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
|Condition or disease||Intervention/treatment||Phase|
|Severe Combined Immunodeficiency||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||Phase 1 Phase 2|
The study is being conducted to assess the following:
- overall survival
- event-free survival (events are defined as: death,non-engraftment/2nd transplant, immune reconstitution failure)
- acute toxicity of the conditioning regimen
- engraftment frequency immune reconstitution frequency and tempo acute and chronic graft-versus-host disease (GVHD), frequency and severity.
The outcome from this protocol will be compared to the retrospective cohort consisting of all patients who have undergone haplo-identical HSCT for SCID at CHLA from 1984-2006 based on the assessment of the above-listed endpoints.
The CliniMACS device will be used for CD34+ selection in place of the Isolex 300i. The CliniMACS CD34 Reagent System is an investigational medical device that has not yet been approved by the FDA. This device is used in vitro to select and enrich specific cell populations. When using the CliniMACS CD34 Reagent, the system selects CD34+ cells from heterogenous hematological cell populations for transplantation in cases where this is clinically indicated.
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||9 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||Phase I/II Trial of Hematopoietic Stem Cell Transplant (HSCT) for Children With Severe Combined Immune Deficiency (SCID) and Without an HLA-Matched Sibling Donor|
|Actual Study Start Date :||January 2, 2007|
|Actual Primary Completion Date :||August 1, 2016|
|Actual Study Completion Date :||August 1, 2016|
unrelated BM with T cell depletion
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.
Biological: unrelated BM with T cell depletion
Remaining unmanipulated bone marrow will be processed to isolate CD34+ cells (T cell depleted).
Other Name: CD34+ selection using CliniMACS
unrelated cord blood
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).
Biological: unrelated cord blood
Cord blood will be thawed (and processed if ABO incompatibility) per institutional SOP.
Other Name: umbilical cord blood
haplo BM with T cell depletion
If there is no unrelated donor available meeting the matching criteria for unrelated bone marrow or unrelated cord blood donors.
Biological: haplo BM with T cell depletion
haplo-identical (parental) bone marrow will be processed for CD34+ cell isolation.
Other Name: CD34+ selection with CliniMACS
unrelated PBSC with T cell depletion
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.
Device: unrelated PBSC with T cell depletion
peripheral blood stem cell will be processed for CD34+ cell isolation.
Other Name: CD34+ selection using CliniMACS
- Number of Participants With Engraftment [ Time Frame: 100 day ]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.
- Number of Participants With Donor-derived CD3+ T Lymphocytes >/= 100/mm3 [ Time Frame: 1 year ]Absolute number of donor-derived CD3+ T lymphocytes >/= 100/mm3 in participating subjects.
- Number of Participants With Veno-occlusive Disease (VOD) - Moderate and Severe [ Time Frame: 100 days ]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
- Number of Participants With Graft Versus Host Disease (GVHD) - Grade III or IV [ Time Frame: 1 year ]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.
- Overall Survival [ Time Frame: 1 year ]Overalls survival of patient at 1 year post transplant
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02127892
|United States, California|
|Children's Hospital Los Angeles|
|Los Angeles, California, United States, 90027|
|Principal Investigator:||Neena Kapoor, M.D.||Children's Hospital Los Angeles, University of Southern California|