LeukemiaPhase 2NCT02159872
Omacetaxine in Patients With Intermediate-1 and Higher Risk Myelodysplastic Syndrome (MDS) Post Hypomethylating Agent (HMA) Failure
A Phase 2 study testing Omacetaxine in people with Leukemia, run by M.D. Anderson Cancer Center at 1 site in United States.
Trial summary
The goal of this clinical research study is learn if omacetaxine can help to control myelodysplastic syndrome (MDS). The safety of this drug will also be studied. This is an investigational study. Omacetaxine is FDA approved and commercially available for the treatment of chronic myelogenous leukemia (CML). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Leukemia
- What is being tested?
- Omacetaxine
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 48 participants
- Where
- 1 siteUnited States
- Study dates
- 18 May 2015to 14 Apr 2020
- Phase
- Phase 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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Age >/= 18 years
- Diagnosis of MDS confirmed within 10 weeks prior to study entry according to WHO criteria. Patients are either not eligible for or choose not to proceed with a stem cell transplant.
- MDS classified as follows: RAEB-1 (5%-9% BM blasts); RAEB-2 (10%-19% BM Blasts); CMML (5%-19% BM blasts); RAEB-t (20%-29% BM blasts) AND/OR by IPSS: intermediate-1 and high risk patients.
- No response, progression, or relapse (according to 2006 IWG criteria) following at least 4 cycles of either azacitidine or decitabine, which were completed within the last 2 years - AND/OR - intolerance to azacitidine or decitabine defined as drug-related >/= grade 3 hepatic or renal toxicity leading to treatment discontinuation during the preceding 2 years.
- Eastern Cooperative Oncology Group (ECOG) performance status of </= 2.
- Willing to adhere to and comply with all prohibitions and restrictions specified in the protocol.
- Patient (or patient's legally authorized representative) must have signed an informed consent document indicating that the patient understands the purpose of and procedures required for the study and is willing to participate in the study.)
Who cannot join
- Uncontrolled intercurrent illness including, but not limited to, symptomatic congestive heart failure, unstable angina pectoris, or cardiac arrhythmia.
- Active infection not adequately responding to appropriate antibiotics (i.e. ongoing temperatures of >/= 38 degree Celsius).
- Total bilirubin >/= 1.5 mg/dL and not related to hemolysis or Gilbert's disease. Patients with total bilirubin >/= 1.5 mg/dL to 3 mg/dL are eligible if at least 75% of the bilirubin is indirect.
- Alanine transaminase (ALT/SGPT) or aspartate transaminase (AST/SGOT) >/= 2.5 x the upper limit of normal.
- Serum creatinine > 1.5 mg/dL.
- Female patients who are pregnant or lactating.
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- Patients with reproductive potential who are unwilling to following contraception requirements (including condom use for males with sexual partners, and for females: prescription oral contraceptives [birth control pills], contraceptive injections, intrauterine devices [IUD], double-barrier method [spermicidal jelly or foam with condoms or diaphragm], contraceptive patch, or surgical sterilization) throughout the study.
- Female patients with reproductive potential who do not have a negative urine or blood beta-human chorionic gonadotropin (beta HCG) pregnancy test at screening.
- Patients receiving any other concurrent investigational agent or chemotherapy, radiotherapy, or immunotherapy.
- Prior hydroxyurea for control of leukocytosis or use of hematopoietic growth factors (eg, G-CSF, GM-CSF, procrit, aranesp, thrombopoietins) is allowed at any time prior to or during study if considered to be in the best interest of the patient.
- Psychiatric illness or social situation that would limit the patient's ability to comply with study requirements.
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) in Patients With Intermediate-1 and Higher Risk Myelodysplastic Syndrome (MDS) Post Hypomethylating Agent (HMA) Failure
The goal of this clinical research study is learn if omacetaxine can help to control myelodysplastic syndrome (MDS). The safety of this drug will also be studied.
This is an investigational study. Omacetaxine is FDA approved and commercially available for the treatment of chronic myelogenous leukemia (CML). It is investigational to use omacetaxine in patients with MDS. The study doctor can explain how the study drug is designed to work.
Up to 80 participants will be enrolled in this study. All will take part at MD Anderson.
Read the detailed description
Study Drug Administration:
Each cycle will be 4-7 weeks, depending on how well the disease responds to the study drug.
If you are found to be eligible to take part in this study, you will receive omacetaxine as an injection under your skin 2 times each day, about 12 hours apart, on Days 1-3 of every 28-day study cycle.
You will receive instructions on how to give these injections to yourself. You will be given a Research Drug Diary to record the drug you take each day. You must bring the Research Drug Diary and any unused drug with you to each study visit. You will also be told how to properly store the drugs.
Depending on how the disease responds to the study drugs, the number of days you receive your injections may stay the same, increase, or decrease. Your doctor will discuss this with you.
During Cycle 1, if the doctor thinks it is needed, you will be given hydroxyurea by mouth to decrease the risk of side effects. You may ask the study staff for information about how the drug is given and its risks.
Study Visits:
At the beginning of every cycle, you will have a physical exam before your dose of study drug.
Every week (+/- 2 days), blood (about 2-3 teaspoons) will be drawn 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.
If you live far from the clinic, this blood can be drawn at a clinic close to your home, and the results will be reported to the study doctor.
If the study doctor thinks it is needed, you may have an additional bone marrow aspirate at any time during the study to check the status of the disease and for cytogenetic testing.
Length of Study:
You may continue taking the study drug for up to 24 cycles of treatment. You will no longer be able to take the study drug 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 at the clinic every 3-6 months for up to 5 years after you stop taking the study drug. You will be asked about your health and any new drugs you may be taking. If you cannot come to the clinic, you will be called by the study staff and asked about your health. These calls should last about 5-10 minutes.
Every 4-8 weeks after your last dose of study drug, blood (about 2-3 teaspoons) will be drawn for routine tests.
- Conditions
- Leukemia
- Being tested
- Omacetaxine
- Keywords
- Leukemia, Myelodysplastic Syndrome, MDS, Omacetaxine, Synribo, Homoharringtonine
- Medical subject headings
- Leukemia, Myelodysplastic Syndromes
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
OmacetaxineDrug
Also called Synribo, Homoharringtonine
1.25 mg/m2 subcutaneously every 12 hours on Days 1-3 of every 28-day study cycle.
| Study group | Role | What they receive |
|---|---|---|
| Omacetaxine | Experimental | Omacetaxine 1.25 mg/m2 subcutaneously every 12 hours on Days 1-3 of every 28-day study cycle. Participant may continue taking the study drug for up to 24 cycles of treatment. Receives: Drug: Omacetaxine |
What is being measured
The primary outcome is the main question the study is designed to answer.
Overall Survival (OS)
Overall survival defined as the time from treatment start to the time of death. Overall survival continuously monitored using the Bayesian method.
Measured: Up to 2.5 Years
Number of Participants With a Response
Response is Complete Response (CR) + Partial Response (PR) + Hematologic Improvement (HI). CR is the normalization of the peripheral blood and bone marrow with </= 5% bone marrow blasts, a peripheral blood granulocyte count >/= (1.0x10^9/L, and a platelet count >/= 100x10^9/L). PR is the same as CR except for the presence of 6-15% marrow blasts, or 50% reduction if <15% at start of treatment. HI meets all of the criteria for CR except for platelet recovery to >/=100x10^9L.
Measured: Up to 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Jun 2014 (estimated)
- Study start18 May 2015
- Primary completion14 Apr 2020
- Study completion14 Apr 2020
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 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 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 48 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
- NCT02159872
- Sponsor's protocol ID
- 2013-0870
- Other IDs
- NCI-2014-01483
- Status last verified
- May 2021
- First submitted
- 6 Jun 2014
- Last updated
- 9 Jun 2021
- Results posted
- Yes, on ClinicalTrials.gov
- 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 9 Jun 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase II Study of Omacetaxine (OM) in Patients With Intermediate-1 and Higher Risk Myelodysplastic Syndrome (MDS) Post Hypomethylating Agent (HMA) Failure
- ClinicalTrials.gov ID
- NCT02159872
- Acronym / sponsor ID
- 2013-0870
- Conditions
- Leukemia
- Keywords
- Leukemia, Myelodysplastic Syndrome, MDS, Omacetaxine, Synribo, Homoharringtonine
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 48 participants (actual)
- Lead sponsor
- M.D. Anderson Cancer CenterUniversity, hospital or other
- Collaborators
- Teva Pharmaceuticals USA
- Study start
- 18 May 2015 (actual)
- Primary completion
- 14 Apr 2020 (actual)
- Study completion
- 14 Apr 2020 (actual)
- First posted
- 10 Jun 2014 (estimated)
- Results
- Posted 9 Jun 2021
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The goal of this clinical research study is learn if omacetaxine can help to control myelodysplastic syndrome (MDS). The safety of this drug will also be studied.
This is an investigational study. Omacetaxine is FDA approved and commercially available for the treatment of chronic myelogenous leukemia (CML). It is investigational to use omacetaxine in patients with MDS. The study doctor can explain how the study drug is designed to work.
Up to 80 participants will be enrolled in this study. All will take part at MD Anderson.
Detailed description
Study Drug Administration:
Each cycle will be 4-7 weeks, depending on how well the disease responds to the study drug.
If you are found to be eligible to take part in this study, you will receive omacetaxine as an injection under your skin 2 times each day, about 12 hours apart, on Days 1-3 of every 28-day study cycle.
You will receive instructions on how to give these injections to yourself. You will be given a Research Drug Diary to record the drug you take each day. You must bring the Research Drug Diary and any unused drug with you to each study visit. You will also be told how to properly store the drugs.
Depending on how the disease responds to the study drugs, the number of days you receive your injections may stay the same, increase, or decrease. Your doctor will discuss this with you.
During Cycle 1, if the doctor thinks it is needed, you will be given hydroxyurea by mouth to decrease the risk of side effects. You may ask the study staff for information about how the drug is given and its risks.
Study Visits:
At the beginning of every cycle, you will have a physical exam before your dose of study drug.
Every week (+/- 2 days), blood (about 2-3 teaspoons) will be drawn 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.
If you live far from the clinic, this blood can be drawn at a clinic close to your home, and the results will be reported to the study doctor.
If the study doctor thinks it is needed, you may have an additional bone marrow aspirate at any time during the study to check the status of the disease and for cytogenetic testing.
Length of Study:
You may continue taking the study drug for up to 24 cycles of treatment. You will no longer be able to take the study drug 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 at the clinic every 3-6 months for up to 5 years after you stop taking the study drug. You will be asked about your health and any new drugs you may be taking. If you cannot come to the clinic, you will be called by the study staff and asked about your health. These calls should last about 5-10 minutes.
Every 4-8 weeks after your last dose of study drug, blood (about 2-3 teaspoons) will be drawn for routine tests.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Omacetaxine | Experimental | Drug: OmacetaxineOmacetaxine 1.25 mg/m2 subcutaneously every 12 hours on Days 1-3 of every 28-day study cycle. Participant may continue taking the study drug for up to 24 cycles of treatment. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- Age >/= 18 years
- Diagnosis of MDS confirmed within 10 weeks prior to study entry according to WHO criteria. Patients are either not eligible for or choose not to proceed with a stem cell transplant.
- MDS classified as follows: RAEB-1 (5%-9% BM blasts); RAEB-2 (10%-19% BM Blasts); CMML (5%-19% BM blasts); RAEB-t (20%-29% BM blasts) AND/OR by IPSS: intermediate-1 and high risk patients.
- No response, progression, or relapse (according to 2006 IWG criteria) following at least 4 cycles of either azacitidine or decitabine, which were completed within the last 2 years - AND/OR - intolerance to azacitidine or decitabine defined as drug-related >/= grade 3 hepatic or renal toxicity leading to treatment discontinuation during the preceding 2 years.
- Eastern Cooperative Oncology Group (ECOG) performance status of </= 2.
Key exclusion criteria (11 listed)
- Uncontrolled intercurrent illness including, but not limited to, symptomatic congestive heart failure, unstable angina pectoris, or cardiac arrhythmia.
- Active infection not adequately responding to appropriate antibiotics (i.e. ongoing temperatures of >/= 38 degree Celsius).
- Total bilirubin >/= 1.5 mg/dL and not related to hemolysis or Gilbert's disease. Patients with total bilirubin >/= 1.5 mg/dL to 3 mg/dL are eligible if at least 75% of the bilirubin is indirect.
- Alanine transaminase (ALT/SGPT) or aspartate transaminase (AST/SGOT) >/= 2.5 x the upper limit of normal.
- Serum creatinine > 1.5 mg/dL.
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, Homoharringtonine1.25 mg/m2 subcutaneously every 12 hours on Days 1-3 of every 28-day study cycle. | Drug | Omacetaxine |
Primary outcome
Overall Survival (OS)
Time frame: Up to 2.5 Years
Overall survival defined as the time from treatment start to the time of death. Overall survival continuously monitored using the Bayesian method.
Number of Participants With a Response
Time frame: Up to 2 years
Response is Complete Response (CR) + Partial Response (PR) + Hematologic Improvement (HI). CR is the normalization of the peripheral blood and bone marrow with </= 5% bone marrow blasts, a peripheral blood granulocyte count >/= (1.0x10^9/L, and a platelet count >/= 100x10^9/L). PR is the same as CR except for the presence of 6-15% marrow blasts, or 50% reduction if <15% at start of treatment. HI meets all of the criteria for CR except for platelet recovery to >/=100x10^9L.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 9 Jun 2021.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- 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 2 study testing Omacetaxine 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 7 inclusion and 11 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 9 Jun 2021. 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 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 9 Jun 2021). 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?
Overall Survival (OS) (time frame: Up to 2.5 Years). The study lists 2 primary outcomes in all. 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 9 Jun 2021.