LeukemiaPhase 1NCT02141477
Omacetaxine and Decitabine in Acute Myelogenous Leukemia (AML) and Myelodysplastic Syndrome (MDS)
A Phase 1 study testing Omacetaxine and Decitabine in people with Leukemia, run by M.D. Anderson Cancer Center at 1 site in United States.
Trial summary
This clinical research study is made up of 2 phases. The goal of Phase 1 of the study is to test the safety of the combination of omacetaxine and decitabine and to find the best dose to give to future patients. The goal of Phase 2 of the study is to learn if this dose can help to control AML and/or MDS. Read more about this study
- Who is it for?
- Ages 70 years and older
- Conditions the study is for
- Leukemia
- What is being tested?
- Omacetaxine and Decitabine
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 2 participants
- Where
- 1 siteUnited States
- Study dates
- 6 May 2015to 22 Jun 2017
- Phase
- Phase 1
- 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 70 years and older
- All sexes
- No healthy volunteers
- Older adult
Who may be able to join
- Previously untreated AML (>/= 20% blasts) or AML M6. Patients with high-risk (intermediate-2 or high by IPSS or >/= 10% blasts) MDS will also be eligible. Prior therapy with hydroxyurea, biological or targeted therapy (e.g. flt3 inhibitors, other kinase inhibitors, azacitidine), or hematopoietic growth factors is allowed. No prior chemotherapy is allowed except for a single or a two day dose of cytarabine (up to 3 g/m2) for emergency use is also allowed as prior therapy.
- Age >/= 70 years.
- Eastern Cooperative Oncology Group (ECOG) performance status </= 2.
- Adequate hepatic (serum total bilirubin </= 1.5 x ULN, serum glutamate pyruvate transaminase (SGPT) and/or SGOT </= 2.5 x ULN) and renal function (creatinine </= 2.0 mg/dL).
- Patients must be willing and able to review, understand, and provide written consent before starting therapy.
- Men of childbearing potential who agree to use contraception prior to study entry and for the duration of participation.
Who cannot join
- New York Heart Association (NYHA) class III or IV heart disease, active ischemia or any other uncontrolled cardiac condition such as angina pectoris, clinically significant cardiac arrhythmia and requiring therapy, uncontrolled hypertension (blood pressure >/= 160 systolic and >/= 110 diastolic not responsive to antihypertensive medication), uncontrolled diabetes mellitus, or congestive heart failure.
- Myocardial infarction in the previous 12 weeks (from the start of treatment).
- Active and uncontrolled disease/infection as judged by the treating physician.
- Acute promyelocytic leukemia (APL).
Trial locations and contacts
1 site in 1 country.
United States1 site
University of Texas MD Anderson Cancer Center
Houston, Texas, 77030
About this study
Official title: A Phase II Study of Omacetaxine (OM) and Decitabine (DAC) in Older Patients With Acute Myelogenous Leukemia (AML) and High-Risk Myelodysplastic Syndrome (MDS)
This clinical research study is made up of 2 phases. The goal of Phase 1 of the study is to test the safety of the combination of omacetaxine and decitabine and to find the best dose to give to future patients. The goal of Phase 2 of the study is to learn if this dose can help to control AML and/or MDS. The safety will then continue to be studied.
Read the detailed description
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. Up to 2 groups of up to 6 participants (combined) will be enrolled in the Phase 1 portion of the study, and up to 60 participants will be enrolled in Phase 2.
If you are enrolled in Phase 1, the dose of decitabine you receive will depend on when you joined this study. If the first group of participants to receive decitabine has intolerable side effects, a second group will receive a lower dose.
If you are enrolled in Phase 2, you will receive decitabine at the highest dose that was tolerated in Phase 1.
All participants will receive the same dose level of omacetaxine.
Study Drug Administration:
Each cycle is 28 days.
Omacetaxine will be given as an injection under your skin 2 times each day, 12 hours apart (+/- 3 hours) on Days 1-3 of every cycle.
Decitabine will be given by vein over about 1 hour on Days 1-5 of every cycle.
Study Visits:
Every week (+/- 2 days), blood (about 2-3 teaspoons) will be collected for routine tests. If the disease appears to get better, this blood will only be drawn every 2-4 weeks while you are still receiving the study drugs, and every 4 to 8 weeks after that as long as you are on study. If you live far from the clinic, this blood and urine can be collected at a clinic close to your home, and the results will be reported to the study doctor.
At the beginning of every cycle, you will have a physical exam.
At Week 3 (+/- 7 days) and then every 4 weeks after that (+/- 7 days), you may (based on the results of your blood tests) have a bone marrow aspirate/biopsy collected to check the status of the disease and for cytogenetic testing.
Length of Study:
You may continue taking the study drugs for up to 3 years or as long as the doctor thinks it is in your best interest. You will no longer be able to take the study drugs if the disease gets worse, if intolerable side effects occur, or if you are unable to follow study directions.
Your participation on the study will be over after the follow-up visits.
Follow Up:
You will have follow-up visits every 3-6 months for up to 5 years after you stop receiving the study drugs. At these visits, you will have a physical exam. If you cannot come to the clinic, you may just be called by the study staff and asked about your health. These calls should last about 5-10 minutes.
This is an investigational study. Omacetaxine is FDA approved and commercially available for the treatment of chronic myelogenous leukemia (CML). Its use in this study is investigational. Decitabine is FDA approved and commercially available for the treatment of MDS.
The study doctor can explain how the study drugs are designed to work.
Up to 66 participants will be enrolled in this study. All will take part at MD Anderson.
- Conditions
- Leukemia
- Being tested
- Omacetaxine, Decitabine
- Keywords
- Leukemia, Acute Myelogenous Leukemia, AML, High Risk Myelodysplastic Syndrome, MDS, Omacetaxine, Synribo, Homoharringtonine, Decitabine, Dacogen
- Medical subject headings
- Leukemia, Leukemia, Myeloid, Acute
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
OmacetaxineDrug
Also called Synribo, Homoharringtonine
Phase I and Phase II Omacetaxine Dose: 1.25 mg/m2 subcutaneously every 12 hours on Days 1 - 3 of a 28 day cycle.
DecitabineDrug
Also called Dacogen
Phase I Starting Decitabine Dose: 20 mg/m2 by vein on Days 1 - 5 of a 28 day cycle.
Phase II Starting Decitabine Dose: Maximum tolerated dose from Phase I.
| Study group | Role | What they receive |
|---|---|---|
| Omacetaxine + Decitabine | Experimental | Phase I and Phase II Omacetaxine Dose: 1.25 mg/m2 subcutaneously every 12 hours on Days 1 - 3 of a 28 day cycle.
Phase I Starting Decitabine Dose: 20 mg/m2 by vein on Days 1 - 5 of a 28 day cycle.
Phase II Starting Decitabine Dose: Maximum tolerated dose from Phase I. Receives: Drug: Omacetaxine; Drug: Decitabine |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safe Dose Combination of Omacetaxine (OM) and Decitabine (DAC)
Safe dose defined as highest dose level with </= 1 out of 6 patients experience a dose limiting toxicity (DLT) during first treatment cycle. DLT defined as clinically significant Grade 3 or 4 adverse event or abnormal laboratory value according to Common Toxicity Criteria for Adverse Effects (CTCAE) criteria assessed by treating physician as related to study drug (and unrelated to disease progression, intercurrent illness, or concomitant medications) occurring during the first 28 days on study.
Measured: 28 days
Secondary outcomes (1)
Complete Response Rate (CRR)
Complete response rate (CRR) is defined as CR or CR with incomplete platelet recovery (CRp).
Measured: 8 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 May 2014 (estimated)
- Study start6 May 2015
- Primary completion22 Jun 2017
- Study completion22 Jun 2017
Why it stopped: Slow Accrual-2 patients were registered Phase I and none in Phase II
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for LeukemiaStudies 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
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 2 participants (actual)
Sponsor and investigators
- Lead sponsor
- M.D. Anderson Cancer Center
- Sponsor type
- University, hospital or other
- Collaborators
- Teva Pharmaceuticals USA
- Responsible party
- Sponsor
- Investigators
- Elias Jabbour, MD, Principal investigator, M.D. Anderson Cancer Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02141477
- Sponsor's protocol ID
- 2013-0812
- Other IDs
- NCI-2014-02157
- Status last verified
- Feb 2018
- First submitted
- 15 May 2014
- Last updated
- 13 Feb 2018
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 13 Feb 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase II Study of Omacetaxine (OM) and Decitabine (DAC) in Older Patients With Acute Myelogenous Leukemia (AML) and High-Risk Myelodysplastic Syndrome (MDS)
- ClinicalTrials.gov ID
- NCT02141477
- Acronym / sponsor ID
- 2013-0812
- Conditions
- Leukemia
- Keywords
- Leukemia, Acute Myelogenous Leukemia, AML, High Risk Myelodysplastic Syndrome, MDS, Omacetaxine, Synribo, Homoharringtonine
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Slow Accrual-2 patients were registered Phase I and none in Phase II
- Enrolment
- 2 participants (actual)
- Lead sponsor
- M.D. Anderson Cancer CenterUniversity, hospital or other
- Collaborators
- Teva Pharmaceuticals USA
- Study start
- 6 May 2015 (actual)
- Primary completion
- 22 Jun 2017 (actual)
- Study completion
- 22 Jun 2017 (actual)
- First posted
- 19 May 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.
This clinical research study is made up of 2 phases. The goal of Phase 1 of the study is to test the safety of the combination of omacetaxine and decitabine and to find the best dose to give to future patients. The goal of Phase 2 of the study is to learn if this dose can help to control AML and/or MDS. The safety will then continue to be studied.
Detailed description
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. Up to 2 groups of up to 6 participants (combined) will be enrolled in the Phase 1 portion of the study, and up to 60 participants will be enrolled in Phase 2.
If you are enrolled in Phase 1, the dose of decitabine you receive will depend on when you joined this study. If the first group of participants to receive decitabine has intolerable side effects, a second group will receive a lower dose.
If you are enrolled in Phase 2, you will receive decitabine at the highest dose that was tolerated in Phase 1.
All participants will receive the same dose level of omacetaxine.
Study Drug Administration:
Each cycle is 28 days.
Omacetaxine will be given as an injection under your skin 2 times each day, 12 hours apart (+/- 3 hours) on Days 1-3 of every cycle.
Decitabine will be given by vein over about 1 hour on Days 1-5 of every cycle.
Study Visits:
Every week (+/- 2 days), blood (about 2-3 teaspoons) will be collected for routine tests. If the disease appears to get better, this blood will only be drawn every 2-4 weeks while you are still receiving the study drugs, and every 4 to 8 weeks after that as long as you are on study. If you live far from the clinic, this blood and urine can be collected at a clinic close to your home, and the results will be reported to the study doctor.
At the beginning of every cycle, you will have a physical exam.
At Week 3 (+/- 7 days) and then every 4 weeks after that (+/- 7 days), you may (based on the results of your blood tests) have a bone marrow aspirate/biopsy collected to check the status of the disease and for cytogenetic testing.
Length of Study:
You may continue taking the study drugs for up to 3 years or as long as the doctor thinks it is in your best interest. You will no longer be able to take the study drugs if the disease gets worse, if intolerable side effects occur, or if you are unable to follow study directions.
Your participation on the study will be over after the follow-up visits.
Follow Up:
You will have follow-up visits every 3-6 months for up to 5 years after you stop receiving the study drugs. At these visits, you will have a physical exam. If you cannot come to the clinic, you may just be called by the study staff and asked about your health. These calls should last about 5-10 minutes.
This is an investigational study. Omacetaxine is FDA approved and commercially available for the treatment of chronic myelogenous leukemia (CML). Its use in this study is investigational. Decitabine is FDA approved and commercially available for the treatment of MDS.
The study doctor can explain how the study drugs are designed to work.
Up to 66 participants will be enrolled in this study. All will take part at MD Anderson.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Omacetaxine + Decitabine | Experimental | Drug: Omacetaxine, Drug: DecitabinePhase I and Phase II Omacetaxine Dose: 1.25 mg/m2 subcutaneously every 12 hours on Days 1 - 3 of a 28 day cycle. Phase I Starting Decitabine Dose: 20 mg/m2 by vein on Days 1 - 5 of a 28 day cycle. Phase II Starting Decitabine Dose: Maximum tolerated dose from Phase I. |
Who may qualify
- Ages
- 70 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Older adult
Key inclusion criteria (6 listed)
- Previously untreated AML (>/= 20% blasts) or AML M6. Patients with high-risk (intermediate-2 or high by IPSS or >/= 10% blasts) MDS will also be eligible. Prior therapy with hydroxyurea, biological or targeted therapy (e.g. flt3 inhibitors, other kinase inhibitors, azacitidine), or hematopoietic growth factors is allowed. No prior chemotherapy is allowed except for a single or a two day dose of cytarabine (up to 3 g/m2) for emergency use is also allowed as prior therapy.
- Age >/= 70 years.
- Eastern Cooperative Oncology Group (ECOG) performance status </= 2.
- Adequate hepatic (serum total bilirubin </= 1.5 x ULN, serum glutamate pyruvate transaminase (SGPT) and/or SGOT </= 2.5 x ULN) and renal function (creatinine </= 2.0 mg/dL).
- Patients must be willing and able to review, understand, and provide written consent before starting therapy.
Key exclusion criteria (4 listed)
- New York Heart Association (NYHA) class III or IV heart disease, active ischemia or any other uncontrolled cardiac condition such as angina pectoris, clinically significant cardiac arrhythmia and requiring therapy, uncontrolled hypertension (blood pressure >/= 160 systolic and >/= 110 diastolic not responsive to antihypertensive medication), uncontrolled diabetes mellitus, or congestive heart failure.
- Myocardial infarction in the previous 12 weeks (from the start of treatment).
- Active and uncontrolled disease/infection as judged by the treating physician.
- Acute promyelocytic leukemia (APL).
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 |
|---|---|---|
| OmacetaxineAlso: Synribo, HomoharringtoninePhase I and Phase II Omacetaxine Dose: 1.25 mg/m2 subcutaneously every 12 hours on Days 1 - 3 of a 28 day cycle. | Drug | Omacetaxine + Decitabine |
| DecitabineAlso: DacogenPhase I Starting Decitabine Dose: 20 mg/m2 by vein on Days 1 - 5 of a 28 day cycle. Phase II Starting Decitabine Dose: Maximum tolerated dose from Phase I. | Drug | Omacetaxine + Decitabine |
Primary outcome
Safe Dose Combination of Omacetaxine (OM) and Decitabine (DAC)
Time frame: 28 days
Safe dose defined as highest dose level with </= 1 out of 6 patients experience a dose limiting toxicity (DLT) during first treatment cycle. DLT defined as clinically significant Grade 3 or 4 adverse event or abnormal laboratory value according to Common Toxicity Criteria for Adverse Effects (CTCAE) criteria assessed by treating physician as related to study drug (and unrelated to disease progression, intercurrent illness, or concomitant medications) occurring during the first 28 days on study.
1 secondary outcome
Complete Response Rate (CRR)
Time frame: 8 weeks
Results and safety
The study was stopped early (reason given: Slow Accrual-2 patients were registered Phase I and none in Phase II). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 13 Feb 2018.
- University of Texas MD Anderson Cancer Center 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.
Elias Jabbour, 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 study testing Omacetaxine and Decitabine in people with 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 6 inclusion and 4 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: stopped early, as of the sponsor's last update on 13 Feb 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 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.
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 was stopped early (reason given: Slow Accrual-2 patients were registered Phase I and none in Phase II). Results are not available in the source record.
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?
Safe Dose Combination of Omacetaxine (OM) and Decitabine (DAC) (time frame: 28 days). 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 13 Feb 2018.