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

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ClinicalTrials.gov Identifier: NCT02327351
Recruitment Status : Unknown
Verified July 2016 by Federal Research Institute of Pediatric Hematology, Oncology and Immunology.
Recruitment status was:  Recruiting
First Posted : December 30, 2014
Last Update Posted : July 26, 2016
Sponsor:
Information provided by (Responsible Party):
Federal Research Institute of Pediatric Hematology, Oncology and Immunology

Brief Summary:
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

Condition or disease Intervention/treatment Phase
Primary Immune Deficiency Disorder Hematopoietic Stem Cell Transplantation Other: Biological: TCR alfa beta T cell depletion Phase 2 Phase 3

Detailed Description:

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  (Clinical Trial)
Estimated Enrollment : 60 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Phase II/III Study of Allogeneic Hematopoietic Stem Cell Transplantation From Unrelated and Haploidentical Donors After TCR Alfa Beta Negative Selection in Pediatric Patients With Primary Immunodeficiency Diseases
Study Start Date : July 2012
Estimated Primary Completion Date : December 2018


Arm Intervention/treatment
Experimental: TCR alfa beta depletion
TCR alfa beta depleted graft, infusion. The leukapheresis product will undergo TCR alfa beta depletion following the standardized protocol.
Other: Biological: TCR alfa beta T cell depletion



Primary Outcome Measures :
  1. Overall survival (OS) [ Time Frame: up to 24 months after transplantation ]
    The OS probability compared with cohort of historical control


Secondary Outcome Measures :
  1. Transplant related mortality (TRM) [ Time Frame: up to 24 months after transplantation ]
    The TRM probability compared with cohort of historical control

  2. Acute graft versus host diseases (аGVHD) [ Time Frame: up to 12 months after transplantation ]
    Cumulative incidence and severity of II-IV grade of aGVHD

  3. Chronic graft versus host diseases (cGVHD) [ Time Frame: up to 24 months after transplantation ]
    Cumulative incidence and severity of cGVHD

  4. Cellular Immunological reconstitution by laboratory evaluations [ Time Frame: up to 24 months after transplantation ]
    The recovery of different lymphocyte's subpopulation (CD3+; CD3+CD45RA+CD197; CD19+; CD4+; CD8+; TCR alfa beta; TCR gamma delta)

  5. Percentage of patients with full/mixed donor chimerism [ Time Frame: up to 12 months after transplantation ]
  6. Viral infections after transplant [ Time Frame: up to 12 months after transplantation ]
    Cumulative incidence on CMV and EBV reactivation/diseases



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Ages Eligible for Study:   1 Month to 18 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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.

Information from the National Library of Medicine

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): NCT02327351


Contacts
Contact: Dmitry Balashov, MD, PhD +7(495)287-6570 ext 6534 bala8@yandex.ru
Contact: Michael Maschan, Professor +7(926)651-2145 mmaschan@yandex.ru

Locations
Russian Federation
Dmitry Rogachev Federal Research and Clinical Centre of Paediatric Haematology, Oncology and Immunology Recruiting
Moscow, Russian Federation, 117997
Contact: Dmitry Balashov, MD, PhD    +7(495)287-6570 ext 6534    bala8@yandex.ru   
Contact: Michael Maschan, Professor       mmaschan@yandex.ru   
Principal Investigator: Dmitry Balashov, MD, PhD         
Sub-Investigator: Michael Maschan, Professor         
Sub-Investigator: Anna Shcherbina, Professor         
Sub-Investigator: Alexandra Laberko, MD         
Sub-Investigator: Yulia Skvortsova, MD, PhD         
Sponsors and Collaborators
Federal Research Institute of Pediatric Hematology, Oncology and Immunology
Investigators
Study Chair: Alexei Maschan, Professor Dmitry Rogachev Federal Research and Clinical Centre of Paediatric Haematology, Oncology and Immunology
Principal Investigator: Dmitry Balashov, MD, PhD Dmitry Rogachev Federal Research and Clinical Centre of Paediatric Haematology, Oncology and Immunology

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: Federal Research Institute of Pediatric Hematology, Oncology and Immunology
ClinicalTrials.gov Identifier: NCT02327351     History of Changes
Other Study ID Numbers: TCRabPID-2012
First Posted: December 30, 2014    Key Record Dates
Last Update Posted: July 26, 2016
Last Verified: July 2016

Additional relevant MeSH terms:
Immunologic Deficiency Syndromes
Immune System Diseases