Clinical trial NCT07015801: CMV-specific Donor-derived T Lymphocytes for the Treatment of Recalcitrant CMV Infection in a Patient With Primary Immunodeficiency
Official title: Open-label Individual Patient Study of Cytomegalovirus (CMV)-Specific Donor-derived T Lymphocytes (DTL) for the Treatment of Recalcitrant CMV Infection in a Patient With Primary Immunodeficiency
Study type: INTERVENTIONAL
Overall status: ACTIVE_NOT_RECRUITING
Phase: NA
Conditions: Cytomegalovirus Viremia
Cytomegalovirus Pneumonia
T-Lymphocyte Immunodeficiency
Keywords: donor-derived T lymphocytes
Lead sponsor: University of Calgary
Sponsor class: OTHER
Collaborators: name: Alberta Precision Laboratories
name: Alberta Health services
name: University of Alberta
First posted: 2025-06-11
Enrollment: 1
Sex: FEMALE
Minimum age (years): 0
Maximum age (years): 20
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: Treatment of CMV in a patient with profound combined immunodeficiency, who has viremia and pneumonia, using CMV-specific donor-derived T lymphocytes (CMV-VST).

Detailed description: 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.

Eligibility criteria: Inclusion Criteria:

* profound combined immunodeficiency
* cytomegalovirus (CMV) infection
* viremia
* pneumonia

Exclusion Criteria:

* Receiving a steroid dose of ≥ 0.5 mg/kg of prednisolone equivalent
* Receiving antithymocyte globulin or similar anti-T-cell antibody therapy, methotrexate, or other antimetabolite-type immunosuppressants that are toxic to proliferating T cells, and extracorporeal
* Receiving checkpoint inhibitor agents (eg, nivolumab, pembrolizumab, ipilimumab) are within 3 drug half-lives of the most recent dose to cycle 1 day 1.
* Administration of another investigational product

Primary outcomes:
measure: Feasibility of study
timeFrame: Enrollment to 24 months
measure: Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability]
timeFrame: Weekly to 3 months
measure: Efficacy of Intervention
timeFrame: Weekly to 3 months
measure: Efficacy of Intervention
timeFrame: Monthly from 3 to 12 months
measure: Efficacy of Intervention
timeFrame: Every 3 months from 12-24 months
Secondary outcomes:
measure: Engraftment failure
timeFrame: Enrollment to 24 months
measure: Graft versus host disease
timeFrame: Enrollment to 24 months
measure: Transplant associated thrombotic microangiopathy
timeFrame: Enrollment to 24 months
measure: Death
timeFrame: Enrollment to 24 months
measure: CMV-reactive T cell number
timeFrame: Weekly to 3 months
measure: CMV-reactive T cell number
timeFrame: Monthly from 3 to 12 months
measure: CMV-reactive T cell number
timeFrame: Every 3 months from 12-24 months
measure: Total CMV-reactive T cell activity
timeFrame: Weekly to 3 months
measure: Total CMV-reactive T cell activity
timeFrame: Monthly from 3 to 12 months
measure: Total CMV-reactive T cell activity
timeFrame: Every 3 months from 12-24 months
measure: CMV-reactive T cell phenotyping
timeFrame: Weekly to 3 months
measure: CMV-reactive T cell phenotyping
timeFrame: Monthly from 3 to 12 months
measure: CMV-reactive T cell phenotyping
timeFrame: Every 3 months from 12-24 months
measure: RNA sequencing
timeFrame: Weekly to 3 months
measure: RNA sequencing
timeFrame: Monthly from 3 to 12 months
measure: RNA sequencing
timeFrame: Every 3 months from 12-24 months
measure: Serum cytokine analysis
timeFrame: Weekly to 3 months
measure: Serum cytokine analysis
timeFrame: Monthly from 3 to 12 months
measure: Serum cytokine analysis
timeFrame: Every 3 months from 12-24 months
Other outcomes:
Locations:
city: Calgary
country: Canada
facility: Alberta Children's Hospital
state: Alberta
Study officials:
References: