Treatment of CMV in a patient with profound combined immunodeficiency, who has viremia and pneumonia, using CMV-specific donor-derived T lymphocytes (CMV-VST).
Treatment of CMV in a patient with profound combined immunodeficiency, who has viremia and pneumonia, using CMV-specific donor-derived T lymphocytes (CMV-VST), a cell therapy product containing a mixture of donor lymphocytes, reactive to peptides derived from cytomegalovirus. After having receipt of therapy, the patient will have clinical assessments twice a week until discharge from the inpatient unit. After discharge, assessments will be performed on a weekly basis for three months. From 3-12 months, the patient will be seen monthly and then every three months till 2 years post planned hematopoietic stem cell transplantation. After 2 years, survival status will be assessed every 6 months through year 15.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
1
30-40 x 10\^3 viable CD3+ cells/kg
Alberta Children's Hospital
Calgary, Alberta, Canada
Feasibility of study
Evaluate the feasibility of conducting this study, evaluated in terms of whether or not the study could be completed as laid out in the protocol in the time allotted.
Time frame: Enrollment to 24 months
Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability]
AEs assessed according to CTCAE grading criteria.
Time frame: Weekly to 3 months
Efficacy of Intervention
Change in viremia from baseline according to PCR testing after the intervention
Time frame: Weekly to 3 months
Efficacy of Intervention
Change in viremia from baseline according to PCR testing after the intervention
Time frame: Monthly from 3 to 12 months
Efficacy of Intervention
Change in viremia from baseline according to PCR testing after the intervention
Time frame: Every 3 months from 12-24 months
Engraftment failure
Assessment of evidence of engraftment failure from clinical evaluations
Time frame: Enrollment to 24 months
Graft versus host disease
Assessment of evidence of graft versus host disease (GVHD) from clinical evaluations
Time frame: Enrollment to 24 months
Transplant associated thrombotic microangiopathy
Assessment of evidence of Transplant associated thrombotic microangiopathy (TA-TMA) from clinical evaluations
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Time frame: Enrollment to 24 months
Death
Death of participant
Time frame: Enrollment to 24 months
CMV-reactive T cell number
The number of CMV-reactive T cells in the patient's blood will be enumerated using an CMV-ELISpot assay.
Time frame: Weekly to 3 months
CMV-reactive T cell number
The number of CMV-reactive T cells in the patient's blood will be enumerated using an CMV-ELISpot assay.
Time frame: Monthly from 3 to 12 months
CMV-reactive T cell number
The number of CMV-reactive T cells in the patient's blood will be enumerated using an CMV-ELISpot assay.
Time frame: Every 3 months from 12-24 months
Total CMV-reactive T cell activity
The total amount of INF γ secreted from all CMV-reactive T cells in the patient's blood to measured using a commercially available ELISA kit (QuantiFERON-CMV). This will allow track the cumulative response to CMV by the reactive T cells over time.
Time frame: Weekly to 3 months
Total CMV-reactive T cell activity
The total amount of INF γ secreted from all CMV-reactive T cells in the patient's blood to measured using a commercially available ELISA kit (QuantiFERON-CMV). This will allow track the cumulative response to CMV by the reactive T cells over time.
Time frame: Monthly from 3 to 12 months
Total CMV-reactive T cell activity
The total amount of INF γ secreted from all CMV-reactive T cells in the patient's blood to measured using a commercially available ELISA kit (QuantiFERON-CMV). This will allow track the cumulative response to CMV by the reactive T cells over time.
Time frame: Every 3 months from 12-24 months
CMV-reactive T cell phenotyping
Flow cytometry will be used to characterize CMV-reactive T cell phenotype
Time frame: Weekly to 3 months
CMV-reactive T cell phenotyping
Flow cytometry will be used to characterize CMV-reactive T cell phenotype
Time frame: Monthly from 3 to 12 months
CMV-reactive T cell phenotyping
Flow cytometry will be used to characterize CMV-reactive T cell phenotype
Time frame: Every 3 months from 12-24 months
RNA sequencing
RNA sequencing, to characterize CMV-reactive T cell biology and track individual VST clones
Time frame: Weekly to 3 months
RNA sequencing
RNA sequencing, to characterize CMV-reactive T cell biology and track individual VST clones
Time frame: Monthly from 3 to 12 months
RNA sequencing
RNA sequencing, to characterize CMV-reactive T cell biology and track individual VST clones
Time frame: Every 3 months from 12-24 months
Serum cytokine analysis
Serum cytokines, to estimate total CMV-reactive T cell activity and host response to treatment
Time frame: Weekly to 3 months
Serum cytokine analysis
Serum cytokines, to estimate total CMV-reactive T cell activity and host response to treatment
Time frame: Monthly from 3 to 12 months
Serum cytokine analysis
Serum cytokines, to estimate total CMV-reactive T cell activity and host response to treatment
Time frame: Every 3 months from 12-24 months