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

10. december 2018 opdateret af: Federal Research Institute of Pediatric Hematology, Oncology and Immunology

A Trial of Plerixafor With G-CSF as Additional Agents in Conditioning Regimen for Prevention of Graft Failure After Transplantation With TCR Alpha/Beta Grafts Depletion in Patients With Wiskott-Aldrich Syndrome.

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.

Studieoversigt

Status

Ukendt

Betingelser

Intervention / Behandling

Detaljeret beskrivelse

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.

Undersøgelsestype

Interventionel

Tilmelding (Forventet)

30

Fase

  • Fase 2

Kontakter og lokationer

Dette afsnit indeholder kontaktoplysninger for dem, der udfører undersøgelsen, og oplysninger om, hvor denne undersøgelse udføres.

Studiekontakt

  • Navn: Dmitry Balashov, MD, PhD
  • Telefonnummer: 6534 +7(495)287-6570
  • E-mail: bala8@yandex.ru

Undersøgelse Kontakt Backup

  • Navn: Michael Maschan, Professor
  • Telefonnummer: +7(926)651-2145
  • E-mail: mmaschan@yandex.ru

Studiesteder

  • Den Russiske Føderation
      • Moscow, Den Russiske Føderation, 117997
        • Rekruttering
        • Dmitry Rogachev Federal Research and Clinical Centre of Paediatric Haematology, Oncology and Immunology
        • Kontakt:
          • Dmitry Balashov, MD, PhD
          • Telefonnummer: 6534 +7(495)287-6570
          • E-mail: bala8@yandex.ru
        • Kontakt:
        • Underforsker:
          • Michael Maschan, Professor
        • Underforsker:
          • Alexandra Laberko, MD
        • Underforsker:
          • Svetlana Kozlovskaya, MD
        • Underforsker:
          • Elena Gutovskaya, MD
        • Underforsker:
          • Anna Shcherbina, Professor

Deltagelseskriterier

Forskere leder efter personer, der passer til en bestemt beskrivelse, kaldet berettigelseskriterier. Nogle eksempler på disse kriterier er en persons generelle helbredstilstand eller tidligere behandlinger.

Berettigelseskriterier

Aldre berettiget til at studere

1 måned til 19 år (Barn, Voksen)

Tager imod sunde frivillige

Ingen

Køn, der er berettiget til at studere

Alle

Beskrivelse

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.

Studieplan

Dette afsnit indeholder detaljer om studieplanen, herunder hvordan undersøgelsen er designet, og hvad undersøgelsen måler.

Hvordan er undersøgelsen tilrettelagt?

Design detaljer

  • Primært formål: Behandling
  • Tildeling: N/A
  • Interventionel model: Enkelt gruppeopgave
  • Maskning: Ingen (Åben etiket)

Våben og indgreb

Deltagergruppe / Arm
Intervention / Behandling
Eksperimentel: Plerixafor/G-CSF for HSCT conditioning
Myeloablative conditioning regimen with Plerixafor and G-CSF as addition agents before stem cell transplantation in WAS patients.
Biologisk: G-CSF for Conditioning before HSCT.
Mobilization of hematopoietic stem (HSC) into circulation
Biologisk: Plerixafor for Conditioning before HSCT.
Directed inhibition of CXC chemokine receptor type 4 (CXCR4) for opening enough BM niches for adequate donor HSC engraftment.

Hvad måler undersøgelsen?

Primære resultatmål

Resultatmål
Foranstaltningsbeskrivelse
Tidsramme
Event free survival (EFS)
Tidsramme: 24 months
The EFS probability compared with historical control. We mean event as patient's death, second transplantation or persistence of severe thrombocytopenia
24 months

Sekundære resultatmål

Resultatmål
Foranstaltningsbeskrivelse
Tidsramme
Overall survival (OS)
Tidsramme: 24 months
The OS probability compared with historical control.
24 months
Percentage of patients with full/mixed donor chimerism
Tidsramme: 12 months
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
12 months
Transplant related mortality (TRM)
Tidsramme: 24 months
The TRM probability compared with historical control.
24 months
Severe thrombocytopenia (ST)
Tidsramme: 24 months
The ST probability after HSCT compared with historical control
24 months
Autoimmune complications (AC)
Tidsramme: 24 months
The AC probability after HSCT compared with historical control
24 months
Acute Graft Versus Host Diseases (aGVHD)
Tidsramme: 12 months
Cumulative Incidence and severity of aGVHD
12 months
Chronic Graft Versus Host Diseases (cGVHD)
Tidsramme: 24 months
Cumulative Incidence and severity of cGVHD
24 months
Plerixafor related complications (PRC)
Tidsramme: 2 week
PRC: severity, features, incidence
2 week

Samarbejdspartnere og efterforskere

Det er her, du vil finde personer og organisationer, der er involveret i denne undersøgelse.

Sponsor

Federal Research Institute of Pediatric Hematology, Oncology and Immunology

Efterforskere

  • Studiestol: Alexei Maschan, Professor, Dmitry Rogachev Federal Research and Clinical Centre of Paediatric Haematology, Oncology and Immunology
  • Ledende efterforsker: Dmitry Balashov, Dmitry Rogachev Federal Research and Clinical Centre of Paediatric Haematology, Oncology and Immunology

Publikationer og nyttige links

Den person, der er ansvarlig for at indtaste oplysninger om undersøgelsen, leverer frivilligt disse publikationer. Disse kan handle om alt relateret til undersøgelsen.

Generelle publikationer

Datoer for undersøgelser

Disse datoer sporer fremskridtene for indsendelser af undersøgelsesrekord og resumeresultater til ClinicalTrials.gov. Studieregistreringer og rapporterede resultater gennemgås af National Library of Medicine (NLM) for at sikre, at de opfylder specifikke kvalitetskontrolstandarder, før de offentliggøres på den offentlige hjemmeside.

Studer store datoer

Studiestart (Faktiske)

1. juni 2016

Primær færdiggørelse (Faktiske)

1. december 2018

Studieafslutning (Forventet)

1. juli 2019

Datoer for studieregistrering

Først indsendt

11. januar 2017

Først indsendt, der opfyldte QC-kriterier

12. januar 2017

Først opslået (Skøn)

13. januar 2017

Opdateringer af undersøgelsesjournaler

Sidste opdatering sendt (Faktiske)

12. december 2018

Sidste opdatering indsendt, der opfyldte kvalitetskontrolkriterier

10. december 2018

Sidst verificeret

1. december 2018

Mere information

Begreber relateret til denne undersøgelse

Yderligere relevante MeSH-vilkår

Andre undersøgelses-id-numre

  • WAS_PG 2016

Disse oplysninger blev hentet direkte fra webstedet clinicaltrials.gov uden ændringer. Hvis du har nogen anmodninger om at ændre, fjerne eller opdatere dine undersøgelsesoplysninger, bedes du kontakte register@clinicaltrials.gov. Så snart en ændring er implementeret på clinicaltrials.gov, vil denne også blive opdateret automatisk på vores hjemmeside .

Kliniske forsøg med G-CSF for Conditioning before HSCT.

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