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Trial record 3 of 25 for:    "Thalassemia" | "Busulfan"

A Pilot Study of HSCT for Patients With High-risk Hemoglobinopathy Using a Nonmyeloablative Preparative Regimen

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ClinicalTrials.gov Identifier: NCT00427661
Recruitment Status : Completed
First Posted : January 29, 2007
Results First Posted : August 18, 2016
Last Update Posted : August 18, 2016
Sponsor:
Information provided by (Responsible Party):
Lakshmanan Krishnamurti, University of Pittsburgh

Study Type Interventional
Study Design Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Conditions Sickle Cell Disease
Thalassemia
Hemoglobinopathies
Intervention Other: Busulfan; Fludarabine; cyclosporine A and MMF
Enrollment 8
Recruitment Details  
Pre-assignment Details  
Arm/Group Title AHSC in Severe SCD
Hide Arm/Group Description The patient population for this study included severe SCD patients, both pediatric and adult, who did not have end-organ failure, and met all of the eligibility criteria.
Period Title: Overall Study
Started 8
Completed 8
Not Completed 0
Arm/Group Title AHSC in Severe SCD
Hide Arm/Group Description The patient population for this study included severe SCD patients, both pediatric and adult, who did not have end-organ failure, and met all of the eligibility criteria.
Overall Number of Baseline Participants 8
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 8 participants
<=18 years
6
  75.0%
Between 18 and 65 years
2
  25.0%
>=65 years
0
   0.0%
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 8 participants
Female
4
  50.0%
Male
4
  50.0%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 8 participants
8
1.Primary Outcome
Title Development of GVHD Within 1 Year of BMT
Hide Description GVHD is assessed by physical exam, bloodwork and biopsy.
Time Frame 1 year
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title AHSC in Severe SCD
Hide Arm/Group Description:
The patient population for this study included severe SCD patients, both pediatric and adult, who did not have end-organ failure, and met all of the eligibility criteria.
Overall Number of Participants Analyzed 7
Measure Type: Number
Unit of Measure: participants
2
2.Primary Outcome
Title Engraftment at 1 Year Post BMT.
Hide Description Measurement of total PBMC chimerism
Time Frame 1 year
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title AHSC in Severe SCD
Hide Arm/Group Description:
The patient population for this study included severe SCD patients, both pediatric and adult, who did not have end-organ failure, and met all of the eligibility criteria.
Overall Number of Participants Analyzed 7
Measure Type: Number
Unit of Measure: participants
6
3.Secondary Outcome
Title Incidence of Grade 2-4 Acute GVHD.
Hide Description [Not Specified]
Time Frame 100 days
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Experimental
Hide Arm/Group Description:
Busulfan, Fludarabine, Cyclosporine, MMF
Overall Number of Participants Analyzed 8
Measure Type: Number
Unit of Measure: participants with grade 2-4 AGVHD
1
Time Frame [Not Specified]
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title AHSC in Severe SCD
Hide Arm/Group Description The patient population for this study included severe SCD patients, both pediatric and adult, who did not have end-organ failure, and met all of the eligibility criteria.
All-Cause Mortality
AHSC in Severe SCD
Affected / at Risk (%)
Total   --/--    
Show Serious Adverse Events Hide Serious Adverse Events
AHSC in Severe SCD
Affected / at Risk (%) # Events
Total   1/8 (12.50%)    
Respiratory, thoracic and mediastinal disorders   
Pulmonary Arterial Hypertension  [1]  1/8 (12.50%) 
Indicates events were collected by systematic assessment
[1]
21 year old female patient developed complications related to sickle cell disease associated progressive pulmonary arterial hypertension and died 10 months post BMT.
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
AHSC in Severe SCD
Affected / at Risk (%) # Events
Total   8/8 (100.00%)    
Gastrointestinal disorders   
Pancreatitis  [1]  1/8 (12.50%)  1
Poor oral intake  [2]  2/8 (25.00%)  2
Infections and infestations   
Line related bacteremia  [3]  1/8 (12.50%)  1
Infection  [4]  1/8 (12.50%)  1
Line related bacteremia  [5]  1/8 (12.50%)  1
Nervous system disorders   
Seizure  [6]  1/7 (14.29%)  1
Headache  [7]  1/8 (12.50%)  1
Renal and urinary disorders   
Hemoglobinuria  [8]  1/8 (12.50%)  1
Indicates events were collected by systematic assessment
[1]
Mild pancreatitis Day +109 (Krishnamurti et al., 2008. Biol Blood Marrow Transplant 14:1270)
[2]
Required transient parenteral alimentation (Krishnamurti et al., 2008. Biol Blood Marrow Transplant 14:1270)
[3]
line related bacteremia with Rhizobium radiobactor d100 (Krishnamurti et al., 2008. Biol Blood Marrow Transplant 14:1270.
[4]
Dermatomal herpes zoster Parainfluenza A infection (Krishnamurti et al., 2008. Biol Blood Marrow Transplant 14:1270)
[5]
Line related bacteremia d244 (Krishnamurti et al., 2008. Biol Blood Marrow Transplant 14:1270)
[6]
Seizure day+11 secondary to low level of phenytoin (Krishnamurti et al., 2008. Biol Blood Marrow Transplant 14:1270.)
[7]
headache day +1-+4, post transplant, requiring analgesics (Krishnamurti et al., 2008. Biol Blood Marrow Transplant 14:1270)
[8]
Hemoglobinuria immediately following stem cell transplant the resolved within 48 hours (Krishnamurti et al., 2008. Biol Blood Marrow Transplant 14:1270)
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: Nancy Harter-Administrative Director of Research
Organization: University of Pittsburgh-Department of Pediatrics
Phone: 412-692-7487
EMail: harterne@upmc.edu
Layout table for additonal information
Responsible Party: Lakshmanan Krishnamurti, University of Pittsburgh
ClinicalTrials.gov Identifier: NCT00427661     History of Changes
Other Study ID Numbers: IRB #0504048
First Submitted: January 18, 2007
First Posted: January 29, 2007
Results First Submitted: February 19, 2016
Results First Posted: August 18, 2016
Last Update Posted: August 18, 2016