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Preventing Stem Cell Transplant Complications With a Blood Separator Machine

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ClinicalTrials.gov Identifier: NCT01866839
Recruitment Status : Completed
First Posted : June 3, 2013
Results First Posted : May 26, 2020
Last Update Posted : July 21, 2020
Sponsor:
Collaborator:
Children's National Research Institute
Information provided by (Responsible Party):
National Institutes of Health Clinical Center (CC) ( National Heart, Lung, and Blood Institute (NHLBI) )

Study Type Interventional
Study Design Allocation: N/A;   Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Conditions MDS (Myelodysplastic Syndrome)
Myeloproliferative Disorder
Lymphoma, Non-Hodgkin
ALL (Acute B-Lymphoblastic Leukemia)
AML (Acute Myelogenous Leukemia
Intervention Device: Graft Manipulation (CD34+ Selection)
Enrollment 41
Recruitment Details  
Pre-assignment Details 1 participant signed consent but did not start study
Arm/Group Title CD34+ Cell Positively Selected Graft Stem Cell Recipient
Hide Arm/Group Description Recipients received a myeloablative conditioning regimen of cyclophosphamide (120 mg/kg total), fludarabine (125 mg/m2 total) and total body irradiation (1200 cGy with lung shielding to 600 cGy), followed by an infusion of a stem cell product selected for CD34+ progenitors using the Miltenyi CliniMACS® system. Older subjects will receive a lower dose of irradiation (800 or 600 cGy based on age) to reduce the regimen intensity.
Period Title: Overall Study
Started 40
Completed 30
Not Completed 10
Arm/Group Title CD34+ Cell Positively Selected Graft Stem Cell Recipient
Hide Arm/Group Description Recipients received a myeloablative conditioning regimen of cyclophosphamide (120 mg/kg total), fludarabine (125 mg/m2 total) and total body irradiation (1200 cGy with lung shielding to 600 cGy), followed by an infusion of a stem cell product selected for CD34+ progenitors using the Miltenyi CliniMACS® system. Older subjects will receive a lower dose of irradiation (800 or 600 cGy based on age) to reduce the regimen intensity.
Overall Number of Baseline Participants 40
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 40 participants
<=18 years
2
   5.0%
Between 18 and 65 years
32
  80.0%
>=65 years
6
  15.0%
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 40 participants
Female
21
  52.5%
Male
19
  47.5%
Ethnicity (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 40 participants
Hispanic or Latino
21
  52.5%
Not Hispanic or Latino
19
  47.5%
Unknown or Not Reported
0
   0.0%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 40 participants
American Indian or Alaska Native
0
   0.0%
Asian
5
  12.5%
Native Hawaiian or Other Pacific Islander
0
   0.0%
Black or African American
3
   7.5%
White
13
  32.5%
More than one race
4
  10.0%
Unknown or Not Reported
15
  37.5%
1.Primary Outcome
Title Overall Survival
Hide Description Determine the rate of overall survival at 200 day
Time Frame 200 days
Hide Outcome Measure Data
Hide Analysis Population Description
Subjects for whom the Miltenyi CliniMACS CD34 selection system was used for graft manipulation in HLA-matched sibling allogeneic stem cell transplantation
Arm/Group Title CD34+ Cell Positively Selected Graft Stem Cell Recipient
Hide Arm/Group Description:
Recipients received a myeloablative conditioning regimen of cyclophosphamide (120 mg/kg total), fludarabine (125 mg/m2 total) and total body irradiation (1200 cGy with lung shielding to 600 cGy), followed by an infusion of a stem cell product selected for CD34+ progenitors using the Miltenyi CliniMACS® system. Older subjects will receive a lower dose of irradiation (800 or 600 cGy based on age) to reduce the regimen intensity.
Overall Number of Participants Analyzed 40
Measure Type: Count of Participants
Unit of Measure: Participants
36
  90.0%
2.Secondary Outcome
Title Disease Free Survival at 2 Years
Hide Description Disease Free Survival at 2 years
Time Frame 2 years
Hide Outcome Measure Data
Hide Analysis Population Description
Subjects for whom the Miltenyi CliniMACS CD34 selection system was used for graft manipulation in HLA-matched sibling allogeneic stem cell transplantation
Arm/Group Title CD34+ Cell Positively Selected Graft Stem Cell Recipient
Hide Arm/Group Description:
Recipients received a myeloablative conditioning regimen of cyclophosphamide (120 mg/kg total), fludarabine (125 mg/m2 total) and total body irradiation (1200 cGy with lung shielding to 600 cGy), followed by an infusion of a stem cell product selected for CD34+ progenitors using the Miltenyi CliniMACS® system. Older subjects will receive a lower dose of irradiation (800 or 600 cGy based on age) to reduce the regimen intensity.
Overall Number of Participants Analyzed 40
Measure Type: Number
Unit of Measure: percentage of participant
60.8
3.Secondary Outcome
Title Disease Progression
Hide Description Incidence of relapse / disease progression
Time Frame 3 years
Hide Outcome Measure Data
Hide Analysis Population Description
Subjects for whom the Miltenyi CliniMACS CD34 selection system was used for graft manipulation in HLA-matched sibling allogeneic stem cell transplantation
Arm/Group Title CD34+ Cell Positively Selected Graft Stem Cell Recipient
Hide Arm/Group Description:
Recipients received a myeloablative conditioning regimen of cyclophosphamide (120 mg/kg total), fludarabine (125 mg/m2 total) and total body irradiation (1200 cGy with lung shielding to 600 cGy), followed by an infusion of a stem cell product selected for CD34+ progenitors using the Miltenyi CliniMACS® system. Older subjects will receive a lower dose of irradiation (800 or 600 cGy based on age) to reduce the regimen intensity.
Overall Number of Participants Analyzed 40
Measure Type: Count of Participants
Unit of Measure: Participants
11
  27.5%
4.Secondary Outcome
Title Incidence of Acute GVHD
Hide Description Participants who develop acute GVHD
Time Frame 100 days
Hide Outcome Measure Data
Hide Analysis Population Description
Subjects for whom the Miltenyi CliniMACS CD34 selection system was used for graft manipulation in HLA-matched sibling allogeneic stem cell transplantation
Arm/Group Title CD34+ Cell Positively Selected Graft Stem Cell Recipient
Hide Arm/Group Description:
Recipients received a myeloablative conditioning regimen of cyclophosphamide (120 mg/kg total), fludarabine (125 mg/m2 total) and total body irradiation (1200 cGy with lung shielding to 600 cGy), followed by an infusion of a stem cell product selected for CD34+ progenitors using the Miltenyi CliniMACS® system. Older subjects will receive a lower dose of irradiation (800 or 600 cGy based on age) to reduce the regimen intensity.
Overall Number of Participants Analyzed 40
Measure Type: Count of Participants
Unit of Measure: Participants
26
  65.0%
5.Secondary Outcome
Title Incidence of Chronic GVHD
Hide Description Participants who develop chronic GVHD
Time Frame 3 years
Hide Outcome Measure Data
Hide Analysis Population Description
Subjects for whom the Miltenyi CliniMACS CD34 selection system was used for graft manipulation in HLA-matched sibling allogeneic stem cell transplantation
Arm/Group Title CD34+ Cell Positively Selected Graft Stem Cell Recipient
Hide Arm/Group Description:
Recipients received a myeloablative conditioning regimen of cyclophosphamide (120 mg/kg total), fludarabine (125 mg/m2 total) and total body irradiation (1200 cGy with lung shielding to 600 cGy), followed by an infusion of a stem cell product selected for CD34+ progenitors using the Miltenyi CliniMACS® system. Older subjects will receive a lower dose of irradiation (800 or 600 cGy based on age) to reduce the regimen intensity.
Overall Number of Participants Analyzed 40
Measure Type: Count of Participants
Unit of Measure: Participants
14
  35.0%
6.Secondary Outcome
Title Median Time to ANC>500/mm3
Hide Description Time in days
Time Frame 3 years
Hide Outcome Measure Data
Hide Analysis Population Description
Subjects for whom the Miltenyi CliniMACS CD34 selection system was used for graft manipulation in HLA-matched sibling allogeneic stem cell transplantation
Arm/Group Title CD34+ Cell Positively Selected Graft Stem Cell Recipient
Hide Arm/Group Description:
Recipients received a myeloablative conditioning regimen of cyclophosphamide (120 mg/kg total), fludarabine (125 mg/m2 total) and total body irradiation (1200 cGy with lung shielding to 600 cGy), followed by an infusion of a stem cell product selected for CD34+ progenitors using the Miltenyi CliniMACS® system. Older subjects will receive a lower dose of irradiation (800 or 600 cGy based on age) to reduce the regimen intensity.
Overall Number of Participants Analyzed 40
Median (95% Confidence Interval)
Unit of Measure: Days
12
(9 to 28)
7.Secondary Outcome
Title Median Time to Platelets>20K/mm3
Hide Description Time in days
Time Frame 3 years
Hide Outcome Measure Data
Hide Analysis Population Description
Subjects for whom the Miltenyi CliniMACS CD34 selection system was used for graft manipulation in HLA-matched sibling allogeneic stem cell transplantation
Arm/Group Title CD34+ Cell Positively Selected Graft Stem Cell Recipient
Hide Arm/Group Description:
Recipients received a myeloablative conditioning regimen of cyclophosphamide (120 mg/kg total), fludarabine (125 mg/m2 total) and total body irradiation (1200 cGy with lung shielding to 600 cGy), followed by an infusion of a stem cell product selected for CD34+ progenitors using the Miltenyi CliniMACS® system. Older subjects will receive a lower dose of irradiation (800 or 600 cGy based on age) to reduce the regimen intensity.
Overall Number of Participants Analyzed 40
Median (95% Confidence Interval)
Unit of Measure: Days
15
(14 to 28)
8.Secondary Outcome
Title Severity of Acute GVHD
Hide Description Grade I
Time Frame 100 days
Hide Outcome Measure Data
Hide Analysis Population Description
Subjects for whom the Miltenyi CliniMACS CD34 selection system was used for graft manipulation in HLA-matched sibling allogeneic stem cell transplantation
Arm/Group Title CD34+ Cell Positively Selected Graft Stem Cell Recipient
Hide Arm/Group Description:
Recipients received a myeloablative conditioning regimen of cyclophosphamide (120 mg/kg total), fludarabine (125 mg/m2 total) and total body irradiation (1200 cGy with lung shielding to 600 cGy), followed by an infusion of a stem cell product selected for CD34+ progenitors using the Miltenyi CliniMACS® system. Older subjects will receive a lower dose of irradiation (800 or 600 cGy based on age) to reduce the regimen intensity.
Overall Number of Participants Analyzed 40
Measure Type: Count of Participants
Unit of Measure: Participants
10
  25.0%
9.Secondary Outcome
Title Severity of Acute GVHD
Hide Description Grade II
Time Frame 100 days
Hide Outcome Measure Data
Hide Analysis Population Description
Subjects for whom the Miltenyi CliniMACS CD34 selection system was used for graft manipulation in HLA-matched sibling allogeneic stem cell transplantation
Arm/Group Title CD34+ Cell Positively Selected Graft Stem Cell Recipient
Hide Arm/Group Description:
Recipients received a myeloablative conditioning regimen of cyclophosphamide (120 mg/kg total), fludarabine (125 mg/m2 total) and total body irradiation (1200 cGy with lung shielding to 600 cGy), followed by an infusion of a stem cell product selected for CD34+ progenitors using the Miltenyi CliniMACS® system. Older subjects will receive a lower dose of irradiation (800 or 600 cGy based on age) to reduce the regimen intensity.
Overall Number of Participants Analyzed 40
Measure Type: Count of Participants
Unit of Measure: Participants
7
  17.5%
10.Secondary Outcome
Title Severity of Acute GVHD
Hide Description Grade III
Time Frame 100 days
Hide Outcome Measure Data
Hide Analysis Population Description
Subjects for whom the Miltenyi CliniMACS CD34 selection system was used for graft manipulation in HLA-matched sibling allogeneic stem cell transplantation
Arm/Group Title CD34+ Cell Positively Selected Graft Stem Cell Recipient
Hide Arm/Group Description:
Recipients received a myeloablative conditioning regimen of cyclophosphamide (120 mg/kg total), fludarabine (125 mg/m2 total) and total body irradiation (1200 cGy with lung shielding to 600 cGy), followed by an infusion of a stem cell product selected for CD34+ progenitors using the Miltenyi CliniMACS® system. Older subjects will receive a lower dose of irradiation (800 or 600 cGy based on age) to reduce the regimen intensity.
Overall Number of Participants Analyzed 40
Measure Type: Count of Participants
Unit of Measure: Participants
8
  20.0%
11.Secondary Outcome
Title Severity of Acute GVHD
Hide Description Grade IV
Time Frame 100 days
Hide Outcome Measure Data
Hide Analysis Population Description
Subjects for whom the Miltenyi CliniMACS CD34 selection system was used for graft manipulation in HLA-matched sibling allogeneic stem cell transplantation
Arm/Group Title CD34+ Cell Positively Selected Graft Stem Cell Recipient
Hide Arm/Group Description:
Recipients received a myeloablative conditioning regimen of cyclophosphamide (120 mg/kg total), fludarabine (125 mg/m2 total) and total body irradiation (1200 cGy with lung shielding to 600 cGy), followed by an infusion of a stem cell product selected for CD34+ progenitors using the Miltenyi CliniMACS® system. Older subjects will receive a lower dose of irradiation (800 or 600 cGy based on age) to reduce the regimen intensity.
Overall Number of Participants Analyzed 40
Measure Type: Count of Participants
Unit of Measure: Participants
1
   2.5%
12.Secondary Outcome
Title Severity of Chronic GVHD
Hide Description Extensive
Time Frame 3 years
Hide Outcome Measure Data
Hide Analysis Population Description
Subjects for whom the Miltenyi CliniMACS CD34 selection system was used for graft manipulation in HLA-matched sibling allogeneic stem cell transplantation
Arm/Group Title CD34+ Cell Positively Selected Graft Stem Cell Recipient
Hide Arm/Group Description:
Recipients received a myeloablative conditioning regimen of cyclophosphamide (120 mg/kg total), fludarabine (125 mg/m2 total) and total body irradiation (1200 cGy with lung shielding to 600 cGy), followed by an infusion of a stem cell product selected for CD34+ progenitors using the Miltenyi CliniMACS® system. Older subjects will receive a lower dose of irradiation (800 or 600 cGy based on age) to reduce the regimen intensity.
Overall Number of Participants Analyzed 40
Measure Type: Count of Participants
Unit of Measure: Participants
10
  25.0%
13.Secondary Outcome
Title Severity of Chronic GVHD
Hide Description Limited
Time Frame 3 years
Hide Outcome Measure Data
Hide Analysis Population Description
Subjects for whom the Miltenyi CliniMACS CD34 selection system was used for graft manipulation in HLA-matched sibling allogeneic stem cell transplantation
Arm/Group Title CD34+ Cell Positively Selected Graft Stem Cell Recipient
Hide Arm/Group Description:
Recipients received a myeloablative conditioning regimen of cyclophosphamide (120 mg/kg total), fludarabine (125 mg/m2 total) and total body irradiation (1200 cGy with lung shielding to 600 cGy), followed by an infusion of a stem cell product selected for CD34+ progenitors using the Miltenyi CliniMACS® system. Older subjects will receive a lower dose of irradiation (800 or 600 cGy based on age) to reduce the regimen intensity.
Overall Number of Participants Analyzed 40
Measure Type: Count of Participants
Unit of Measure: Participants
4
  10.0%
Time Frame 3 years
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title CD34+ Cell Positively Selected Graft Stem Cell Recipient
Hide Arm/Group Description Recipients received a myeloablative conditioning regimen of cyclophosphamide (120 mg/kg total), fludarabine (125 mg/m2 total) and total body irradiation (1200 cGy with lung shielding to 600 cGy), followed by an infusion of a stem cell product selected for CD34+ progenitors using the Miltenyi CliniMACS® system. Older subjects will receive a lower dose of irradiation (800 or 600 cGy based on age) to reduce the regimen intensity.
All-Cause Mortality
CD34+ Cell Positively Selected Graft Stem Cell Recipient
Affected / at Risk (%)
Total   11/40 (27.50%) 
Hide Serious Adverse Events
CD34+ Cell Positively Selected Graft Stem Cell Recipient
Affected / at Risk (%)
Total   38/40 (95.00%) 
Blood and lymphatic system disorders   
Anaemia   1/40 (2.50%) 
Engraftment syndrome   1/40 (2.50%) 
Febrile neutropenia   1/40 (2.50%) 
Thrombocytopenia   1/40 (2.50%) 
Cardiac disorders   
Cardiac failure congestive   1/40 (2.50%) 
Cardiomyopathy acute   1/40 (2.50%) 
Diastolic dysfunction   1/40 (2.50%) 
Fluid overload   2/40 (5.00%) 
Tachycardia   1/40 (2.50%) 
Gastrointestinal disorders   
Abdominal discomfort   1/40 (2.50%) 
Appendicitis   1/40 (2.50%) 
Clostridium difficile colitis   1/40 (2.50%) 
Constipation   1/40 (2.50%) 
Cytomegalovirus gastrointestinal infection   1/40 (2.50%) 
Diarrhoea   3/40 (7.50%) 
Gastritis   1/40 (2.50%) 
Gastroenteritis astroviral   1/40 (2.50%) 
Gastrointestinal haemorrhage   1/40 (2.50%) 
Nausea   1/40 (2.50%) 
Viral diarrhoea   1/40 (2.50%) 
Vomiting   1/40 (2.50%) 
General disorders   
Adult failure to thrive   1/40 (2.50%) 
Catheter site pain   1/40 (2.50%) 
Drug withdrawal convulsions   1/40 (2.50%) 
Drug withdrawal syndrome   1/40 (2.50%) 
Incarcerated hernia   1/40 (2.50%) 
Pain   1/40 (2.50%) 
Immune system disorders   
Acute graft versus host disease in intestine   7/40 (17.50%) 
Acute graft versus host disease in skin   3/40 (7.50%) 
Chronic graft versus host disease   1/40 (2.50%) 
Chronic graft versus host disease in intestine   3/40 (7.50%) 
Food allergy   1/40 (2.50%) 
Reaction to preservatives   1/40 (2.50%) 
Transplant rejection   1/40 (2.50%) 
Infections and infestations   
Adenovirus infection   2/40 (5.00%) 
Bacteraemia   6/40 (15.00%) 
BK virus infection   2/40 (5.00%) 
Candida infection   1/40 (2.50%) 
Cellulitis   1/40 (2.50%) 
Clostridium difficile infection   5/40 (12.50%) 
Cystitis   1/40 (2.50%) 
Cytomegalovirus infection   7/40 (17.50%) 
Epstein-Barr viraemia   1/40 (2.50%) 
Escherichia bacteraemia   3/40 (7.50%) 
Escherichia sepsis   2/40 (5.00%) 
Fungaemia   1/40 (2.50%) 
Influenza   1/40 (2.50%) 
Klebsiella infection   1/40 (2.50%) 
Lung infection   1/40 (2.50%) 
Pneumonia   5/40 (12.50%) 
Pneumonia fungal   1/40 (2.50%) 
Pyelonephritis   1/40 (2.50%) 
Salmonella sepsis   1/40 (2.50%) 
Sepsis   4/40 (10.00%) 
Septic shock   1/40 (2.50%) 
Stenotrophomonas infection   1/40 (2.50%) 
suspected infection   5/40 (12.50%) 
Upper respiratory tract infection   1/40 (2.50%) 
Urinary tract infection   3/40 (7.50%) 
Varicella zoster virus infection   1/40 (2.50%) 
Vulvovaginal human papilloma virus infection   1/40 (2.50%) 
Wound abscess   1/40 (2.50%) 
Injury, poisoning and procedural complications   
Delayed engraftment   1/40 (2.50%) 
Engraft failure   1/40 (2.50%) 
Humerus fracture   1/40 (2.50%) 
Procedural headache   1/40 (2.50%) 
Investigations   
Anion gap   1/40 (2.50%) 
Body temperature increased   12/40 (30.00%) 
Hyperkalaemia   1/40 (2.50%) 
Nervous system disorders   
Headache   1/40 (2.50%) 
Orthostatic hypotension   1/40 (2.50%) 
Posterior reversible encephalopathy syndrome   1/40 (2.50%) 
Psychiatric disorders   
Altered state of consciousness   1/40 (2.50%) 
Mental status changes   1/40 (2.50%) 
Respiratory, thoracic and mediastinal disorders   
Pulmonary embolism   1/40 (2.50%) 
Surgical and medical procedures   
Mucositis management   1/40 (2.50%) 
Vascular disorders   
Orthostatic hypotension   1/40 (2.50%) 
Syncope   1/40 (2.50%) 
Indicates events were collected by systematic assessment
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
CD34+ Cell Positively Selected Graft Stem Cell Recipient
Affected / at Risk (%)
Total   0/40 (0.00%) 
1 participant signed consent but did not start study.
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: Aue, Georg
Organization: National Heart Lung and Blood Institute
Phone: +1 301 451 7141
EMail: aueg@nhlbi.nih.gov
Layout table for additonal information
Responsible Party: National Institutes of Health Clinical Center (CC) ( National Heart, Lung, and Blood Institute (NHLBI) )
ClinicalTrials.gov Identifier: NCT01866839    
Other Study ID Numbers: 130144
13-H-0144
First Submitted: May 29, 2013
First Posted: June 3, 2013
Results First Submitted: May 1, 2020
Results First Posted: May 26, 2020
Last Update Posted: July 21, 2020