LeukemiaPhase 1NCT00624936
Azacytidine and Bortezomib in Treating Patients With Relapsed or Refractory Acute Myeloid Leukemia or Myelodysplastic Syndromes
A Phase 1 study testing Vidaza and Velcade in people with Leukemia and Myelodysplastic Syndromes, run by Ohio State University Comprehensive Cancer Center at 1 site in United States.
Trial summary
RATIONALE: Drugs used in chemotherapy, such as azacytidine work in different ways to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Leukemia, Myelodysplastic Syndromes, Myelodysplastic/Myeloproliferative Neoplasms
- What is being tested?
- Vidaza and Velcade
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 23 participants
- Where
- 1 siteUnited States
- Study dates
- Apr 2008to Jun 2014
- 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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Patients must be >18 with relapsed or refractory acute myeloid leukemia (AML) and high risk (by IPSS scoring) Myelodysplastic Syndromes (MDS)
- Patients with secondary AML or therapy related disease (t-AML) are eligible If decitabine or Vidaza was a prior treatment for MDS or AML patient is eligible.Prior Velcade is also permitted.
- ECOG performance status 0-2
- Life expectancy > 6 months for patients with a co-morbid medical illness
- Total bilirubin < 2.0mg/dL
- AST/ALT < 2.5 times upper limit of normal (ULN)
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- Creatinine < 2.0 mg/dL
- Not pregnant or nursing
- Negative pregnancy test
- Fertile patients must use effective contraception prior to and during study treatment
- Ability to understand and willingness to sign the written informed consent document
- Active infection is allowed provided it is under control
Who cannot join
- History of allergic reactions attributed to compounds of similar chemical or biologic composition to azacytidine or bortezomib that are not easily managed
- Hypersensitivity to bortezomib, boron, or mannitol
- Uncontrolled intercurrent illness including, but not limited to:
- Symptomatic congestive heart failure
- Unstable angina pectoris
- Serious cardiac arrhythmia
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- Psychiatric illness/social situations that would limit compliance with study
- Myocardial infarction within 6 months prior to enrollment
- New York Heart Association (NYHA) Class III or IV congestive heart failure
- Uncontrolled angina
- Severe uncontrolled ventricular arrhythmia
- Electrocardiographic evidence of acute ischemia
- Active conduction system abnormalities
- ECG abnormality that is medically relevant
- Psychiatric conditions that prevent compliance with protocol or consent.
- Pre-existing neuropathy grade 2 or higher or other serious neurologic toxicity that would significantly increase risk of complications from bevacizumab therapy
- Serious medical or psychiatric illness likely to interfere with participation in this clinical study
- Diagnosis or treatment for another malignancy within 3 years of enrollment, with the exception of any of the following:
- Complete resected basal cell carcinoma
- Squamous cell carcinoma of the skin
- Any in situ malignancy
- Low-risk prostate cancer after curative therapy
- PRIOR CONCURRENT THERAPY:
- Prior decitabine or azacytidine for MDS or AML is allowed
- Prior bortezomib allowed
- More than 2 weeks since prior chemotherapy or radiotherapy (6 weeks for nitrosoureas or mitomycin C)
- More than 14 days since prior and no concurrent investigational agents
- No concurrent combination antiretroviral therapy for HIV-positive patients
Trial locations and contacts
1 site in 1 country.
United States1 site
Ohio State University Medical Center
Columbus, Ohio, 43210
About this study
Official title: Phase I Study of Vidaza and Velcade (Bortezomib) in Acute Myeloid Leukemia
RATIONALE: Drugs used in chemotherapy, such as azacytidine work in different ways to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. Bortezomib may stop the growth of cancer cells by blocking blood flow to the cancer and by blocking some of the enzymes needed for cell growth. Giving azacytidine together with bortezomib may kill more cancer cells.
PURPOSE: This phase I trial is studying the side effects and best dose of bortezomib when giving together with azacytidine in treating patients with relapsed or refractory acute myeloid leukemia or myelodysplastic syndromes.
Read the detailed description
OBJECTIVES:
Primary
- To determine the maximum tolerated dose (MTD) bortezomib in combination with Azacytidine in patients with relapsed/refractory acute myeloid leukemia (AML) and myelodysplastic syndromes (MDS).
- To define the specific toxicities and the dose limiting toxicity (DLT) of Azacytidine plus bortezomib combination.
Secondary
- To determine the overall response rate (ORR).
- To determine the rate of complete remission (CR) of Azacytidine plus bortezomib in relapsed/refractory AML and MDS.
- To correlate the biological activity of Azacytidine as demethylating agent (changes in target gene methylation and gene expression, DNMT1 protein expression, global methylation) with clinical endpoints and plasma pharmacokinetics of azacytidine.
- To characterize the biological activity of bortezomib as a potential demethylating agent.
- To correlate intracellular concentration of azacytidine-triphosphate with global DNA methylation and other biological endpoints as well as clinical response.
- To explore the biologic role of microRNAs in determining clinical response to the azacytidine plus bortezomib combination and achievement of the other pharmacodynamic endpoints.
OUTLINE: This is a dose-escalation study of bortezomib.
Patients receive azacytidine IV over 30 minutes on days 1-7 and bortezomib IV on days 2 and 5 or on days 2, 5, and 9 or on days 2, 5, 9, and 12. Treatment repeats every 28 days for up to 12 courses in the absence of disease progression or unacceptable toxicity.
Cohorts of 3-6 patients receive escalating doses of bortezomib and tipifarnib until the maximum tolerated dose (MTD) is determined. The MTD is defined as the dose preceding that at which 2 of 3 or 2 of 6 patients experience dose-limiting toxicity.
After completion of study treatment, patients are followed for at least 30 days.
- Being tested
- Vidaza, Velcade
- Keywords
- adult acute myeloid leukemia with 11q23 (MLL) abnormalities, adult acute myeloid leukemia with inv(16)(p13;q22), adult acute myeloid leukemia with t(15;17)(q22;q12), adult acute myeloid leukemia with t(16;16)(p13;q22), adult acute myeloid leukemia with t(8;21)(q22;q22), recurrent adult acute myeloid leukemia, myelodysplastic/myeloproliferative neoplasm, unclassifiable, myelodysplastic syndromes, secondary acute myeloid leukemia
- Medical subject headings
- Leukemia, Myelodysplastic Syndromes, Myelodysplastic-Myeloproliferative Diseases, Congenital Abnormalities, Leukemia, Myeloid, Acute, Myeloproliferative Disorders
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
VidazaDrug
Also called Azacitidina, Azacitidinum, Azacytidine, Mylosar
Vidaza 75mg/m2 IV over 30min daily on days 1-7. This dose is the same for all dose levels.
VelcadeDrug
Also called Bortezomib
Dose level 1 Velcade 0.7mg/m2 IVP on days 2 and 5 Dose level 2 Velcade 0.7mg/m2 IVP on days 2, 5, 9, 12 Dose level 3 Velcade 1.0 mg/m2 IVP on days 2, 5, 9, 12 Dose level 4 Velcade 1.3 mg/m2 IVP on days 2, 5, 9, 12
| Study group | Role | What they receive |
|---|---|---|
| Vidaza and Velcade | Experimental | Vidaza 75mg/m2 IV over 30 min daily on days 1-7 This dose is the same for all dose levels. Velcade will be given immediately after Vidaza is completed at one of the following dose levels: 1, 2, 3, 4 Receives: Drug: Vidaza; Drug: Velcade |
What is being measured
The primary outcome is the main question the study is designed to answer.
Maximum tolerated dose of bortezomib in combination with azacytidine
Determine the maximum tolerated dose (MTD) of Velcade (bortezomib, PS-341) in combination with Vidaza in patients with relapsed/refractory acute myeloid leukemia (AML) and Myelodysplastic Syndrome (MDS)
Measured: Up to 1 year
Overall response rate
Determine the overall response rate (ORR)
Measured: Up to 1 year
Secondary outcomes (5)
Rate of complete remission
Determine the rate of complete remission (CR) of Vidaza plus Velcade in relapsed/refractory AML and MDS
Measured: Up to 1 year
Biological activity of azacytidine and bortezomib as demethylating agents
Correlate the biological activity of Vidaza as demethylating agent (changes in target gene methylation and gene expression, DNMT1 protein expression, global methylation) with clinical endpoints and plasma pharmacokinetics of Vidaza
Measured: Up to 1 year
Correlation of intracellular concentration of azacytidine-triphosphate with global DNA methylation and other biological endpoints as well as clinical response
Characterize the biological activity of Velcade as a potential demethylating
Measured: Up to 1 year
Biologic role of microRNAs in determining clinical response to study drugs
Correlate intracellular concentration of Vidaza-triphosphate with global DNA methylation and other biological endpoints as well as clinical response
Measured: Up to 1 year
Achievement of other pharmacodynamic endpoints
Explore the biologic role of microRNAs in determining clinical response to the Vidaza plus Velcade combination and achievement of the other pharmacodynamic endpoints.
Measured: Up to 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Feb 2008 (estimated)
- Study startApr 2008
- Primary completionDec 2009
- Study completionJun 2014
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 Ohio State University Comprehensive 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
- 23 participants (actual)
Sponsor and investigators
- Sponsor type
- University, hospital or other
- Collaborators
- Millennium Pharmaceuticals, Inc., Celgene Corporation
- Responsible party
- Sponsor
- Investigators
- William G. Blum, MD, Principal investigator, Ohio State UniversityGuido Marcucci, MD, Principal investigator, Ohio State University Comprehensive Cancer Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00624936
- Sponsor's protocol ID
- OSU-07095
- Other IDs
- NCI-2011-03146
- Status last verified
- Aug 2017
- First submitted
- 27 Feb 2008
- Last updated
- 22 Aug 2017
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 22 Aug 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase I Study of Vidaza and Velcade (Bortezomib) in Acute Myeloid Leukemia
- ClinicalTrials.gov ID
- NCT00624936
- Acronym / sponsor ID
- OSU-07095
- Keywords
- adult acute myeloid leukemia with 11q23 (MLL) abnormalities, adult acute myeloid leukemia with inv(16)(p13;q22), adult acute myeloid leukemia with t(15;17)(q22;q12), adult acute myeloid leukemia with t(16;16)(p13;q22), adult acute myeloid leukemia with t(8;21)(q22;q22), recurrent adult acute myeloid leukemia, myelodysplastic/myeloproliferative neoplasm, unclassifiable, myelodysplastic syndromes
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 23 participants (actual)
- Lead sponsor
- Ohio State University Comprehensive Cancer CenterUniversity, hospital or other
- Collaborators
- Millennium Pharmaceuticals, Inc., Celgene Corporation
- Study start
- Apr 2008 (actual)
- Primary completion
- Dec 2009 (actual)
- Study completion
- Jun 2014 (actual)
- First posted
- 28 Feb 2008 (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.
RATIONALE: Drugs used in chemotherapy, such as azacytidine work in different ways to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. Bortezomib may stop the growth of cancer cells by blocking blood flow to the cancer and by blocking some of the enzymes needed for cell growth. Giving azacytidine together with bortezomib may kill more cancer cells.
PURPOSE: This phase I trial is studying the side effects and best dose of bortezomib when giving together with azacytidine in treating patients with relapsed or refractory acute myeloid leukemia or myelodysplastic syndromes.
Detailed description
OBJECTIVES:
Primary
- To determine the maximum tolerated dose (MTD) bortezomib in combination with Azacytidine in patients with relapsed/refractory acute myeloid leukemia (AML) and myelodysplastic syndromes (MDS).
- To define the specific toxicities and the dose limiting toxicity (DLT) of Azacytidine plus bortezomib combination.
Secondary
- To determine the overall response rate (ORR).
- To determine the rate of complete remission (CR) of Azacytidine plus bortezomib in relapsed/refractory AML and MDS.
- To correlate the biological activity of Azacytidine as demethylating agent (changes in target gene methylation and gene expression, DNMT1 protein expression, global methylation) with clinical endpoints and plasma pharmacokinetics of azacytidine.
- To characterize the biological activity of bortezomib as a potential demethylating agent.
- To correlate intracellular concentration of azacytidine-triphosphate with global DNA methylation and other biological endpoints as well as clinical response.
- To explore the biologic role of microRNAs in determining clinical response to the azacytidine plus bortezomib combination and achievement of the other pharmacodynamic endpoints.
OUTLINE: This is a dose-escalation study of bortezomib.
Patients receive azacytidine IV over 30 minutes on days 1-7 and bortezomib IV on days 2 and 5 or on days 2, 5, and 9 or on days 2, 5, 9, and 12. Treatment repeats every 28 days for up to 12 courses in the absence of disease progression or unacceptable toxicity.
Cohorts of 3-6 patients receive escalating doses of bortezomib and tipifarnib until the maximum tolerated dose (MTD) is determined. The MTD is defined as the dose preceding that at which 2 of 3 or 2 of 6 patients experience dose-limiting toxicity.
After completion of study treatment, patients are followed for at least 30 days.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Vidaza and Velcade | Experimental | Drug: Vidaza, Drug: VelcadeVidaza 75mg/m2 IV over 30 min daily on days 1-7 This dose is the same for all dose levels. Velcade will be given immediately after Vidaza is completed at one of the following dose levels: 1, 2, 3, 4 |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (12 listed)
- Patients must be >18 with relapsed or refractory acute myeloid leukemia (AML) and high risk (by IPSS scoring) Myelodysplastic Syndromes (MDS)
- Patients with secondary AML or therapy related disease (t-AML) are eligible If decitabine or Vidaza was a prior treatment for MDS or AML patient is eligible.Prior Velcade is also permitted.
- ECOG performance status 0-2
- Life expectancy > 6 months for patients with a co-morbid medical illness
- Total bilirubin < 2.0mg/dL
Key exclusion criteria (28 listed)
- History of allergic reactions attributed to compounds of similar chemical or biologic composition to azacytidine or bortezomib that are not easily managed
- Hypersensitivity to bortezomib, boron, or mannitol
- Uncontrolled intercurrent illness including, but not limited to:
- Myocardial infarction within 6 months prior to enrollment
- New York Heart Association (NYHA) Class III or IV congestive heart failure
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 |
|---|---|---|
| VidazaAlso: Azacitidina, Azacitidinum, Azacytidine, MylosarVidaza 75mg/m2 IV over 30min daily on days 1-7. This dose is the same for all dose levels. | Drug | Vidaza and Velcade |
| VelcadeAlso: BortezomibDose level 1 Velcade 0.7mg/m2 IVP on days 2 and 5 Dose level 2 Velcade 0.7mg/m2 IVP on days 2, 5, 9, 12 Dose level 3 Velcade 1.0 mg/m2 IVP on days 2, 5, 9, 12 Dose level 4 Velcade 1.3 mg/m2 IVP on days 2, 5, 9, 12 | Drug | Vidaza and Velcade |
Primary outcome
Maximum tolerated dose of bortezomib in combination with azacytidine
Time frame: Up to 1 year
Determine the maximum tolerated dose (MTD) of Velcade (bortezomib, PS-341) in combination with Vidaza in patients with relapsed/refractory acute myeloid leukemia (AML) and Myelodysplastic Syndrome (MDS)
Overall response rate
Time frame: Up to 1 year
Determine the overall response rate (ORR)
5 secondary outcomes
Rate of complete remission
Time frame: Up to 1 year
Biological activity of azacytidine and bortezomib as demethylating agents
Time frame: Up to 1 year
Correlation of intracellular concentration of azacytidine-triphosphate with global DNA methylation and other biological endpoints as well as clinical response
Time frame: Up to 1 year
Biologic role of microRNAs in determining clinical response to study drugs
Time frame: Up to 1 year
Achievement of other pharmacodynamic endpoints
Time frame: Up to 1 year
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 22 Aug 2017.
- JameslineLink 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.
William G. Blum, MD principal investigator
Ohio State University
Guido Marcucci, MD principal investigator
Ohio State University Comprehensive 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 Vidaza and Velcade in people with Leukemia and Myelodysplastic Syndromes, run by Ohio State University Comprehensive 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 12 inclusion and 28 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 22 Aug 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 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 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?
Maximum tolerated dose of bortezomib in combination with azacytidine (time frame: Up to 1 year). 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 22 Aug 2017.