Study Stopped
Slow Accrual.
Autologous Stem Cell Rescue With CD133+ Selected Cells in High-Risk Neuroblastoma
1 other identifier
interventional
3
1 country
1
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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for phase_2
Started Oct 2007
Longer than P75 for phase_2
1 active site
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
Study Start
First participant enrolled
October 1, 2007
CompletedFirst Submitted
Initial submission to the registry
October 2, 2007
CompletedFirst Posted
Study publicly available on registry
October 4, 2007
CompletedPrimary Completion
Last participant's last visit for primary outcome
September 5, 2012
CompletedStudy Completion
Last participant's last visit for all outcomes
September 5, 2012
CompletedResults Posted
Study results publicly available
May 10, 2019
CompletedJuly 26, 2019
July 1, 2019
4.9 years
October 2, 2007
June 23, 2016
July 10, 2019
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Engraftment Failure Rate
Engraftment Failure Rate is number of participants with engraftment failure out of total participants. Engraftment failure is defined as failure to achieve an absolute neutrophil count (ANC) \>500/ul by day 42, and has no evidence of donor chimerism on bone marrow examination.
Participant evaluation at Day 42, total study up to 3 Years
Secondary Outcomes (1)
Number of Participants With Device-related Toxicity Associated With Transplantation of CD133+ Cells
3 Years
Other Outcomes (1)
Number of Treated Participants With Engraftment Failure at Day 42
Participant evaluation at Day 42
Study Arms (1)
Transplantation CD133+ cells
EXPERIMENTALStem Cell Transplantation of CD133+ cells using the ClinicMACS in combination with Carboplatin + Etoposide + Melphalan
Interventions
Carboplatin by vein over 24 hours for 4 days, dosing as determined at day 1.
300 mg/m\^2 by vein over 24 hours for 4 days
70 mg/m\^2 Intravenous Bolus for 3 Days
Stem Cell Infusion (approximately 5x10\^8 TNC cells/kg CD133+ selected) on Day 0.
Device used to process the blood and separate the CD 133+ cells needed for transplantation
Eligibility Criteria
You may qualify if:
- 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.
- Pre-transplant modalities may include surgery, chemotherapy, or radiation therapy. Radiation must not include lung fields. Only patients in complete response (CR), or partial response (PR) at the primary site will be eligible.
- Any recurrent neuroblastoma with at least a partial response to salvage therapy.
- 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.
- No symptomatic pulmonary disease. forced expiratory volume in 1 second (FEV1), forced vital capacity (FVC), and Carbon Monoxide Diffusing Capacity (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.
- Adequate cardiac function as demonstrated by left ventricular ejection fraction \>/= 50% by echocardiogram.
- Adequate hepatic function as defined as serum glutamic-oxaloacetic transaminase, SGOT (AST) and serum glutamic-pyruvic transaminase, SGPT (ALT)\< 5 times upper limits of normal.
- All patients and/or their parents or legal guardians must sign a written informed consent.
- 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.
You may not qualify if:
- Patient is pregnant or breast-feeding.
- Active infection not controlled by antibiotics after seven days of therapy.
- Brain metastases.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (1)
UT MD Anderson Cancer Center
Houston, Texas, 77030, United States
Related Links
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Limitations and Caveats
Early termination leading to small numbers of subjects analyzed, rare patient population.
Results Point of Contact
- Title
- Laura L. Worth, MD / Professor, Pediatrics
- Organization
- University of Texas MD Anderson Cancer Center
Study Officials
- PRINCIPAL INVESTIGATOR
Laura L. Worth, MD, PhD
M.D. Anderson Cancer Center
Publication Agreements
- PI is Sponsor Employee
- Yes
- Restrictive Agreement
- No
Study Design
- Study Type
- interventional
- Phase
- phase 2
- Allocation
- NA
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SINGLE GROUP
- Sponsor Type
- OTHER
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
October 2, 2007
First Posted
October 4, 2007
Study Start
October 1, 2007
Primary Completion
September 5, 2012
Study Completion
September 5, 2012
Last Updated
July 26, 2019
Results First Posted
May 10, 2019
Record last verified: 2019-07