High Risk Solid TumorsNCT00966498
Peripheral Blood Stem Cell Transplant (PBSCT) in Children With High Risk or Recurrent Solid Tumors
A study testing Peripheral Blood Stem Cell Transplant in people with High Risk Solid Tumors and Recurrent Solid Tumors, run by Emory University at 2 sites in United States.
Trial summary
Before the transplant, the patient will have a pre-transplant evaluation. This will help find out whether there are health problems that will prevent the transplant. It also provides "baseline" tests that will be used later to see whether or not organs have gotten better or worse after the transplant. Read more about this study
- Who is it for?
- Ages Up to 35 years
- Conditions the study is for
- High Risk Solid Tumors, Recurrent Solid Tumors
- What is being tested?
- Peripheral Blood Stem Cell Transplant
- Type of treatment
- Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 3 participants
- Where
- 2 sitesUnited States
- Study dates
- May 2003to Jul 2011
- 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 35 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Patients diagnosed with high risk recurrent or refractory solid tumors following initial chemotherapy
- Tumor must be sensitive to chemotherapy (maximum 8 cycles) and/or radiation defined as a greater than 50% reduction in size of the primary and/or metastatic sites.
- Patients who are under 35 years of age.
- Patients with a life expectancy of at least 8 weeks and performance status (Karnofsky or Lansky score) of at least 70%.
- Patients who are acceptable candidates for peripheral blood stem cell transplantation based on their pre-transplant evaluation.
Who cannot join
- Patients will not be excluded based on sex, race.
- Patients with central nervous system tumors are not eligible for this protocol.
- Patients have significant functional deficits in major organs which would interfere with successful outcome following PBSCT.
- Patients who have been treated for infections must have appropriate responses as documented by negative cultures and/or a normal radiographic examination.
- Patients may not have active CNS disease or marrow involvement with the tumor at the time of transplant.
- Patients with disease progression after tandem PBSC #1 will not be eligible for tandem PBSC #2.
- Patients will be excluded if they are women of childbearing potential who are currently pregnant (HCG+) or who are not practicing adequate contraception.
- Patients who have had a previous stem cell transplant.
Trial locations and contacts
2 sites in 1 country.
United States2 sites
Children's Healthcare of Atlanta
Atlanta, Georgia, 30322
Emory University
Atlanta, Georgia, 30322
About this study
Official title: Peripheral Blood Stem Cell Transplantation in Children With High-Risk or Recurrent Solid Tumors
Before the transplant, the patient will have a pre-transplant evaluation. This will help find out whether there are health problems that will prevent the transplant. It also provides "baseline" tests that will be used later to see whether or not organs have gotten better or worse after the transplant. Prior to the stem cell collection, the patient will get chemotherapy to help try to put him/her in remission and to push more stem cells into the peripheral blood (mobilization). The study doctor will decide which chemotherapy will be used for this part of the study. Once mobilization is completed, the peripheral blood stem cell collection (apheresis) will be done in the clinic. The apheresis machine will draw blood out of the central line. The blood then passes through the apheresis machine and the stem cells are separated out. The remaining blood is sent back through the central line. If the investigators are unable to collect enough peripheral blood stem cells, a bone marrow harvest may be necessary to collect more stem cells.
The patient will then be admitted to the hospital for the first transplant. He/she will get Thiotepa and Cyclophosphamide. Then the patient will be given back the cells that were collected. The cells are given in the same manner as a blood transfusion. The patient will be kept in the hospital until he/she is stable and blood counts are increasing. Approximately 6 to 8 weeks after Day 0 of the 1st transplant, the patient will be admitted for the second transplant. At this time, he/she will get Busulfan and Melphalan and then the collected cells will be given back. The patient will be kept in the hospital until he/she is stable and blood counts are increasing. Frequent clinic follow-up is required.
This study is open to patients who are less then 21 years of age with refractory or relapsed high-risk, solid tumors, excluding neuroblastoma (there is a cooperative group trial for these patients). Patients will be identified by the Transplant team and eligibility will be verified by a member of the clinical research team. Patients will be cared for by members of the Transplant team and various other subspecialty physicians.
Read the detailed description
The optimal treatment for refractory (disease won't go away with standard treatment) or relapsed (disease comes back after going away) solid tumors is unknown. Recent studies have shown some benefit to tandem peripheral blood stem cell transplantation (PBSCT). In a tandem transplant, two transplants are done, one after the other. To do a PBSCT, stem cells ("mother cells" that can become any other type of cell) are first collected from the patient's circulating blood. The patient then undergoes high-dose chemotherapy called the preparative regimen. The preparative regimen destroys not only the tumor cells, but it also destroys all of the normal blood making cells. The collected cells are then given back to the patient to "rescue" the patient from the devastating effects of the preparative regimen. By using a stem cell rescue will are able to give much higher doses of chemotherapy than we would be able to give without the stem cell rescue. To make sure that all of the tumor cells are destroyed, patients in this study will undergo two separate transplants using two different preparative regimens. The preparative regimens will use the best agents that have been found to work against recurrent and refractory solid tumors: Busulfan, Thiotepa, Cyclophosphamide and Melphalan.
- Conditions
- High Risk Solid Tumors, Recurrent Solid Tumors
- Being tested
- Peripheral Blood Stem Cell Transplant
- Keywords
- malignancy, solid tumor, recurrent, high risk
- Medical subject headings
- Neoplasms, Recurrence
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Peripheral Blood Stem Cell TransplantProcedure or surgery
Peripheral Blood Stem Cell Transplant
| Study group | Role | What they receive |
|---|---|---|
| 1 | Experimental | This study is a single-arm, non-randomized trial. Peripheral blood stem cells will be harvested by mobilization with chemotherapy followed by G-CSF. Following adequate peripheral blood stem cell collection, the patients would be transplanted using the conditioning regimen consisting of thiotepa and cyclophosphamide (tandem one) and busulfan and melphalan (tandem two). They will receive G-CSF post transplant. There will be 6-8 weeks interval between tandem transplants. All patients would be carefully observed for any toxicity, transplant-related complications, relapse and disease-free survival. Receives: Procedure: Peripheral Blood Stem Cell Transplant |
What is being measured
The primary outcome is the main question the study is designed to answer.
To determine the feasibility and toxicity of tandem high dose chemotherapy and peripheral blood stem cells rescue in patients with high-risk or recurrent solid tumors.
Measured: 1 year after last patient is enrolled
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Aug 2009 (estimated)
- Study startMay 2003
- Primary completionJul 2011
- Study completionJul 2011
Why it stopped: poor enrollment
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for High Risk Solid TumorsStudies of the same condition, open and under way.
- More trials run by Emory UniversityThe 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
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 3 participants (actual)
Sponsor and investigators
- Lead sponsor
- Emory University
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Kuang-Yueh Chiang, Emory University
- Investigators
- Kuang-Yueh Chiang, MD, Principal investigator, Emory University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00966498
- Sponsor's protocol ID
- 1023-2003
- Other IDs
- PBSCT for ST
- Status last verified
- Dec 2013
- First submitted
- 11 Aug 2009
- Last updated
- 11 Dec 2013
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 11 Dec 2013. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Peripheral Blood Stem Cell Transplantation in Children With High-Risk or Recurrent Solid Tumors
- ClinicalTrials.gov ID
- NCT00966498
- Acronym / sponsor ID
- 1023-2003
- Conditions
- High Risk Solid Tumors, Recurrent Solid Tumors
- Keywords
- malignancy, solid tumor, recurrent, high risk
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: poor enrollment
- Enrolment
- 3 participants (actual)
- Lead sponsor
- Emory UniversityUniversity, hospital or other
- Study start
- May 2003
- Primary completion
- Jul 2011 (actual)
- Study completion
- Jul 2011 (actual)
- First posted
- 27 Aug 2009 (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.
Before the transplant, the patient will have a pre-transplant evaluation. This will help find out whether there are health problems that will prevent the transplant. It also provides "baseline" tests that will be used later to see whether or not organs have gotten better or worse after the transplant. Prior to the stem cell collection, the patient will get chemotherapy to help try to put him/her in remission and to push more stem cells into the peripheral blood (mobilization). The study doctor will decide which chemotherapy will be used for this part of the study. Once mobilization is completed, the peripheral blood stem cell collection (apheresis) will be done in the clinic. The apheresis machine will draw blood out of the central line. The blood then passes through the apheresis machine and the stem cells are separated out. The remaining blood is sent back through the central line. If the investigators are unable to collect enough peripheral blood stem cells, a bone marrow harvest may be necessary to collect more stem cells.
The patient will then be admitted to the hospital for the first transplant. He/she will get Thiotepa and Cyclophosphamide. Then the patient will be given back the cells that were collected. The cells are given in the same manner as a blood transfusion. The patient will be kept in the hospital until he/she is stable and blood counts are increasing. Approximately 6 to 8 weeks after Day 0 of the 1st transplant, the patient will be admitted for the second transplant. At this time, he/she will get Busulfan and Melphalan and then the collected cells will be given back. The patient will be kept in the hospital until he/she is stable and blood counts are increasing. Frequent clinic follow-up is required.
This study is open to patients who are less then 21 years of age with refractory or relapsed high-risk, solid tumors, excluding neuroblastoma (there is a cooperative group trial for these patients). Patients will be identified by the Transplant team and eligibility will be verified by a member of the clinical research team. Patients will be cared for by members of the Transplant team and various other subspecialty physicians.
Detailed description
The optimal treatment for refractory (disease won't go away with standard treatment) or relapsed (disease comes back after going away) solid tumors is unknown. Recent studies have shown some benefit to tandem peripheral blood stem cell transplantation (PBSCT). In a tandem transplant, two transplants are done, one after the other. To do a PBSCT, stem cells ("mother cells" that can become any other type of cell) are first collected from the patient's circulating blood. The patient then undergoes high-dose chemotherapy called the preparative regimen. The preparative regimen destroys not only the tumor cells, but it also destroys all of the normal blood making cells. The collected cells are then given back to the patient to "rescue" the patient from the devastating effects of the preparative regimen. By using a stem cell rescue will are able to give much higher doses of chemotherapy than we would be able to give without the stem cell rescue. To make sure that all of the tumor cells are destroyed, patients in this study will undergo two separate transplants using two different preparative regimens. The preparative regimens will use the best agents that have been found to work against recurrent and refractory solid tumors: Busulfan, Thiotepa, Cyclophosphamide and Melphalan.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| 1 | Experimental | Procedure: Peripheral Blood Stem Cell TransplantThis study is a single-arm, non-randomized trial. Peripheral blood stem cells will be harvested by mobilization with chemotherapy followed by G-CSF. Following adequate peripheral blood stem cell collection, the patients would be transplanted using the conditioning regimen consisting of thiotepa and cyclophosphamide (tandem one) and busulfan and melphalan (tandem two). They will receive G-CSF post transplant. There will be 6-8 weeks interval between tandem transplants. All patients would be carefully observed for any toxicity, transplant-related complications, relapse and disease-free survival. |
Who may qualify
- Ages
- Up to 35 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (5 listed)
- Patients diagnosed with high risk recurrent or refractory solid tumors following initial chemotherapy
- Tumor must be sensitive to chemotherapy (maximum 8 cycles) and/or radiation defined as a greater than 50% reduction in size of the primary and/or metastatic sites.
- Patients who are under 35 years of age.
- Patients with a life expectancy of at least 8 weeks and performance status (Karnofsky or Lansky score) of at least 70%.
- Patients who are acceptable candidates for peripheral blood stem cell transplantation based on their pre-transplant evaluation.
Key exclusion criteria (8 listed)
- Patients will not be excluded based on sex, race.
- Patients with central nervous system tumors are not eligible for this protocol.
- Patients have significant functional deficits in major organs which would interfere with successful outcome following PBSCT.
- Patients who have been treated for infections must have appropriate responses as documented by negative cultures and/or a normal radiographic examination.
- Patients may not have active CNS disease or marrow involvement with the tumor at the time of transplant.
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 |
|---|---|---|
| Peripheral Blood Stem Cell TransplantPeripheral Blood Stem Cell Transplant | Procedure or surgery | 1 |
Primary outcome
To determine the feasibility and toxicity of tandem high dose chemotherapy and peripheral blood stem cells rescue in patients with high-risk or recurrent solid tumors.
Time frame: 1 year after last patient is enrolled
Results and safety
The study was stopped early (reason given: poor enrollment). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 11 Dec 2013.
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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.
Kuang-Yueh Chiang, MD principal investigator
Emory University
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Peripheral Blood Stem Cell Transplant in people with High Risk Solid Tumors and Recurrent Solid Tumors, run by Emory University at 2 sites 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 5 inclusion and 8 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: stopped early, as of the sponsor's last update on 11 Dec 2013. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites 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 was stopped early (reason given: poor enrollment). Results are not available in the source record.
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 feasibility and toxicity of tandem high dose chemotherapy and peripheral blood stem cells rescue in patients with high-risk or recurrent solid tumors. (time frame: 1 year after last patient is enrolled). 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 Dec 2013.