Autologous Stem Cell Rescue With CD133+ Selected Cells in High-Risk Neuroblastoma

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00539500
Recruitment Status : Terminated (Slow Accrual.)
First Posted : October 4, 2007
Last Update Posted : September 11, 2012
Information provided by (Responsible Party):
M.D. Anderson Cancer Center

Brief Summary:
The goal of this clinical research study is to learn how long it takes for certain types of transplanted stem cells to produce new blood cells. The safety of this treatment will also be studied. Finally, researchers want to learn if collecting the cells with the CliniMACS device can decrease the possibility of tumor cells contaminating (appearing in) the stem cells that are reinfused into participants.

Condition or disease Intervention/treatment Phase
Neuroblastoma Drug: Carboplatin Drug: Etoposide Drug: Melphalan Procedure: Stem Cell Infusion Device: ClinicMACS Phase 2 Phase 3

  Show Detailed Description

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 3 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Autologous Stem Cell Rescue With CD133+ Selected Hematopoietic Progenitor Cells in Patients With High-Risk Neuroblastoma
Study Start Date : October 2007
Actual Primary Completion Date : September 2012

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Neuroblastoma
Drug Information available for: Carboplatin
U.S. FDA Resources

Arm Intervention/treatment
Experimental: Transplantation CD133+ cells
Transplantation of CD133+ cells using the ClinicMACS in combination with Carboplatin + Etoposide + Melphalan
Drug: Carboplatin
Carboplatin by vein over 24 hours x 4 days, dosing as determined at day 1.
Other Name: Paraplatin®
Drug: Etoposide
300 mg/m^2 by vein over 24 Hours x 4 Days
Other Name: VePesid®
Drug: Melphalan
70 mg/m^2 Intravenous Bolus x 3 Days
Other Name: Alkeran
Procedure: Stem Cell Infusion
Stem Cell Infusion (approximately 5x10^8 TNC cells/kg CD133+ selected) on Day 0.
Device: ClinicMACS
Device used to process the blood and separate the CD 133+ cells needed for transplantation

Primary Outcome Measures :
  1. Engraftment Failure Rate [ Time Frame: 3 Years ]

Secondary Outcome Measures :
  1. Device-related toxicity associated with transplantation of CD133+ cells [ Time Frame: 3 Years ]

Information from the National Library of Medicine

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Ages Eligible for Study:   Child, Adult, Senior
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  1. Newly diagnosed high-risk Neuroblastoma defined as: a. INSS 2A/2B older then 365 days with MYCN amplified, unfavorable histology, and any ploidy. b. INSS Stage 3, older than 365 days with MYCN amplification and/or unfavorable histology. c. INSS Stage 4 or 4S, less than 365 days of age, with MYCN amplification d. INSS Stage 4, over 365, regardless of MYCN amplification or histology.
  2. Pre-transplant modalities may include surgery, chemotherapy, or radiation therapy. Radiation must not include lung fields. Only patients in CR, or PR at the primary site will be eligible.
  3. Any recurrent neuroblastoma with at least a partial response to salvage therapy.
  4. Lansky performance score greater than or equal to 50 for patients </= 16 years of age, or Zubrod performance status score of 0-2 for patients > 16 years of age.
  5. No symptomatic pulmonary disease. FEV1, FVC, and DLCO >/= 50% of expected corrected for hemoglobin. If unable to perform pulmonary function test (most children < 6 years of age), pulse oximetry >/= 92% on room air.
  6. Adequate cardiac function as demonstrated by left ventricular ejection fraction >/= 50% by echocardiogram.
  7. Adequate hepatic function as defined as SGOT (AST) and SGPT (ALT)< 5 X upper limits of normal.
  8. All patients and/or their parents or legal guardians must sign a written informed consent.
  9. Females of childbearing potential defined as not post-menopausal for 12 months or no previous surgical sterilization must have a negative urine pregnancy test within 30 days of registering. Patients will be informed of the risk of not using adequate contraception.

Exclusion Criteria:

  1. Patient is pregnant or breast-feeding.
  2. Active infection not controlled by antibiotics after seven days of therapy.
  3. Brain metastases.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT00539500

United States, Texas
UT MD Anderson Cancer Center
Houston, Texas, United States, 77030
Sponsors and Collaborators
M.D. Anderson Cancer Center
Principal Investigator: Laura L. Worth, MD, PhD M.D. Anderson Cancer Center

Additional Information:
Responsible Party: M.D. Anderson Cancer Center Identifier: NCT00539500     History of Changes
Other Study ID Numbers: 2006-0374
First Posted: October 4, 2007    Key Record Dates
Last Update Posted: September 11, 2012
Last Verified: September 2012

Keywords provided by M.D. Anderson Cancer Center:
Stem Cell Transplantation
Blood And Marrow Transplantation
Solid Tumors
CD133+ Cells

Additional relevant MeSH terms:
Neuroectodermal Tumors, Primitive, Peripheral
Neuroectodermal Tumors, Primitive
Neoplasms, Neuroepithelial
Neuroectodermal Tumors
Neoplasms, Germ Cell and Embryonal
Neoplasms by Histologic Type
Neoplasms, Glandular and Epithelial
Neoplasms, Nerve Tissue
Etoposide phosphate
Antineoplastic Agents
Antineoplastic Agents, Phytogenic
Topoisomerase II Inhibitors
Topoisomerase Inhibitors
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action
Antineoplastic Agents, Alkylating
Alkylating Agents
Myeloablative Agonists
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs