Plerixafor/G-CSF as Additional Agents for Conditioning Before HSCT in CGD Patients

Federal Research Institute of Pediatric Hematology, Oncology and Immunology

Status and phase: Unknown; Phase 2

Conditions: Chronic Granulomatous Disease

Treatments: Drug: Plerixafor; Drug: Gcsf

Study type: Interventional

Funder types: Other

Identifiers

NCT03547830

NCPHOI-2018-02

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 in patients with chronic granulomatous disease

Full description

Severe primary or secondary graft dysfunction is one of major problem in patients with Chronic granulomatous disease (CGD). In this study the hypothesis is that the use of plerixafor and G-CSF as additional agents in conditioning regimen would offers advantages. The effect is based on mobilizing bone marrow stem cells into the peripheral blood and blocking CXCR4 chemokine receptors to prevent stem cell homing. Thus, some have hypothesized that plerixafor and G-CSF make free stromal space of the bone marrow available for donor stem cell engraftment. 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. 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 stem cell transplantation in patients with chronic granulomatous disease.

Enrollment: 17 estimated patients

Sex: All

Ages: 1 month to 24 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

Patients aged ≥ 1 months and < 25 years Patients diagnosed with CGD eligible for an allogeneic transplantation Signed written informed consent

Exclusion criteria

Lack of informed consent.

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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17 participants in 1 patient group

Plerixafor/G-CSF

Experimental group

Description:

Plerixafor/G-CSF for HSCT conditioning Myeloablative conditioning regimen with Plerixafor as addition agent before stem cell transplantation in CGD patients

Treatment:

Drug: Gcsf

Drug: Plerixafor