A Trial of Plerixafor/G-CSF as Additional Agents for Conditioning Before TCR Alpha/Beta Depleted HSCT in WAS Patients

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Brief Title

A Trial of Plerixafor/G-CSF as Additional Agents for Conditioning Before TCR Alpha/Beta Depleted HSCT in WAS Patients

Official Title

A Trial of Plerixafor With G-CSF as Additional Agents in Conditioning Regimen for Prevention of Graft Failure After Transplantation With TCR Alpha/Beta Grafts Depletion in Patients With Wiskott-Aldrich Syndrome.

Brief Summary

      Treatment Study to assess of safety and efficiency of conditioning with Plerixafor and G-CSF
      as additional agents for prevention of graft failure after transplantation with TCR
      alpha/beta grafts depletion in patient with Wiskott-Aldrich syndrome.
    

Detailed Description

      Severe graft dysfunction, such as the degree of donor chimerism predominantly in the myeloid
      compartment is one of major problem in patients with Wiskott-Aldrich syndrome (WAS),
      especially after hematopoietic stem cell transplantation (HSCT) from alternative donor. It
      often leads to the development of severe thrombocytopenia or even transplants rejection. In
      this study the hypothesis is that the use of plerixafor and G-CSF as additional agents in
      conditioning regimen would offers advantages due to lowing risk of mixed chimerism after
      HSCT. This effect is based on the fact that simultaneous use of plerixafor with G-CSF is
      efficient in inducing stem cell release and opening of bone marrow (BM) niches. Moreover,
      stem cell release probably leads to liberation of host stem cells from the anti-apoptotic
      effects of the BM stroma for the more powerful effect of chemotherapy.

      In this study, the investigators use TCR alpha/beta grafts depletion of the grafts as basic
      technology for HSCT from haploidentical and unrelated donors approved in Institution.

      Thus, the purpose of this study is to evaluate the safety and efficiency of myeloablative
      conditioning with Plerixafor and G-CSF as additional agents for prevention of graft failure
      after transplantation with TCR alpha/beta grafts depletion in patients with Wiskott-Aldrich
      syndrome.
    

Study Phase

Phase 2

Study Type

Interventional


Primary Outcome

Event free survival (EFS)

Secondary Outcome

 Overall survival (OS)

Condition

Wiskott-Aldrich Syndrome

Intervention

G-CSF for Conditioning before HSCT.

Study Arms / Comparison Groups

 Plerixafor/G-CSF for HSCT conditioning
Description:  Myeloablative conditioning regimen with Plerixafor and G-CSF as addition agents before stem cell transplantation in WAS patients.

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information


Recruitment Status

Biological

Estimated Enrollment

30

Start Date

June 2016

Completion Date

July 2019

Primary Completion Date

December 2018

Eligibility Criteria

        Inclusion Criteria:

          -  Patients aged ≥ 1 months and < 19 years

          -  Patients diagnosed with Wiskott-Aldrich syndrome 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

          -  Lack of parents' informed consent.
      

Gender

All

Ages

1 Month - 19 Years

Accepts Healthy Volunteers

No

Contacts

Alexei Maschan, Professor, +7(495)287-6570, [email protected]

Location Countries

Russian Federation

Location Countries

Russian Federation

Administrative Informations


NCT ID

NCT03019809

Organization ID

WAS_PG 2016


Responsible Party

Sponsor

Study Sponsor

Federal Research Institute of Pediatric Hematology, Oncology and Immunology


Study Sponsor

Alexei Maschan, Professor, Study Chair, Dmitry Rogachev Federal Research and Clinical Centre of Paediatric Haematology, Oncology and Immunology


Verification Date

December 2018