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Study of Molecular and Genetic Abnormalities in Patients With Myeloid Neoplasms

7 november 2016 bijgewerkt door: Fabio Pires de Souza Santos, Hospital Israelita Albert Einstein

Evaluation of the Incidence and Prognostic Impact of Molecular and Genetic Abnormalities in Patients With Acute Myeloid Leukemia, Myelodysplastic Syndrome and Myeloproliferative Neoplasms

The objective of this study is to describe the prevalence and prognostic impact of the most common genetic abnormalities in patients with Myeloid Neoplasms, including Acute Myeloid Leukemia (AML), Myeloproliferative Neoplasms (MPN), Myelodysplastic Syndromes (MDS) and Myeloproliferative/Myelodysplastic Neoplasms. Patients will have samples of blood and/or bone marrow collected and sent to Hospital Israelita Albert Einstein for analysis and storage.

Patients with a diagnosis of Acute Myeloid Leukemia will be treated according to an uniform protocol.

Studie Overzicht

Studietype

Ingrijpend

Inschrijving (Werkelijk)

455

Fase

  • Fase 2

Contacten en locaties

In dit gedeelte vindt u de contactgegevens van degenen die het onderzoek uitvoeren en informatie over waar dit onderzoek wordt uitgevoerd.

Studie Locaties

    • SP
      • Sao Paulo, SP, Brazilië, 05651901
        • Hospital israelita Albert Einstein

Deelname Criteria

Onderzoekers zoeken naar mensen die aan een bepaalde beschrijving voldoen, de zogenaamde geschiktheidscriteria. Enkele voorbeelden van deze criteria zijn iemands algemene gezondheidstoestand of eerdere behandelingen.

Geschiktheidscriteria

Leeftijden die in aanmerking komen voor studie

18 jaar en ouder (Volwassen, Oudere volwassene)

Accepteert gezonde vrijwilligers

Nee

Geslachten die in aanmerking komen voor studie

Allemaal

Beschrijving

Acute Myeloid Leukemia-Intensive Chemotherapy

Inclusion Criteria:

  • Diagnosis of AML according to WHO criteria
  • Age greater than 18 years
  • Performance status (ECOG) between 0-2
  • Adequate liver and kidney function
  • Signed Informed Consent form
  • No prior therapy for AML, except use of hydroxyurea for control of elevated white blood cell counts
  • Adequate contraception for fertile men and women
  • Eligible for intensive chemotherapy (as judged by the treating physician)

Exclusion Criteria:

  • Acute myeloid leukemia with retinoic acid receptor alpha (RARA) translocations (APL, acute promyelocytic leukemia)
  • Pregnant women
  • HIV-positivity
  • New York Heart Association class III and IV congestive heart failure
  • Patient refuses to use adequate contraception
  • History of hypersensibility to any of the used chemotherapy drugs
  • Patient refuses to sign informed consent form

Acute Myeloid Leukemia-Non-Intensive Chemotherapy

Inclusion Criteria:

  • Diagnosis of AML according to WHO criteria
  • Age greater than 18 years
  • Signed Informed Consent form
  • No prior therapy for AML, except use of hydroxyurea for control of elevated white blood cell counts
  • Adequate contraception for fertile men and women
  • Non-eligible for intensive chemotherapy (as judged by the treating physician)

Exclusion Criteria:

  • Acute myeloid leukemia with RARA translocations (APL, acute promyelocytic leukemia)
  • Pregnant women
  • HIV-positivity
  • Patient refuses to use adequate contraception
  • History of hypersensibility to any of the used chemotherapy drugs
  • Patient refuses to sign informed consent form

Chronic Myeloid Disorders:

Inclusion Criteria:

  • Diagnosis of Myeloproliferative Neoplasm or Myelodysplastic Syndrome or Myeloproliferative/Myelodysplastic Neoplasm according to WHO criteria
  • Age greater than 18 years
  • Signed Informed Consent form

Exclusion Criteria:

  • Patient refuses to sign informed consent form

Studie plan

Dit gedeelte bevat details van het studieplan, inclusief hoe de studie is opgezet en wat de studie meet.

Hoe is de studie opgezet?

Ontwerpdetails

  • Primair doel: Fundamentele wetenschap
  • Toewijzing: Niet-gerandomiseerd
  • Interventioneel model: Parallelle opdracht
  • Masker: Geen (open label)

Wapens en interventies

Deelnemersgroep / Arm
Interventie / Behandeling
Ander: AML-Intensive Chemotherapy

Patients with Acute Myeloid Leukemia fit for intensive chemotherapy Patients will receive Induction Chemotherapy, and CR will be evaluated after 28 days.

Patients who achieve CR post-induction chemotherapy will receive post-remission therapy according to risk:

  • Low risk patients: Consolidation chemotherapy or Autologous stem cell transplantation
  • Intermediate and high-risk patients: Allogeneic stem cell transplantation Patients who do not achieve CR may receive one second induction cycle, and if CR is achieved may proceed to post-remission therapy as per above. Patients who do not achieve CR after two cycles of induction will be deemed refractory and removed from the study.

Induction chemotherapy for patients with AML eligible for intensive chemotherapy:

  • Cytarabine 200 mg/m2 IV continuous infusion days 1-7
  • Daunorubicin 90 mg/m2 intravenous piggyback days 1-3
Andere namen:
  • Ara-C
  • Antracycline
  • 7+3
  • Daunorubicine
  • 3+7

Consolidation chemotherapy for patients eligible for intensive chemotherapy with low-risk AML or patients with intermediate-/high-risk AML who do not have matched donors:

-Cytarabine 1.5 g/m2 IV in 3 hours days 1, 3 and 5 for 3 cycles

Andere namen:
  • Ara-C
  • Cytarabine

Autologous Stem Cell Transplantation for consolidation of patients eligible for intensive chemotherapy with low-risk AML or patients with intermediate-/high-risk AML who do not have matched donors:

  • Busulfan 1 mg/Kg PO q6h or 130 mg/m2 IV once daily days -7 to -4
  • Cyclophosphamide 60 mg/Kg IV once daily days -3 and -2
Andere namen:
  • ASCT
  • ABMT
  • Autologous Bone Marrow Transplantation

Allogeneic Stem Cell Transplantation for consolidation of patients eligible for intensive chemotherapy with intermediate-/high-risk AML

Conditioning regimen:

  • Busulfan 1 mg/Kg PO q6h or 130 mg/m2 IV once daily days -7 to -4
  • Cyclophosphamide 60 mg/Kg IV once daily days -3 and -2 or Fludarabine 40 mg/m2 IV once daily days -7 to -4
Andere namen:
  • AlloSCT
  • Allogene beenmergtransplantatie
Ander: AML-Non-intensive chemotherapy

Patients with acute myeloid leukemia not fit for intensive chemotherapy Patients will receive induction chemotherapy with either low dose cytarabine or decitabine. Assignment to each drug will depend on drug availability and physician discretion. No randomization will be done between the drugs.

Cycles will be repeated every 28 days. Patients who achieve CR will continue to post-consolidation therapy with either cytarabine or decitabine, based on the induction therapy received. Patients will receive a maximum of 4 cycles until achieving CR, if no response is seen after 4 cycles patients will be deemed refractory.

Chemotherapy for patients with AML who are not fit for intensive chemotherapy:

  • Cytarabine 60 mg/m2 subcutaneous (SQ) bid days 1-5 (until CR or maximum 4 cycles)
  • Cytarabine 40 mg/m2 SQ bid days 1-5 (after CR, until a maximum of 3 years of therapy or relapse, whichever comes first)
Andere namen:
  • Low dose ara-C
  • LDAC

Chemotherapy for patients with AML who are not fit for intensive chemotherapy:

  • Decitabine 20 mg/m2 IV once daily days 1-10 (until CR or maximum 4 cycles)
  • Decitabine 20 mg/m2 IV once daily days 1-5 (after CR, until a maximum of 3 years of therapy or relapse, whichever comes first)
Andere namen:
  • Dacogen
  • DAC
  • 2-aza-5´-deoxycytidine
Geen tussenkomst: Chronic Myeloid Disorders

Patients with Chronic Myeloid Disorders:

  • Myeloproliferative Neoplasms
  • Myelodysplastic Syndromes
  • Myeloproliferative/Myelodysplastic Neoplasms

Wat meet het onderzoek?

Primaire uitkomstmaten

Uitkomstmaat
Maatregel Beschrijving
Tijdsspanne
Prevalence of molecular and cytogenetic abnormalities
Tijdsspanne: 2 years
As assessed by results of molecular and cytogenetic tests and frequency in the population studied
2 years

Secundaire uitkomstmaten

Uitkomstmaat
Maatregel Beschrijving
Tijdsspanne
Overall survival
Tijdsspanne: 5 years
Evaluation of 5-years overall survival in patients with Acute Myeloid Leukemia, Myeloproliferative Neoplasms, Myelodysplastic Syndromes and Myeloproliferative/Myelodysplastic Neoplasms
5 years
Response rate
Tijdsspanne: 1 month
Evaluate complete remission (CR) rate at 1 month for patients with Acute Myeloid Leukemia who received induction chemotherapy. Complete remission was defined by the presence of < 5% blasts in the bone marrow (BM) with > 1 x 10^9/L neutrophils and >100x10^9/L platelets in the peripheral blood (PB)
1 month
Disease Free Survival
Tijdsspanne: 5 years
Evaluate rate of 5-years disease-free survival in patients with Acute Myeloid Leukemia who enter complete remission after induction chemotherapy
5 years
Cumulative incidence of relapse and non-relapse mortality
Tijdsspanne: 5 years
Evaluate 5-years cumulative incidence of relapse and non-relapse mortality in patients with Acute Myeloid Leukemia who achieve complete remission following induction chemotherapy
5 years
Number of participants with adverse events as a measure of safety and tolerability
Tijdsspanne: 1 year
Evaluate hematological and non-hematological toxicity in patients with Acute Myeloid Leukemia treated according to the protocol. Toxicity will be graded as per the National Cancer Institute Common Toxicity Criteria for Adverse Events v4.0.3
1 year
Cumulative Incidence of Transformation to Acute Myeloid Leukemia
Tijdsspanne: 5 years
Evaluate 5-years incidence of transformation to Acute Myeloid Leukemia in patients with Myeloproliferative Neoplasms, Myelodysplastic Syndromes and Myeloproliferative/Myelodysplastic Neoplasms
5 years

Medewerkers en onderzoekers

Hier vindt u mensen en organisaties die betrokken zijn bij dit onderzoek.

Onderzoekers

  • Studie directeur: Fabio P Santos, MD, Hospital israelita Albert Einstein
  • Studie stoel: Nelson Hamerschlak, MD, PhD, Hospital israelita Albert Einstein

Studie record data

Deze datums volgen de voortgang van het onderzoeksdossier en de samenvatting van de ingediende resultaten bij ClinicalTrials.gov. Studieverslagen en gerapporteerde resultaten worden beoordeeld door de National Library of Medicine (NLM) om er zeker van te zijn dat ze voldoen aan specifieke kwaliteitscontrolenormen voordat ze op de openbare website worden geplaatst.

Bestudeer belangrijke data

Studie start

1 oktober 2012

Primaire voltooiing (Werkelijk)

1 december 2014

Studie voltooiing (Werkelijk)

1 november 2016

Studieregistratiedata

Eerst ingediend

2 januari 2013

Eerst ingediend dat voldeed aan de QC-criteria

10 maart 2014

Eerst geplaatst (Schatting)

12 maart 2014

Updates van studierecords

Laatste update geplaatst (Schatting)

8 november 2016

Laatste update ingediend die voldeed aan QC-criteria

7 november 2016

Laatst geverifieerd

1 november 2016

Meer informatie

Deze informatie is zonder wijzigingen rechtstreeks van de website clinicaltrials.gov gehaald. Als u verzoeken heeft om uw onderzoeksgegevens te wijzigen, te verwijderen of bij te werken, neem dan contact op met register@clinicaltrials.gov. Zodra er een wijziging wordt doorgevoerd op clinicaltrials.gov, wordt deze ook automatisch bijgewerkt op onze website .

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