NCT03547830

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

Trial Health

43
At Risk

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Trial has exceeded expected completion date
Enrollment
17

participants targeted

Target at below P25 for phase_2

Timeline
Completed

Started Apr 2019

Typical duration for phase_2

Geographic Reach
1 country

1 active site

Status
unknown

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

May 24, 2018

Completed
13 days until next milestone

First Posted

Study publicly available on registry

June 6, 2018

Completed
10 months until next milestone

Study Start

First participant enrolled

April 13, 2019

Completed
3.7 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

January 1, 2023

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

January 1, 2023

Completed
Last Updated

September 17, 2019

Status Verified

September 1, 2019

Enrollment Period

3.7 years

First QC Date

May 24, 2018

Last Update Submit

September 16, 2019

Conditions

Keywords

chronic granulomatous diseasegraft failurestem cell transplantationplerixaforconditioning regimen

Outcome Measures

Primary Outcomes (1)

  • Event free survival

    The EFS probability compared with historical control. We mean event as primary (non-engraftment) and secondary (rejection) graft dysfunction.

    1 year

Secondary Outcomes (5)

  • 1. Overall survival

    1 year

  • Proportion of patients with full/mixed donor chimerism

    30 days

  • 3. Transplant related mortality

    1 year

  • 4. Acute Graft Versus Host Diseases

    100 days

  • 5. Incidence of Plerixafor related toxicity

    100 days

Study Arms (1)

Plerixafor/G-CSF

EXPERIMENTAL

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

Drug: PlerixaforDrug: Gcsf

Interventions

Plerixafor for Conditioning before HSCT.

Plerixafor/G-CSF
GcsfDRUG

GCSF for Conditioning before HSCT.

Plerixafor/G-CSF

Eligibility Criteria

Age1 Month - 24 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64)

You may qualify if:

  • Patients aged ≥ 1 months and \< 25 years Patients diagnosed with CGD eligible for an allogeneic transplantation Signed written informed consent

You may not qualify if:

  • Lack of informed consent.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Dmitry Rogachev National Research Center of Pediatric Hematology, Oncology and Immunology

Moscow, 117997, Russia

RECRUITING

Related Publications (1)

  • 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.

    PMID: 29550630BACKGROUND

MeSH Terms

Conditions

Granulomatous Disease, Chronic

Interventions

plerixaforGranulocyte Colony-Stimulating Factor

Condition Hierarchy (Ancestors)

Phagocyte Bactericidal DysfunctionLeukocyte DisordersHematologic DiseasesHemic and Lymphatic DiseasesGenetic Diseases, X-LinkedGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesImmunologic Deficiency SyndromesImmune System DiseasesChronic DiseaseDisease AttributesPathologic ProcessesPathological Conditions, Signs and Symptoms

Intervention Hierarchy (Ancestors)

Colony-Stimulating FactorsGlycoproteinsGlycoconjugatesCarbohydratesHematopoietic Cell Growth FactorsCytokinesIntercellular Signaling Peptides and ProteinsPeptidesAmino Acids, Peptides, and ProteinsProteinsBiological Factors

Central Study Contacts

Dmitry Balashov, MD

CONTACT

Svetlana Kozlovskaya, MD

CONTACT

Study Design

Study Type
interventional
Phase
phase 2
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

May 24, 2018

First Posted

June 6, 2018

Study Start

April 13, 2019

Primary Completion

January 1, 2023

Study Completion

January 1, 2023

Last Updated

September 17, 2019

Record last verified: 2019-09

Locations