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

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ClinicalTrials.gov Identifier: NCT01182675
Recruitment Status : Terminated (Insufficient evidence of efficacy)
First Posted : August 17, 2010
Results First Posted : November 10, 2014
Last Update Posted : July 31, 2018
Sponsor:
Information provided by (Responsible Party):
Christopher Dvorak, University of California, San Francisco

Study Type Interventional
Study Design Allocation: Non-Randomized;   Intervention Model: Parallel Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Severe Combined Immunodeficiency
Interventions Drug: Transplant Conditioning with Mobilization Only
Drug: Transplant Conditioning with Mobilization and Alemtuzumab
Enrollment 7
Recruitment Details  
Pre-assignment Details  
Arm/Group Title T-cell Graft Permissive SCID T-cell Graft Resistant SCID
Hide Arm/Group Description

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

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

Period Title: Overall Study
Started 7 0
Completed 7 0
Not Completed 0 0
Arm/Group Title T-cell Graft Permissive SCID T-cell Graft Resistant SCID Total
Hide Arm/Group Description

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

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 of all reporting groups
Overall Number of Baseline Participants 7 0 7
Hide Baseline Analysis Population Description
No patients with T-cell Graft Resistant SCID were enrolled during the time the trial was open.
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 7 participants 0 participants 7 participants
<=18 years
7
 100.0%
7
 100.0%
Between 18 and 65 years
0
   0.0%
0
   0.0%
>=65 years
0
   0.0%
0
   0.0%
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 7 participants 0 participants 7 participants
Female
2
  28.6%
2
  28.6%
Male
5
  71.4%
5
  71.4%
1.Primary Outcome
Title Engraftment of Donor B-cells in Blood by STR Testing
Hide Description Number of participants in whom donor B cells were detected in the patient's blood after HSCT.
Time Frame 1 Year
Hide Outcome Measure Data
Hide Analysis Population Description
No patients with T-cell Graft Resistant SCID were enrolled during the time the trial was open.
Arm/Group Title T-cell Graft Permissive SCID T-cell Graft Resistant SCID
Hide Arm/Group Description:

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

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

Overall Number of Participants Analyzed 7 0
Measure Type: Number
Unit of Measure: participants
0
2.Secondary Outcome
Title Incidence of Acute GVHD
Hide Description [Not Specified]
Time Frame 100 Days
Hide Outcome Measure Data
Hide Analysis Population Description
No patients with T-cell Graft Resistant SCID were enrolled during the time the trial was open.
Arm/Group Title T-cell Graft Permissive SCID T-cell Graft Resistant SCID
Hide Arm/Group Description:

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 Intervention: Transplant Conditioning with Mobilization Only

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

Patients with SCID with NK+ phenotype with HLA-mismatched donor Intervention: Transplant Conditioning with Mobilization and Alemtuzumab

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

Overall Number of Participants Analyzed 7 0
Measure Type: Number
Unit of Measure: participants
1
3.Secondary Outcome
Title Incidence of Chronic GVHD
Hide Description [Not Specified]
Time Frame 2 Years
Hide Outcome Measure Data
Hide Analysis Population Description
No patients with T-cell Graft Resistant SCID were enrolled during the time the trial was open.
Arm/Group Title T-cell Graft Permissive SCID T-cell Graft Resistant SCID
Hide Arm/Group Description:

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 Intervention: Transplant Conditioning with Mobilization Only

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

Patients with SCID with NK+ phenotype with HLA-mismatched donor Intervention: Transplant Conditioning with Mobilization and Alemtuzumab

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

Overall Number of Participants Analyzed 7 0
Measure Type: Count of Participants
Unit of Measure: Participants
1
  14.3%
4.Secondary Outcome
Title Percentage of Patients Who Become Independent From Regular IVIG Infusion
Hide Description Based on B-cell function assays from the patient's blood, we will be able to determine if patients are able to successfully discontinue IVIG infusions.
Time Frame 2 Years
Hide Outcome Measure Data
Hide Analysis Population Description
No patients with T-cell Graft Resistant SCID were enrolled during the time the trial was open.
Arm/Group Title T-cell Graft Permissive SCID T-cell Graft Resistant SCID
Hide Arm/Group Description:

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 Intervention: Transplant Conditioning with Mobilization Only

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

Patients with SCID with NK+ phenotype with HLA-mismatched donor Intervention: Transplant Conditioning with Mobilization and Alemtuzumab

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

Overall Number of Participants Analyzed 7 0
Measure Type: Count of Participants
Unit of Measure: Participants
0
   0.0%
5.Secondary Outcome
Title Number of Patients With Engraftment of Donor Stem Cells in Bone Marrow by STR Testing
Hide Description We will measure whether we are able to detect donor stem cells in the patient's bone marrow after HSCT.
Time Frame 1 Year
Hide Outcome Measure Data
Hide Analysis Population Description
No patients with T-cell Graft Resistant SCID were enrolled during the time the trial was open.
Arm/Group Title T-cell Graft Permissive SCID T-cell Graft Resistant SCID
Hide Arm/Group Description:

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 Intervention: Transplant Conditioning with Mobilization Only

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

Patients with SCID with NK+ phenotype with HLA-mismatched donor Intervention: Transplant Conditioning with Mobilization and Alemtuzumab

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

Overall Number of Participants Analyzed 7 0
Measure Type: Count of Participants
Unit of Measure: Participants
0
   0.0%
6.Secondary Outcome
Title Number of Patients Who Achieve Engraftment of Donor T-cells in Blood by STR Testing
Hide Description We will measure whether we are able to detect donor T-cells in the patient's blood after HSCT.
Time Frame 1 Year
Hide Outcome Measure Data
Hide Analysis Population Description
No patients with T-cell Graft Resistant SCID were enrolled during the time the trial was open.
Arm/Group Title T-cell Graft Permissive SCID T-cell Graft Resistant SCID
Hide Arm/Group Description:

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 Intervention: Transplant Conditioning with Mobilization Only

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

Patients with SCID with NK+ phenotype with HLA-mismatched donor Intervention: Transplant Conditioning with Mobilization and Alemtuzumab

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

Overall Number of Participants Analyzed 7 0
Measure Type: Count of Participants
Unit of Measure: Participants
7
 100.0%
Time Frame [Not Specified]
Adverse Event Reporting Description No patients with T-cell Graft Resistant SCID were enrolled during the time the trial was open.
 
Arm/Group Title T-cell Graft Permissive SCID T-cell Graft Resistant SCID
Hide Arm/Group Description

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

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

All-Cause Mortality
T-cell Graft Permissive SCID T-cell Graft Resistant SCID
Affected / at Risk (%) Affected / at Risk (%)
Total   0/7 (0.00%)   0/0 
Show Serious Adverse Events Hide Serious Adverse Events
T-cell Graft Permissive SCID T-cell Graft Resistant SCID
Affected / at Risk (%) Affected / at Risk (%)
Total   0/7 (0.00%)   0/0 
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
T-cell Graft Permissive SCID T-cell Graft Resistant SCID
Affected / at Risk (%) Affected / at Risk (%)
Total   0/7 (0.00%)   0/0 
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
Responsible Party: Christopher Dvorak, University of California, San Francisco
ClinicalTrials.gov Identifier: NCT01182675     History of Changes
Other Study ID Numbers: UCSF10-00701
First Submitted: August 9, 2010
First Posted: August 17, 2010
Results First Submitted: October 28, 2014
Results First Posted: November 10, 2014
Last Update Posted: July 31, 2018