Acute Myeloid LeukemiaPhase 2NCT02084563
Study of Molecular and Genetic Abnormalities in Patients With Myeloid Neoplasms
A Phase 2 study testing Induction Chemotherapy, Consolidation Chemotherapy and Autologous Stem Cell Transplantation in people with Acute Myeloid Leukemia and Myeloproliferative Neoplasms, run by Hospital Israelita Albert Einstein at 1 site in Brazil.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Acute Myeloid Leukemia, Myeloproliferative Neoplasms, Myelodysplastic Syndromes
- What is being tested?
- Induction Chemotherapy, Consolidation Chemotherapy and Autologous Stem Cell Transplantation
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 455 participants
- Where
- 1 siteBrazil
- Study dates
- Oct 2012to Nov 2016
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
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- Adequate contraception for fertile men and women
- Eligible for intensive chemotherapy (as judged by the treating physician)
- 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)
- Diagnosis of Myeloproliferative Neoplasm or Myelodysplastic Syndrome or Myeloproliferative/Myelodysplastic Neoplasm according to WHO criteria
- Age greater than 18 years
- Signed Informed Consent form
Who cannot join
- 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
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- Patient refuses to sign informed consent form
- Acute Myeloid Leukemia-Non-Intensive Chemotherapy
- 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:
- Patient refuses to sign informed consent form
Trial locations and contacts
1 site in 1 country.
About this study
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
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.
- Conditions
- Acute Myeloid Leukemia, Myeloproliferative Neoplasms, Myelodysplastic Syndromes, Myeloproliferative/Myelodysplastic Neoplasm
- Being tested
- Induction Chemotherapy, Consolidation Chemotherapy, Autologous Stem Cell Transplantation, Allogeneic Stem Cell Transplantation, Low Dose Cytarabine
- 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)
- Medical subject headings
- Leukemia, Myeloid, Acute, Myeloproliferative Disorders, Myelodysplastic Syndromes, Anemia, Refractory, with Excess of Blasts, Leukemia, Leukemia, Myelomonocytic, Chronic, Polycythemia Vera, Thrombocythemia, Essential
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Induction ChemotherapyDrug
Also called 7+3, Ara-C, Daunorubicin, Anthracycline
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
Consolidation ChemotherapyDrug
Also called Ara-C, Cytarabine
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
Autologous Stem Cell TransplantationDrug
Also called ASCT, Autologous Bone Marrow Transplantation, ABMT
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
Allogeneic Stem Cell TransplantationDrug
Also called AlloSCT, Allogeneic 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
Low Dose CytarabineDrug
Also called Low dose ara-C, LDAC
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)
DecitabineDrug
Also called 2-aza-5´-deoxycytidine, Dacogen, DAC
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)
| Study group | Role | What they receive |
|---|---|---|
| AML-Intensive Chemotherapy | Other | 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. Receives: Drug: Induction Chemotherapy; Drug: Consolidation Chemotherapy; Drug: Autologous Stem Cell Transplantation; Drug: Allogeneic Stem Cell Transplantation |
| AML-Non-intensive chemotherapy | Other | 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. Receives: Drug: Low Dose Cytarabine; Drug: Decitabine |
| Chronic Myeloid Disorders | No intervention | Patients with Chronic Myeloid Disorders: * Myeloproliferative Neoplasms * Myelodysplastic Syndromes * Myeloproliferative/Myelodysplastic Neoplasms |
What is being measured
The primary outcome is the main question the study is designed to answer.
Prevalence of molecular and cytogenetic abnormalities
As assessed by results of molecular and cytogenetic tests and frequency in the population studied
Measured: 2 years
Secondary outcomes (6)
Overall survival
Evaluation of 5-years overall survival in patients with Acute Myeloid Leukemia, Myeloproliferative Neoplasms, Myelodysplastic Syndromes and Myeloproliferative/Myelodysplastic Neoplasms
Measured: 5 years
Response rate
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)
Measured: 1 month
Disease Free Survival
Evaluate rate of 5-years disease-free survival in patients with Acute Myeloid Leukemia who enter complete remission after induction chemotherapy
Measured: 5 years
Cumulative incidence of relapse and non-relapse mortality
Evaluate 5-years cumulative incidence of relapse and non-relapse mortality in patients with Acute Myeloid Leukemia who achieve complete remission following induction chemotherapy
Measured: 5 years
Number of participants with adverse events as a measure of safety and tolerability
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
Measured: 1 year
Cumulative Incidence of Transformation to Acute Myeloid Leukemia
Evaluate 5-years incidence of transformation to Acute Myeloid Leukemia in patients with Myeloproliferative Neoplasms, Myelodysplastic Syndromes and Myeloproliferative/Myelodysplastic Neoplasms
Measured: 5 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Mar 2014 (estimated)
- Study startOct 2012
- Primary completionDec 2014
- Study completionNov 2016
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Acute Myeloid LeukemiaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Basic science
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 455 participants (actual)
Sponsor and investigators
- Lead sponsor
- Hospital Israelita Albert Einstein
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Fabio Pires de Souza Santos, Hospital Israelita Albert Einstein
- Investigators
- Fabio P Santos, MD, Study director, Hospital Israelita Albert EinsteinNelson Hamerschlak, MD, PhD, Study chair, Hospital Israelita Albert Einstein
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02084563
- Sponsor's protocol ID
- LMA Brasil
- Other IDs
- 11/1714
- Status last verified
- Nov 2016
- First submitted
- 2 Jan 2013
- Last updated
- 8 Nov 2016
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 8 Nov 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT02084563
- Acronym / sponsor ID
- LMA Brasil
- 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
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 455 participants (actual)
- Lead sponsor
- Hospital Israelita Albert EinsteinUniversity, hospital or other
- Study start
- Oct 2012
- Primary completion
- Dec 2014 (actual)
- Study completion
- Nov 2016 (actual)
- First posted
- 12 Mar 2014 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Basic science
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| AML-Intensive Chemotherapy | Other | Drug: Induction Chemotherapy, Drug: Consolidation Chemotherapy, Drug: Autologous Stem Cell Transplantation, Drug: Allogeneic Stem Cell TransplantationPatients 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. |
| AML-Non-intensive chemotherapy | Other | Drug: Low Dose Cytarabine, Drug: DecitabinePatients 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. |
| Chronic Myeloid Disorders | No intervention | –Patients with Chronic Myeloid Disorders: * Myeloproliferative Neoplasms * Myelodysplastic Syndromes * Myeloproliferative/Myelodysplastic Neoplasms |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (17 listed)
- 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
Key exclusion criteria (16 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Induction ChemotherapyAlso: 7+3, Ara-C, Daunorubicin, AnthracyclineInduction 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 | Drug | AML-Intensive Chemotherapy |
| Consolidation ChemotherapyAlso: Ara-C, CytarabineConsolidation 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 | Drug | AML-Intensive Chemotherapy |
| Autologous Stem Cell TransplantationAlso: ASCT, Autologous Bone Marrow Transplantation, ABMTAutologous 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 | Drug | AML-Intensive Chemotherapy |
| Allogeneic Stem Cell TransplantationAlso: AlloSCT, Allogeneic Bone Marrow TransplantationAllogeneic 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 | Drug | AML-Intensive Chemotherapy |
| Low Dose CytarabineAlso: Low dose ara-C, LDACChemotherapy 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) | Drug | AML-Non-intensive chemotherapy |
| DecitabineAlso: 2-aza-5´-deoxycytidine, Dacogen, DACChemotherapy 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) | Drug | AML-Non-intensive chemotherapy |
Primary outcome
Prevalence of molecular and cytogenetic abnormalities
Time frame: 2 years
As assessed by results of molecular and cytogenetic tests and frequency in the population studied
6 secondary outcomes
Overall survival
Time frame: 5 years
Response rate
Time frame: 1 month
Disease Free Survival
Time frame: 5 years
Cumulative incidence of relapse and non-relapse mortality
Time frame: 5 years
Number of participants with adverse events as a measure of safety and tolerability
Time frame: 1 year
Cumulative Incidence of Transformation to Acute Myeloid Leukemia
Time frame: 5 years
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 Nov 2016.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Fabio P Santos, MD study director
Hospital Israelita Albert Einstein
Nelson Hamerschlak, MD, PhD study chair
Hospital Israelita Albert Einstein
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Induction Chemotherapy, Consolidation Chemotherapy and Autologous Stem Cell Transplantation in people with Acute Myeloid Leukemia and Myeloproliferative Neoplasms, run by Hospital Israelita Albert Einstein at 1 site in Brazil.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 17 inclusion and 16 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 8 Nov 2016. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Brazil. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Prevalence of molecular and cytogenetic abnormalities (time frame: 2 years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 8 Nov 2016.