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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03547830
Recruitment Status : Recruiting
First Posted : June 6, 2018
Last Update Posted : September 17, 2019
Sponsor:
Information provided by (Responsible Party):
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

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.

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Study Type : Interventional  (Clinical Trial)
Estimated 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 : April 13, 2019
Estimated Primary Completion Date : January 1, 2023
Estimated Study Completion Date : January 1, 2023


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.




Primary Outcome Measures :
  1. Event free survival [ Time Frame: 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. 1. Overall survival [ Time Frame: 1 year ]
    The OS probability compared with historical control

  2. Proportion of patients with full/mixed donor chimerism [ Time Frame: 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. 3. Transplant related mortality [ Time Frame: 1 year ]
    The TRM probability compared with historical control.

  4. 4. Acute Graft Versus Host Diseases [ Time Frame: 100 days ]
    Cumulative Incidence of aGVHD

  5. 5. Incidence of Plerixafor related toxicity [ Time Frame: 100 days ]
    severity, features, incidence



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Ages Eligible for Study:   1 Month to 24 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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.


Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03547830


Contacts
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Contact: Dmitry Balashov, MD +79265791817 bala8@yandex.ru
Contact: Svetlana Kozlovskaya, MD +79165587891 lana.n.kozlovskaya@gmail.com

Locations
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Russian Federation
Dmitry Rogachev National Research Center of Pediatric Hematology, Oncology and Immunology Recruiting
Moscow, Russian Federation, 117997
Contact: Zhanna Shekhovtsova, MD    4956647078 ext 7538    zhanna.shekhovtsova@fccho-moscow.ru   
Contact: Eugene Pashanov, PhD    +79262205578    e.pashanov@gmail.com   
Principal Investigator: Dmitry Balashov, MD         
Sponsors and Collaborators
Federal Research Institute of Pediatric Hematology, Oncology and Immunology
Publications:
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Responsible Party: Federal Research Institute of Pediatric Hematology, Oncology and Immunology
ClinicalTrials.gov Identifier: NCT03547830    
Other Study ID Numbers: NCPHOI-2018-02
First Posted: June 6, 2018    Key Record Dates
Last Update Posted: September 17, 2019
Last Verified: September 2019

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Federal Research Institute of Pediatric Hematology, Oncology and Immunology:
chronic granulomatous disease
graft failure
stem cell transplantation
plerixafor
conditioning regimen
Additional relevant MeSH terms:
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Granuloma
Granulomatous Disease, Chronic
Lymphoproliferative Disorders
Lymphatic Diseases
Pathologic Processes
Phagocyte Bactericidal Dysfunction
Leukocyte Disorders
Hematologic Diseases
Genetic Diseases, X-Linked
Genetic Diseases, Inborn
Immunologic Deficiency Syndromes
Immune System Diseases
Plerixafor octahydrochloride
Anti-HIV Agents
Anti-Retroviral Agents
Antiviral Agents
Anti-Infective Agents