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Allo-HCT MUD for Non-malignant Red Blood Cell (RBC) Disorders: Sickle Cell, Thal, and DBA: Reduced Intensity Conditioning, Co-tx MSCs

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ClinicalTrials.gov Identifier: NCT00957931
Recruitment Status : Completed
First Posted : August 13, 2009
Results First Posted : August 6, 2018
Last Update Posted : August 6, 2018
Sponsor:
Collaborators:
University of Minnesota
University of Alabama at Birmingham
Information provided by (Responsible Party):
Sandhya Kharbanda, Stanford University

Study Type Interventional
Study Design Allocation: N/A;   Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Conditions Sickle Cell Disease
Thalassemia
Diamond-Blackfan Anemia
Interventions Procedure: Bone marrow transplantation
Biological: Mesenchymal Stromal Cells
Enrollment 6
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Mesenchymal Stromal Cells
Hide Arm/Group Description Patients received bone marrow transplantation using matched unrelated donors, reduced intensity conditioning regimen, and co-transplanting mesenchymal stromal cells derived from parental bone marrow.
Period Title: Overall Study
Started 6
Completed 2
Not Completed 4
Reason Not Completed
Death             4
Arm/Group Title Mesenchymal Stromal Cells
Hide Arm/Group Description Patients received bone marrow transplantation using matched unrelated donors, reduced intensity conditioning regimen, and co-transplanting mesenchymal stromal cells derived from parental bone marrow.
Overall Number of Baseline Participants 6
Hide Baseline Analysis Population Description
[Not Specified]
Age, Continuous  
Median (Full Range)
Unit of measure:  Years
Number Analyzed 6 participants
10
(8 to 18)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 6 participants
Female
1
  16.7%
Male
5
  83.3%
Number of known transfusions prior to HSCT   [1] 
Median (Full Range)
Unit of measure:  Transfusions
Number Analyzed 6 participants
47.5
(4 to 220)
[1]
Measure Description: HSCT = hematopoietic stem cell transplantation
Diagnosis  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 6 participants
Sickle cell disease
4
  66.7%
Thalassemia major
2
  33.3%
1.Primary Outcome
Title Count of Participants With Stable Engraftment Post Hematopoietic Cell Transplantation (HCT)
Hide Description Stable engraftment was defined as absolute neutrophil count (ANC) >500 cells /µL for 3 consecutive days and platelet count >50,000 for one week without transfusion; subsequently stable engraftment was measured by percentage of donor cells.
Time Frame Up to 1 year
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Mesenchymal Stromal Cells
Hide Arm/Group Description:
Patients received bone marrow transplantation using matched unrelated donors, reduced intensity conditioning regimen, and co-transplanting mesenchymal stromal cells derived from parental bone marrow.
Overall Number of Participants Analyzed 6
Measure Type: Count of Participants
Unit of Measure: Participants
3
  50.0%
2.Secondary Outcome
Title Overall Survival 6 Months Following HCT
Hide Description Overall survival is reported at the count of participants alive 6 months following HCT.
Time Frame 6 months
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Mesenchymal Stromal Cells
Hide Arm/Group Description:
Patients received bone marrow transplantation using matched unrelated donors, reduced intensity conditioning regimen, and co-transplanting mesenchymal stromal cells derived from parental bone marrow.
Overall Number of Participants Analyzed 6
Measure Type: Count of Participants
Unit of Measure: Participants
2
  33.3%
3.Secondary Outcome
Title Overall Survival 1 Year Following HCT
Hide Description Overall survival is reported at the count of participants alive 1 year following HCT.
Time Frame 1 year
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Mesenchymal Stromal Cells
Hide Arm/Group Description:
Patients received bone marrow transplantation using matched unrelated donors, reduced intensity conditioning regimen, and co-transplanting mesenchymal stromal cells derived from parental bone marrow.
Overall Number of Participants Analyzed 6
Measure Type: Count of Participants
Unit of Measure: Participants
2
  33.3%
4.Secondary Outcome
Title Count of Participants With Disease-free Survival 6 Months Following HCT
Hide Description Disease-free survival is defined as alive without underlying disease.
Time Frame 6 months
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Mesenchymal Stromal Cells
Hide Arm/Group Description:
Patients received bone marrow transplantation using matched unrelated donors, reduced intensity conditioning regimen, and co-transplanting mesenchymal stromal cells derived from parental bone marrow.
Overall Number of Participants Analyzed 6
Measure Type: Count of Participants
Unit of Measure: Participants
0
   0.0%
5.Secondary Outcome
Title Count of Participants With Disease-free Survival 1 Year Following HCT
Hide Description Disease-free survival is defined as alive without underlying disease.
Time Frame 1 year
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Mesenchymal Stromal Cells
Hide Arm/Group Description:
Patients received bone marrow transplantation using matched unrelated donors, reduced intensity conditioning regimen, and co-transplanting mesenchymal stromal cells derived from parental bone marrow.
Overall Number of Participants Analyzed 6
Measure Type: Count of Participants
Unit of Measure: Participants
0
   0.0%
Time Frame 1 year
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Mesenchymal Stromal Cells
Hide Arm/Group Description Patients received bone marrow transplantation using matched unrelated donors, reduced intensity conditioning regimen, and co-transplanting mesenchymal stromal cells derived from parental bone marrow.
All-Cause Mortality
Mesenchymal Stromal Cells
Affected / at Risk (%)
Total   4/6 (66.67%) 
Hide Serious Adverse Events
Mesenchymal Stromal Cells
Affected / at Risk (%)
Total   6/6 (100.00%) 
Blood and lymphatic system disorders   
Graft failure with autologous recovery   2/6 (33.33%) 
Hepatobiliary disorders   
SOS  [1]  1/6 (16.67%) 
Immune system disorders   
EBV-PTLD  [2]  1/6 (16.67%) 
Grade II GVHD  [3]  1/6 (16.67%) 
Grade III acute GVHD   1/6 (16.67%) 
Infections and infestations   
Adenovirus reactivation   1/6 (16.67%) 
BK virus reactivation   1/6 (16.67%) 
CMV pneumonitis  [4]  1/6 (16.67%) 
CMV reactivation   1/6 (16.67%) 
CMV viremia   2/6 (33.33%) 
Disseminated toxoplasmosis   1/6 (16.67%) 
Gram-negative septic shock   1/6 (16.67%) 
HHV-6 viremia  [5]  1/6 (16.67%) 
Klebsiella and enterobacter   1/6 (16.67%) 
Klebsiella bacteremia   1/6 (16.67%) 
Nervous system disorders   
Intracranial bleeding   1/6 (16.67%) 
Intracranial hemorrhage   1/6 (16.67%) 
PRES  [6]  1/6 (16.67%) 
Indicates events were collected by systematic assessment
[1]
SOS = Sinusoidal obstruction syndrome
[2]
EPV = Epstein-Barr virus post-transplant lymphoproliferative disorder
[3]
GVHD = Graft-versus-host disease
[4]
CMV = Cytomegalovirus
[5]
HHV-6 = Human Herpesvirus 6
[6]
PRES = Posterior reversible encephalopathy syndrome
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Mesenchymal Stromal Cells
Affected / at Risk (%)
Total   0/6 (0.00%) 
Certain Agreements
All Principal Investigators ARE employed by the organization sponsoring the study.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Dr. Sandhya Kharbanda, MD
Organization: University of California, San Francisco
EMail: Sandhya.Kharbanda@ucsf.edu
Layout table for additonal information
Responsible Party: Sandhya Kharbanda, Stanford University
ClinicalTrials.gov Identifier: NCT00957931    
Other Study ID Numbers: MSC01
First Submitted: August 12, 2009
First Posted: August 13, 2009
Results First Submitted: May 30, 2018
Results First Posted: August 6, 2018
Last Update Posted: August 6, 2018