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Combined Haploidentical-Cord Blood Transplantation for Adults and Children

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ClinicalTrials.gov Identifier: NCT00943800
Recruitment Status : Completed
First Posted : July 22, 2009
Results First Posted : October 27, 2020
Last Update Posted : January 27, 2021
Sponsor:
Information provided by (Responsible Party):
University of Chicago

Study Type Interventional
Study Design Allocation: Non-Randomized;   Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Conditions Leukemia
Myelodysplastic Syndrome
Multiple Myeloma
Lymphoma
Interventions Drug: Fludarabine-Melphalan & Rabbit antithymocyte globulin (r-ATG)
Procedure: Stem Cell Transplant
Procedure: Stem Cells Collections
Drug: Fludarabine, Thiotepa, Antithymocyte globulin (ATG), and Total Body Irradiation (TBI)
Drug: Fludarabine, Busulfan, and ATG
Enrollment 87
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Treatment Group
Hide Arm/Group Description

Although the protocol initially had different disease intensities, ultimately, moved to one regimen for all patients.

Subjects received 30mg/m^2 of Fludarabine from Day-7 until Day-3, 140mg/m^2 of Melphalan on Day-2, and 1.5mg/kg of Rabbit antithymocyte globulin at Day-7,-5,-3 and -1."

Period Title: Overall Study
Started 87
Completed 87
Not Completed 0
Arm/Group Title Treatment Group
Hide Arm/Group Description Although the protocol initially had different disease intensities, ultimately, moved to one regimen for all patients. Subjects received 30mg/m^2 of Fludarabine from Day-7 until Day-3, 140mg/m^2 of Melphalan on Day-2, and 1.5mg/kg of Rabbit antithymocyte globulin at Day-7,-5,-3 and -1."
Overall Number of Baseline Participants 87
Hide Baseline Analysis Population Description
[Not Specified]
Age, Continuous  
Mean (Full Range)
Unit of measure:  Years
Number Analyzed 87 participants
45.5
(10 to 69)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 87 participants
Female
36
  41.4%
Male
51
  58.6%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 87 participants
American Indian or Alaska Native
1
   1.1%
Asian
3
   3.4%
Native Hawaiian or Other Pacific Islander
0
   0.0%
Black or African American
21
  24.1%
White
53
  60.9%
More than one race
0
   0.0%
Unknown or Not Reported
9
  10.3%
1.Primary Outcome
Title Percentage of Participants With Neutrophil Engraftment
Hide Description Cumulative incidence of graft failure (neutrophil) by day 28 was reported. Patients who did not have neutrophil engraftment before death was considered as a competing risk. Failure to engraft was defined as lack of evidence of hematopoietic recovery (ANC <500/mm3 and platelet count < 20,000/mm3) by day +35, confirmed by a biopsy revealing a marrow cellularity < 5%. Graft failure was also defined as initial myeloid engraftment by day +35, documented to be of donor origin, followed by a drop in the ANC to < 500/mm3 for more than three days, independent of any myelosuppressive drugs, severe GVHD, CMV, or other infection.
Time Frame Transplant (Day 0) through Day +28
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Treatment Group
Hide Arm/Group Description:
Although the protocol initially had different disease intensities, ultimately, moved to one regimen for all patients. Subjects received 30mg/m^2 of Fludarabine from Day-7 until Day-3, 140mg/m^2 of Melphalan on Day-2, and 1.5mg/kg of Rabbit antithymocyte globulin at Day-7,-5,-3 and -1."
Overall Number of Participants Analyzed 87
Measure Type: Number
Number (95% Confidence Interval)
Unit of Measure: percentage of patients
85.1
(75.4 to 91.1)
2.Secondary Outcome
Title Percentage of Participants With Incidence of Acute (Grade II-IV) and Chronic Graft-vs-host Disease(GVHD)
Hide Description

Acute GVHD is defined by the Przepiorka criteria, which stages the degree of organ involvement in the skin, liver, and gastrointestinal (GI) tract, based on severity, with Stage 1+ being least severe and stage 4+ being the most severe. Grading of acute GVHD is as follows: Grade II (skin involvement stages 1+ to 3+, liver 1+, GI tract 1+), Grade III (skin involvement stages 2+ to 3+, liver 1+, GI tract 2+ to 4+), Grade IV (skin involvement stages 4+, Liver 4+).

Chronic GVHD is assessed by NIH consensus development project on criteria for clinical trials in chronic graft-versus-host disease: I. Diagnosis and staging working group report. Biol Blood Marrow Transplant. 2005; 11:945-56., for grading criteria. (See Citation: Filipovich AH et al)

The incidence of patients with acute GVHD (Grade II-IV) was determined at 180 days. The incidence of Chronic GVHD by 2 years was reported

Time Frame Up to 2 years
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Treatment Group
Hide Arm/Group Description:
Although the protocol initially had different disease intensities, ultimately, moved to one regimen for all patients. Subjects received 30mg/m^2 of Fludarabine from Day-7 until Day-3, 140mg/m^2 of Melphalan on Day-2, and 1.5mg/kg of Rabbit antithymocyte globulin at Day-7,-5,-3 and -1."
Overall Number of Participants Analyzed 87
Measure Type: Number
Number (95% Confidence Interval)
Unit of Measure: percentage of patients
Acute GVHD (grade II-IV)
16.1
(9.3 to 24.6)
Chronic GVHD
3.4
(0.9 to 9.0)
3.Secondary Outcome
Title Overall Survival- Percentage of Participants Who Survived at 2 Years and 5 Years
Hide Description We reported overall survival at 2 years and 5 years after transplant
Time Frame up to 5 years
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Treatment Group
Hide Arm/Group Description:
Although the protocol initially had different disease intensities, ultimately, moved to one regimen for all patients. Subjects received 30mg/m^2 of Fludarabine from Day-7 until Day-3, 140mg/m^2 of Melphalan on Day-2, and 1.5mg/kg of Rabbit antithymocyte globulin at Day-7,-5,-3 and -1."
Overall Number of Participants Analyzed 87
Measure Type: Number
Number (95% Confidence Interval)
Unit of Measure: percentage of patients
Two-year overall survival
43.7
(34.4 to 55.4)
Five-year overall survival
32.9
(24.3 to 44.5)
Time Frame 100 days
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Treatment Group
Hide Arm/Group Description Although the protocol initially had different disease intensities, ultimately, moved to one regimen for all patients. Subjects received 30mg/m^2 of Fludarabine from Day-7 until Day-3, 140mg/m^2 of Melphalan on Day-2, and 1.5mg/kg of Rabbit antithymocyte globulin at Day-7,-5,-3 and -1."
All-Cause Mortality
Treatment Group
Affected / at Risk (%)
Total   18/87 (20.69%) 
Hide Serious Adverse Events
Treatment Group
Affected / at Risk (%)
Total   24/87 (27.59%) 
Blood and lymphatic system disorders   
Anemia   1/87 (1.15%) 
Febrile neutropenia   1/87 (1.15%) 
Cardiac disorders   
Heart failure   1/87 (1.15%) 
Pericardial tamponade   1/87 (1.15%) 
Gastrointestinal disorders   
Diarrhea   1/87 (1.15%) 
Fever   1/87 (1.15%) 
General disorders   
Gait disturbance   1/87 (1.15%) 
Immune system disorders   
Cytokine release syndrome   1/87 (1.15%) 
Infections and infestations   
Abdominal infection   2/87 (2.30%) 
Epstein-barr virus infection   3/87 (3.45%) 
Esophageal infection   1/87 (1.15%) 
Lung infection   2/87 (2.30%) 
Sepsis   11/87 (12.64%) 
Skin infection   1/87 (1.15%) 
Investigations   
Neutrophil count decreased   1/87 (1.15%) 
White blood cell decreased   1/87 (1.15%) 
Metabolism and nutrition disorders   
Hypernatremia   1/87 (1.15%) 
Respiratory, thoracic and mediastinal disorders   
Hypoxia   3/87 (3.45%) 
Respiratory failure   4/87 (4.60%) 
Indicates events were collected by systematic assessment
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Treatment Group
Affected / at Risk (%)
Total   0/87 (0.00%) 
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: Hongtao Liu
Organization: University of Chicago
Phone: 773-834-8980
EMail: hliu2@medicine.bsd.uchicago.edu
Layout table for additonal information
Responsible Party: University of Chicago
ClinicalTrials.gov Identifier: NCT00943800    
Other Study ID Numbers: 14736B
First Submitted: July 20, 2009
First Posted: July 22, 2009
Results First Submitted: October 5, 2020
Results First Posted: October 27, 2020
Last Update Posted: January 27, 2021