A Phase 2 interventional study of G-CSF for Conditioning before HSCT. and Plerixafor for Conditioning before HSCT. in Wiskott-Aldrich Syndrome, Hematopoietic Stem Cell Transplantation and Graft Failure, sponsored by Federal Research Institute of Pediatric Hematology, Oncology and Immunology. Status unknown at 1 site in Russian Federation. Open to participants aged 1 Month to 19 Years. Per ClinicalTrials.gov, last updated 2018-12-12.
Sponsored by Federal Research Institute of Pediatric Hematology, Oncology and Immunology · Phase 2, Interventional, and Treatment
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.
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.
34 studies on the registry are indexed under Wiskott-Aldrich Syndrome; 4 are open to participants now.
This study's planned enrollment of 30 is above the median of 20 across 24 interventional studies indexed under Wiskott-Aldrich Syndrome.
Browse Wiskott-Aldrich Syndrome studies →Federal Research Institute of Pediatric Hematology, Oncology and Immunology is the lead sponsor of 55 studies on the registry; 17 are open to participants now.
Counted across the registry records on this site, refreshed daily.
Exclusion Criteria:
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. · Biological: Plerixafor for Conditioning before HSCT.
Mobilization of hematopoietic stem (HSC) into circulation
Directed inhibition of CXC chemokine receptor type 4 (CXCR4) for opening enough BM niches for adequate donor HSC engraftment.
Event free survival (EFS)
The EFS probability compared with historical control. We mean event as patient's death, second transplantation or persistence of severe thrombocytopenia
Time frame: 24 months
Overall survival (OS)
The OS probability compared with historical control.
Time frame: 24 months
Percentage of patients with full/mixed donor chimerism
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
Time frame: 12 months
Transplant related mortality (TRM)
The TRM probability compared with historical control.
Time frame: 24 months
Severe thrombocytopenia (ST)
The ST probability after HSCT compared with historical control
Time frame: 24 months
Autoimmune complications (AC)
The AC probability after HSCT compared with historical control
Time frame: 24 months
Acute Graft Versus Host Diseases (aGVHD)
Cumulative Incidence and severity of aGVHD
Time frame: 12 months
Chronic Graft Versus Host Diseases (cGVHD)
Cumulative Incidence and severity of cGVHD
Time frame: 24 months
Plerixafor related complications (PRC)
PRC: severity, features, incidence
Time frame: 2 week
This study is status unknown, as verified in Dec 2018. You cannot join it, but the record below documents what was studied.
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Federal Research Institute of Pediatric Hematology, Oncology and Immunology