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Stem Cell Transplantation for Fanconi Anemia

21. august 2019 oppdatert av: Masonic Cancer Center, University of Minnesota

A Study of Thymic Shielding in Recipients of Total Body Irradiation, Cyclophosphamide, and Fludarabine Followed by Alternate Donor Hematopoietic Stem Cell Transplantation in Patients With Fanconi Anemia

The purpose of this study is to determine whether thymic shielding during total body irradiation can be given and whether it will reduce the risk of infections in Fanconi Anemia patients undergoing alternate donor (not a matched sibling) stem cell transplants.

Studieoversikt

Status

Avsluttet

Forhold

Intervensjon / Behandling

Detaljert beskrivelse

All subjects will be given the same treatment regimen of total body irradiation (TBI), Fludarabine, Cyclophosphamide, and anti-thymocyte globulin (ATG), followed by an alternate donor stem cell transplant. Since this treatment regimen has been given before, without thymic shielding, we will compare the outcomes of these patients with the historical data from subjects who did not receive thymic shielding.

Studietype

Intervensjonell

Registrering (Faktiske)

16

Fase

  • Fase 2
  • Fase 1

Kontakter og plasseringer

Denne delen inneholder kontaktinformasjon for de som utfører studien, og informasjon om hvor denne studien blir utført.

Studiesteder

  • Forente stater
    • Minnesota
      • Minneapolis, Minnesota, Forente stater, 55455
        • Masonic Cancer Center, University of Minnesota

Deltakelseskriterier

Forskere ser etter personer som passer til en bestemt beskrivelse, kalt kvalifikasjonskriterier. Noen eksempler på disse kriteriene er en persons generelle helsetilstand eller tidligere behandlinger.

Kvalifikasjonskriterier

Alder som er kvalifisert for studier

1 dag til 18 år (Barn, Voksen)

Tar imot friske frivillige

Nei

Kjønn som er kvalifisert for studier

Alle

Beskrivelse

Inclusion Criteria:

  • Patients must be less than (<) 18 years of age with a diagnosis of Fanconi anemia.
  • Patients must have an HLA-A, B, DRB1 identical unrelated donor or less than or equal to (≤)1 antigen mismatched related (non-HLA-matched sibling) or <1 antigen mismatched unrelated UCB donor. Patients and donors will be typed for HLA-A and B using serological or molecular techniques and for DRB1 using high resolution molecular typing.

  • Patients with FA must have aplastic anemia (AA), myelodysplastic syndrome without excess blasts, or high risk genotype as defined below.

    • Aplastic anemia is defined as having at least one of the following when not receiving growth factors or transfusions
    • Platelet count <20 x 10^9/L
    • ANC <5 x 10^8/L
    • Hgb <8 g/dL
    • Myelodysplastic syndrome with multilineage dysplasia with or without chromosomal anomalies
    • High risk genotype (e.g. IVS-4 or exon 14 FANCC mutations, or BRCA1 or 2 mutations)

  • Adequate major organ function including

    • Cardiac: ejection fraction greater than (>)45%
    • Hepatic: bilirubin, AST/ALT, ALP <2 x normal
    • Karnofsky performance status >70% or Lansky performance status >50%
  • Women of child-bearing age must be using adequate birth control and have a negative pregnancy test

Exclusion Criteria:

  • Available HLA-genotypically identical related donor
  • History of gram negative sepsis or systemic fungal infection (proven or suspected based on radiographic studies)
  • Refractory anemia with excess blasts, or leukemia
  • Active central nervous system (CNS) leukemia at time of hematopoietic cell transplant (HCT)
  • History of squamous cell carcinoma of the head/neck/cervix within 2 years of HCT
  • Pregnant or lactating female
  • Prior radiation therapy preventing use of total body irradiation (TBI) 450 centigray (cGy)

Studieplan

Denne delen gir detaljer om studieplanen, inkludert hvordan studien er utformet og hva studien måler.

Hvordan er studiet utformet?

Designdetaljer

  • Primært formål: Behandling
  • Tildeling: Ikke-randomisert
  • Intervensjonsmodell: Enkeltgruppeoppdrag
  • Masking: Ingen (Open Label)

Våpen og intervensjoner

Deltakergruppe / Arm
Intervensjon / Behandling
Eksperimentell: HSCT Patients
Patients who received total body irradiation (450 cGy [centigray]) with thymic shielding prior to chemotherapy regimen and Hematopoietic Stem Cell Transplant (HSCT)
Fremgangsmåte: Hematopoietic Stem Cell Transplant
Bone marrow failure may be treated by giving patients stem cells that come from someone else. This is called a stem-cell transplant. As part of the transplant process, patients receive high doses of chemotherapy and/or radiation to treat their underlying disease. As one of its effects, this treatment also kills the healthy stem cells that are already in the marrow. The transplant provides new stem cells for the patient from a healthy donor; that replace the bone marrow and allow the blood counts to recover.
Andre navn:
  • Beinmargstransplantasjon
Fremgangsmåte: Thymic Shielding During Radiation
protecting the thymus during total body radiation (450 cGy administered)
Andre navn:
  • TBI
Fremgangsmåte: Total Body Irradiation
Six days before the stem cells are given (day -6), subjects will receive total body irradiation with thymic shielding. Thymic shielding is done by placing a piece of lead on the chest during the irradiation treatment so that the irradiation beams do not go to the thymus.
Andre navn:
  • Radiation Therapy, Therapuetic Radiation
Legemiddel: Cyclophosphamide, Fludarabine
Day -5 through Day -2, subjects will receive a chemotherapy regimen of Fludarabine, Cyclophosphamide via central line
Andre navn:
  • Cytoxan, Fludara

Hva måler studien?

Primære resultatmål

Resultatmål
Tiltaksbeskrivelse
Tidsramme
Number of Patients Who Exhibited Hematopoietic Recovery and Engraftment
Tidsramme: Day 42 after hematopoietic cell transplant
Calculated from Day 1 of hematopoietic cell transplant to Day 42 post-transplant. Hematopoietic recovery and engraftment is defined as the first of three consecutive days the patient's absolute neutrophil count is greater than or equal to 0.5X10^9/Liter.
Day 42 after hematopoietic cell transplant

Sekundære resultatmål

Resultatmål
Tiltaksbeskrivelse
Tidsramme
Number of Patients Who Exhibited Secondary Graft Failure
Tidsramme: Day 100 after hematopoietic cell transplant
Calculated from Day 1 of hematopoietic cell transplant to Day 100 after transplant. A complication after Bone Marrow Transplant in which the transplanted stem cells do not grow in the recipient's bone marrow and thus do not produce new blood cells.
Day 100 after hematopoietic cell transplant
Number of Patients With Acute Graft Versus-Host Disease (aGVHD)
Tidsramme: Day 100 after hematopoietic cell transplant
Calculated from Day 1 of hematopoietic cell transplant to Day 100 after transplant. GVHD is a common complication of allogeneic bone marrow transplantation in which functional immune cells in the transplanted marrow recognize the recipient as "foreign" and mount an immunologic attack.
Day 100 after hematopoietic cell transplant
Number of Patients With Chronic Graft Versus-Host Disease (GVHD)
Tidsramme: 1 year after hematopoietic cell transplant
Calculated from Day 1 of hematopoietic cell transplant to 1 year after transplant. GVHD is a common complication of allogeneic bone marrow transplantation in which functional immune cells in the transplanted marrow recognize the recipient as "foreign" and mount an immunologic attack.
1 year after hematopoietic cell transplant
Number of Patients Who Exhibited Regimen-related Toxicity (RRT)
Tidsramme: 1 year after hematopoietic cell transplant
Calculated from Day 1 of hematopoietic cell transplant to 1 year after transplant. Regimen-related toxicity involves harmful effects in an organism through exposure to the treatment given.
1 year after hematopoietic cell transplant
Immune Reconstitution - Mean Value (1 Year)
Tidsramme: 1 year post-transplant.
Calculated mean value of patient CD4 values collected at intervals from Day 30 through 1 year post-transplant.
1 year post-transplant.
Immune Reconstitution - Mean Value (2 Years)
Tidsramme: at 2 years after transplant
Calculated mean value of patient CD4 values collected at intervals from Day 30 through 2 years post-transplant.
at 2 years after transplant
Number of Patients Alive at 1 Year
Tidsramme: 1 year after transplant
Calculated from Day 1 of hematopoietic cell transplant to 1 year post-transplant.
1 year after transplant
Number of Patients Alive at 2 Years
Tidsramme: 2 years after transplant
Calculated from Day 1 of hematopoietic cell transplant to 2 years post-transplant.
2 years after transplant

Samarbeidspartnere og etterforskere

Det er her du vil finne personer og organisasjoner som er involvert i denne studien.

Sponsor

Masonic Cancer Center, University of Minnesota

Etterforskere

  • Hovedetterforsker: Margaret MacMillan, MD, Masonic Cancer Center, University of Minnesota

Publikasjoner og nyttige lenker

Den som er ansvarlig for å legge inn informasjon om studien leverer frivillig disse publikasjonene. Disse kan handle om alt relatert til studiet.

Generelle publikasjoner

Studierekorddatoer

Disse datoene sporer fremdriften for innsending av studieposter og sammendragsresultater til ClinicalTrials.gov. Studieposter og rapporterte resultater gjennomgås av National Library of Medicine (NLM) for å sikre at de oppfyller spesifikke kvalitetskontrollstandarder før de legges ut på det offentlige nettstedet.

Studer hoveddatoer

Studiestart

1. mars 2004

Primær fullføring (Faktiske)

1. desember 2008

Studiet fullført (Faktiske)

1. desember 2008

Datoer for studieregistrering

Først innsendt

9. september 2005

Først innsendt som oppfylte QC-kriteriene

9. september 2005

Først lagt ut (Anslag)

14. september 2005

Oppdateringer av studieposter

Sist oppdatering lagt ut (Faktiske)

26. august 2019

Siste oppdatering sendt inn som oppfylte QC-kriteriene

21. august 2019

Sist bekreftet

1. august 2019

Mer informasjon

Begreper knyttet til denne studien

Nøkkelord

Ytterligere relevante MeSH-vilkår

Andre studie-ID-numre

  • 0312M54991
  • MT2003-18 (Annen identifikator: Blood and Marrow Transplantation Program)

Denne informasjonen ble hentet direkte fra nettstedet clinicaltrials.gov uten noen endringer. Hvis du har noen forespørsler om å endre, fjerne eller oppdatere studiedetaljene dine, vennligst kontakt register@clinicaltrials.gov. Så snart en endring er implementert på clinicaltrials.gov, vil denne også bli oppdatert automatisk på nettstedet vårt. .

Kliniske studier på Fanconi anemi

Kliniske studier på Hematopoietic Stem Cell Transplant

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