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SCID Bu/Flu/ATG Study With T Cell Depletion

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ClinicalTrials.gov Identifier: NCT02127892
Recruitment Status : Terminated (Closed due to slow accrual. Nine subjects enrolled over 7 years.)
First Posted : May 1, 2014
Results First Posted : September 18, 2017
Last Update Posted : September 18, 2017
Sponsor:
Information provided by (Responsible Party):
Neena Kapoor, M.D., Children's Hospital Los Angeles

Study Type Interventional
Study Design Allocation: Non-Randomized;   Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Severe Combined Immunodeficiency
Interventions Biological: unrelated BM with T cell depletion
Biological: unrelated cord blood
Biological: haplo BM with T cell depletion
Device: unrelated PBSC with T cell depletion
Enrollment 9
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Unrelated BM With T Cell Depletion Unrelated Cord Blood Haplo BM With T Cell Depletion Unrelated PBSC With T Cell Depletion
Hide Arm/Group Description

Acceptable matching for matched unrelated donor (MUD) bone marrow will be genotypic matches at 10 of 10 HLA alleles (HLA-A, B, C, DR and DQ) or 9 of 10 HLA alleles.

unrelated BM with T cell depletion: Remaining unmanipulated bone marrow will be processed to isolate CD34+ cells (T cell depleted).

Acceptable matching for unrelated cord blood will be a genotypic match at 6 of 6 alleles (HLA A, B and DR) or 5 of 6 alleles, but not with mismatches at both alleles of a single locus (e.g. not mismatched for both HLA A alleles).

unrelated cord blood: Cord blood will be thawed (and processed if ABO incompatibility) per institutional SOP.

If there is no unrelated donor available meeting the matching criteria for unrelated bone marrow or unrelated cord blood donors.

haplo BM with T cell depletion: haplo-identical (parental) bone marrow will be processed for CD34+ cell isolation.

The preferred source will be bone marrow, however, if a donor is unable or unwilling to donate bone marrow, peripheral blood stem cells (PBSC) will be allowed.

unrelated PBSC with T cell depletion: peripheral blood stem cell will be processed for CD34+ cell isolation.

Period Title: Overall Study
Started 7 2 0 0
Completed 6 1 0 0
Not Completed 1 1 0 0
Arm/Group Title Unrelated BM With T Cell Depletion Unrelated Cord Blood Haplo BM With T Cell Depletion Unrelated PBSC With T Cell Depletion Total
Hide Arm/Group Description

Acceptable matching for matched unrelated donor (MUD) bone marrow will be genotypic matches at 10 of 10 HLA alleles (HLA-A, B, C, DR and DQ) or 9 of 10 HLA alleles.

unrelated BM with T cell depletion: Remaining unmanipulated bone marrow will be processed to isolate CD34+ cells (T cell depleted).

Acceptable matching for unrelated cord blood will be a genotypic match at 6 of 6 alleles (HLA A, B and DR) or 5 of 6 alleles, but not with mismatches at both alleles of a single locus (e.g. not mismatched for both HLA A alleles).

unrelated cord blood: Cord blood will be thawed (and processed if ABO incompatibility) per institutional SOP.

If there is no unrelated donor available meeting the matching criteria for unrelated bone marrow or unrelated cord blood donors.

haplo BM with T cell depletion: haplo-identical (parental) bone marrow will be processed for CD34+ cell isolation.

The preferred source will be bone marrow, however, if a donor is unable or unwilling to donate bone marrow, peripheral blood stem cells (PBSC) will be allowed.

unrelated PBSC with T cell depletion: peripheral blood stem cell will be processed for CD34+ cell isolation.

Total of all reporting groups
Overall Number of Baseline Participants 7 2 0 0 9
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 7 participants 2 participants 0 participants 0 participants 9 participants
<=18 years
7
 100.0%
2
 100.0%
0 0
9
 100.0%
Between 18 and 65 years
0
   0.0%
0
   0.0%
0 0
0
   0.0%
>=65 years
0
   0.0%
0
   0.0%
0 0
0
   0.0%
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 7 participants 2 participants 0 participants 0 participants 9 participants
Female
1
  14.3%
0
   0.0%
1
  11.1%
Male
6
  85.7%
2
 100.0%
8
  88.9%
Ethnicity (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 7 participants 2 participants 0 participants 0 participants 9 participants
Hispanic or Latino
3
  42.9%
2
 100.0%
0 0
5
  55.6%
Not Hispanic or Latino
3
  42.9%
0
   0.0%
0 0
3
  33.3%
Unknown or Not Reported
1
  14.3%
0
   0.0%
0 0
1
  11.1%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 7 participants 2 participants 0 participants 0 participants 9 participants
American Indian or Alaska Native
0
   0.0%
0
   0.0%
0
   0.0%
Asian
1
  14.3%
0
   0.0%
1
  11.1%
Native Hawaiian or Other Pacific Islander
0
   0.0%
0
   0.0%
0
   0.0%
Black or African American
0
   0.0%
0
   0.0%
0
   0.0%
White
2
  28.6%
0
   0.0%
2
  22.2%
More than one race
0
   0.0%
0
   0.0%
0
   0.0%
Unknown or Not Reported
4
  57.1%
2
 100.0%
6
  66.7%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 7 participants 2 participants 0 participants 0 participants 9 participants
7 2 9
1.Primary Outcome
Title Number of Participants With Engraftment
Hide Description Engraftment is defined as recovery of blood counts (neutrophil and platelet engraftment) with cells of donor origin, documented by either bone marrow or peripheral blood chimerism assays after hematopoietic stem cell transplant.
Time Frame 100 day
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Unrelated BM With T Cell Depletion Unrelated Cord Blood Haplo BM With T Cell Depletion Unrelated PBSC With T Cell Depletion
Hide Arm/Group Description:

Acceptable matching for matched unrelated donor (MUD) bone marrow will be genotypic matches at 10 of 10 HLA alleles (HLA-A, B, C, DR and DQ) or 9 of 10 HLA alleles.

unrelated BM with T cell depletion: Remaining unmanipulated bone marrow will be processed to isolate CD34+ cells (T cell depleted).

Acceptable matching for unrelated cord blood will be a genotypic match at 6 of 6 alleles (HLA A, B and DR) or 5 of 6 alleles, but not with mismatches at both alleles of a single locus (e.g. not mismatched for both HLA A alleles).

unrelated cord blood: Cord blood will be thawed (and processed if ABO incompatibility) per institutional SOP.

If there is no unrelated donor available meeting the matching criteria for unrelated bone marrow or unrelated cord blood donors.

haplo BM with T cell depletion: haplo-identical (parental) bone marrow will be processed for CD34+ cell isolation.

The preferred source will be bone marrow, however, if a donor is unable or unwilling to donate bone marrow, peripheral blood stem cells (PBSC) will be allowed.

unrelated PBSC with T cell depletion: peripheral blood stem cell will be processed for CD34+ cell isolation.

Overall Number of Participants Analyzed 7 2 0 0
Measure Type: Count of Participants
Unit of Measure: Participants
7
 100.0%
2
 100.0%
2.Secondary Outcome
Title Number of Participants With Donor-derived CD3+ T Lymphocytes >/= 100/mm3
Hide Description Absolute number of donor-derived CD3+ T lymphocytes >/= 100/mm3 in participating subjects.
Time Frame 1 year
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Unrelated BM With T Cell Depletion Unrelated Cord Blood Haplo BM With T Cell Depletion Unrelated PBSC With T Cell Depletion
Hide Arm/Group Description:

Acceptable matching for matched unrelated donor (MUD) bone marrow will be genotypic matches at 10 of 10 HLA alleles (HLA-A, B, C, DR and DQ) or 9 of 10 HLA alleles.

unrelated BM with T cell depletion: Remaining unmanipulated bone marrow will be processed to isolate CD34+ cells (T cell depleted).

Acceptable matching for unrelated cord blood will be a genotypic match at 6 of 6 alleles (HLA A, B and DR) or 5 of 6 alleles, but not with mismatches at both alleles of a single locus (e.g. not mismatched for both HLA A alleles).

unrelated cord blood: Cord blood will be thawed (and processed if ABO incompatibility) per institutional SOP.

If there is no unrelated donor available meeting the matching criteria for unrelated bone marrow or unrelated cord blood donors.

haplo BM with T cell depletion: haplo-identical (parental) bone marrow will be processed for CD34+ cell isolation.

The preferred source will be bone marrow, however, if a donor is unable or unwilling to donate bone marrow, peripheral blood stem cells (PBSC) will be allowed.

unrelated PBSC with T cell depletion: peripheral blood stem cell will be processed for CD34+ cell isolation.

Overall Number of Participants Analyzed 7 2 0 0
Measure Type: Count of Participants
Unit of Measure: Participants
7
 100.0%
1
  50.0%
0 0
3.Other Pre-specified Outcome
Title Number of Participants With Veno-occlusive Disease (VOD) - Moderate and Severe
Hide Description Evaluation of veno-occlusive disease determined by the presence of the following features; fluid retention, weight gain, leaky capillary syndrome, painful liver enlargement, refractoriness to platelet tranfusion and hyperbilirubinemia
Time Frame 100 days
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Unrelated BM With T Cell Depletion Unrelated Cord Blood Haplo BM With T Cell Depletion Unrelated PBSC With T Cell Depletion
Hide Arm/Group Description:

Acceptable matching for matched unrelated donor (MUD) bone marrow will be genotypic matches at 10 of 10 HLA alleles (HLA-A, B, C, DR and DQ) or 9 of 10 HLA alleles.

unrelated BM with T cell depletion: Remaining unmanipulated bone marrow will be processed to isolate CD34+ cells (T cell depleted).

Acceptable matching for unrelated cord blood will be a genotypic match at 6 of 6 alleles (HLA A, B and DR) or 5 of 6 alleles, but not with mismatches at both alleles of a single locus (e.g. not mismatched for both HLA A alleles).

unrelated cord blood: Cord blood will be thawed (and processed if ABO incompatibility) per institutional SOP.

If there is no unrelated donor available meeting the matching criteria for unrelated bone marrow or unrelated cord blood donors.

haplo BM with T cell depletion: haplo-identical (parental) bone marrow will be processed for CD34+ cell isolation.

The preferred source will be bone marrow, however, if a donor is unable or unwilling to donate bone marrow, peripheral blood stem cells (PBSC) will be allowed.

unrelated PBSC with T cell depletion: peripheral blood stem cell will be processed for CD34+ cell isolation.

Overall Number of Participants Analyzed 7 2 0 0
Measure Type: Count of Participants
Unit of Measure: Participants
2
  28.6%
1
  50.0%
0 0
4.Other Pre-specified Outcome
Title Number of Participants With Graft Versus Host Disease (GVHD) - Grade III or IV
Hide Description GVHD disease surveillance done by clinical evaluation, to include history, physical examination, specifically for rash, jaundice, liver dysfunction, nausea and vomiting, diarrhea and failure to thrive.
Time Frame 1 year
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Unrelated BM With T Cell Depletion Unrelated Cord Blood Haplo BM With T Cell Depletion Unrelated PBSC With T Cell Depletion
Hide Arm/Group Description:

Acceptable matching for matched unrelated donor (MUD) bone marrow will be genotypic matches at 10 of 10 HLA alleles (HLA-A, B, C, DR and DQ) or 9 of 10 HLA alleles.

unrelated BM with T cell depletion: Remaining unmanipulated bone marrow will be processed to isolate CD34+ cells (T cell depleted).

Acceptable matching for unrelated cord blood will be a genotypic match at 6 of 6 alleles (HLA A, B and DR) or 5 of 6 alleles, but not with mismatches at both alleles of a single locus (e.g. not mismatched for both HLA A alleles).

unrelated cord blood: Cord blood will be thawed (and processed if ABO incompatibility) per institutional SOP.

If there is no unrelated donor available meeting the matching criteria for unrelated bone marrow or unrelated cord blood donors.

haplo BM with T cell depletion: haplo-identical (parental) bone marrow will be processed for CD34+ cell isolation.

The preferred source will be bone marrow, however, if a donor is unable or unwilling to donate bone marrow, peripheral blood stem cells (PBSC) will be allowed.

unrelated PBSC with T cell depletion: peripheral blood stem cell will be processed for CD34+ cell isolation.

Overall Number of Participants Analyzed 7 2 0 0
Measure Type: Count of Participants
Unit of Measure: Participants
1
  14.3%
0
   0.0%
0 0
5.Other Pre-specified Outcome
Title Overall Survival
Hide Description Overalls survival of patient at 1 year post transplant
Time Frame 1 year
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Unrelated BM With T Cell Depletion Unrelated Cord Blood Haplo BM With T Cell Depletion Unrelated PBSC With T Cell Depletion
Hide Arm/Group Description:

Acceptable matching for matched unrelated donor (MUD) bone marrow will be genotypic matches at 10 of 10 HLA alleles (HLA-A, B, C, DR and DQ) or 9 of 10 HLA alleles.

unrelated BM with T cell depletion: Remaining unmanipulated bone marrow will be processed to isolate CD34+ cells (T cell depleted).

Acceptable matching for unrelated cord blood will be a genotypic match at 6 of 6 alleles (HLA A, B and DR) or 5 of 6 alleles, but not with mismatches at both alleles of a single locus (e.g. not mismatched for both HLA A alleles).

unrelated cord blood: Cord blood will be thawed (and processed if ABO incompatibility) per institutional SOP.

If there is no unrelated donor available meeting the matching criteria for unrelated bone marrow or unrelated cord blood donors.

haplo BM with T cell depletion: haplo-identical (parental) bone marrow will be processed for CD34+ cell isolation.

The preferred source will be bone marrow, however, if a donor is unable or unwilling to donate bone marrow, peripheral blood stem cells (PBSC) will be allowed.

unrelated PBSC with T cell depletion: peripheral blood stem cell will be processed for CD34+ cell isolation.

Overall Number of Participants Analyzed 7 2 0 0
Measure Type: Count of Participants
Unit of Measure: Participants
6
  85.7%
1
  50.0%
0 0
Time Frame Adverse event data was collected over a period of 1 year post transplant.
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Unrelated BM With T Cell Depletion Unrelated Cord Blood Haplo BM With T Cell Depletion Unrelated PBSC With T Cell Depletion
Hide Arm/Group Description

Acceptable matching for matched unrelated donor (MUD) bone marrow will be genotypic matches at 10 of 10 HLA alleles (HLA-A, B, C, DR and DQ) or 9 of 10 HLA alleles.

unrelated BM with T cell depletion: Remaining unmanipulated bone marrow will be processed to isolate CD34+ cells (T cell depleted).

Acceptable matching for unrelated cord blood will be a genotypic match at 6 of 6 alleles (HLA A, B and DR) or 5 of 6 alleles, but not with mismatches at both alleles of a single locus (e.g. not mismatched for both HLA A alleles).

unrelated cord blood: Cord blood will be thawed (and processed if ABO incompatibility) per institutional SOP.

If there is no unrelated donor available meeting the matching criteria for unrelated bone marrow or unrelated cord blood donors.

haplo BM with T cell depletion: haplo-identical (parental) bone marrow will be processed for CD34+ cell isolation.

The preferred source will be bone marrow, however, if a donor is unable or unwilling to donate bone marrow, peripheral blood stem cells (PBSC) will be allowed.

unrelated PBSC with T cell depletion: peripheral blood stem cell will be processed for CD34+ cell isolation.

All-Cause Mortality
Unrelated BM With T Cell Depletion Unrelated Cord Blood Haplo BM With T Cell Depletion Unrelated PBSC With T Cell Depletion
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   1/7 (14.29%)      1/2 (50.00%)      0/0      0/0    
Show Serious Adverse Events Hide Serious Adverse Events
Unrelated BM With T Cell Depletion Unrelated Cord Blood Haplo BM With T Cell Depletion Unrelated PBSC With T Cell Depletion
Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   1/7 (14.29%)      1/2 (50.00%)      0/0      0/0    
Hepatobiliary disorders         
Sinusoidal obstruction syndrome  1 [1]  1/7 (14.29%)  1 1/2 (50.00%)  1 0/0  0 0/0  0
1
Term from vocabulary, SOS
Indicates events were collected by systematic assessment
[1]
Sinusoidal obstruction syndrome (SOS), previously known as veno-occlusive disease (VOD), is a distinctive and potentially fatal form of hepatic injury that occurs predominantly, if not only, after drug or toxin exposure
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Unrelated BM With T Cell Depletion Unrelated Cord Blood Haplo BM With T Cell Depletion Unrelated PBSC With T Cell Depletion
Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   5/7 (71.43%)      1/2 (50.00%)      0/0      0/0    
Infections and infestations         
Infection - Aspergillis  [1]  1/7 (14.29%)  1 1/2 (50.00%)  1 0/0  0 0/0  0
Infection - Fungal  [2]  1/7 (14.29%)  1 0/2 (0.00%)  0 0/0  0 0/0  0
Nervous system disorders         
Seizure  [3]  1/7 (14.29%)  1 0/2 (0.00%)  0 0/0  0 0/0  0
Respiratory, thoracic and mediastinal disorders         
Pulmonary Hemorrhage   0/7 (0.00%)  0 1/2 (50.00%)  1 0/0  0 0/0  0
Skin and subcutaneous tissue disorders         
Graft Versus Host Disease  [4]  5/7 (71.43%)  5 1/2 (50.00%)  1 0/0  0 0/0  0
Indicates events were collected by systematic assessment
[1]
Pulmonary Aspergillis infection
[2]
Pulmonary fungal infection
[3]
Busulfan toxicity
[4]
Graft versus host disease - condition post transplant the donor's immune cells in the transplant (graft) make antibodies against the patient's tissues (host) and attack vital organs (skin, GI track and liver common organs attacked)
Certain Agreements
All Principal Investigators ARE employed by the organization sponsoring the study.
Results Point of Contact
Name/Title: Neena Kapoor, M.D.
Organization: Children's Hospital Los Angeles
Phone: 323-361-2546
Responsible Party: Neena Kapoor, M.D., Children's Hospital Los Angeles
ClinicalTrials.gov Identifier: NCT02127892     History of Changes
Other Study ID Numbers: CCI-06-00243
First Submitted: October 19, 2012
First Posted: May 1, 2014
Results First Submitted: July 17, 2017
Results First Posted: September 18, 2017
Last Update Posted: September 18, 2017