A Phase 2 interventional study of Plerixafor and Gcsf in Chronic Granulomatous Disease, sponsored by Federal Research Institute of Pediatric Hematology, Oncology and Immunology. Status unknown at 1 site in Russian Federation. Open to participants aged 1 Month to 24 Years. Per ClinicalTrials.gov, last updated 2019-09-17.
Sponsored by Federal Research Institute of Pediatric Hematology, Oncology and Immunology · Phase 2, Interventional, and Treatment
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
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.
79 studies on the registry are indexed under Granuloma; 8 are open to participants now.
This study's planned enrollment of 17 is below the median of 25 across 56 interventional studies indexed under Granuloma.
Browse Granuloma studies →Federal Research Institute of Pediatric Hematology, Oncology and Immunology is the lead sponsor of 55 studies on the registry; 17 are open to participants now.
Counted across the registry records on this site, refreshed daily.
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.
Plerixafor/G-CSF for HSCT conditioning Myeloablative conditioning regimen with Plerixafor as addition agent before stem cell transplantation in CGD patients
Drug: Plerixafor · Drug: Gcsf
Plerixafor for Conditioning before HSCT.
GCSF for Conditioning before HSCT.
Event free survival
The EFS probability compared with historical control. We mean event as primary (non-engraftment) and secondary (rejection) graft dysfunction.
Time frame: 1 year
1. Overall survival
The OS probability compared with historical control
Time frame: 1 year
Proportion of patients with full/mixed donor chimerism
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
Time frame: 30 days
3. Transplant related mortality
The TRM probability compared with historical control.
Time frame: 1 year
4. Acute Graft Versus Host Diseases
Cumulative Incidence of aGVHD
Time frame: 100 days
5. Incidence of Plerixafor related toxicity
severity, features, incidence
Time frame: 100 days
This study is status unknown, as verified in Sep 2019. You cannot join it, but the record below documents what was studied.
Get an email when the registry record changes — status, dates, results — or when someone posts here.
Sign in to followQuestions and observations about this study, from anyone following it. Not medical advice, and not a channel to the study team — their contact details are on the registry record.
Sign in to join the discussion. Reading takes no account; posting does. You choose a display name, and a pseudonym is the default.
Nothing here yet. If you are running this trial, taking part in it, or weighing whether to, this is the place to say so.
Federal Research Institute of Pediatric Hematology, Oncology and Immunology