NCT02327351

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

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
98

participants targeted

Target at P50-P75 for phase_2

Timeline
Completed

Started Jul 2012

Longer than P75 for phase_2

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

July 1, 2012

Completed
2.5 years until next milestone

First Submitted

Initial submission to the registry

December 18, 2014

Completed
12 days until next milestone

First Posted

Study publicly available on registry

December 30, 2014

Completed
2.9 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

December 1, 2017

Completed
6 months until next milestone

Study Completion

Last participant's last visit for all outcomes

June 1, 2018

Completed
2.7 years until next milestone

Results Posted

Study results publicly available

February 26, 2021

Completed
Last Updated

February 26, 2021

Status Verified

February 1, 2021

Enrollment Period

5.4 years

First QC Date

December 18, 2014

Results QC Date

November 30, 2020

Last Update Submit

February 9, 2021

Conditions

Primary Immune Deficiency DisorderHematopoietic Stem Cell Transplantation

Outcome Measures

Primary Outcomes (1)

  • Overall Survival

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

    1 year after HSCT

Secondary Outcomes (6)

  • Transplant Related Mortality (TRM)

    24 months after transplantation

  • Acute Graft Versus Host Diseases (аGVHD)

    12 months after transplantation

  • Chronic Graft Versus Host Diseases (cGVHD)

    1 year after HSCT

  • Cellular Immunological Reconstitution

    2 years after HSCT

  • Percentage of Patients With Full Donor Chimerism

    last follow-up

  • +1 more secondary outcomes

Study Arms (1)

TCR alfa beta depletion

EXPERIMENTAL

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

Interventions

Biological: TCR alfa beta T cell depletionOTHER
TCR alfa beta depletion

Eligibility Criteria

Age1 Month - 18 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64)

You may qualify if:

  • 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

You may not qualify if:

  • 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.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

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

Moscow, 117997, Russia

Location

Related Publications (1)

  • Balashov D, Shcherbina A, Maschan M, Trakhtman P, Skvortsova Y, Shelikhova L, Laberko A, Livshits A, Novichkova G, Maschan A. Single-Center Experience of Unrelated and Haploidentical Stem Cell Transplantation with TCRalphabeta and CD19 Depletion in Children with Primary Immunodeficiency Syndromes. Biol Blood Marrow Transplant. 2015 Nov;21(11):1955-62. doi: 10.1016/j.bbmt.2015.07.008. Epub 2015 Jul 15.

    PMID: 26187864DERIVED

MeSH Terms

Conditions

Primary Immunodeficiency Diseases

Condition Hierarchy (Ancestors)

Genetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesImmunologic Deficiency SyndromesImmune System Diseases

Results Point of Contact

Title
Balashov Dmitriy, head of HSCT department
Organization
National Research Center for Pediatric Hematology , Moscow, Russian Federation
Dmitriy.Balashov@fccho-moscow.ru
84956647078

Study Officials

  • Alexei Maschan, Professor

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

    STUDY CHAIR

  • Dmitry Balashov, MD, PhD

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

    PRINCIPAL INVESTIGATOR

Publication Agreements

PI is Sponsor Employee
Yes

Study Design

Study Type
interventional
Phase
phase 2
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

December 18, 2014

First Posted

December 30, 2014

Study Start

July 1, 2012

Primary Completion

December 1, 2017

Study Completion

June 1, 2018

Last Updated

February 26, 2021

Results First Posted

February 26, 2021

Record last verified: 2021-02

Locations

RU(1)