AMLPhase 1/2NCT00542971
Phase I-II Study of Idarubicin, Cytarabine, and Sorafenib (BAY43-9006)
A Phase 1/2 study testing Idarubicin, Sorafenib and Ara-C in people with AML and Acute Myeloid Leukemia, run by M.D. Anderson Cancer Center at 1 site in United States.
Trial summary
A primary goal of this clinical research study is to find the highest safe dose of sorafenib that can be given in combination with idarubicin and Ara-C for the treatment of acute myelogenous leukemia (AML) and high-risk, myelodysplastic syndrome (MDS). Read more about this study
- Who is it for?
- Ages 15 to 60 years
- Conditions the study is for
- AML, Acute Myeloid Leukemia, Myelodysplastic Disorders
- What is being tested?
- Idarubicin, Sorafenib and Ara-C
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 78 participants
- Where
- 1 siteUnited States
- Study dates
- Oct 2007to May 2011
- Phase
- Phase 1/2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 15 to 60 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Diagnosis of 1) AML (World Health Organization classification definition of > 20% blasts), or 2) high risk MDS (defined as the presence of > 10% blasts).
- Patients aged 15 to 60 years are eligible. Patients older than 60 who are deemed fit to receive intensive chemotherapy by the treating physician are eligible after discussion with the Principal Investigator (PI). For the Phase II portion of the study, patients must be chemo-naïve, i.e. not have received any prior chemotherapy (except hydrea) for AML or MDS. They could have received hypomethylator agents, transfusions, hematopoietic growth factors or vitamins. Temporary prior measures such as pheresis or hydrea are allowed. In the Phase I portion, patients with relapsed or refractory AML/MDS are also eligible.
- Serum biochemical values with the following limits unless considered due to leukemia: 1) creatinine less than or equal to 2 mg/dl, 2) total bilirubin less than or equal to 2 mg/dL, unless increase is due to hemolysis or congenital disorder, and 3) transaminases (SG PT) less than or equal to 2.5 times upper limit of normal (ULN)
- Ability to take oral medication.
- Ability to understand and provide signed informed consent.
- Baseline test of ejection fraction must be >/=50%.
- Performance status < 3, unless directly related to the disease process as determined by the principal investigator.
Who cannot join
- Patients with Acute promyelocytic leukemia (APL).
- Any coexisting medical condition that in the judgment of the treating physician is likely to interfere with study procedures or results.
- Nursing women, women of childbearing potential with positive urine pregnancy test, or women of childbearing potential who are not willing to maintain adequate contraception (such as birth control pills, Intrauterine Device (IUD), diaphragm, abstinence, or condoms by their partner) over the entire course of the study.
- Any significant, uncontrolled hypertension.
- Cardiac disease: Congestive heart failure > class II The New York Heart Association (NYHA). Patients must not have unstable angina (anginal symptoms at rest) or new onset angina (began within the last 3 months) or myocardial infarction within the past 6 months.
- Cardiac ventricular arrhythmias requiring anti-arrhythmic therapy. Sorafenib is contraindicated in patients with known severe hypersensitivity to sorafenib or any of the excipients.
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- Known human immunodeficiency virus (HIV) infection or active Hepatitis B or C.
- Thrombotic or embolic events such as a cerebrovascular accident including transient ischemic attacks within the past 6 months.
- Pulmonary hemorrhage/bleeding event > or = to Common Terminology Criteria for Adverse Events (CTCAE) Grade 2 within 4 weeks of first dose of study drug.
- Any other hemorrhage/bleeding event > or = to CTCAE Grade 3 within 4 weeks of first dose of study drug.
- Major surgery, open biopsy or significant traumatic injury within 4 weeks of first study drug.
- Use of St. John's Wort or rifampin.
- Known or suspected allergy to sorafenib or any agent given in the course of this trial.
- Active clinically serious and uncontrolled infection > CTCAE Grade 2
- Serious non-healing wound, ulcer, or bone fracture
Trial locations and contacts
1 site in 1 country.
United States1 site
The University of Texas M.D. Anderson Cancer Center
Houston, Texas, 77030
About this study
Official title: Phase I-II Study of Idarubicin, Cytarabine, and Sorafenib (BAY43-9006), an Oral Vascular Endothelial Growth Factor (VEGF), Rapidly Accelerated Fibrosarcoma (RAF) and FMS-like Tyrosine Kinase 3 (FLT3), in Patients With High-risk MDS and AML
A primary goal of this clinical research study is to find the highest safe dose of sorafenib that can be given in combination with idarubicin and Ara-C for the treatment of acute myelogenous leukemia (AML) and high-risk, myelodysplastic syndrome (MDS).
Once the highest safe dose is found, researchers will then try to learn if this combination treatment can help control AML and high-risk MDS in newly diagnosed patients. The safety of this treatment combination will also be studied.
Read the detailed description
The Study Drugs:
Sorafenib is designed to block the function of important proteins in cancer cells. When active, these proteins help cause abnormal growth and behavior of leukemia cells.
Idarubicin and Ara-C are both designed to insert themselves into DNA (the genetic material of cells) and to stop DNA from repairing itself.
Study Groups:
If you are found to be eligible to take part in this study, you will be assigned to a study group based on when you join this study.
If you are enrolled in the Phase I part of this study, the dose of sorafenib you receive with Ara-C and idarubicin will depend on when you enrolled in this study. Each new group of 3 participants will receive a higher dose level of sorafenib than the previous group until the highest safe dose of sorafenib that can be given in combination with Ara-C and idarubicin is reached.
If you are enrolled in the Phase II portion of this study, you will receive the highest safe dose of sorafenib in combination with Ara-C and idarubicin found in the Phase I part.
Study Drug Administration:
During the study, you will receive treatment over 28-day "cycles." The doses of idarubicin and Ara-C are the same for all study participants.
On Days 1-4 (Days 1-3 if you are older than 60) of each induction cycle (#1 and possibly #2), you will receive Ara-C through a nonstop infusion (through a needle in your vein). You will also receive steroids by mouth or through a vein every day to reduce the risks of side effects, such as rash.
On Days 1-3 of each induction cycle (#1 and possibly #2), you will receive idarubicin by vein over 1 hour once a day.
In general, sorafenib will be taken by mouth twice daily on Days 1-7, unless otherwise instructed. However, the dose of sorafenib may differ among study participants. Sorafenib should be taken with water on an empty stomach. Your study doctor will give you complete instructions on when and how to take sorafenib.
Depending on the side effects that you may have, the dose of sorafenib may be decreased, stopped until side effects go away, or even stopped completely, if your doctor thinks that this is in your best interest.
Study Visits:
During Cycles 1-2, you will have study visits about 2 times a week. You may need to have study visits more often when the study doctor thinks it is necessary. At these visits, you will have the following procedures performed.
- You will be asked about any side effects you may have experienced and any medications you may be taking.
- Blood (about 2 teaspoons) will be drawn for routine tests.
During Cycles 1 and 2, between Days 21-28, you will have a bone marrow aspirate and/or biopsy performed to check the status of the disease.
For Cycle 3 and in further cycles, you will have a bone marrow aspirate as determined necessary by your doctor. If your doctor thinks you have responded to the treatment, you will then have bone marrow aspirates collected every 3-6 months, if your doctor feels this to be necessary.
Consolidation Therapy:
If it is found that the disease is responding to treatment, you may receive 5 additional cycles of therapy. These cycles are called "consolidation" therapy and will be given every 4-6 weeks.
On Days 1-3, Ara-C will be given as a nonstop infusion. You will also receive steroids either by mouth or by vein to help reduce the risk of side effects.
On Days 1-2, idarubicin will be given by vein over 1 hour.
Sorafenib will be taken twice daily by mouth for 7 or more days, at a schedule determined by your study doctor.
After each cycle, about every 4-6 weeks, blood (about 2 teaspoons) will be drawn to check your blood count levels.
Maintenance Therapy:
After 5 cycles of consolidation therapy, and according to your response to consolidation therapy, you may continue on this study to receive "maintenance" treatment, as needed, and as determined by your study doctor for up to 7 more cycles.
You will take sorafenib twice a day every day during the 28-day cycle.
Every 1-4 weeks, blood (about 2 teaspoons) will be drawn for routine tests.
Length of Study:
You may receive up to 14 cycles in total of therapy. You may be taken off this study if the leukemia gets worse, develop another illness that interferes with taking the study drugs, or you have intolerable side effects.
This is an investigational study. Sorafenib is FDA approved and commercially available for the treatment of metastatic renal cancer only. Idarubicin is FDA approved for use in combination with other approved antileukemic drugs for the treatment of acute myeloid leukemia (AML) in adults. Cytarabine is FDA approved for use in the treatment of leukemia. The use of sorafenib combined with idarubicin and Ara-C in the treatment of AML and high-risk MDS is investigational and authorized for use in research only. Up to 75 patients will take part in this study. All patients will be enrolled at M. D. Anderson Cancer Center.
- Conditions
- AML, Acute Myeloid Leukemia, Myelodysplastic Disorders
- Being tested
- Idarubicin, Sorafenib, Ara-C
- Keywords
- Leukemia, AML, High-Risk MDS, Acute Myeloid Leukemia, High-Risk Myelodysplastic Disorder, Idarubicin, Ara-C, Cytarabine, Sorafenib, BAY43-9006
- Medical subject headings
- Leukemia, Myeloid, Acute, Leukemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
IdarubicinDrug
Also called Idamycin®
12 mg/m^2 IV over 1 hour daily (days 1-3)
SorafenibDrug
Also called Nexavar®
Starting dose 400 mg by mouth for 7 days
Ara-CDrug
Also called Cytarabine, Cytosar-U®, Arabinosylcytosine
1.5 g/m^2 IV over 24 hours daily (days 1-4)
| Study group | Role | What they receive |
|---|---|---|
| Sorafenib + Idarubicin + Ara-C | Experimental | Sorafenib starting dose 400 mg orally for 7 days; Idarubicin 12 mg/m^2 intravenous (IV) daily (days 1-3); and Ara-C 1.5 g/m^2 IV over 24 hours daily (days 1-4) Receives: Drug: Idarubicin; Drug: Sorafenib; Drug: Ara-C |
What is being measured
The primary outcome is the main question the study is designed to answer.
Maximum Tolerated Dose (MTD)
MTD is dose level where grade 3-4 sorafenib-attributable toxicity in <2 of 6 participants. Dose-Limiting Toxicity graded according to the NCI Common Toxicity Criteria version 3.0.
Measured: Twice a week for first two 28 day cycles
Secondary outcomes (1)
Number of Participants With Complete Response
Complete response was defined by the presence of < 5% blasts in the bone marrow (BM) with > 1 x 10^9/L platelets in the peripheral blood (PB).
Measured: Baseline to 2 years or disease progression.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Oct 2007 (estimated)
- Study startOct 2007
- Primary completionMay 2011
- Study completionMay 2011
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for AMLStudies of the same condition, open and under way.
- More trials run by M.D. Anderson Cancer CenterThe sponsor's other registered studies.
- 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 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 78 participants (actual)
Sponsor and investigators
- Lead sponsor
- M.D. Anderson Cancer Center
- Sponsor type
- University, hospital or other
- Collaborators
- Bayer
- Responsible party
- Sponsor
- Investigators
- Farhad Ravandi-Kashani, MD, Principal investigator, M.D. Anderson Cancer Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00542971
- Sponsor's protocol ID
- 2006-0977
- Status last verified
- Jul 2018
- First submitted
- 10 Oct 2007
- Last updated
- 23 Aug 2018
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 23 Aug 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase I-II Study of Idarubicin, Cytarabine, and Sorafenib (BAY43-9006), an Oral Vascular Endothelial Growth Factor (VEGF), Rapidly Accelerated Fibrosarcoma (RAF) and FMS-like Tyrosine Kinase 3 (FLT3), in Patients With High-risk MDS and AML
- ClinicalTrials.gov ID
- NCT00542971
- Acronym / sponsor ID
- 2006-0977
- Conditions
- AML, Acute Myeloid Leukemia, Myelodysplastic Disorders
- Keywords
- Leukemia, AML, High-Risk MDS, Acute Myeloid Leukemia, High-Risk Myelodysplastic Disorder, Idarubicin, Ara-C, Cytarabine
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 78 participants (actual)
- Lead sponsor
- M.D. Anderson Cancer CenterUniversity, hospital or other
- Collaborators
- Bayer
- Study start
- Oct 2007
- Primary completion
- May 2011 (actual)
- Study completion
- May 2011 (actual)
- First posted
- 12 Oct 2007 (estimated)
- Results
- Posted 13 Jul 2012
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
A primary goal of this clinical research study is to find the highest safe dose of sorafenib that can be given in combination with idarubicin and Ara-C for the treatment of acute myelogenous leukemia (AML) and high-risk, myelodysplastic syndrome (MDS).
Once the highest safe dose is found, researchers will then try to learn if this combination treatment can help control AML and high-risk MDS in newly diagnosed patients. The safety of this treatment combination will also be studied.
Detailed description
The Study Drugs:
Sorafenib is designed to block the function of important proteins in cancer cells. When active, these proteins help cause abnormal growth and behavior of leukemia cells.
Idarubicin and Ara-C are both designed to insert themselves into DNA (the genetic material of cells) and to stop DNA from repairing itself.
Study Groups:
If you are found to be eligible to take part in this study, you will be assigned to a study group based on when you join this study.
If you are enrolled in the Phase I part of this study, the dose of sorafenib you receive with Ara-C and idarubicin will depend on when you enrolled in this study. Each new group of 3 participants will receive a higher dose level of sorafenib than the previous group until the highest safe dose of sorafenib that can be given in combination with Ara-C and idarubicin is reached.
If you are enrolled in the Phase II portion of this study, you will receive the highest safe dose of sorafenib in combination with Ara-C and idarubicin found in the Phase I part.
Study Drug Administration:
During the study, you will receive treatment over 28-day "cycles." The doses of idarubicin and Ara-C are the same for all study participants.
On Days 1-4 (Days 1-3 if you are older than 60) of each induction cycle (#1 and possibly #2), you will receive Ara-C through a nonstop infusion (through a needle in your vein). You will also receive steroids by mouth or through a vein every day to reduce the risks of side effects, such as rash.
On Days 1-3 of each induction cycle (#1 and possibly #2), you will receive idarubicin by vein over 1 hour once a day.
In general, sorafenib will be taken by mouth twice daily on Days 1-7, unless otherwise instructed. However, the dose of sorafenib may differ among study participants. Sorafenib should be taken with water on an empty stomach. Your study doctor will give you complete instructions on when and how to take sorafenib.
Depending on the side effects that you may have, the dose of sorafenib may be decreased, stopped until side effects go away, or even stopped completely, if your doctor thinks that this is in your best interest.
Study Visits:
During Cycles 1-2, you will have study visits about 2 times a week. You may need to have study visits more often when the study doctor thinks it is necessary. At these visits, you will have the following procedures performed.
- You will be asked about any side effects you may have experienced and any medications you may be taking.
- Blood (about 2 teaspoons) will be drawn for routine tests.
During Cycles 1 and 2, between Days 21-28, you will have a bone marrow aspirate and/or biopsy performed to check the status of the disease.
For Cycle 3 and in further cycles, you will have a bone marrow aspirate as determined necessary by your doctor. If your doctor thinks you have responded to the treatment, you will then have bone marrow aspirates collected every 3-6 months, if your doctor feels this to be necessary.
Consolidation Therapy:
If it is found that the disease is responding to treatment, you may receive 5 additional cycles of therapy. These cycles are called "consolidation" therapy and will be given every 4-6 weeks.
On Days 1-3, Ara-C will be given as a nonstop infusion. You will also receive steroids either by mouth or by vein to help reduce the risk of side effects.
On Days 1-2, idarubicin will be given by vein over 1 hour.
Sorafenib will be taken twice daily by mouth for 7 or more days, at a schedule determined by your study doctor.
After each cycle, about every 4-6 weeks, blood (about 2 teaspoons) will be drawn to check your blood count levels.
Maintenance Therapy:
After 5 cycles of consolidation therapy, and according to your response to consolidation therapy, you may continue on this study to receive "maintenance" treatment, as needed, and as determined by your study doctor for up to 7 more cycles.
You will take sorafenib twice a day every day during the 28-day cycle.
Every 1-4 weeks, blood (about 2 teaspoons) will be drawn for routine tests.
Length of Study:
You may receive up to 14 cycles in total of therapy. You may be taken off this study if the leukemia gets worse, develop another illness that interferes with taking the study drugs, or you have intolerable side effects.
This is an investigational study. Sorafenib is FDA approved and commercially available for the treatment of metastatic renal cancer only. Idarubicin is FDA approved for use in combination with other approved antileukemic drugs for the treatment of acute myeloid leukemia (AML) in adults. Cytarabine is FDA approved for use in the treatment of leukemia. The use of sorafenib combined with idarubicin and Ara-C in the treatment of AML and high-risk MDS is investigational and authorized for use in research only. Up to 75 patients will take part in this study. All patients will be enrolled at M. D. Anderson Cancer Center.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Sorafenib + Idarubicin + Ara-C | Experimental | Drug: Idarubicin, Drug: Sorafenib, Drug: Ara-CSorafenib starting dose 400 mg orally for 7 days; Idarubicin 12 mg/m^2 intravenous (IV) daily (days 1-3); and Ara-C 1.5 g/m^2 IV over 24 hours daily (days 1-4) |
Who may qualify
- Ages
- 15 to 60 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (7 listed)
- Diagnosis of 1) AML (World Health Organization classification definition of > 20% blasts), or 2) high risk MDS (defined as the presence of > 10% blasts).
- Patients aged 15 to 60 years are eligible. Patients older than 60 who are deemed fit to receive intensive chemotherapy by the treating physician are eligible after discussion with the Principal Investigator (PI). For the Phase II portion of the study, patients must be chemo-naïve, i.e. not have received any prior chemotherapy (except hydrea) for AML or MDS. They could have received hypomethylator agents, transfusions, hematopoietic growth factors or vitamins. Temporary prior measures such as pheresis or hydrea are allowed. In the Phase I portion, patients with relapsed or refractory AML/MDS are also eligible.
- Serum biochemical values with the following limits unless considered due to leukemia: 1) creatinine less than or equal to 2 mg/dl, 2) total bilirubin less than or equal to 2 mg/dL, unless increase is due to hemolysis or congenital disorder, and 3) transaminases (SG PT) less than or equal to 2.5 times upper limit of normal (ULN)
- Ability to take oral medication.
- Ability to understand and provide signed informed consent.
Key exclusion criteria (15 listed)
- Patients with Acute promyelocytic leukemia (APL).
- Any coexisting medical condition that in the judgment of the treating physician is likely to interfere with study procedures or results.
- Nursing women, women of childbearing potential with positive urine pregnancy test, or women of childbearing potential who are not willing to maintain adequate contraception (such as birth control pills, Intrauterine Device (IUD), diaphragm, abstinence, or condoms by their partner) over the entire course of the study.
- Any significant, uncontrolled hypertension.
- Cardiac disease: Congestive heart failure > class II The New York Heart Association (NYHA). Patients must not have unstable angina (anginal symptoms at rest) or new onset angina (began within the last 3 months) or myocardial infarction within the past 6 months.
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 |
|---|---|---|
| IdarubicinAlso: Idamycin®12 mg/m^2 IV over 1 hour daily (days 1-3) | Drug | Sorafenib + Idarubicin + Ara-C |
| SorafenibAlso: Nexavar®Starting dose 400 mg by mouth for 7 days | Drug | Sorafenib + Idarubicin + Ara-C |
| Ara-CAlso: Cytarabine, Cytosar-U®, Arabinosylcytosine1.5 g/m^2 IV over 24 hours daily (days 1-4) | Drug | Sorafenib + Idarubicin + Ara-C |
Primary outcome
Maximum Tolerated Dose (MTD)
Time frame: Twice a week for first two 28 day cycles
MTD is dose level where grade 3-4 sorafenib-attributable toxicity in <2 of 6 participants. Dose-Limiting Toxicity graded according to the NCI Common Toxicity Criteria version 3.0.
1 secondary outcome
Number of Participants With Complete Response
Time frame: Baseline to 2 years or disease progression.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Aug 2018.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- M.D. Anderson's websiteLink provided by the sponsor.
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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.
Farhad Ravandi-Kashani, MD principal investigator
M.D. Anderson Cancer Center
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Idarubicin, Sorafenib and Ara-C in people with AML and Acute Myeloid Leukemia, run by M.D. Anderson Cancer Center at 1 site in United States.
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 7 inclusion and 15 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 23 Aug 2018. 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 United States. 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 combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
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?
Yes. Results are posted in the registry (first posted 13 Jul 2012). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Maximum Tolerated Dose (MTD) (time frame: Twice a week for first two 28 day cycles). 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 23 Aug 2018.