Ewing's SarcomaPhase 1/2NCT00179816
Tandem Peripheral Blood Stem Cell (PBSC) Rescue for High Risk Solid Tumors
A Phase 1/2 study testing High-Dose Chemotherapy with Tandem PBSC Rescue. in people with Ewing's Sarcoma and Soft Tissue Sarcoma, run by Ann & Robert H Lurie Children's Hospital of Chicago at 1 site in United States.
Trial summary
This study uses a double autologous peripheral blood stem cell rescue (PBSC) following dose-intensive chemotherapy for the treatment of high-risk pediatric solid tumors. Read more about this study
- Who is it for?
- Ages Up to 21 years
- Conditions the study is for
- Ewing's Sarcoma, Soft Tissue Sarcoma, Hepatoblastoma
- What is being tested?
- High-Dose Chemotherapy with Tandem PBSC Rescue.
- How many people
- 12 participants
- Where
- 1 siteUnited States
- Study dates
- Apr 1999to Sep 2012
- Phase
- Phase 1/2
- 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 21 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Malignant Diseases:
- Ewing's sarcoma/PNET:
- CR1 - Metastatic disease at diagnosis, tumor volume > 100 ml, pelvic bone primary
- CR2 - Locally recurrent disease
- Soft tissue sarcoma
- CR1 - Metastatic disease at diagnosis or locally advanced disease where local control is suboptimal (i.e., inability to provide radiation therapy due to extent of disease).
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- CR2 - Locally recurrent disease (VGPR2 acceptable)
- Hepatoblastoma:
- VGPR1 - Patients with metastatic disease at diagnosis who have a persistently elevated alpha FP, or unresectable primary as a way of converting to resectable.
- CR2/VGPR2
- Hodgkin's Disease:
- VGPR1 - Progression on primary therapy/Refractory disease
- CR2/VGPR2
- Germ Cell Tumor:
- CR2/VGPR2 - recurrent disease
- Wilms Tumor:
- CR2/VGPR2 - recurrent disease
- IRB approved signed written informed consent by patient and/or their legally authorized guardian.
- Patients 21 years of age or younger at initial diagnosis, with older patients considered individually for primary pediatric disease diagnosis.
- Adequate central venous access (double lumen CVL or 2 single lumen PCVC).
- Adequate PBSC harvests with a minimum of 2.0 x 108 MNC/kg available for each PBSC rescue.
- Organ Function:
- Platelets > 50,000/ml
- SGOT < 10 x upper limits of normal
- Creatinine < 1.5 x normal baseline
- Normal cardiac function in accordance with institutional policies
- Normal pulmonary function in accordance with institutional policies.
- Physiologic status:
- No active infections
- Adequate performance status as measured by Karnofsky (> 70%) or Lansky scale (> 60%) as appropriate for age.
- Bone Marrow Status
- No evidence of morphologic involvement with tumor at the time of transplant
- Off Study Criteria:
- Severe toxicity. Contact the Study Coordinator immediately and complete Adverse Reaction Form.
- Disease progression or relapse prior to PBSC #1 or between PBSC rescue # 1 and #2.
- Inability to collect adequate numbers of PBSC for successful transplantation.
- Patient or parent/guardian refusal to remain on study.
Who cannot join
None listed.
Trial locations and contacts
1 site in 1 country.
United States1 site, 1 recruiting
- Recruiting
Children's Memorial Hospital
Chicago, Illinois, 60614
About this study
Official title: High-Dose Chemotherapy With Tandem Peripheral Blood Stem Cell (PBSC) Rescue for the Treatment of High-Risk Pediatric Solid Tumors.
This study uses a double autologous peripheral blood stem cell rescue (PBSC) following dose-intensive chemotherapy for the treatment of high-risk pediatric solid tumors.
Read the detailed description
Significant advances have been made in recent years in the treatment of solid tumors of childhood. However, much of the improvement in survival has been made in low stage and localized disease. Of significance is the fact that the improvements have come in up-front remission rates without translation into significantly high event-free survival(EFS) or overall survival (OS). This is despite the fact that these tumors as a whole are largely chemotherapy responsive.
Recent advances in the understanding of the biology of hematopoeitic stem cells have driven the design of treatment regimens that allow for dose intensification without unacceptable hematologic toxicity. Protocol development has focused on active agents that have a broad range between hematologic and non-hematologic toxicities. This study uses a double autologous peripheral blood stem cell rescue (PBSC) following dose-intensive chemotherapy for the treatment of high-risk pediatric solid tumors. This study utilizes PBSC to limit the risk of tumor cell contamination while retaining prompt hematologic recovery from these highly intensified treatments.
- Being tested
- High-Dose Chemotherapy with Tandem PBSC Rescue.
- Keywords
- wilm's tumor, stem cell transplantation, solid tumor
- Medical subject headings
- Sarcoma, Ewing, Sarcoma, Hepatoblastoma, Hodgkin Disease, Neoplasms, Germ Cell and Embryonal, Wilms Tumor
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
High-Dose Chemotherapy with Tandem PBSC Rescue.Drug
Also called Cyclophosphamide call also be referred to as Cytoxan
Patients on this study will undergo a tandem Peripheral Blood Stem Cell Rescue following high-dose chemotherapy. The first Peripheral Blood Stem Cell Rescue will consist of Etoposide, Carboplatin, Cyclophosphamide, and Mesna. Once the patient recovers, the patient will be evaluated again and will then undergo a second stem cell transplant consisting of the chemotherapy drugs; Melphalan, Cyclophosphamide, and Mesna.
What is being measured
The primary outcome is the main question the study is designed to answer.
Determine the feasibility and toxicity of tandem PBSC rescue following high dose chemotherapy as consolidation in pediatric patients with high risk solid tumors.
Measured: annually
Secondary outcomes (2)
Evaluate length of remission and long term disease free survival in chemotherapy responsive high-risk pediatric solid tumor patients treated using this approach.
Measured: Annually
Evaluate correlation between cell dose and time to engraftment in high-risk pediatric solid tumor patients treated using this approach.
Measured: Time to engraftment
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Sep 2005 (estimated)
- Study startApr 1999
- Primary completionSep 2012 (estimated)
- Study completionSep 2012 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Ewing's SarcomaStudies of the same condition, open and under way.
- 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 1/2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 12 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Ann & Robert H Lurie Children's Hospital of Chicago
- Sponsor type
- University, hospital or other
- Investigators
- Morris Kletzel, M.D., Principal investigator, Ann & Robert H Lurie Children's Hospital of Chicago
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00179816
- Sponsor's protocol ID
- BMT 0499 Solid
- Status last verified
- Oct 2010
- First submitted
- 10 Sep 2005
- Last updated
- 11 Oct 2010
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 11 Oct 2010. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- High-Dose Chemotherapy With Tandem Peripheral Blood Stem Cell (PBSC) Rescue for the Treatment of High-Risk Pediatric Solid Tumors.
- ClinicalTrials.gov ID
- NCT00179816
- Acronym / sponsor ID
- BMT 0499 Solid
- Keywords
- wilm's tumor, stem cell transplantation, solid tumor
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 12 participants (estimated)
- Lead sponsor
- Ann & Robert H Lurie Children's Hospital of ChicagoUniversity, hospital or other
- Study start
- Apr 1999
- Primary completion
- Sep 2012 (estimated)
- Study completion
- Sep 2012 (estimated)
- First posted
- 16 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.
This study uses a double autologous peripheral blood stem cell rescue (PBSC) following dose-intensive chemotherapy for the treatment of high-risk pediatric solid tumors.
Detailed description
Significant advances have been made in recent years in the treatment of solid tumors of childhood. However, much of the improvement in survival has been made in low stage and localized disease. Of significance is the fact that the improvements have come in up-front remission rates without translation into significantly high event-free survival(EFS) or overall survival (OS). This is despite the fact that these tumors as a whole are largely chemotherapy responsive.
Recent advances in the understanding of the biology of hematopoeitic stem cells have driven the design of treatment regimens that allow for dose intensification without unacceptable hematologic toxicity. Protocol development has focused on active agents that have a broad range between hematologic and non-hematologic toxicities. This study uses a double autologous peripheral blood stem cell rescue (PBSC) following dose-intensive chemotherapy for the treatment of high-risk pediatric solid tumors. This study utilizes PBSC to limit the risk of tumor cell contamination while retaining prompt hematologic recovery from these highly intensified treatments.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
Who may qualify
- Ages
- Up to 21 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (37 listed)
- Malignant Diseases:
- IRB approved signed written informed consent by patient and/or their legally authorized guardian.
- Patients 21 years of age or younger at initial diagnosis, with older patients considered individually for primary pediatric disease diagnosis.
- Adequate central venous access (double lumen CVL or 2 single lumen PCVC).
- Adequate PBSC harvests with a minimum of 2.0 x 108 MNC/kg available for each PBSC rescue.
Key exclusion criteria (0 listed)
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 |
|---|---|---|
| High-Dose Chemotherapy with Tandem PBSC Rescue.Also: Cyclophosphamide call also be referred to as CytoxanPatients on this study will undergo a tandem Peripheral Blood Stem Cell Rescue following high-dose chemotherapy. The first Peripheral Blood Stem Cell Rescue will consist of Etoposide, Carboplatin, Cyclophosphamide, and Mesna. Once the patient recovers, the patient will be evaluated again and will then undergo a second stem cell transplant consisting of the chemotherapy drugs; Melphalan, Cyclophosphamide, and Mesna. | Drug | – |
Primary outcome
Determine the feasibility and toxicity of tandem PBSC rescue following high dose chemotherapy as consolidation in pediatric patients with high risk solid tumors.
Time frame: annually
2 secondary outcomes
Evaluate length of remission and long term disease free survival in chemotherapy responsive high-risk pediatric solid tumor patients treated using this approach.
Time frame: Annually
Evaluate correlation between cell dose and time to engraftment in high-risk pediatric solid tumor patients treated using this approach.
Time frame: Time to engraftment
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 11 Oct 2010.
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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.
Morris Kletzel, M.D. principal investigator
Ann & Robert H Lurie Children's Hospital of Chicago
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing High-Dose Chemotherapy with Tandem PBSC Rescue. in people with Ewing's Sarcoma and Soft Tissue Sarcoma, run by Ann & Robert H Lurie Children's Hospital of Chicago 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 37 inclusion and 0 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: status not confirmed, as of the sponsor's last update on 11 Oct 2010. 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 combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
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?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Morris Kletzel, M.D. (773.880.4000 ext. 4564, mkletzel@northwestern.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Determine the feasibility and toxicity of tandem PBSC rescue following high dose chemotherapy as consolidation in pediatric patients with high risk solid tumors. (time frame: annually). 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 11 Oct 2010; AzTrials last refreshed its copy on 6 Nov 2026.