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A Trial of Plerixafor/G-CSF as Additional Agents for Conditioning Before TCR Alpha/Beta Depleted HSCT in WAS Patients

NCT ID: NCT03019809

Last Updated: 2018-12-12

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

UNKNOWN

Clinical Phase

PHASE2

Total Enrollment

30 participants

Study Classification

INTERVENTIONAL

Study Start Date

2016-06-30

Study Completion Date

2019-07-31

Brief Summary

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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 with TCR alpha/beta grafts depletion in patient with Wiskott-Aldrich syndrome.

Detailed Description

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Severe graft dysfunction, such as the degree of donor chimerism predominantly in the myeloid compartment is one of major problem in patients with Wiskott-Aldrich syndrome (WAS), especially after hematopoietic stem cell transplantation (HSCT) from alternative donor. It often leads to the development of severe thrombocytopenia or even transplants rejection. In this study the hypothesis is that the use of plerixafor and G-CSF as additional agents in conditioning regimen would offers advantages due to lowing risk of mixed chimerism after HSCT. This effect is based on the fact that simultaneous use of plerixafor with G-CSF is efficient in inducing stem cell release and opening of bone marrow (BM) niches. 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.

In this study, the investigators use TCR alpha/beta grafts depletion of the grafts as basic technology for HSCT from haploidentical and unrelated donors approved in Institution.

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 transplantation with TCR alpha/beta grafts depletion in patients with Wiskott-Aldrich syndrome.

Conditions

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Wiskott-Aldrich Syndrome Hematopoietic Stem Cell Transplantation Graft Failure

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Plerixafor/G-CSF for HSCT conditioning

Myeloablative conditioning regimen with Plerixafor and G-CSF as addition agents before stem cell transplantation in WAS patients.

Group Type EXPERIMENTAL

G-CSF for Conditioning before HSCT.

Intervention Type BIOLOGICAL

Mobilization of hematopoietic stem (HSC) into circulation

Plerixafor for Conditioning before HSCT.

Intervention Type BIOLOGICAL

Directed inhibition of CXC chemokine receptor type 4 (CXCR4) for opening enough BM niches for adequate donor HSC engraftment.

Interventions

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G-CSF for Conditioning before HSCT.

Mobilization of hematopoietic stem (HSC) into circulation

Intervention Type BIOLOGICAL

Plerixafor for Conditioning before HSCT.

Directed inhibition of CXC chemokine receptor type 4 (CXCR4) for opening enough BM niches for adequate donor HSC engraftment.

Intervention Type BIOLOGICAL

Eligibility Criteria

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Inclusion Criteria

* Patients aged ≥ 1 months and \< 19 years
* Patients diagnosed with Wiskott-Aldrich syndrome eligible for an allogeneic transplantation and lacking a related HLA-matched donor
* Lansky/Karnofsky score \> 40, WHO \> 4
* Signed written informed consent

Exclusion Criteria

* Dysfunction of liver (ALT/AST \> 5 times normal value, or bilirubin \> 3 times normal value), or of renal function (creatinine clearance \< 30 ml / min)
* Severe cardiovascular disease (arrhythmias requiring chronic treatment, congestive heart failure or left ventricular ejection fraction \<40%)
* Serious concurrent uncontrolled medical disorder
* Lack of parents' informed consent.
Minimum Eligible Age

1 Month

Maximum Eligible Age

19 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Federal Research Institute of Pediatric Hematology, Oncology and Immunology

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Alexei Maschan, Professor

Role: STUDY_CHAIR

Dmitry Rogachev Federal Research and Clinical Centre of Paediatric Haematology, Oncology and Immunology

Dmitry Balashov

Role: PRINCIPAL_INVESTIGATOR

Dmitry Rogachev Federal Research and Clinical Centre of Paediatric Haematology, Oncology and Immunology

Locations

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Dmitry Rogachev Federal Research and Clinical Centre of Paediatric Haematology, Oncology and Immunology

Moscow, , Russia

Site Status RECRUITING

Countries

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Russia

Central Contacts

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Dmitry Balashov, MD, PhD

Role: CONTACT

Phone: +7(495)287-6570

Email: [email protected]

Michael Maschan, Professor

Role: CONTACT

Phone: +7(926)651-2145

Email: [email protected]

Facility Contacts

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Dmitry Balashov, MD, PhD

Role: primary

Michael Maschan, Professor

Role: backup

References

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Balashov D, Laberko A, Shcherbina A, Trakhtman P, Abramov D, Gutovskaya E, Kozlovskaya S, Shelikhova L, Novichkova G, Maschan M, Rumiantsev A, Maschan A. A Conditioning Regimen with Plerixafor Is Safe and Improves the Outcome of TCRalphabeta+ and CD19+ Cell-Depleted Stem Cell Transplantation in Patients with Wiskott-Aldrich Syndrome. Biol Blood Marrow Transplant. 2018 Jul;24(7):1432-1440. doi: 10.1016/j.bbmt.2018.03.006. Epub 2018 Mar 14.

Reference Type DERIVED
PMID: 29550630 (View on PubMed)

Other Identifiers

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WAS_PG 2016

Identifier Type: -

Identifier Source: org_study_id