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

Phase 2

• Conditions: Chronic Granulomatous Disease
• Interventions: Drug: Plerixafor; Drug: Gcsf

• Registration Number: NCT03547830
• Lead Sponsor: Federal Research Institute of Pediatric Hematology, Oncology and Immunology

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

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 Timeline

• Study First Submitted: 2018-05-24
• Study First Submitted QC: 2018-05-24
• Study First Posted: 2018-06-06
• Study Start: 2019-04-13
• Status Verified: 2019-09
• Last Update Submitted: 2019-09-16
• Last Update Posted: 2019-09-17
• Primary Completion: 2023-01-01
• Study Completion: 2023-01-01

Recruitment & Eligibility

• Status: UNKNOWN
• Sex: All
• Target Recruitment: 17

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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

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

Primary Outcome Measures

Name	Time	Method
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

Name	Time	Method
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
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
1. Overall survival	1 year	The OS probability compared with historical control

Trial Locations

Locations (1)

Dmitry Rogachev National Research Center of Pediatric Hematology, Oncology and Immunology; 🇷🇺 Moscow, Russian Federation