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Evaluating the Safety and Effectiveness of Bone Marrow Transplants in Children With Sickle Cell Disease (BMT CTN 0601) (SCURT)

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.
ClinicalTrials.gov Identifier: NCT00745420
Recruitment Status : Completed
First Posted : September 3, 2008
Results First Posted : December 25, 2017
Last Update Posted : December 25, 2017
Sponsor:
Collaborators:
Information provided by (Responsible Party):

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition: Sickle Cell Disease
Intervention: Biological: Hematopoietic Stem Cell Transplantation

  Participant Flow

Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
Participants were enrolled between August 2008 and April 2014 from 19 different transplant centers.

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
Hematopoietic Stem Cell Transplantation Bone Marrow Transplant with GVHD Prophylaxis Regimen

Participant Flow:   Overall Study
    Hematopoietic Stem Cell Transplantation
STARTED   30 
COMPLETED   29 
NOT COMPLETED   1 
Ineligible                1 



  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
Hematopoietic Stem Cell Transplantation Bone Marrow Transplant with GVHD Prophylaxis Regimen

Baseline Measures
   Hematopoietic Stem Cell Transplantation 
Overall Participants Analyzed 
[Units: Participants]
 29 
Age 
[Units: Years]
Median (Full Range)
 14 
 (6 to 19) 
Sex: Female, Male 
[Units: Participants]
Count of Participants
 
Female      13  44.8% 
Male      16  55.2% 
Race/Ethnicity, Customized 
[Units: Participants]
Count of Participants
 
Black or African American   26 
Hispanic or Latino   3 
Indications for Transplant [1] 
[Units: Participants]
Count of Participants
 
Stroke   12 
Trans cranial doppler velocity > 200cm/s   2 
Acute chest syndrome   4 
Vaso-occlusive pain crisis   12 
Chronic blood transfusion prior to transplant   14 
[1] Some patients had more than 1 indication
Karnofsky Performance Score [1] 
[Units: Participants]
Count of Participants
 
100   17 
90   9 
80   2 
70   1 
[1] Assesses patient self-perceived global quality of life and functioning on a scale of 0 - 100; where 100 equals normal quality of life with no evidence of disease, 90 equals ability to carry on normal activity with minor signs/symptoms of disease, 80 equals normal activity with effort and some signs/symptoms of disease, and 70 equals ability to care for oneself but unable to carry on normal activity or to do active work.
Cytomegalovirus (CMV) Status 
[Units: Participants]
Count of Participants
 
CMV - donor and recipient   8 
CMV + donor and recipient   9 
CMV - donor and CMV + recipient   3 
CMV + donor and CMV - recipient   9 
Donor Blood Type Match 
[Units: Participants]
Count of Participants
 
ABO Blood group matched   15 
ABO Blood group major mismatch   9 
ABO Blood group minor mismatch   5 
Donor Gender Match 
[Units: Participants]
Count of Participants
 
Gender matched transplants   15 
Female donor, male recipient   7 
Male donor, female recipient   7 


  Outcome Measures

1.  Primary:   Percentage of Participants With Event-Free Survival (EFS)   [ Time Frame: 2 years ]

2.  Secondary:   Percentage of Participants With Overall Survival (OS)   [ Time Frame: 2 years ]

3.  Secondary:   Neutrophil and Platelet Recovery   [ Time Frame: Up to 100 days ]

4.  Secondary:   Percentage of Participants With Acute Graft-vs-Host-Disease (GVHD)   [ Time Frame: 100 days ]

5.  Secondary:   Percentage of Participants With Chronic GVHD   [ Time Frame: 1 year ]

6.  Secondary:   Health-Related Quality of Life (HRQL) - Change From Baseline to Day 100   [ Time Frame: 100 days ]

7.  Secondary:   Health-Related Quality of Life (HRQL) - Change From Baseline to Day 180   [ Time Frame: 180 days ]

8.  Secondary:   Health-Related Quality of Life (HRQL) - Change From Baseline to 1 Year   [ Time Frame: 1 year ]


  Serious Adverse Events


  Other Adverse Events


  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

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: Adam Mendizabal, PhD
Organization: The Emmes Corporation
phone: 301-251-1161
e-mail: amendizabal@emmes.com


Publications of Results:

Responsible Party: Medical College of Wisconsin
ClinicalTrials.gov Identifier: NCT00745420     History of Changes
Other Study ID Numbers: BMTCTN0601
U01HL069294 ( U.S. NIH Grant/Contract )
5U24CA076518 ( U.S. NIH Grant/Contract )
First Submitted: August 29, 2008
First Posted: September 3, 2008
Results First Submitted: August 23, 2017
Results First Posted: December 25, 2017
Last Update Posted: December 25, 2017