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Clinical Trial Details — Status: Recruiting

Administrative data

NCT number NCT03019809
Other study ID # WAS_PG 2016
Secondary ID
Status Recruiting
Phase Phase 2
First received
Last updated
Start date June 2016
Est. completion date July 2019

Study information

Verified date December 2018
Source Federal Research Institute of Pediatric Hematology, Oncology and Immunology
Contact Dmitry Balashov, MD, PhD
Phone +7(495)287-6570
Email bala8@yandex.ru
Is FDA regulated No
Health authority
Study type Interventional

Clinical Trial 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.


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.


Recruitment information / eligibility

Status Recruiting
Enrollment 30
Est. completion date July 2019
Est. primary completion date December 2018
Accepts healthy volunteers No
Gender All
Age group 1 Month to 19 Years
Eligibility 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.

Study Design


Related Conditions & MeSH terms


Intervention

Biological:
G-CSF for Conditioning before HSCT.
Mobilization of hematopoietic stem (HSC) into circulation
Plerixafor for Conditioning before HSCT.
Directed inhibition of CXC chemokine receptor type 4 (CXCR4) for opening enough BM niches for adequate donor HSC engraftment.

Locations

Country Name City State
Russian Federation Dmitry Rogachev Federal Research and Clinical Centre of Paediatric Haematology, Oncology and Immunology Moscow

Sponsors (1)

Lead Sponsor Collaborator
Federal Research Institute of Pediatric Hematology, Oncology and Immunology

Country where clinical trial is conducted

Russian Federation, 

Outcome

Type Measure Description Time frame Safety issue
Primary 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 24 months
Secondary Overall survival (OS) The OS probability compared with historical control. 24 months
Secondary 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 12 months
Secondary Transplant related mortality (TRM) The TRM probability compared with historical control. 24 months
Secondary Severe thrombocytopenia (ST) The ST probability after HSCT compared with historical control 24 months
Secondary Autoimmune complications (AC) The AC probability after HSCT compared with historical control 24 months
Secondary Acute Graft Versus Host Diseases (aGVHD) Cumulative Incidence and severity of aGVHD 12 months
Secondary Chronic Graft Versus Host Diseases (cGVHD) Cumulative Incidence and severity of cGVHD 24 months
Secondary Plerixafor related complications (PRC) PRC: severity, features, incidence 2 week
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