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Reduced Intensity Transplant Conditioning Regimen for Severe Thalassemia (URTH)

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ClinicalTrials.gov Identifier: NCT01005576
Recruitment Status : Completed
First Posted : November 1, 2009
Results First Posted : October 20, 2017
Last Update Posted : December 13, 2017
Sponsor:
Collaborators:
HealthCore-NERI
Pediatric Blood and Marrow Transplant Consortium
Information provided by (Responsible Party):
Washington University School of Medicine

Study Type Interventional
Study Design Allocation: N/A;   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
Enrollment 21
Recruitment Details 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  
Arm/Group Title Conditioning Regimen
Hide Arm/Group Description

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

Period Title: Overall Study
Started 21
Completed 21 [1]
Not Completed 0
[1]
All participants completed the conditioning regimen and received their stem cell transplant
Arm/Group Title Conditioning Regimen
Hide Arm/Group Description

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

Overall Number of Baseline Participants 21
Hide Baseline Analysis Population Description
[Not Specified]
Age, Continuous  
Median (Full Range)
Unit of measure:  Years
Number Analyzed 21 participants
10
(1.8 to 17)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 21 participants
Female
10
  47.6%
Male
11
  52.4%
Race/Ethnicity, Customized  
Measure Type: Count of Participants
Unit of measure:  Participants
Race/Ethnicity Number Analyzed 21 participants
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  
Measure Type: Count of Participants
Unit of measure:  Participants
United States Number Analyzed 21 participants
21
 100.0%
1.Primary Outcome
Title Primary Objective: Event-free Survival at 1 Year.
Hide Description [Not Specified]
Time Frame 1 year
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[Not Specified]
Arm/Group Title Conditioning Regimen
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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

Overall Number of Participants Analyzed 21
Measure Type: Count of Participants
Unit of Measure: Participants
17
  81.0%
2.Secondary Outcome
Title Development of Graft Versus Host Disease (GVHD)
Hide Description [Not Specified]
Time Frame 2 years
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Conditioning Regimen
Hide Arm/Group Description:

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

Overall Number of Participants Analyzed 21
Measure Type: Count of Participants
Unit of Measure: Participants
Participants who developed any GVHD
12
  57.1%
Participants who did not develop any GVHD
9
  42.9%
3.Secondary Outcome
Title Median Time to ANC Engraftment
Hide Description [Not Specified]
Time Frame 100 days
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Conditioning Regimen
Hide Arm/Group Description:

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

Overall Number of Participants Analyzed 21
Median (Full Range)
Unit of Measure: days
14
(10 to 46)
4.Secondary Outcome
Title Median Time to Platelet Engraftment
Hide Description [Not Specified]
Time Frame 100 days
Hide Outcome Measure Data
Hide Analysis Population Description
3 participants did not engraft platelets
Arm/Group Title Conditioning Regimen
Hide Arm/Group Description:

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

Overall Number of Participants Analyzed 18
Median (Full Range)
Unit of Measure: days
27.5
(18 to 234)
5.Secondary Outcome
Title Incidence of Disease Recurrence
Hide Description [Not Specified]
Time Frame 2 years
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Conditioning Regimen
Hide Arm/Group Description:

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

Overall Number of Participants Analyzed 21
Measure Type: Count of Participants
Unit of Measure: Participants
1
   4.8%
Time Frame [Not Specified]
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Conditioning Regimen
Hide Arm/Group Description

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

All-Cause Mortality
Conditioning Regimen
Affected / at Risk (%)
Total   4/21 (19.05%) 
Hide Serious Adverse Events
Conditioning Regimen
Affected / at Risk (%)
Total   10/21 (47.62%) 
General disorders   
Pain   1/21 (4.76%) 
Graft rejection  1/21 (4.76%) 
Edema [1]  1/21 (4.76%) 
Infections and infestations   
Sepsis   1/21 (4.76%) 
Nervous system disorders   
Posterior Reversible Encephalopathy Syndrome  1/21 (4.76%) 
Headache   1/21 (4.76%) 
Respiratory, thoracic and mediastinal disorders   
Pulmonary toxicity  [2]  4/21 (19.05%) 
Indicates events were collected by systematic assessment
[1]
Head and neck edema
[2]
Respiratory distress, pulmonary failure, pulmonary hemorrhage
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Conditioning Regimen
Affected / at Risk (%)
Total   21/21 (100.00%) 
Hepatobiliary disorders   
Veno Occlusive Disease  1/21 (4.76%) 
Infections and infestations   
CMV reactivation   14/21 (66.67%) 
Infection (other than CMV)  21/21 (100.00%) 
Investigations   
Hyperbilirubinemia  1/21 (4.76%) 
Hypercalcemia  1/21 (4.76%) 
Renal and urinary disorders   
Acute Kidney Injury  1/21 (4.76%) 
Indicates events were collected by systematic assessment
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
Layout table for Results Point of Contact information
Name/Title: Dr. Shalini Shenoy
Organization: Washington University School of Medicine
Phone: 314-454-6018
EMail: shalinishenoy@wustl.edu
Layout table for additonal information
Responsible Party: Washington University School of Medicine
ClinicalTrials.gov Identifier: NCT01005576    
Other Study ID Numbers: TCRN-NMD 0901
First Submitted: October 29, 2009
First Posted: November 1, 2009
Results First Submitted: September 13, 2017
Results First Posted: October 20, 2017
Last Update Posted: December 13, 2017