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

Sponsor

Federal Research Institute of Pediatric Hematology, Oncology and Immunology (Other)

Overall Status

Recruiting

CT.gov ID

NCT03547830

Collaborator

(none)

Enrollment

Location

Arm

44.6

Anticipated Duration (Months)

0.4

Patients Per Site Per Month

Study Details

Study Description

Brief 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 in patients with chronic granulomatous disease

Condition or Disease	Intervention/Treatment	Phase
Chronic Granulomatous Disease	Drug: Plerixafor Drug: Gcsf	Phase 2

Detailed 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.

Study Design

Study Type:

Interventional

Anticipated Enrollment :

17 participants

Allocation:

N/A

Intervention Model:

Single Group Assignment

Masking:

None (Open Label)

Primary Purpose:

Treatment

Official Title:

A Clinical Trial of Plerixafor With G-CSF as Additional Agents in Conditioning Regimen for Prevention of Graft Failure in Patients With Chronic Granulomatous Disease

Actual Study Start Date :

Apr 13, 2019

Anticipated Primary Completion Date :

Jan 1, 2023

Anticipated Study Completion Date :

Jan 1, 2023

Arms and Interventions

Arm	Intervention/Treatment
Experimental: Plerixafor/G-CSF Plerixafor/G-CSF for HSCT conditioning Myeloablative conditioning regimen with Plerixafor as addition agent before stem cell transplantation in CGD patients	Drug: Plerixafor Plerixafor for Conditioning before HSCT. Drug: Gcsf GCSF for Conditioning before HSCT.

Arm

Intervention/Treatment

Experimental: Plerixafor/G-CSF

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

Drug: Plerixafor

Plerixafor for Conditioning before HSCT.

Drug: Gcsf

GCSF for Conditioning before HSCT.

Outcome Measures

Primary Outcome Measures

Event free survival [1 year]
The EFS probability compared with historical control. We mean event as primary (non-engraftment) and secondary (rejection) graft dysfunction.

Secondary Outcome Measures

1. Overall survival [1 year]
The OS probability compared with historical control
Proportion of patients with full/mixed donor chimerism [30 days]
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
3. Transplant related mortality [1 year]
The TRM probability compared with historical control.
4. Acute Graft Versus Host Diseases [100 days]
Cumulative Incidence of aGVHD
5. Incidence of Plerixafor related toxicity [100 days]
severity, features, incidence