Hematopoietic Stem Cell Transplantation (HSCT) for Children With SCID Utilizing Alemtuzumab, Plerixafor & Filgrastim

This study has been terminated.
(Insufficient evidence of efficacy)
Sponsor:
Information provided by (Responsible Party):
Christopher Dvorak, University of California, San Francisco
ClinicalTrials.gov Identifier:
NCT01182675
First received: August 9, 2010
Last updated: November 6, 2014
Last verified: November 2014
Results First Received: October 28, 2014  
Study Type: Interventional
Study Design: Allocation: Non-Randomized;   Endpoint Classification: Safety/Efficacy Study;   Intervention Model: Parallel Assignment;   Masking: Open Label;   Primary Purpose: Treatment
Condition: Severe Combined Immunodeficiency
Interventions: Drug: Transplant Conditioning with Mobilization Only
Drug: Transplant Conditioning with Mobilization and Alemtuzumab

  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
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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
T-cell Graft Permissive SCID

Patients with SCID with:

i. NK- phenotype; ii. NK+ phenotype with 10/10 HLA-matched relative or unrelated donor; or iii. NK+ phenotype with maternal engraftment by STR analysis and undergoing haplocompatible HSCT from maternal donor

Transplant Conditioning with Mobilization Only: Day -4: Filgrastim (G-CSF) 5 mcg/kg IV q12 hours; Day -3: Filgrastim (G-CSF) 5 mcg/kg IV q12 hours; Day -2: Filgrastim (G-CSF) 5 mcg/kg IV q12 hours; Day -1: Filgrastim (G-CSF) 5 mcg/kg IV q12 hours; Day 0: Plerixafor 240 mcg/kg subcutaneous 9-12 hours prior to transplant; Day 0 Transplant

T-cell Graft Resistant SCID

Patients with SCID with NK+ phenotype with HLA-mismatched donor

Transplant Conditioning with Mobilization and Alemtuzumab: Day -7: Alemtuzumab 0.3 mg test dose then 0.3 mg/kg IV; Day -6: Alemtuzumab 0.3 mg/kg IV; Day -5: Alemtuzumab 0.3 mg/kg IV; Day -4: Filgrastim (G-CSF) 5 mcg/kg IV q12 hours; Day -3: Filgrastim (G-CSF) 5 mcg/kg IV q12 hours; Day -2: Filgrastim (G-CSF) 5 mcg/kg IV q12 hours; Day -1: Filgrastim (G-CSF) 5 mcg/kg IV q12 hours; Day 0: Plerixafor 240 mcg/kg subcutaneous 9-12 hours prior to transplant; Day 0: Transplant


Participant Flow:   Overall Study
    T-cell Graft Permissive SCID     T-cell Graft Resistant SCID  
STARTED     7     0  
COMPLETED     7     0  
NOT COMPLETED     0     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.
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Reporting Groups
  Description
T-cell Graft Permissive SCID

Patients with SCID with:

i. NK- phenotype; ii. NK+ phenotype with 10/10 HLA-matched relative or unrelated donor; or iii. NK+ phenotype with maternal engraftment by STR analysis and undergoing haplocompatible HSCT from maternal donor

Transplant Conditioning with Mobilization Only: Day -4: Filgrastim (G-CSF) 5 mcg/kg IV q12 hours; Day -3: Filgrastim (G-CSF) 5 mcg/kg IV q12 hours; Day -2: Filgrastim (G-CSF) 5 mcg/kg IV q12 hours; Day -1: Filgrastim (G-CSF) 5 mcg/kg IV q12 hours; Day 0: Plerixafor 240 mcg/kg subcutaneous 9-12 hours prior to transplant; Day 0 Transplant

T-cell Graft Resistant SCID

Patients with SCID with NK+ phenotype with HLA-mismatched donor

Transplant Conditioning with Mobilization and Alemtuzumab: Day -7: Alemtuzumab 0.3 mg test dose then 0.3 mg/kg IV; Day -6: Alemtuzumab 0.3 mg/kg IV; Day -5: Alemtuzumab 0.3 mg/kg IV; Day -4: Filgrastim (G-CSF) 5 mcg/kg IV q12 hours; Day -3: Filgrastim (G-CSF) 5 mcg/kg IV q12 hours; Day -2: Filgrastim (G-CSF) 5 mcg/kg IV q12 hours; Day -1: Filgrastim (G-CSF) 5 mcg/kg IV q12 hours; Day 0: Plerixafor 240 mcg/kg subcutaneous 9-12 hours prior to transplant; Day 0: Transplant

Total Total of all reporting groups

Baseline Measures
    T-cell Graft Permissive SCID     T-cell Graft Resistant SCID     Total  
Number of Participants  
[units: participants]
  7     0     7  
Age  
[units: participants]
     
<=18 years     7         7  
Between 18 and 65 years     0         0  
>=65 years     0         0  
Gender  
[units: participants]
     
Female     2         2  
Male     5         5  



  Outcome Measures

1.  Primary:   Engraftment of Donor B-cells in Blood by STR Testing   [ Time Frame: 1 Year ]

2.  Secondary:   Incidence of Acute GVHD   [ Time Frame: 100 Days ]
Results not yet reported.   Anticipated Reporting Date:   No text entered.   Safety Issue:   Yes

3.  Secondary:   Incidence of Chronic GVHD   [ Time Frame: 2 Years ]
Results not yet reported.   Anticipated Reporting Date:   No text entered.   Safety Issue:   Yes

4.  Secondary:   Percentage of Patients Who Become Independent From Regular IVIG Infusion   [ Time Frame: 2 Years ]
Results not yet reported.   Anticipated Reporting Date:   No text entered.   Safety Issue:   No

5.  Secondary:   Percent Engraftment of Donor Stem Cells in Bone Marrow by STR Testing   [ Time Frame: 1 Year ]
Results not yet reported.   Anticipated Reporting Date:   No text entered.   Safety Issue:   No

6.  Secondary:   Percent Engraftment of Donor T-cells in Blood by STR Testing   [ Time Frame: 1 Year ]
Results not yet reported.   Anticipated Reporting Date:   No text entered.   Safety Issue:   No


  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
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  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: Christopher C. Dvorak, MD
Organization: UCSF
phone: 415-476-2188
e-mail: dvorakc@peds.ucsf.edu


Publications:

Responsible Party: Christopher Dvorak, University of California, San Francisco
ClinicalTrials.gov Identifier: NCT01182675     History of Changes
Study First Received: August 9, 2010
Results First Received: October 28, 2014
Last Updated: November 6, 2014
Health Authority: United States: Institutional Review Board