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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):
Terry Fry, M.D., National Institutes of Health Clinical Center (CC)
ClinicalTrials.gov Identifier:
NCT00043979
First received: August 15, 2002
Last updated: April 11, 2017
Last verified: April 2017
Results First Received: April 3, 2013  
Study Type: Interventional
Study Design: Allocation: Non-Randomized;   Intervention Model: Parallel Assignment;   Masking: No masking;   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
  Hide Participant Flow

Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
No text entered.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
No text entered.

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
  Hide Baseline Characteristics

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.
No text entered.

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]
Count of Participants
     
Participants Analyzed 
[Units: Participants]
 30   30   60 
<=18 years      17  56.7%      14  46.7%      31  51.7% 
Between 18 and 65 years      13  43.3%      16  53.3%      29  48.3% 
>=65 years      0   0.0%      0   0.0%      0   0.0% 
Age 
[Units: Years]
Mean (Standard Deviation)
     
Participants Analyzed 
[Units: Participants]
 30   30   60 
   21.12  (10.9)   19.98  (5.8)   20.55  (8.35) 
Sex: Female, Male 
[Units: Participants]
Count of Participants
     
Participants Analyzed 
[Units: Participants]
 30   30   60 
Female      19  63.3%      10  33.3%      29  48.3% 
Male      11  36.7%      20  66.7%      31  51.7% 
Ethnicity (NIH/OMB) 
[Units: Participants]
Count of Participants
     
Participants Analyzed 
[Units: Participants]
 30   30   60 
Hispanic or Latino      1   3.3%      1   3.3%      2   3.3% 
Not Hispanic or Latino      29  96.7%      29  96.7%      58  96.7% 
Unknown or Not Reported      0   0.0%      0   0.0%      0   0.0% 
Race (NIH/OMB) 
[Units: Participants]
Count of Participants
     
Participants Analyzed 
[Units: Participants]
 30   30   60 
American Indian or Alaska Native      0   0.0%      0   0.0%      0   0.0% 
Asian      0   0.0%      0   0.0%      0   0.0% 
Native Hawaiian or Other Pacific Islander      0   0.0%      0   0.0%      0   0.0% 
Black or African American      0   0.0%      0   0.0%      0   0.0% 
White      30 100.0%      30 100.0%      60 100.0% 
More than one race      0   0.0%      0   0.0%      0   0.0% 
Unknown or Not Reported      0   0.0%      0   0.0%      0   0.0% 
Region of Enrollment 
[Units: Participants]
Count of Participants
     
United States       
Participants Analyzed 
[Units: Participants]
 30   30   60 
United States   30   30   60 
Number of Prior Regimens of Participants that Received Stem Cell Transplant [1] [2] 
[Units: Regimens]
     
Patient # 1       
Participants Analyzed 
[Units: Participants]
 0   23   23 
Patient # 1      5   5 
Patient # 2       
Participants Analyzed 
[Units: Participants]
 0   23   23 
Patient # 2      3   3 
Patient # 3       
Participants Analyzed 
[Units: Participants]
 0   23   23 
Patient # 3      2   2 
Patient # 4       
Participants Analyzed 
[Units: Participants]
 0   23   23 
Patient # 4      3   3 
Patient # 6       
Participants Analyzed 
[Units: Participants]
 0   23   23 
Patient # 6      2   2 
Patient # 8       
Participants Analyzed 
[Units: Participants]
 0   23   23 
Patient # 8      1   1 
Patient # 9       
Participants Analyzed 
[Units: Participants]
 0   23   23 
Patient # 9      2   2 
Patient # 10       
Participants Analyzed 
[Units: Participants]
 0   23   23 
Patient # 10      2   2 
Patient # 12       
Participants Analyzed 
[Units: Participants]
 0   23   23 
Patient # 12      2   2 
Patient # 13       
Participants Analyzed 
[Units: Participants]
 0   23   23 
Patient # 13      3   3 
Patient # 14       
Participants Analyzed 
[Units: Participants]
 0   23   23 
Patient # 14      1   1 
Patient # 15       
Participants Analyzed 
[Units: Participants]
 0   23   23 
Patient # 15      2   2 
Patient # 17       
Participants Analyzed 
[Units: Participants]
 0   23   23 
Patient # 17      2   2 
Patient # 18       
Participants Analyzed 
[Units: Participants]
 0   23   23 
Patient # 18      2   2 
Patient # 20       
Participants Analyzed 
[Units: Participants]
 0   23   23 
Patient # 20      3   3 
Patient # 21       
Participants Analyzed 
[Units: Participants]
 0   23   23 
Patient # 21      1   1 
Patient # 22       
Participants Analyzed 
[Units: Participants]
 0   23   23 
Patient # 22      1   1 
Patient # 24       
Participants Analyzed 
[Units: Participants]
 0   23   23 
Patient # 24      2   2 
Patient # 25       
Participants Analyzed 
[Units: Participants]
 0   23   23 
Patient # 25      2   2 
Patient # 27       
Participants Analyzed 
[Units: Participants]
 0   23   23 
Patient # 27      1   1 
Patient # 28 (c)       
Participants Analyzed 
[Units: Participants]
 0   23   23 
Patient # 28 (c)      1   1 
Patient # 29       
Participants Analyzed 
[Units: Participants]
 0   23   23 
Patient # 29      2   2 
Patient # 30       
Participants Analyzed 
[Units: Participants]
 0   23   23 
Patient # 30      1   1 
[1] (c) Up-front regimen included autologous stem cell transplantation.
[2] This baseline measure only applies to recipients.
Number of Prior Regimens of Participants that Did Not Receive Stem Cell Transplant [1] 
[Units: Regimens]
     
Patient # 5       
Participants Analyzed 
[Units: Participants]
 0   7   7 
Patient # 5      2   2 
Patient # 7       
Participants Analyzed 
[Units: Participants]
 0   7   7 
Patient # 7      1   1 
Patient # 11       
Participants Analyzed 
[Units: Participants]
 0   7   7 
Patient # 11      3   3 
Patient # 16       
Participants Analyzed 
[Units: Participants]
 0   7   7 
Patient # 16      2   2 
Patient # 19       
Participants Analyzed 
[Units: Participants]
 0   7   7 
Patient # 19      2   2 
Patient # 23       
Participants Analyzed 
[Units: Participants]
 0   7   7 
Patient # 23      4   4 
Patient # 26       
Participants Analyzed 
[Units: Participants]
 0   7   7 
Patient # 26      3   3 
[1] This baseline measure applies to recipients only.
Participants who Received Stem Cell Transplant Disease Status at Enrollment [1] 
[Units: Participants]
Count of Participants
     
Progressive Disease (PD)       
Participants Analyzed 
[Units: Participants]
 0   23   23 
Progressive Disease (PD)      18   18 
No Evidence of Disease (NED)       
Participants Analyzed 
[Units: Participants]
 0   23   23 
No Evidence of Disease (NED)      4   4 
Stable Disease (SD)       
Participants Analyzed 
[Units: Participants]
 0   23   23 
Stable Disease (SD)      1   1 
[1] This baseline measure applies to recipients only.
Participants who Did Not Receive Stem Cell Transplant Disease Status(Progressive Disease)/Enrollment [1] 
[Units: Participants]
Count of Participants
     
Participants Analyzed 
[Units: Participants]
 0   7   7 
      7   7 
[1] This baseline measure applies to recipients only.
Disease Status at Hematopoietic Stem Cell Transplant [1] [2] 
[Units: Participants]
Count of Participants
     
Partial Response (PR)       
Participants Analyzed 
[Units: Participants]
 0   23   23 
Partial Response (PR)      4   4 
Stable Disease (SD)       
Participants Analyzed 
[Units: Participants]
 0   23   23 
Stable Disease (SD)      9   9 
No Evidence of Disease (NED)       
Participants Analyzed 
[Units: Participants]
 0   23   23 
No Evidence of Disease (NED)      4   4 
Progressive Disease (PD)       
Participants Analyzed 
[Units: Participants]
 0   23   23 
Progressive Disease (PD)      3   3 
Complete Response (CR)/(NED)       
Participants Analyzed 
[Units: Participants]
 0   23   23 
Complete Response (CR)/(NED)      3   3 
[1] This list includes the disease status of participants who received stem cell transplant.
[2] This baseline measure applies to recipients only.
Disease Status (Progressive Disease) at Hematopoietic Stem Cell Transplant [1] [2] 
[Units: Participants]
Count of Participants
     
Participants Analyzed 
[Units: Participants]
 0   7   7 
      7   7 
[1] This list includes the disease status of participants who did not receive stem cell transplant.
[2] This baseline measure applies to recipients only.
Diagnosis (Participants that Received Stem Cell Transplant) [1] [2] 
[Units: Participants]
Count of Participants
     
aRMS       
Participants Analyzed 
[Units: Participants]
 0   23   23 
aRMS      7   7 
ESFT       
Participants Analyzed 
[Units: Participants]
 0   23   23 
ESFT      5   5 
DSRCT       
Participants Analyzed 
[Units: Participants]
 0   23   23 
DSRCT      11   11 
[1] aRMS indicates alveolar rhabdomyosarcoma; ESRT, Ewings sarcoma; DSRCT, desmoplastic small round cell tumor.
[2] This baseline measure applies to recipients only.
Diagnosis (Participants that Did Not Receive Stem Cell Transplant) [1] [2] 
[Units: Participants]
Count of Participants
     
ESFT       
Participants Analyzed 
[Units: Participants]
 0   7   7 
ESFT      6   6 
DSRCT       
Participants Analyzed 
[Units: Participants]
 0   23   23 
DSRCT      1   1 
[1] ESRT, Ewings sarcoma; DSRCT, desmoplastic small round cell tumor.
[2] This baseline measure applies to recipients only.
High-Risk Features (Participants that Received Stem Cell Transplant) [1] [2] 
[Units: Participants]
Count of Participants
     
MR       
Participants Analyzed 
[Units: Participants]
 0   23   23 
MR      6   6 
ER       
Participants Analyzed 
[Units: Participants]
 0   23   23 
ER      2   2 
ER, B/BM, MR       
Participants Analyzed 
[Units: Participants]
 0   23   23 
ER, B/BM, MR      1   1 
PP       
Participants Analyzed 
[Units: Participants]
 0   23   23 
PP      1   1 
B/BM       
Participants Analyzed 
[Units: Participants]
 0   23   23 
B/BM      7   7 
Metastatic       
Participants Analyzed 
[Units: Participants]
 0   23   23 
Metastatic      4   4 
PP/Metastatic       
Participants Analyzed 
[Units: Participants]
 0   23   23 
PP/Metastatic      1   1 
PP, B/BM       
Participants Analyzed 
[Units: Participants]
 0   23   23 
PP, B/BM      1   1 
[1] B/BM indicates bone or bone marrow metastases; ER, early recurrence; MR, multiply recurrent; PP, primary progressive disease.
[2] This baseline measure applies to recipients only.
High-Risk Features (Participants that Did Not Receive Stem Cell Transplant) [1] [2] 
[Units: Participants]
Count of Participants
     
ER, B/BM       
Participants Analyzed 
[Units: Participants]
 0   7   7 
ER, B/BM      1   1 
PP, B/BM       
Participants Analyzed 
[Units: Participants]
 0   7   7 
PP, B/BM      1   1 
ER       
Participants Analyzed 
[Units: Participants]
 0   7   7 
ER      3   3 
PP       
Participants Analyzed 
[Units: Participants]
 0   7   7 
PP      2   2 
[1] B/BM indicates bone or bone marrow metastases; ER, early recurrence; MR, multiply recurrent; PP, primary progressive disease.
[2] This baseline measure applies to recipients only.


  Outcome Measures
  Show All Outcome Measures

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 ]

4.  Secondary:   Median Time to Reach Absolute Neutrophil Count of 500/mm(3)   [ Time Frame: up to 12 days ]

5.  Secondary:   Median Time to Reach a Platelet Count of 50,000/mm(3)   [ Time Frame: up to 43 days ]

6.  Secondary:   Early Post Transplantation Relapse   [ Time Frame: up to 300 days ]

7.  Secondary:   Median Progression Free Survival   [ Time Frame: up to 77 months ]

8.  Secondary:   Two Year Survival Rate for Patients Undergoing Allo-Hematopoietic Stem Cell Transplant   [ Time Frame: 2 years ]

9.  Secondary:   Number of Participants to Complete Conversion to >95% Donor Chimerism   [ Time Frame: up to 30 days ]

10.  Secondary:   Cluster of Differentiation 4 (CD4) Reconstitution   [ Time Frame: Day +28-42 ]

11.  Secondary:   Best Response Post-Hematopoietic Stem Cell Transplant EOCH (Etoposide, Vincristine, Cyclophosphamide, and Doxorubicin)   [ Time Frame: up to 10 cycles of therapy or 280 days ]

12.  Secondary:   Median Survival From Date of Progression   [ Time Frame: up to 77 months ]

13.  Other Pre-specified:   Number of Participants Who Experienced Graft Versus Tumor Effect (GVT)   [ Time Frame: up to day 100 ]

14.  Other Pre-specified:   Post-Hematopoietic Stem Cell Transplant (HSCT) Radiotherapy   [ Time Frame: up to 6 cycles or 168 days ]


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats
  Hide Limitations and Caveats

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
No text entered.


  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. Terry Fry
Organization: National Cancer Institute, National Institutes of Health
phone: 301-402-0215
e-mail: fryt@mail.nih.gov


Publications of Results:
Other Publications:

Responsible Party: Terry Fry, 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: April 11, 2017