-Farber Cancer Institute 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 10 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 2 Nov 2009. 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.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
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?
To determine the toxicity and feasibility of double dose chemo-radiotherapy with blood progenitor cell rescue in this patient population. (time frame: 5 years). 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 2 Nov 2009.