TCR Alpha/Beta Depletion for HSCT From Haploidentical and Unrelated Donors in the Treatment of PID

Federal Research Institute of Pediatric Hematology, Oncology and Immunology98 enrolled

Overview

Treatment Study to assess of safety and efficiency of T cells receptor (TCR) alfa beta depleted graft for hematopoietic stem cell transplantation (HSCT) from haploidentical and unrelated donors in patients with primary immunodeficiency diseases

Infections, graft versus host diseases (GVHD) and associated morbidity and mortality remains significant problems after unrelated and haploidentical hematopoietic stem sell transplantation (HSCT) in patients with primary immunodeficiency diseases (PID). In this study the hypothesis is that the transplantation of TCR alfa beta depleted peripheral blood stem cells (PBSC) would offers advantages over the use of positively selected CD34+ stem cells in haploidentical HSCT and non-manipulated graft in unrelated HSCT. The purpose of this study is to evaluate the safety and efficiency of the selective infusion of TCR alfa beta T cell depleted graft in pediatric patients with PID receiving HSCT from haploidentical and unrelated donors.

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Primary Immune Deficiency Disorder Hematopoietic Stem Cell Transplantation

Interventions

Biological: TCR alfa beta T cell depletionOTHER

Eligibility

Sex: ALLMin age: 1 MonthMax age: 18 Years

Medical Language ↔ Plain English

Inclusion Criteria: * Patients aged ≥ 1 months and \< 19 years * Patients diagnosed with Primary Immunodeficiency Diseases eligible for an allogeneic transplantation and lacking a related HLA-matched donor * Lansky/Karnofsky score \> 40, WHO \> 4 * Signed written informed consent Exclusion Criteria: * Dysfunction of liver (ALT/AST \> 5 times normal value, or bilirubin \> 3 times normal value), or of renal function (creatinine clearance \< 30 ml / min) * Severe cardiovascular disease (arrhythmias requiring chronic treatment, congestive heart failure or left ventricular ejection fraction \<40%) * Serious concurrent uncontrolled medical disorder * Pregnant or breast feeding female patient * Lack of parents' informed consent.

Locations (1)

Dmitry Rogachev Federal Research and Clinical Centre of Paediatric Haematology, Oncology and Immunology

Moscow, Russia

Outcomes

Primary Outcomes

Overall Survival

The probability of overall survival estimated by the Kaplan-Meier method at 1 year after HSCT

Time frame: 1 year after HSCT

Secondary Outcomes

Transplant Related Mortality (TRM)

transplant-related mortality estimated with cumulative incidence curve, considering relapse as a competitive risk

Time frame: 24 months after transplantation

Acute Graft Versus Host Diseases (аGVHD)

incidence of aGVHD II-IV stage estimated with cumulative incidence curve, considering graft rejection and death as competitive risks

Time frame: 12 months after transplantation

Chronic Graft Versus Host Diseases (cGVHD)

incidence of cGVHD estimated with cumulative incidence curve, considering graft rejection and death as competitive risks

Time frame: 1 year after HSCT

Cellular Immunological Reconstitution

Number of participants, who reached immune recovery - CD19+ lymphocytes subsets

Time frame: 2 years after HSCT

Percentage of Patients With Full Donor Chimerism

Percentage of patients with full (more than 90%) donor chimerism among survivals

Time frame: last follow-up

Viral Infections After Transplant

number of patients with CMV reactivation (detection of any grade of CMV viremia after HSCT)

Time frame: 12 months after transplantation

Data from ClinicalTrials.gov

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