A Trial of Plerixafor/G-CSF as Additional Agents for Conditioning Before TCR Alpha/Beta Depleted HSCT in WAS Patients
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|ClinicalTrials.gov Identifier: NCT03019809|
Recruitment Status : Recruiting
First Posted : January 13, 2017
Last Update Posted : December 12, 2018
|Condition or disease||Intervention/treatment||Phase|
|Wiskott-Aldrich Syndrome Hematopoietic Stem Cell Transplantation Graft Failure||Biological: G-CSF for Conditioning before HSCT. Biological: Plerixafor for Conditioning before HSCT.||Phase 2|
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 Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||30 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|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.|
|Actual Study Start Date :||June 2016|
|Actual Primary Completion Date :||December 2018|
|Estimated Study Completion Date :||July 2019|
Experimental: Plerixafor/G-CSF for HSCT conditioning
Myeloablative conditioning regimen with Plerixafor and G-CSF as addition agents before stem cell transplantation in WAS patients.
Biological: G-CSF for Conditioning before HSCT.
Mobilization of hematopoietic stem (HSC) into circulation
Biological: Plerixafor for Conditioning before HSCT.
Directed inhibition of CXC chemokine receptor type 4 (CXCR4) for opening enough BM niches for adequate donor HSC engraftment.
- Event free survival (EFS) [ Time Frame: 24 months ]The EFS probability compared with historical control. We mean event as patient's death, second transplantation or persistence of severe thrombocytopenia
- Overall survival (OS) [ Time Frame: 24 months ]The OS probability compared with historical control.
- Percentage of patients with full/mixed donor chimerism [ Time Frame: 12 months ]Evaluation of the percentage of patients with the full/mixed donor chimerism (whole blood and CD3+ lineage). In addition, patients will be divided in accordance with % of donors cells: >95%; 50%-95%; 10%-49%; <10%. All data will be compared with historical control
- Transplant related mortality (TRM) [ Time Frame: 24 months ]The TRM probability compared with historical control.
- Severe thrombocytopenia (ST) [ Time Frame: 24 months ]The ST probability after HSCT compared with historical control
- Autoimmune complications (AC) [ Time Frame: 24 months ]The AC probability after HSCT compared with historical control
- Acute Graft Versus Host Diseases (aGVHD) [ Time Frame: 12 months ]Cumulative Incidence and severity of aGVHD
- Chronic Graft Versus Host Diseases (cGVHD) [ Time Frame: 24 months ]Cumulative Incidence and severity of cGVHD
- Plerixafor related complications (PRC) [ Time Frame: 2 week ]PRC: severity, features, incidence
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): NCT03019809
|Contact: Dmitry Balashov, MD, PhD||+7(495)287-6570 ext email@example.com|
|Contact: Michael Maschan, Professor||+7(926)firstname.lastname@example.org|
|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 email@example.com|
|Contact: Michael Maschan, Professor +7(926)287-6570 firstname.lastname@example.org|
|Sub-Investigator: Michael Maschan, Professor|
|Sub-Investigator: Alexandra Laberko, MD|
|Sub-Investigator: Svetlana Kozlovskaya, MD|
|Sub-Investigator: Elena Gutovskaya, MD|
|Sub-Investigator: Anna Shcherbina, Professor|
|Study Chair:||Alexei Maschan, Professor||Dmitry Rogachev Federal Research and Clinical Centre of Paediatric Haematology, Oncology and Immunology|
|Principal Investigator:||Dmitry Balashov||Dmitry Rogachev Federal Research and Clinical Centre of Paediatric Haematology, Oncology and Immunology|