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

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

Status

UNKNOWN

Phases

Phase 2

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT03547830

Enrollment

Registered

2018-06-06

Start date

2019-04-13

Completion date

2023-01-01

Last updated

2019-09-17

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Chronic Granulomatous Disease

Keywords

chronic granulomatous disease, graft failure, stem cell transplantation, plerixafor, conditioning regimen

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.

Interventions

DRUGPlerixafor

Plerixafor for Conditioning before HSCT.

DRUGGcsf

GCSF for Conditioning before HSCT.

Study design

Allocation

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Masking

NONE

Eligibility

Sex/Gender

ALL

Age

1 Months to 24 Years

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.

Design outcomes

Primary

Measure	Time frame	Description
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

Measure	Time frame	Description
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

Countries

Russia

Contacts

Primary ContactDmitry Balashov, MD

bala8@yandex.ru+79265791817

Backup ContactSvetlana Kozlovskaya, MD

lana.n.kozlovskaya@gmail.com+79165587891

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 4, 2026