NeuroblastomaNCT00152126
Chemotherapy With CD133+ Select Autologous Hematopoietic Stem Cells for Children With Solid Tumors and Lymphomas
A study testing Stem Cell Transplantation and Busulfan, Melphalan in people with Neuroblastoma and Central Nervous System Tumors, run by St. Jude Children's Research Hospital at 1 site in United States.
Trial summary
Studies have provided evidence that residual microscopic malignant cells in autologous bone marrow or blood stem cell grafts can contribute to posttransplant relapse. Researchers are currently exploring different methods in an attempt to purify or "purge" the stem cell product to minimize the risk of tumor… Read more about this study
- Who is it for?
- Ages Up to 25 years
- Conditions the study is for
- Neuroblastoma, Central Nervous System Tumors, Lymphomas
- What is being tested?
- Stem Cell Transplantation and Busulfan, Melphalan
- Type of treatment
- Injection or infusion, Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 26 participants
- Where
- 1 siteUnited States
- Study dates
- Aug 2003to Feb 2009
- Phase
- Interventional
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 25 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Eligibility will be determined separately for Part I and Part II of this study:
- Part I ( Part I Eligibility criteria (eligibility for undergoing apheresis procedure)
- Age ≤ 25 years at initial diagnosis.
- Must have one of the following diagnoses:
- High risk neuroblastoma
- Metastatic or recurrent retinoblastoma
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- High risk rain tumors
- Recurrent or refractory Hodgkin disease
- Recurrent or advanced stage Wilms tumor
- Recurrent or metastatic sarcomas
- Recurrent or refractory non-Hodgkin lymphoma
- Desmoplastic small round cell tumor.
- Lansky or Karnofsky Performance Score ≥ 70.
- Creatinine ≤ 2.0 mg/dl.
- Direct bilirubin ≤ 2.0 mg/dl.
- SGPT ≤ 2 x upper limit of normal
- HIV testing
- Negative pregnancy test
- Patients with significant prior radiation therapy to the liver will be excluded.
- Part II eligibility criteria (criteria for transplantation of CD133 select stem cell product)
- Successfully completed Part I of protocol treatment plan and has the following available:
- Stored autologous bone marrow or peripheral blood stem cells (i.e. 2 x 106 unselected CD34+ cells/ kg PBSC or 1 x 106 CD34+ cells/ kg BM) for back up.
- Stored autologous bone marrow or peripheral blood stem cells (2 x 106 CD133+ cells/ kg PBSC or 2 x 106 CD133+ cells/ kg BM) for infusion.
- Forced vital capacity greater than or equal to 40% normal or pulse oximetry greater than or equal to 92% on room air.
- Lansky or Karnofsky Performance Score ≥ 70.
- Creatinine ≤ 2.0 mg/dl.
- Direct bilirubin ≤ 2.0 mg/dl.
- SGPT ≤ 2 x upper limit of normal
- Negative pregnancy test
- Patients with significant prior radiation therapy (in opinion of the PI) to the liver will be excluded.
Who cannot join
None listed.
Trial locations and contacts
1 site in 1 country.
United States1 site
St. Jude Children's Research Hospital
Memphis, Tennessee, 38105
About this study
Official title: Busulfan and Melphalan With Autologous Hematopoietic Stem Cell Support With Positively-Selected CD133+ Hematopoietic Cells for Children With High Risk Solid Tumors and Lymphomas
Studies have provided evidence that residual microscopic malignant cells in autologous bone marrow or blood stem cell grafts can contribute to posttransplant relapse. Researchers are currently exploring different methods in an attempt to purify or "purge" the stem cell product to minimize the risk of tumor contamination.
The CD133+ antigen is a protein contained on or "expressed" on numerous cells in the human body including specific hematopoietic progenitor (blood forming) cells. However, this antigen is not expressed on certain cancer cells including neuroblastoma. A technique using the investigational CliniMACS cell sorting device has been developed in an effort to filter out only those stem cells that express this CD133+ antigen in order to infuse a hematopoietic stem cell product with no tumor contamination potential.
The primary objective of this study is to establish safety of treating patients with a high dose chemotherapy regimen of Busulfan and Melphalan followed by autologous CD133+ hematopoietic stem cell support. Transplants recipients are expected to achieve engraftment as defined by an absolute neutrophil count of greater than or equal to 500/mm3 for three consecutive days by day 42-post infusion. Thus, safety of the treatment plan will be evaluated in terms of failure to engraft by this specific time period.
Read the detailed description
Secondary objectives for this protocol include the following:
- To describe CD133+ graft content post-selection and to describe the yield and purity of CD133+ content of the graft obtained.
- To describe the negative selection efficiency of this strategy by assessing the processed product for tumor specific markers, when applicable.
- To characterize the proliferation of clonal progeny of CD133+ cells.
- To characterize lymphocyte and hematopoietic reconstitution (including the kinetics of platelet engraftment) in these patients.
- To estimate one-year disease-free and overall survival in these transplant recipients.
- Conditions
- Neuroblastoma, Central Nervous System Tumors, Lymphomas, Wilms Tumor
- Being tested
- Stem Cell Transplantation, Busulfan, Melphalan
- Keywords
- Autologous stem cell transplantation, CD133 cell selection, CliniMACS device, Tumor marker, Tumor purging
- Medical subject headings
- Neuroblastoma, Central Nervous System Neoplasms, Lymphoma, Wilms Tumor
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Stem Cell TransplantationProcedure or surgery
Autologous stem cell transplantation
Busulfan, MelphalanDrug
Also called Autologous stem cell transplant, CD133+ antigen specific selection, Apheresis
Transplant recipients will receive high dose Busulfan and Melphalan followed by autologous CD133+ antigen specific hematopoietic stem cell infusion. The autologous graft product will be selected using the investigational CliniMACS device.
| Study group | Role | What they receive |
|---|---|---|
| 1 | Other | Not described Receives: Procedure: Stem Cell Transplantation; Drug: Busulfan, Melphalan |
What is being measured
The primary outcome is the main question the study is designed to answer.
To determine the safety of the treatment plan using Busulfan and Melphalan followed by infusion of CD133+ selected hematopoietic cells in patients with high-risk malignancies.
Measured: August 2005
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Sep 2005 (estimated)
- Study startAug 2003
- Primary completionAug 2005
- 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 St. Jude Children's Research HospitalThe 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
- 26 participants (actual)
Sponsor and investigators
- Lead sponsor
- St. Jude Children's Research Hospital
- Sponsor type
- University, hospital or other
- Collaborators
- University of Miami
- Investigators
- Gregory Hale, M.D., Principal investigator, St. Jude Children's Research Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00152126
- Sponsor's protocol ID
- ST133
- Status last verified
- Feb 2009
- First submitted
- 7 Sep 2005
- Last updated
- 13 Feb 2009
- Results posted
- No
- Data monitoring committee
- No