NCT03019809

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

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
30

participants targeted

Target at P25-P50 for phase_2

Timeline
Completed

Started Jun 2016

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

Study Start

First participant enrolled

June 1, 2016

Completed
7 months until next milestone

First Submitted

Initial submission to the registry

January 11, 2017

Completed
2 days until next milestone

First Posted

Study publicly available on registry

January 13, 2017

Completed
1.9 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

December 1, 2018

Completed
7 months until next milestone

Study Completion

Last participant's last visit for all outcomes

July 1, 2019

Completed
Last Updated

December 12, 2018

Status Verified

December 1, 2018

Enrollment Period

2.5 years

First QC Date

January 11, 2017

Last Update Submit

December 10, 2018

Conditions

Wiskott-Aldrich SyndromeHematopoietic Stem Cell TransplantationGraft Failure

Outcome Measures

Primary Outcomes (1)

  • Event free survival (EFS)

    The EFS probability compared with historical control. We mean event as patient's death, second transplantation or persistence of severe thrombocytopenia

    24 months

Secondary Outcomes (8)

  • Overall survival (OS)

    24 months

  • Percentage of patients with full/mixed donor chimerism

    12 months

  • Transplant related mortality (TRM)

    24 months

  • Severe thrombocytopenia (ST)

    24 months

  • Autoimmune complications (AC)

    24 months

  • +3 more secondary outcomes

Study Arms (1)

Plerixafor/G-CSF for HSCT conditioning

EXPERIMENTAL

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

Biological: G-CSF for Conditioning before HSCT.Biological: Plerixafor for Conditioning before HSCT.

Interventions

G-CSF for Conditioning before HSCT.BIOLOGICAL

Mobilization of hematopoietic stem (HSC) into circulation

Plerixafor/G-CSF for HSCT conditioning
Plerixafor for Conditioning before HSCT.BIOLOGICAL

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

Plerixafor/G-CSF for HSCT conditioning

Eligibility Criteria

Age1 Month - 19 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64)

You may qualify if:

  • 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

You may not qualify if:

  • 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.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Dmitry Rogachev Federal Research and Clinical Centre of Paediatric Haematology, 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: 29550630DERIVED

MeSH Terms

Conditions

Wiskott-Aldrich Syndrome

Interventions

Granulocyte Colony-Stimulating Factorplerixafor

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, InheritedBlood Coagulation DisordersHematologic DiseasesHemic and Lymphatic DiseasesLymphopeniaLeukopeniaCytopeniaHemorrhagic DisordersLeukocyte DisordersGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesGenetic Diseases, X-LinkedPrimary Immunodeficiency DiseasesImmunologic Deficiency SyndromesImmune System Diseases

Intervention Hierarchy (Ancestors)

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

Study Officials

  • Alexei Maschan, Professor

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

    STUDY CHAIR

  • Dmitry Balashov

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

    PRINCIPAL INVESTIGATOR

Central Study Contacts

Dmitry Balashov, MD, PhD

CONTACT

+7(495)287-6570bala8@yandex.ru

Michael Maschan, Professor

CONTACT

+7(926)651-2145mmaschan@yandex.ru

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

January 11, 2017

First Posted

January 13, 2017

Study Start

June 1, 2016

Primary Completion

December 1, 2018

Study Completion

July 1, 2019

Last Updated

December 12, 2018

Record last verified: 2018-12

Locations

RU(1)