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Pilot and Feasibility Study of Reduced-Intensity Hematopoietic Stem Cell Transplant for MonoMAC

This study has been completed.
Sponsor:
ClinicalTrials.gov Identifier:
NCT00923364
First Posted: June 18, 2009
Last Update Posted: November 30, 2017
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
Information provided by (Responsible Party):
Dennis Hickstein, M.D., National Institutes of Health Clinical Center (CC)
Results First Submitted: May 3, 2017  
Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Conditions: MDS
Immunodeficiency
GATA2
Interventions: Drug: Cyclophosphamide (CTX, Cytoxan)
Drug: Fludarabine(Fludara,Berlex Laboratories)
Procedure: Total Body Irradiation (TBI)
Procedure: Allogeneic Hematopoietic Stem Cell (HSC)
Drug: Equine Anti-Thymocyte Globulin

  Participant Flow
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Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
No text entered.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
No text entered.

Reporting Groups
  Description
Recipients

Hematopoietic Stem Cell Transplant for MonoMAC: 10/10 Human Leukocyte Antigen (HLA) Matched Related Donor (MRD) or Unrelated Donor (URD) Transplant. 9/10 HLA Matched Related Donor or Unrelated Donor Transplant. Haploidentical Related Donor Transplant. Umbilical Cord Blood Transplant.

Fludarabine(Fludara,Berlex Laboratories): 40 mg/m(2) intravenous (IV) (in the vein) over 30 minutes (in the vein) once daily on Days -6, -5, -4, and -3 or 30 mg/m(2) IV over 30 minutes (in the vein) once daily on Days -6, -5, -4, -3, and -2

Total Body Irradiation (TBI): 200 centigray (cGy) on Day -1 or 300 cGy on Day -1 (for 9/10 URD and Haplo patients)

Allogeneic hematopoietic stem cell (HSC): stem cell transplant

Healthy Related Donors No text entered.

Participant Flow:   Overall Study
    Recipients   Healthy Related Donors
STARTED   14   5 
COMPLETED   10   5 
NOT COMPLETED   4   0 
Death                3                0 
Did not go to transplant                1                0 



  Baseline Characteristics
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Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
Data were not collected separately for donors and recipients.

Reporting Groups
  Description
Recipients and Healthy Related Donors

Hematopoietic Stem Cell Transplant for MonoMAC: 10/10 Human Leukocyte Antigen (HLA) Matched Related Donor (MRD) or Unrelated Donor (URD) Transplant. 9/10 HLA Matched Related Donor or Unrelated Donor Transplant. Haploidentical Related Donor Transplant. Umbilical Cord Blood Transplant.

Fludarabine(Fludara,Berlex Laboratories): 40 mg/m(2) intravenous (IV) (in the vein) over 30 minutes (in the vein) once daily on Days -6, -5, -4, and -3 or 30 mg/m(2) IV over 30 minutes (in the vein) once daily on Days -6, -5, -4, -3, and -2

Total Body Irradiation (TBI): 200 centigray (cGy) on Day -1 or 300 cGy on Day -1 (for 9/10 URD and Haplo patients)

Allogeneic hematopoietic stem cell (HSC): stem cell transplant Healthy related donors =5; recipients = 14.


Baseline Measures
   Recipients and Healthy Related Donors 
Overall Participants Analyzed 
[Units: Participants]
 19 
Age 
[Units: Participants]
Count of Participants
 
<=18 years      1   5.3% 
Between 18 and 65 years      18  94.7% 
>=65 years      0   0.0% 
Age 
[Units: Years]
Mean (Standard Deviation)
 30.21  (7.61) 
Sex: Female, Male 
[Units: Participants]
Count of Participants
 
Female      11  57.9% 
Male      8  42.1% 
Ethnicity (NIH/OMB) 
[Units: Participants]
Count of Participants
 
Hispanic or Latino      3  15.8% 
Not Hispanic or Latino      16  84.2% 
Unknown or Not Reported      0   0.0% 
Race (NIH/OMB) 
[Units: Participants]
Count of Participants
 
American Indian or Alaska Native      0   0.0% 
Asian      2  10.5% 
Native Hawaiian or Other Pacific Islander      0   0.0% 
Black or African American      2  10.5% 
White      15  78.9% 
More than one race      0   0.0% 
Unknown or Not Reported      0   0.0% 
Region of Enrollment 
[Units: Participants]
Count of Participants
 
United States   19 


  Outcome Measures
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1.  Primary:   Days to Neutrophil Engraftment   [ Time Frame: 30 days ]

2.  Primary:   Days to Platelet Engraftment   [ Time Frame: 30 days ]

3.  Secondary:   Percentage of Donor Cells at Last Follow Up in Patients With Mutations GATA Binding Protein 2 (GATA2)   [ Time Frame: 2 years ]

4.  Secondary:   Incidence of Acute and Chronic Graft-versus-host Disease (GVHD)   [ Time Frame: 2 years ]

5.  Secondary:   Overall Survival   [ Time Frame: 2 years ]

6.  Secondary:   Number of Participants With Disease Free Survival   [ Time Frame: 2 years ]

7.  Secondary:   Number of Participants With Serious and Non-serious Adverse Events   [ Time Frame: 91 months ]


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats
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Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
No text entered.


  More Information
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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:  
Name/Title: Dr. Dennis Hickstein
Organization: National Cancer Institute
phone: 301-594-1718
e-mail: hicksted@mail.nih.gov


Publications of Results:
Other Publications:
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

Responsible Party: Dennis Hickstein, M.D., National Institutes of Health Clinical Center (CC)
ClinicalTrials.gov Identifier: NCT00923364     History of Changes
Obsolete Identifiers: NCT00884923
Other Study ID Numbers: 090096
09-C-0096
First Submitted: June 17, 2009
First Posted: June 18, 2009
Results First Submitted: May 3, 2017
Results First Posted: November 30, 2017
Last Update Posted: November 30, 2017