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

Federal Research Institute of Pediatric Hematology, Oncology and Immunology

Status and phase

Unknown

Phase 2

Conditions

Graft Failure

Wiskott-Aldrich Syndrome

Hematopoietic Stem Cell Transplantation

Treatments

Biological: Plerixafor for Conditioning before HSCT.

Biological: G-CSF for Conditioning before HSCT.

Study type

Interventional

Funder types

Other

Identifiers

NCT03019809

WAS_PG 2016

Details and patient eligibility

About

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.

Full 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.

Enrollment

30 estimated patients

Sex

All

Ages

1 month to 19 years old

Volunteers

No Healthy Volunteers

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.