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A Two-Step Approach to Bone Marrow Transplant Using Cells From A Partially-Matched Relative

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ClinicalTrials.gov Identifier: NCT00429143
Recruitment Status : Completed
First Posted : January 31, 2007
Results First Posted : May 31, 2013
Last Update Posted : November 29, 2016
Sponsor:
Information provided by (Responsible Party):
Thomas Jefferson University ( Sidney Kimmel Cancer Center at Thomas Jefferson University )

Study Type Interventional
Study Design Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Hematologic Malignancies
Interventions Radiation: Total Body Irradiation (TBI)
Biological: Donor Lymphocyte Infusion (DLI)
Drug: Cyclophosphamide (CY)
Drug: Tacrolimus
Drug: Mycophenolate Mofetil (MMF)
Biological: Hematopoietic Stem Cell Transplant (HSCT)
Enrollment 27
Recruitment Details Patients presenting to Thomas Jefferson University with hematological malignancies requiring hematopoeitic stem cell transplantation without matched related donors. Opened January, 2006 through August, 2009
Pre-assignment Details  
Arm/Group Title Haploidentical Allogeneic Transplantation
Hide Arm/Group Description Patients undergoing hematopoietic stem cell transplant from a partially matched related donor
Period Title: Overall Study
Started 27
Completed 27
Not Completed 0
Arm/Group Title Haploidentical Allogeneic Transplantation
Hide Arm/Group Description Patients undergoing hematopoietic stem cell transplant from a partially matched related donor
Overall Number of Baseline Participants 27
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 27 participants
<=18 years
0
   0.0%
Between 18 and 65 years
24
  88.9%
>=65 years
3
  11.1%
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 27 participants
50  (12.9)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 27 participants
Female
16
  59.3%
Male
11
  40.7%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 27 participants
27
1.Primary Outcome
Title Overall Survival of Participants
Hide Description To determine overall survival at 6 months post-transplant.
Time Frame 6 months
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
27 Patients undergoing haploidentical transplant at Thomas Jefferson University
Arm/Group Title Haploidentical Allogeneic Transplantation
Hide Arm/Group Description:
Patients undergoing hematopoietic stem cell transplant from a partially matched related donor
Overall Number of Participants Analyzed 27
Measure Type: Number
Unit of Measure: participants
13
2.Primary Outcome
Title Optimal Dose of CD3+ Donor Lymphocytes (T-cells) for Consistent Engraftment Without GVHD
Hide Description

To determine the optimal dose of CD3+ donor lymphocytes required for consistent engraftment without the development of grade III/IV GVHD.

Measured as CD3+ donor lymphocytes given as n x 10^8/kg.

"n" was found to be 2 and was found to be the optimal dose and was the only dose given.

Time Frame 6 months
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Two patients died prior to expected day of engraftment
Arm/Group Title Haploidentical Allogeneic Transplantation
Hide Arm/Group Description:
Patients undergoing hematopoietic stem cell transplant from a partially matched related donor
Overall Number of Participants Analyzed 25
Measure Type: Number
Unit of Measure: lymphocytes x 10^8/kg
2
3.Secondary Outcome
Title Engraftment Rates
Hide Description To assess hematopoietic engraftment rates.
Time Frame 6 months
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Two patients died prior to expected engraftment day
Arm/Group Title Haploidentical Allogeneic Transplantation
Hide Arm/Group Description:
Patients undergoing hematopoietic stem cell transplant from a partially matched related donor
Overall Number of Participants Analyzed 25
Measure Type: Number
Unit of Measure: participants
23
4.Secondary Outcome
Title Lymphoid Recovery
Hide Description To assess the pace of lymphoid recovery in this patient population.
Time Frame 6 months
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Two patients did not engraft, two patients died prior to expected day of engraftment
Arm/Group Title Haploidentical Allogeneic Transplantation
Hide Arm/Group Description:
Patients undergoing hematopoietic stem cell transplant from a partially matched related donor
Overall Number of Participants Analyzed 23
Measure Type: Number
Unit of Measure: participants
23
5.Secondary Outcome
Title Incidence of Grades III-IV GVHD
Hide Description

To determine the incidence and severity of GVHD in these patients using a combination of cyclophosphamide, tacrolimus and mycophenolate mofetil (MMF) as GVHD prophylaxis.'

Severity was graded using CTCAE 3.0 (1=mild, 2=moderate, 3=severe, 4=life threatening/disabling, 5=death)

Time Frame 6 months
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Two patients died prior to expected engraftment. Two patients who rejected were retransplanted and were evaluable for GVHD.
Arm/Group Title Haploidentical Allogeneic Transplantation
Hide Arm/Group Description:
Patients undergoing hematopoietic stem cell transplant from a partially matched related donor
Overall Number of Participants Analyzed 25
Measure Type: Number
Unit of Measure: participants
2
Time Frame 6 Months
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Haploidentical Allogeneic Transplantation
Hide Arm/Group Description Patients undergoing hematopoietic stem cell transplant from a partially matched related donor
All-Cause Mortality
Haploidentical Allogeneic Transplantation
Affected / at Risk (%)
Total   --/--    
Show Serious Adverse Events Hide Serious Adverse Events
Haploidentical Allogeneic Transplantation
Affected / at Risk (%) # Events
Total   23/27 (85.19%)    
Gastrointestinal disorders   
Diarrhea  1  2/27 (7.41%)  2
Nausea  1  1/27 (3.70%)  1
General disorders   
Death - Disease progression NOS  1  5/27 (18.52%)  5
Death - Multi-organ failure  1  3/27 (11.11%)  3
Constitutional Symptoms – Other  1  1/27 (3.70%)  1
Death - Death NOS  1  2/27 (7.41%)  2
Hepatobiliary disorders   
Liver dysfunction/failure  1 [1]  1/27 (3.70%)  1
Immune system disorders   
Cytokine release syndrome/acute infusion reaction  1  1/27 (3.70%)  1
Infections and infestations   
Opportunistic infection associated with >=Grade 2 Lymphopenia  1  8/27 (29.63%)  9
Infection - Other  1  4/27 (14.81%)  4
Infection with normal ANC or Grade 1 or 2 neutrophils  1  2/27 (7.41%)  2
Renal and urinary disorders   
Renal/Genitourinary – Other  1  1/27 (3.70%)  1
Respiratory, thoracic and mediastinal disorders   
Pneumonitis/pulmonary infiltrates  1  1/27 (3.70%)  1
Hypoxia  1  1/27 (3.70%)  1
Vascular disorders   
Thrombosis/embolism  1  1/27 (3.70%)  1
Indicates events were collected by systematic assessment
1
Term from vocabulary, CTCAE (3.0)
[1]
Hepatic failure
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Haploidentical Allogeneic Transplantation
Affected / at Risk (%) # Events
Total   27/27 (100.00%)    
Gastrointestinal disorders   
Mucositis/stomatitis  1  23/27 (85.19%)  23
Metabolism and nutrition disorders   
Bilirubin  1  3/27 (11.11%)  3
Renal and urinary disorders   
Renal/Genitourinary – Other  1  2/27 (7.41%)  2
Indicates events were collected by systematic assessment
1
Term from vocabulary, CTCAE (3.0)
Limitations of the trial include small sample size and single institution
Certain Agreements
All Principal Investigators ARE employed by the organization sponsoring the study.
Results Point of Contact
Name/Title: Neal Flomenberg, MD
Organization: Thomas Jefferson University
Phone: 215-955-4367
Responsible Party: Thomas Jefferson University ( Sidney Kimmel Cancer Center at Thomas Jefferson University )
ClinicalTrials.gov Identifier: NCT00429143     History of Changes
Other Study ID Numbers: 06U.20
2005-84 ( Other Identifier: CCRRC )
First Submitted: January 29, 2007
First Posted: January 31, 2007
Results First Submitted: June 4, 2012
Results First Posted: May 31, 2013
Last Update Posted: November 29, 2016