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Stem Cell Transplantation in Patients With High-Risk and Recurrent Pediatric Sarcomas

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Kristin Baird, M.D., National Institutes of Health Clinical Center (CC)
ClinicalTrials.gov Identifier:
NCT00043979
First received: August 15, 2002
Last updated: September 12, 2013
Last verified: September 2013
Results First Received: April 3, 2013  
Study Type: Interventional
Study Design: Allocation: Non-Randomized;   Endpoint Classification: Safety/Efficacy Study;   Intervention Model: Parallel Assignment;   Masking: Open Label;   Primary Purpose: Treatment
Condition: Sarcoma
Interventions: Drug: F-18 Fluorodeoxyglucose
Biological: therapeutic allogeneic lymphocytes
Drug: cyclophosphamide
Drug: cyclosporine
Drug: doxorubicin hydrochloride
Drug: etoposide
Drug: fludarabine phosphate
Drug: melphalan
Drug: prednisone
Drug: sirolimus
Drug: tacrolimus
Drug: vincristine sulfate
Procedure: peripheral blood stem cell transplantation
Drug: Filgrastim
Procedure: Peripheral Blood Stem Cell donation

  Participant Flow
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Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
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Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
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Reporting Groups
  Description
Sibling Donors Donors (n = 30) were matched first degree relatives who were eligible to donate peripheral blood stem cells.In period 1 they donated cells.
Recipients Cyclosporine GVHD Prophylaxis

In period 1 recipients received EPOCH-F/chemotherapy. In period 2 recipients received peripheral blood stem transplant.

Post transplant recipients received cyclosporine for GVHD prophylaxis.

Recipients Tacrolimus /Sirolimus Prophylaxis

In period 1 recipients received EPOCH-F/chemotherapy. In period 2 recipients received peripheral blood stem transplant.

Post transplant recipients received tacrolimus & sirolimus for GVHD prophylaxis.


Participant Flow for 2 periods

Period 1:   Period 1
    Sibling Donors   Recipients Cyclosporine GVHD Prophylaxis   Recipients Tacrolimus /Sirolimus Prophylaxis
STARTED   30   17   13 
COMPLETED   23   13   10 
NOT COMPLETED   7   4   3 
recipient with progressive disease                7                4                3 

Period 2:   Period 2
    Sibling Donors   Recipients Cyclosporine GVHD Prophylaxis   Recipients Tacrolimus /Sirolimus Prophylaxis
STARTED   0   13   10 
COMPLETED   0   13   10 
NOT COMPLETED   0   0   0 



  Baseline Characteristics
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Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
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Reporting Groups
  Description
Arm 1-Sibling Donors Donors (n=30) were matched first degree relatives who were eligible to donate peripheral blood stem cells.
Arm 2-Recipients Recipients (n=30) were enrolled to receive peripheral blood stem cells (PBSC) and receive either cyclosporine or tacrolimus and sirolimus for graft versus host disease (GVHD) prophylaxis.
Total Total of all reporting groups

Baseline Measures
   Arm 1-Sibling Donors   Arm 2-Recipients   Total 
Overall Participants Analyzed 
[Units: Participants]
 30   30   60 
Age 
[Units: Participants]
     
<=18 years   17   14   31 
Between 18 and 65 years   13   16   29 
>=65 years   0   0   0 
Age 
[Units: Years]
Mean (Standard Deviation)
 21.12  (10.9)   19.98  (5.8)   20.55  (8.35) 
Gender 
[Units: Participants]
     
Female   19   10   29 
Male   11   20   31 
Ethnicity (NIH/OMB) 
[Units: Participants]
     
Hispanic or Latino   1   1   2 
Not Hispanic or Latino   29   29   58 
Unknown or Not Reported   0   0   0 
Race (NIH/OMB) 
[Units: Participants]
     
American Indian or Alaska Native   0   0   0 
Asian   0   0   0 
Native Hawaiian or Other Pacific Islander   0   0   0 
Black or African American   0   0   0 
White   30   30   60 
More than one race   0   0   0 
Unknown or Not Reported   0   0   0 
Region of Enrollment 
[Units: Participants]
     
United States   30   30   60 


  Outcome Measures
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1.  Primary:   Number of Participants With Engraftment   [ Time Frame: 100 days ]

2.  Primary:   Toxicity   [ Time Frame: 16.5 months ]

3.  Secondary:   Number of Participants With Acute and Chronic GVHD   [ Time Frame: up to 5 years or death ]


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats
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Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
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  More Information
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Certain Agreements:  
Principal Investigators are NOT employed by the organization sponsoring the study.
There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.


Results Point of Contact:  
Name/Title: Dr. Kristin Baird
Organization: National Cancer Institute, National Institutes of Health
phone: 301-451-0391
e-mail: kbaird@mail.nih.gov


Publications of Results:
Other Publications:

Responsible Party: Kristin Baird, M.D., National Institutes of Health Clinical Center (CC)
ClinicalTrials.gov Identifier: NCT00043979     History of Changes
Obsolete Identifiers: NCT00047372
Other Study ID Numbers: 020259
02-C-0259
Study First Received: August 15, 2002
Results First Received: April 3, 2013
Last Updated: September 12, 2013
Health Authority: United States: Federal Government