LeukemiaPhase 2NCT00948064

Vorinostat in Combination With Azacitidine in Patients With Newly-Diagnosed Acute Myelogenous Leukemia (AML) or Myelodysplastic Syndrome (MDS)

A Phase 2 study testing Vorinostat and Azacitidine in people with Leukemia, run by M.D. Anderson Cancer Center at 1 site in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

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Trial summary

The goal of this clinical research study is to learn if the combination of azacitidine and vorinostat can help to control AML or MDS better than azacitidine alone. The safety of this drug combination will also be studied. Read more about this study

Who is it for?: Ages 18 years and older
Conditions the study is for: Leukemia
What is being tested?: Vorinostat and Azacitidine
Type of treatment: Pill or oral medicine, Injection or infusion
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 110 participants
Where: 1 siteUnited States
Study dates: 8 Sep 2009to 3 Aug 2017
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

Patients with newly diagnosed AML or MDS (Intermediate 1 or higher risk)
Patient must have at least one of the following: a. Creatinine >/= 2 mg/dL; b. total Bilirubin >/= 2 mg/dL; c.ECOG Performance Status equal to 3 or 4; and d. is ineligible for participation on a protocol of higher priority
Patients must provide written informed consent.
Patients must be age > 18 years due to lack of safety information with these agents in children.
Patient agrees to: 1) Use 2 adequate methods of contraception to prevent pregnancy (either 2 barrier methods or a barrier method plus a hormonal contraceptive method) or 2) abstain from heterosexual activity throughout the study starting with Visit 1.
Female patients of childbearing potential should have a negative pregnancy test (serum) within 72 hrs. of study enrollment.

Who cannot join

Patients must not have the favorable cytogenetic abnormalities of inv (16), t (16;16), t (8;21), or t (15;17).
Patients receiving any anti-leukemic therapy with the exception of Hydroxyurea prior to study enrollment. Prior growth factor therapy is acceptable. Hydroxyurea could be used at the discretion of the treating physician. A single or a two day dose of cytarabine (up to 3 g/m^2) for emergency use is allowed as prior therapy.
Patient has a prior history of treatment with HDAC inhibitors. Patients who have received valproic acid (VPA) for the treatment of seizures may be enrolled on this study, but must not have received VPA within 30 days of study enrollment.
Patient is unable to take and/or tolerate oral medications on a continuous basis, examples include patients on a ventilator, or have altered mental status that precludes safe oral route of administration.
Patient has active hepatitis A, B, or C infection.
Patient is pregnant or breast-feeding.
Patient has a known allergy or hypersensitivity to any component of vorinostat or azacitidine.
History of any psychiatric condition that might impair the patient's ability to understand or to comply with the requirements of the study or to provide informed consent.

Trial locations and contacts

1 site in 1 country.

United States1 site

University of Texas MD Anderson Cancer Center
Houston, Texas, 77030

Other trials in United States

About this study

Official title: A Phase 2 Trial of Vorinostat in Combination With Azacitidine in Patients With Newly-Diagnosed Acute Myelogenous Leukemia (AML) or Myelodysplastic Syndrome (MDS) Who Are Ineligible for Other Leukemia Protocols

Read the detailed description

The Study Drugs:

Azacitidine is designed to block certain genes in cancer cells whose job is to stop the function of the tumor-fighting genes. By blocking the "bad" genes, the tumor-fighting genes may be able to work better.

Vorinostat is designed to cause chemical changes in different groups of proteins that are attached to DNA (the genetic material of cells), which may slow the growth of cancer cells or cause the cancer cells to die.

Study Groups:

If you are found to be eligible to take part in this study, you will be randomly assigned (as in the flip of a coin) to 1 of 2 groups.

If you are in Group 1, you will receive azacitidine and vorinostat.
If you are in Group 2, you will receive azacitidine alone.

For the first 40 patients, you will have an equal chance of being in either group. After the first 40 patients, you will have a higher chance of being assigned to a group based on the results from previous participants.

Study Drug Administration:

On Days 1-5 of every cycle, you will receive azacitidine by vein over 15-30 minutes.

If you are in Group 1, you will also take vorinostat by mouth 3 times a day with food on Days 1-5 of every cycle.

If you cannot take vorinostat by mouth during a cycle, you will receive only azacitidine during that cycle. You will begin taking vorinostat by mouth again when you are able.

Your dose of study drugs may be lowered if you experience side effects.

You may receive a drug such as ondansetron before each dose of azacitidine to prevent nausea and vomiting.

If you have diarrhea, you will take a drug such as Imodium (loperamide) to prevent diarrhea.

Study Visits:

Once a week of Cycle 1, the following tests and procedures will be performed:

Your complete medical history will be recorded.
You will have a physical exam.
You will be asked if you have experienced any intolerable side effects.
Blood (about 1-2 tablespoons) will be drawn for routine tests.

On Day 28 of Cycle 1 (+/- 3 days), you will have bone marrow aspiration to check the status of the disease. To collect a bone marrow aspirate, an area of the hip is numbed with anesthetic, and a small amount of bone marrow is withdrawn through a large needle.

If the doctor thinks it is needed, you will have extra bone marrow aspirations during the later cycles to check the status of the disease.

One (1) time each cycle of Cycles 2 and beyond, the following tests and procedures will be performed:

Your complete medical history will be recorded.
You will have a physical exam.
You will be asked if you have experienced any intolerable side effects.
If the doctor thinks it is needed, blood (about 2 tablespoons) will be drawn for routine tests.

Length of Study:

You will be on active study for up to 12 cycles (about 12-18 months). You will be taken off study if the disease gets worse or you experience intolerable side effects.

This is an investigational study. Vorinostat is FDA approved and commercially available for the treatment of cutaneous T-cell lymphoma. Azacitidine is FDA approved commercially available for the treatment of MDS. The combination of these drugs for use in patients with intermediate-1 or higher risk MDS and AML is investigational.

Up to 80 participants will take part in this study. All will be enrolled at MD Anderson.

Conditions: Leukemia
Being tested: Vorinostat, Azacitidine
Keywords: Leukemia, Acute Myelogenous Leukemia, AML, Myelodysplastic Syndrome, MDS, Vorinostat, SAHA, Suberoylanilide Hydroxamic Acid, MSK-390, Zolinza, Azacitidine, 5-Azacitidine, 5-Aza, Vidaza, 5-AZC, AZA-CR, Ladakamycin, NSC-102816
Medical subject headings: Leukemia, Leukemia, Myeloid, Acute, Myelodysplastic Syndromes

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

VorinostatDrug
Also called SAHA, Suberoylanilide Hydroxamic Acid, MSK-390, Zolinza
200 mg by mouth three (3) times per day with food for 5 days (Days 1 - 5)
AzacitidineDrug
Also called 5-Azacitidine, 5-Aza, Vidaza, 5-AZC
75 mg/m^2/day given intravenously over 15 - 30 minutes daily for 5 days (Days 1 - 5)

Study groups in NCT00948064
Study group	Role	What they receive
Vorinostat with Azacitidine	Experimental	ARM A: Azacitidine 75 mg/m^2/day by vein over 15 - 30 minutes daily for 5 days (Days 1 - 5). Vorinostat 200 mg by mouth three time a day with food for 5 days (Days 1 - 5). Courses repeated every 3 to 8 weeks. Receives: Drug: Vorinostat; Drug: Azacitidine
Azacitidine	Experimental	ARM B: Azacitidine 75 mg/m^2 /day by vein over 15 - 30 minutes daily for 5 days (Days 1 - 5). Courses repeated every 3 to 8 weeks. Receives: Drug: Azacitidine

What is being measured

The primary outcome is the main question the study is designed to answer.

Survival at Day 60
Assessment of survival for outcome done on 60 days following therapy and includes participants alive for at least 60 days. Survival is calculated from start of therapy until death from any cause.
Measured: Phase I, Baseline to 60 days following first treatment.
Response Rate
Number of participants with Complete Response (CR) in AML requiring disappearance of all signs and symptoms related to disease, normalization of peripheral counts (absolute neutrophil count 10^9/L or more, platelet count 100 x 10^9/L or more), and a marrow with 5% or less marrow blasts; a hematologic improvement (HI) defined as a CR except for a platelet count increase by 50% to above 30 x 10^9/L. For MDS, the International Working Group criteria used to assess response.
Measured: 12-18 Months
Survival at Day 60
Assessment of survival for outcome done on 60 days following therapy and includes participants alive for at least 60 days. Survival is calculated from start of therapy until death from any cause.
Measured: Phase II, Baseline to 60 days following first treatment.

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

8 Sep 20093 Aug 2017

First posted29 Jul 2009 (estimated)
Study start8 Sep 2009
Primary completionApr 2015
Study completion3 Aug 2017

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.
Publications (1)Papers the sponsor has linked to this study.
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: Randomised
Intervention model: Parallel groups
Masking: None (open label)
Enrolment: 110 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT00948064
Sponsor's protocol ID: 2007-0685
Other IDs: NCI-2009-01495
Status last verified: Feb 2020
First submitted: 28 Jul 2009
Last updated: 24 Feb 2020
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: No
FDA-regulated drug: Yes
FDA-regulated device: No

Publications

Montalban-Bravo G, Huang X, Naqvi K, Jabbour E, Borthakur G, DiNardo CD, Pemmaraju N, Cortes J, Verstovsek S, Kadia T, Daver N, Wierda W, Alvarado Y, Konopleva M, Ravandi F, Estrov Z, Jain N, Alfonso A, Brandt M, Sneed T, Chen HC, Yang H, Bueso-Ramos C, Pierce S, Estey E, Bohannan Z, Kantarjian HM, Garcia-Manero G. A clinical trial for patients with acute myeloid leukemia or myelodysplastic syndromes not eligible for standard clinical trials. Leukemia. 2017 Feb;31(2):318-324. doi: 10.1038/leu.2016.303. Epub 2016 Oct 31.
PubMed 27795561

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

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Study at a glance

From the registry record, last updated by the sponsor on 24 Feb 2020. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: A Phase 2 Trial of Vorinostat in Combination With Azacitidine in Patients With Newly-Diagnosed Acute Myelogenous Leukemia (AML) or Myelodysplastic Syndrome (MDS) Who Are Ineligible for Other Leukemia Protocols
ClinicalTrials.gov ID: NCT00948064
Acronym / sponsor ID: 2007-0685
Conditions: Leukemia
Keywords: Leukemia, Acute Myelogenous Leukemia, AML, Myelodysplastic Syndrome, MDS, Vorinostat, SAHA, Suberoylanilide Hydroxamic Acid
Phase: Phase 2
Study type: Interventional
Recruitment status: Completed
Enrolment: 110 participants (actual)
Lead sponsor: M.D. Anderson Cancer CenterUniversity, hospital or other
Collaborators: Merck Sharp & Dohme LLC, Celgene Corporation
Study start: 8 Sep 2009 (actual)
Primary completion: Apr 2015 (actual)
Study completion: 3 Aug 2017 (actual)
First posted: 29 Jul 2009 (estimated)
Results: Posted 2 Jul 2015

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Detailed description

The Study Drugs:

Study Groups:

If you are found to be eligible to take part in this study, you will be randomly assigned (as in the flip of a coin) to 1 of 2 groups.

If you are in Group 1, you will receive azacitidine and vorinostat.
If you are in Group 2, you will receive azacitidine alone.

Study Drug Administration:

On Days 1-5 of every cycle, you will receive azacitidine by vein over 15-30 minutes.

If you are in Group 1, you will also take vorinostat by mouth 3 times a day with food on Days 1-5 of every cycle.

If you cannot take vorinostat by mouth during a cycle, you will receive only azacitidine during that cycle. You will begin taking vorinostat by mouth again when you are able.

Your dose of study drugs may be lowered if you experience side effects.

You may receive a drug such as ondansetron before each dose of azacitidine to prevent nausea and vomiting.

If you have diarrhea, you will take a drug such as Imodium (loperamide) to prevent diarrhea.

Study Visits:

Once a week of Cycle 1, the following tests and procedures will be performed:

Your complete medical history will be recorded.
You will have a physical exam.
You will be asked if you have experienced any intolerable side effects.
Blood (about 1-2 tablespoons) will be drawn for routine tests.

If the doctor thinks it is needed, you will have extra bone marrow aspirations during the later cycles to check the status of the disease.

One (1) time each cycle of Cycles 2 and beyond, the following tests and procedures will be performed:

Your complete medical history will be recorded.
You will have a physical exam.
You will be asked if you have experienced any intolerable side effects.
If the doctor thinks it is needed, blood (about 2 tablespoons) will be drawn for routine tests.

Length of Study:

You will be on active study for up to 12 cycles (about 12-18 months). You will be taken off study if the disease gets worse or you experience intolerable side effects.

Up to 80 participants will take part in this study. All will be enrolled at MD Anderson.

Study design

Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: None (open label)
Number of arms: 2

Arm	Type	Receives
Vorinostat with Azacitidine	Experimental	Drug: Vorinostat, Drug: AzacitidineARM A: Azacitidine 75 mg/m^2/day by vein over 15 - 30 minutes daily for 5 days (Days 1 - 5). Vorinostat 200 mg by mouth three time a day with food for 5 days (Days 1 - 5). Courses repeated every 3 to 8 weeks.
Azacitidine	Experimental	Drug: AzacitidineARM B: Azacitidine 75 mg/m^2 /day by vein over 15 - 30 minutes daily for 5 days (Days 1 - 5). Courses repeated every 3 to 8 weeks.

Who may qualify

Ages: 18 years and older
Sex: All
Healthy volunteers: Not accepted
Age groups: Adult, Older adult

Key inclusion criteria (6 listed)

Patients with newly diagnosed AML or MDS (Intermediate 1 or higher risk)
Patient must have at least one of the following: a. Creatinine >/= 2 mg/dL; b. total Bilirubin >/= 2 mg/dL; c.ECOG Performance Status equal to 3 or 4; and d. is ineligible for participation on a protocol of higher priority
Patients must provide written informed consent.
Patients must be age > 18 years due to lack of safety information with these agents in children.
Patient agrees to: 1) Use 2 adequate methods of contraception to prevent pregnancy (either 2 barrier methods or a barrier method plus a hormonal contraceptive method) or 2) abstain from heterosexual activity throughout the study starting with Visit 1.

Key exclusion criteria (8 listed)

Patients must not have the favorable cytogenetic abnormalities of inv (16), t (16;16), t (8;21), or t (15;17).
Patients receiving any anti-leukemic therapy with the exception of Hydroxyurea prior to study enrollment. Prior growth factor therapy is acceptable. Hydroxyurea could be used at the discretion of the treating physician. A single or a two day dose of cytarabine (up to 3 g/m^2) for emergency use is allowed as prior therapy.
Patient has a prior history of treatment with HDAC inhibitors. Patients who have received valproic acid (VPA) for the treatment of seizures may be enrolled on this study, but must not have received VPA within 30 days of study enrollment.
Patient is unable to take and/or tolerate oral medications on a continuous basis, examples include patients on a ventilator, or have altered mental status that precludes safe oral route of administration.
Patient has active hepatitis A, B, or C infection.

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
VorinostatAlso: SAHA, Suberoylanilide Hydroxamic Acid, MSK-390, Zolinza200 mg by mouth three (3) times per day with food for 5 days (Days 1 - 5)	Drug	Vorinostat with Azacitidine
AzacitidineAlso: 5-Azacitidine, 5-Aza, Vidaza, 5-AZC75 mg/m^2/day given intravenously over 15 - 30 minutes daily for 5 days (Days 1 - 5)	Drug	Azacitidine, Vorinostat with Azacitidine

Primary outcome

Survival at Day 60
Time frame: Phase I, Baseline to 60 days following first treatment.
Assessment of survival for outcome done on 60 days following therapy and includes participants alive for at least 60 days. Survival is calculated from start of therapy until death from any cause.
Response Rate
Time frame: 12-18 Months
Number of participants with Complete Response (CR) in AML requiring disappearance of all signs and symptoms related to disease, normalization of peripheral counts (absolute neutrophil count 10^9/L or more, platelet count 100 x 10^9/L or more), and a marrow with 5% or less marrow blasts; a hematologic improvement (HI) defined as a CR except for a platelet count increase by 50% to above 30 x 10^9/L. For MDS, the International Working Group criteria used to assess response.
Survival at Day 60
Time frame: Phase II, Baseline to 60 days following first treatment.
Assessment of survival for outcome done on 60 days following therapy and includes participants alive for at least 60 days. Survival is calculated from start of therapy until death from any cause.

Results and safety

Results posted to the registry on 2 Jul 2015, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Vorinostat With Azacitidine, Phase I	31	30
Vorinostat With Azacitidine, Phase II	0	0
Azacitidine, Phase II	0	0

Primary outcome: Survival at Day 60

Time frame: Phase I, Baseline to 60 days following first treatment.. Number. Unit: participants

Measure	Vorinostat With Azacitidine, Phase I n = 30
Value	24

Population analysed: Of the 31 enrolled participants, 30 were evaluable and 1 participant never received treatment.

Primary outcome: Response Rate

Time frame: 12-18 Months. Number. Unit: participants

Measure	Vorinostat With Azacitidine, Phase I n = 30	Vorinostat With Azacitidine, Phase II n = 51	Azacitidine, Phase II n = 26
Complete Response	9	11	8
Hematologic Improvement	0	1	1
No Response	21	39	17
Not Evaluable	0	1	1

Population analysed: Out of 79 participants enrolled in Phase II, 2 participants were not evaluable - 1 participant was removed from study per treating physician discretion and the other was removed per participant's request.

Primary outcome: Survival at Day 60

Time frame: Phase II, Baseline to 60 days following first treatment.. Number. Unit: participants

Measure	Vorinostat With Azacitidine, Phase II n = 51	Azacitidine, Phase II n = 27
Value	43	18

Population analysed: Of the 79 participants enrolled, 78 were evaluable and 1 participant withdrew from study.

Adverse events

Time frame: Adverse event collected when occur within 30 days of discontinuation of dosing or completion of participation in study if the last scheduled visit occurs at a later time. Overall study period: January 2009 to April 2015.. Other adverse events reported at 0% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Vorinostat With Azacitidine, Phase I	15 of 30 (50%)	21 of 30 (70%)	Not reported
Vorinostat With Azacitidine, Phase II	36 of 52 (69.2%)	45 of 52 (86.5%)	Not reported
Azacitidine, Phase II	18 of 27 (66.7%)	26 of 27 (96.3%)	Not reported

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 24 Feb 2020.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Derived from this study: PubMed 27795561Montalban-Bravo G, Huang X, Naqvi K, Jabbour E, Borthakur G, DiNardo CD, Pemmaraju N, Cortes J, Verstovsek S, Kadia T, Daver N, Wierda W, Alvarado Y, Konopleva M, Ravandi F, Estrov Z, Jain N, Alfonso A, Brandt M, Sneed T, Chen HC, Yang H, Bueso-Ramos C, Pierce S, Estey E, Bohannan Z, Kantarjian HM, Garcia-Manero G. A clinical trial for patients with acute myeloid leukemia or myelodysplastic syndromes not eligible for standard clinical trials. Leukemia. 2017 Feb;31(2):318-324. doi: 10.1038/leu.2016.303. Epub 2016 Oct 31.
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.

Guillermo Garcia-Manero, 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 Vorinostat and Azacitidine 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 8 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 24 Feb 2020. 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 2 Jul 2015). 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?

Survival at Day 60 (time frame: Phase I, Baseline to 60 days following first treatment.). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT00948064, which also lists 1 linked publication.

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 24 Feb 2020.