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Study Summary
No. 2006-0374:.......Blood And Marrow Transplantation; Pediatrics; Solid Tumors......Laura L. Worth......Pediatrics
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Study Summary Title
Study Summary
Number:
2006-0374
Study Title:Autologous Stem Cell Rescue with CD133+ Selected Hematopoietic Progenitor Cells in Patients with High-risk Neuroblastoma
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Physician New Patient Referral
Name:Laura L. WorthPatients Call:800-392-1611 (in U.S.A.) 713-792-6161 (outside U.S.A.)
Dept:PediatricsReferring MD
Call:
800-392-1611 (in U.S.A.) 713-792-6161 (outside U.S.A.)
Phone:713-792-6620
Contact us about clinical trials
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General Information
Disease Group:Blood And Marrow Transplantation
Pediatrics
Solid Tumors
Supported By:N/A
Phase of Study:Phase II/Phase IIIReturn
Visit:
Post discharge patients come 1-2 x/ week for about 60 days post infusion.
Yearly followup assess for disease recurrence includes CT scan of original
tumor site &/or metastatic disease, CBC, diff, plt, electrolytes & chem survey;
history & phys. exam
Treatment
Agents:
Carboplatin
CD133
Etoposide
Filgrastim
Melphalan
Home Care:GCSF
Treatment Loc:Only at MDACC
Estimated
Length of Stay
in Houston:
Treatment length is approximately 12 weeks from time of cell collection as an
outpatient to completion of high dose chemotherapy, cell infusion, and
engraftment.
Description/
Intervention:
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.
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Study Objectives / Outcomes
Primary Objectives:
1.1 To determine the engraftment failure rate.
1.2 To evaluate the device-related toxicity associated with transplantation of CD133+ cells in high risk neuroblastoma.

Secondary Objectives:
2.1 To evaluate the time to failure, one-year overall and disease-free survival.
2.2 To evaluate the efficiency of purging of CD133+ selection by immunohistochemistry, flow and/or RT-PCR for tumor specific transcripts.
2.3 To document ClinicMACS device performance as assessed by yield and purity of CD133+ in a clinical cohort.
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Study Status Information
Study Activation / Registration Date:10/01/2007
IRB Review and Approval Date:12/08/2006
Study Type:Phase Ii Or Phase I/Ii
Recruitment Status:Terminated
Projected Accrual:N/A
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Enrollment Eligibility
If you do not meet the enrollment eligibility, there may be other treatment options for you. Please Contact the Referral Office for more information.

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.

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Links
Registration Number: NCT00539500
Study Information on Clinical Trials Registry (clinicaltrials.gov)

Other Links:
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Results


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