Clinical trial NCT02084563: Study of Molecular and Genetic Abnormalities in Patients With Myeloid Neoplasms
Official title: Evaluation of the Incidence and Prognostic Impact of Molecular and Genetic Abnormalities in Patients With Acute Myeloid Leukemia, Myelodysplastic Syndrome and Myeloproliferative Neoplasms
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Acute Myeloid Leukemia
Myeloproliferative Neoplasms
Myelodysplastic Syndromes
Myeloproliferative/Myelodysplastic Neoplasm
Keywords: Acute Myeloid Leukemia
Myeloproliferative Neoplasms
Myelodysplastic Syndromes
Myeloproliferative/Myelodysplastic Neoplasm
Leukemia
MDS
MPN
AML
Chronic myelomonocytic leukemia (CMML)
Polycythemia Vera (PV)
Essential Thrombocythemia (ET)
Myelofibrosis (MF)
Lead sponsor: Hospital Israelita Albert Einstein
Sponsor class: OTHER
First posted: 2014-03-12
Enrollment: 455
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: PARALLEL
Primary purpose: BASIC_SCIENCE

Brief summary: 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.


Eligibility criteria: 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

Primary outcomes:
measure: Prevalence of molecular and cytogenetic abnormalities
timeFrame: 2 years
Secondary outcomes:
measure: Overall survival
timeFrame: 5 years
measure: Response rate
timeFrame: 1 month
measure: Disease Free Survival
timeFrame: 5 years
measure: Cumulative incidence of relapse and non-relapse mortality
timeFrame: 5 years
measure: Number of participants with adverse events as a measure of safety and tolerability
timeFrame: 1 year
measure: Cumulative Incidence of Transformation to Acute Myeloid Leukemia
timeFrame: 5 years
Other outcomes:
Locations:
city: Sao Paulo
country: Brazil
facility: Hospital Israelita Albert Einstein
state: SP
Study officials:
affiliation: Hospital Israelita Albert Einstein
name: Fabio P Santos, MD
role: STUDY_DIRECTOR
affiliation: Hospital Israelita Albert Einstein
name: Nelson Hamerschlak, MD, PhD
role: STUDY_CHAIR
References: