- ICH GCP
- US Clinical Trials Registry
- Klinisk forsøg NCT00167206
Stem Cell Transplantation for Fanconi Anemia
21. august 2019 opdateret af: 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.
Studieoversigt
Status
Afsluttet
Betingelser
Detaljeret 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.
Undersøgelsestype
Interventionel
Tilmelding (Faktiske)
16
Fase
- Fase 2
- Fase 1
Kontakter og lokationer
Dette afsnit indeholder kontaktoplysninger for dem, der udfører undersøgelsen, og oplysninger om, hvor denne undersøgelse udføres.
Studiesteder
-
-
Minnesota
-
Minneapolis, Minnesota, Forenede Stater, 55455
- Masonic Cancer Center, University of Minnesota
-
-
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 dag til 18 år (Barn, Voksen)
Tager imod sunde frivillige
Ingen
Køn, der er berettiget til at studere
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
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: Ikke-randomiseret
- Interventionel model: Enkelt gruppeopgave
- Maskning: Ingen (Åben etiket)
Våben og indgreb
Deltagergruppe / Arm |
Intervention / Behandling |
---|---|
Eksperimentel: HSCT Patients
Patients who received total body irradiation (450 cGy [centigray]) with thymic shielding prior to chemotherapy regimen and Hematopoietic Stem Cell Transplant (HSCT)
|
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 navne:
protecting the thymus during total body radiation (450 cGy administered)
Andre navne:
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 navne:
Day -5 through Day -2, subjects will receive a chemotherapy regimen of Fludarabine, Cyclophosphamide via central line
Andre navne:
|
Hvad måler undersøgelsen?
Primære resultatmål
Resultatmål |
Foranstaltningsbeskrivelse |
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 |
Foranstaltningsbeskrivelse |
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
|
Samarbejdspartnere og efterforskere
Det er her, du vil finde personer og organisationer, der er involveret i denne undersøgelse.
Efterforskere
- Ledende efterforsker: Margaret MacMillan, MD, Masonic Cancer Center, University of Minnesota
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.
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
1. marts 2004
Primær færdiggørelse (Faktiske)
1. december 2008
Studieafslutning (Faktiske)
1. december 2008
Datoer for studieregistrering
Først indsendt
9. september 2005
Først indsendt, der opfyldte QC-kriterier
9. september 2005
Først opslået (Skøn)
14. september 2005
Opdateringer af undersøgelsesjournaler
Sidste opdatering sendt (Faktiske)
26. august 2019
Sidste opdatering indsendt, der opfyldte kvalitetskontrolkriterier
21. august 2019
Sidst verificeret
1. august 2019
Mere information
Begreber relateret til denne undersøgelse
Yderligere relevante MeSH-vilkår
- Metaboliske sygdomme
- Nyresygdomme
- Urologiske sygdomme
- Knoglemarvssygdomme
- Hæmatologiske sygdomme
- Genetiske sygdomme, medfødte
- DNA-reparation-mangellidelser
- Anæmi, hypoplastisk, medfødt
- Anæmi, aplastisk
- Medfødte knoglemarvssvigtsyndromer
- Knoglemarvssvigtsforstyrrelser
- Renal Tubular Transport, Medfødte Fejl
- Anæmi
- Fanconi syndrom
- Fanconi Anæmi
- Lægemidlers fysiologiske virkninger
- Molekylære mekanismer for farmakologisk virkning
- Antirheumatiske midler
- Antineoplastiske midler
- Immunsuppressive midler
- Immunologiske faktorer
- Antineoplastiske midler, Alkylering
- Alkyleringsmidler
- Myeloablative agonister
- Cyclofosfamid
- Fludarabin
Andre undersøgelses-id-numre
- 0312M54991
- MT2003-18 (Anden identifikator: Blood and Marrow Transplantation Program)
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 Fanconi Anæmi
-
Rocket Pharmaceuticals Inc.Aktiv, ikke rekrutterendeFanconi Anæmi Komplementeringsgruppe ASpanien
-
Rocket Pharmaceuticals Inc.UkendtFanconi Anæmi Komplementeringsgruppe AForenede Stater
-
Rocket Pharmaceuticals Inc.Tilmelding efter invitationFanconi Anæmi | Fanconi Anæmi Komplementeringsgruppe ASpanien
-
Baylor College of MedicineCenter for Cell and Gene Therapy, Baylor College of Medicine; University...AfsluttetFANCONI AnæmiForenede Stater
-
Cystinosis Research FoundationUkendtCystinose | Nefropatisk cystinose | Renal Fanconi syndromForenede Stater
-
Rocket Pharmaceuticals Inc.Aktiv, ikke rekrutterendeFanconi Anæmi Komplementeringsgruppe AForenede Stater
-
CENTOGENE GmbH RostockTrukket tilbageHypofosfatæmi | Fotofobi | Renal Fanconi syndromTyskland, Indien, Sri Lanka
-
Masonic Cancer Center, University of MinnesotaAfsluttet
-
OHSU Knight Cancer InstituteNational Cancer Institute (NCI)AfsluttetFanconi AnæmiForenede Stater
-
Memorial Sloan Kettering Cancer CenterNational Cancer Institute (NCI); Miltenyi Biotec, Inc.Afsluttet