Myelodysplastic SyndromesPhase 1/2NCT02835794
A Clinical Trial of Omacetaxine, Azacitidine, and Growth-Colony Stimulating Factor (G-CSF) for Myelodysplastic Syndromes (MDS)
A Phase 1/2 study testing Omacetaxine, Azacitidine and G-CSF in people with Myelodysplastic Syndromes, run by University of Florida at 1 site in United States.
Trial summary
The purpose of this study is to determine the safety and establish the maximum tolerated dose (MTD) of omacetaxine in combination with azacitidine and G-CSF in patients with relapsed and/or refractory MDS. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Myelodysplastic Syndromes
- What is being tested?
- Omacetaxine, Azacitidine and G-CSF
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 0 participants
- Where
- 1 siteUnited States
- Study dates
- Aug 2016to Dec 2018
- Phase
- Phase 1/2
- 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
- Age >= 18 years;
- Informed consent;
- Low- and intermediate-risk MDS that has failed to achieve any hematologic improvement after at least 4 cycles of induction therapy or has relapsed after any duration of any hematologic response. Prior therapy with azanucleosides (i.e., azacitidine, decitabine), biologic therapies (i.e., lenalidomide, rigosertib) and hematopoietic cell transplant are permissible;
- Performance status must be Eastern Cooperative Oncology Group (ECOG) 0, 1, or 2;
- Women of child-bearing potential (i.e., women who are pre-menopausal or not surgically sterile) may participate, provided they meet the following conditions:
- Must agree to use physician-approved contraceptive methods throughout the study and for three months following the last dose of omacetaxine and
- Must have a negative serum or urine pregnancy test within 7 days prior to beginning treatment on this trial;
- Males with female partners of child-bearing potential must agree to use physician-approved contraceptive methods throughout the study and should avoid conceiving a child for 6 months following the last dose of omacetaxine.
Who cannot join
- Subjects who are eligible for hematopoietic stem cell transplant;
- History of atrial fibrillation related to azanucleoside therapy in the past;
- Active, uncontrolled, clinically significant infection;
- Pregnant and nursing patients are excluded because the effects of omacetaxine on a fetus or nursing child are unknown;
- Treatment with any anticancer therapy (standard or investigational) within the previous 14 days prior to the first dose of study drug or less than full recovery from the clinically significant toxic effects of that treatment.
Trial locations and contacts
1 site in 1 country.
United States1 site
University of Florida Health Shands Cancer Hospital
Gainesville, Florida, 32608
About this study
Official title: A Phase I/II Clinical Trial of Omacetaxine, Azacitidine, and G-CSF for Relapsed and/or Refractory Myelodysplastic Syndromes
The purpose of this study is to determine the safety and establish the maximum tolerated dose (MTD) of omacetaxine in combination with azacitidine and G-CSF in patients with relapsed and/or refractory MDS.
- Conditions
- Myelodysplastic Syndromes
- Being tested
- Omacetaxine, Azacitidine, G-CSF
- Keywords
- Myelodysplastic Syndromes
- Medical subject headings
- 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
Comparison of different doses of omacetaxine in combination with azacitidine and G-CSF
AzacitidineDrug
Also called Vidaza
Comparison of different doses of omacetaxine in combination with azacitidine and G-CSF
G-CSFDrug
Also called Neupogen
Comparison of different doses of omacetaxine in combination with azacitidine and G-CSF
| Study group | Role | What they receive |
|---|---|---|
| Arm 1 | Experimental | Omacetaxine - escalating doses subcutaneous twice daily on Days 1-5 and 8-12 Azacitidine 50 mg/m2 subcutaneous/intravenous daily on Days 8-12 G-CSF 5mcg/kg subcutaneous daily on Days 15-19 and 22-26 Receives: Drug: Omacetaxine; Drug: Azacitidine; Drug: G-CSF |
What is being measured
The primary outcome is the main question the study is designed to answer.
The maximum tolerated dose (MTD) of omacetaxine in combination with azacitidine and G-CSF in patients with relapsed and/or refractory low- and intermediate-risk MDS.
Measured: 28 days
Secondary outcomes (5)
Number of participants with Hematologic Improvement (HI) as measured by hemoglobin, platelet count and neutrophil count.
Measured: 12 months
Number of participants with disease response as defined by International Working Group (IWG) 2006 criteria.
Measured: 12 months
Number of participants who achieve complete remission and how long the response lasts
Measured: 24 months
Length of time of survival for participants
Measured: 24 months
Incidences of Grade 3/4 adverse events directly related to the drug combination
Measured: 24 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Jul 2016 (estimated)
- Study startAug 2016
- Primary completionDec 2018 (estimated)
- Study completionDec 2018 (estimated)
Why it stopped: Lack of funding to support clinical trial
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Myelodysplastic SyndromesStudies of the same condition, open and under way.
- More trials run by University of FloridaThe 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
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of Florida
- Sponsor type
- University, hospital or other
- Collaborators
- Teva Pharmaceutical Industries, Ltd.
- Responsible party
- Sponsor
- Investigators
- Maxim N. Norkin, MD, PhD, Principal investigator, University of Florida
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02835794
- Sponsor's protocol ID
- IRB201601194
- Other IDs
- UF-MDS-OAG-101
- Status last verified
- May 2017
- First submitted
- 30 Jun 2016
- Last updated
- 3 May 2017
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 3 May 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase I/II Clinical Trial of Omacetaxine, Azacitidine, and G-CSF for Relapsed and/or Refractory Myelodysplastic Syndromes
- ClinicalTrials.gov ID
- NCT02835794
- Acronym / sponsor ID
- IRB201601194
- Conditions
- Myelodysplastic Syndromes
- Keywords
- Myelodysplastic Syndromes
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: Lack of funding to support clinical trial
- Enrolment
- 0 participants (actual)
- Lead sponsor
- University of FloridaUniversity, hospital or other
- Collaborators
- Teva Pharmaceutical Industries, Ltd.
- Study start
- Aug 2016
- Primary completion
- Dec 2018 (estimated)
- Study completion
- Dec 2018 (estimated)
- First posted
- 18 Jul 2016 (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 purpose of this study is to determine the safety and establish the maximum tolerated dose (MTD) of omacetaxine in combination with azacitidine and G-CSF in patients with relapsed and/or refractory MDS.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Arm 1 | Experimental | Drug: Omacetaxine, Drug: Azacitidine, Drug: G-CSFOmacetaxine - escalating doses subcutaneous twice daily on Days 1-5 and 8-12 Azacitidine 50 mg/m2 subcutaneous/intravenous daily on Days 8-12 G-CSF 5mcg/kg subcutaneous daily on Days 15-19 and 22-26 |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (8 listed)
- Age >= 18 years;
- Informed consent;
- Low- and intermediate-risk MDS that has failed to achieve any hematologic improvement after at least 4 cycles of induction therapy or has relapsed after any duration of any hematologic response. Prior therapy with azanucleosides (i.e., azacitidine, decitabine), biologic therapies (i.e., lenalidomide, rigosertib) and hematopoietic cell transplant are permissible;
- Performance status must be Eastern Cooperative Oncology Group (ECOG) 0, 1, or 2;
- Women of child-bearing potential (i.e., women who are pre-menopausal or not surgically sterile) may participate, provided they meet the following conditions:
Key exclusion criteria (5 listed)
- Subjects who are eligible for hematopoietic stem cell transplant;
- History of atrial fibrillation related to azanucleoside therapy in the past;
- Active, uncontrolled, clinically significant infection;
- Pregnant and nursing patients are excluded because the effects of omacetaxine on a fetus or nursing child are unknown;
- Treatment with any anticancer therapy (standard or investigational) within the previous 14 days prior to the first dose of study drug or less than full recovery from the clinically significant toxic effects of that treatment.
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: SynriboComparison of different doses of omacetaxine in combination with azacitidine and G-CSF | Drug | Arm 1 |
| AzacitidineAlso: VidazaComparison of different doses of omacetaxine in combination with azacitidine and G-CSF | Drug | Arm 1 |
| G-CSFAlso: NeupogenComparison of different doses of omacetaxine in combination with azacitidine and G-CSF | Drug | Arm 1 |
Primary outcome
The maximum tolerated dose (MTD) of omacetaxine in combination with azacitidine and G-CSF in patients with relapsed and/or refractory low- and intermediate-risk MDS.
Time frame: 28 days
5 secondary outcomes
Number of participants with Hematologic Improvement (HI) as measured by hemoglobin, platelet count and neutrophil count.
Time frame: 12 months
Number of participants with disease response as defined by International Working Group (IWG) 2006 criteria.
Time frame: 12 months
Number of participants who achieve complete remission and how long the response lasts
Time frame: 24 months
Length of time of survival for participants
Time frame: 24 months
Incidences of Grade 3/4 adverse events directly related to the drug combination
Time frame: 24 months
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 May 2017.
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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.
Maxim N. Norkin, MD, PhD principal investigator
University of Florida
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 Omacetaxine, Azacitidine and G-CSF in people with Myelodysplastic Syndromes, run by University of Florida 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 8 inclusion and 5 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: withdrawn, as of the sponsor's last update on 3 May 2017. 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?
The study was withdrawn before enrolling participants, so there are no results.
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?
The maximum tolerated dose (MTD) of omacetaxine in combination with azacitidine and G-CSF in patients with relapsed and/or refractory low- and intermediate-risk MDS. (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 3 May 2017.