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Reduced Intensity Transplant Conditioning Regimen for Severe Thalassemia (URTH)

This study has been completed.
Sponsor:
ClinicalTrials.gov Identifier:
NCT01005576
First Posted: November 2, 2009
Last Update Posted: October 20, 2017
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.
Collaborators:
New England Research Institutes
Pediatric Blood and Marrow Transplant Consortium
Information provided by (Responsible Party):
Washington University School of Medicine
Results First Submitted: September 13, 2017  
Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition: Severe Thalassemia
Intervention: Drug: Transplant conditioning regimen of alemtuzumab, fludarabine, and melphalan

  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
Participants were recruited based on physician referral at 11 academic medical centers. The first participant was enrolled in May 2010 and the last participant was enrolled in April 2012.

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
Conditioning Regimen

Transplant conditioning regimen of alemtuzumab, fludarabine, and melphalan:

Days -50 to -21: Hydroxyurea 30mg/kg po Day -22: Alemtuzumab 3mg IV Day -21: Alemtuzumab 10mg IV Day -20: Alemtuzumab 15mg IV Day -19: Alemtuzumab 20mg IV Day -8: Fludarabine 30mg/m2 IV Day -7: Fludarabine 30mg/m2 IV Day -6: Fludarabine 30mg/m2 IV Day -5: Fludarabine 30mg/m2 IV Day -4: Fludarabine 30mg/m2 IV Day -4: Thiotepa 8mg/kg IV Day -3: Melphalan 140mg/m2 IV Day 0: Stem cell infusion


Participant Flow:   Overall Study
    Conditioning Regimen
STARTED   21 
COMPLETED   21 [1] 
NOT COMPLETED   0 
[1] All participants completed the conditioning regimen and received their stem cell transplant



  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.
No text entered.

Reporting Groups
  Description
Conditioning Regimen

Hydroxyurea days -50 to -21 Alemtuzumab days -21 to -19 Fludarabine days -8 to -4 Thiotepa day -4 Melphalan day -3 Stem cell infusion day 0

Transplant conditioning regimen of alemtuzumab, fludarabine, and melphalan: Days -50 to -21: Hydroxyurea 30mg/kg po Day -22: Alemtuzumab 3mg IV Day -21: Alemtuzumab 10mg IV Day -20: Alemtuzumab 15mg IV Day -19: Alemtuzumab 20mg IV Day -8: Fludarabine 30mg/m2 IV Day -7: Fludarabine 30mg/m2 IV Day -6: Fludarabine 30mg/m2 IV Day -5: Fludarabine 30mg/m2 IV Day -4: Fludarabine 30mg/m2 IV Day -4: Thiotepa 8mg/kg IV Day -3: Melphalan 140mg/m2 IV Day 0: Stem cell infusion


Baseline Measures
   Conditioning Regimen 
Overall Participants Analyzed 
[Units: Participants]
 21 
Age 
[Units: Years]
Median (Full Range)
 10 
 (1.8 to 17) 
Sex: Female, Male 
[Units: Participants]
Count of Participants
 
Female      10  47.6% 
Male      11  52.4% 
Race/Ethnicity, Customized 
[Units: Participants]
Count of Participants
 
Race/Ethnicity   
Asian      5  23.8% 
Middle Eastern      2   9.5% 
African American      1   4.8% 
Hispanic      2   9.5% 
Pakistani      2   9.5% 
Caucasian      3  14.3% 
Vietnamese      1   4.8% 
More than 1 race reported      5  23.8% 
Region of Enrollment 
[Units: Participants]
Count of Participants
 
United States   21 


  Outcome Measures
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1.  Primary:   Primary Objective: Event-free Survival at 1 Year.   [ Time Frame: 1 year ]

2.  Secondary:   Secondary Objectives: Incidence of Transplant-related Outcomes for 2 Years.   [ Time Frame: 2 years ]


  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
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. Shalini Shenoy
Organization: Washington University School of Medicine
phone: 314-454-6018
e-mail: shalinishenoy@wustl.edu



Responsible Party: Washington University School of Medicine
ClinicalTrials.gov Identifier: NCT01005576     History of Changes
Other Study ID Numbers: TCRN-NMD 0901
First Submitted: October 29, 2009
First Posted: November 2, 2009
Results First Submitted: September 13, 2017
Results First Posted: October 20, 2017
Last Update Posted: October 20, 2017