Myelodysplastic SyndromesPhase 1NCT01913951
Vosaroxin and Azacitidine in Treating Patients With Myelodysplastic Syndromes
A Phase 1 study testing vosaroxin and Azacitidine in people with Myelodysplastic Syndromes, run by Washington University School of Medicine at 1 site in United States.
Trial summary
This phase I trial studies the side effects and the best dose of vosaroxin when given together with azacitidine in treating patients with myelodysplastic syndromes. Drugs used in chemotherapy, such as vosaroxin and azacitidine, work in different ways to stop the growth of cancer cells, either by killing the cells or… 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?
- vosaroxin and Azacitidine
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 35 participants
- Where
- 1 siteUnited States
- Study dates
- 22 Nov 2013to 29 Apr 2024
- 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
- Diagnosis of myelodysplastic syndrome and one of the following:
- Cytopenias requiring red blood cell and/or platelet transfusions or neutropenia (ANC <1 X109/L)
- IPSS score of INT-1 or higher at screening
- MDS with excess blasts in transformation as defined by FAB criteria (20-29% bone marrow blasts) or
- Chronic myelomonocytic leukemia
- Age ≥18 years old
Show all 15Show fewer
- Adequate renal and hepatic function defined as all of the following:
- total bilirubin ≤ 2.0 mg/dl, except in cases of Gilbert's disease;
- AST and ALT ≤2.5 institutional ULN;
- serum creatinine within normal institutional limits or estimated creatinine clearance ≥60 mL/min/1.73 m2 by the Cockcroft-Gault equation
- ECOG performance status ≤2
- Ability to understand and willingness to sign an IRB approved written informed consent document (or that of legally authorized representative, if applicable).
- Females must be surgically or biologically sterile or postmenopausal or if of childbearing potential, must agree to use an adequate method of contraception during the study until 30 days after the last treatment. Males must be surgically or biologically sterile or agree to use an adequate method of contraception during the study until 30 days after the last treatment. Should a woman become pregnant or suspect she is pregnant while participating in this study, she must inform her treating physician immediately.
- Patients may have received up to 3 prior cycles of hypomethylator therapy (i.e. decitabine or azacitidine) prior to enrollment and may have received supportive care measures (growth factors, erythropoietin stimulating agents, transfusion, etc.
- Either enrolled in HRPO# 201011766 ("Tissue Acquisition for Analysis of Genetic Progression Factors in Hematologic Diseases"), which facilitates collection of blood, bone marrow, and skin for correlative studies, or consents to collection of blood, bone marrow, and skin as part of this protocol.
Who cannot join
- Prior treatment with four or more cycles of hypomethylator therapy.
- Receiving concomitant chemotherapy, radiation therapy, or immunotherapy during the duration of treatment on protocol.
- Known seropositivity for or active viral infection with human immunodeficiency virus (HIV), hepatitis B virus (HBV), or hepatitis C virus (HCV). Patients who are seropositive because of hepatitis B virus vaccine are eligible. Patients who are seropositive for HCV, but have a negative viral load are also eligible. Documentation that the patients have completed a course of therapy for HCV is required and will be obtained.
- Uncontrolled intercurrent illness including, but not limited to, ongoing or active infection, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, or psychiatric illness/social situations that would limit compliance with study requirements
- Pregnant and/or breastfeeding.
Trial locations and contacts
1 site in 1 country.
United States1 site
Washington University School of Medicine
St Louis, Missouri, 63110
About this study
Official title: A Phase I Study of Vosaroxin Plus Azacitidine for Patients With Myelodysplastic Syndrome
This phase I trial studies the side effects and the best dose of vosaroxin when given together with azacitidine in treating patients with myelodysplastic syndromes. Drugs used in chemotherapy, such as vosaroxin and azacitidine, work in different ways to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing.
- Conditions
- Myelodysplastic Syndromes
- Being tested
- vosaroxin, Azacitidine
- Medical subject headings
- Myelodysplastic Syndromes
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
vosaroxinDrug
Also called voreloxin
Given IV over 10 minutes on Day 1 and 4
AzacitidineDrug
Also called Vidaza, Ladakamycin
Given SC or IV over 15 minutes on days 1-7
| Study group | Role | What they receive |
|---|---|---|
| Treatment (vosaroxin, azacitidine) | Experimental | Patients receive vosaroxin IV over 10 minutes on days 1 and 4 and azacitidine SC or IV over 15 minutes on days 1-7. Treatment repeats every 28 days for up to 6 cycles in the absence of disease progression or unacceptable toxicity. Receives: Drug: vosaroxin; Drug: Azacitidine |
What is being measured
The primary outcome is the main question the study is designed to answer.
MTD of vosaroxin in combination with azacitidine
Defined as the highest dose of vosaroxin that results in a DLT in =< 1 of 6 patients graded according to the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) 4.0
Measured: 28 days
Secondary outcomes (9)
Best response (including hematologic improvement)
According to the modified IWG criteria. Summarized as proportion with 95% confidence intervals.
Measured: At 3 cycles
Best overall response
According to the modified IWG criteria. Summarized as proportion with 95% confidence intervals
Measured: Up to 7 months
Incidence of adverse events
Graded according to NCI CTCAE v. 4.0. summarized by grade, type and patient.
Measured: Up to 7 months
Time to response
According to the modified IWG criteria. Described using Kaplan-Meier models to plot these endpoints and estimate median times with a 95% confidence interval.
Measured: Up to 7 months
Event-free survival
From the date of first dose of study drug to date of treatment failure, recurrence, or death due to any cause. Described using Kaplan-Meier models to plot these endpoints and estimate median times with a 95% confidence interval.
Measured: up to 5 years
Progression-free survival (PFS)
From the date of first dose of study drug to disease progression or death from MDS. Described using Kaplan-Meier models to plot these endpoints and estimate median times with a 95% confidence interval.
Measured: up to 5 years
Disease-free survival (DFS)
From the date of first documentation of a complete remission (CR) to date of relapse. Described using Kaplan-Meier models to plot these endpoints and estimate median times with a 95% confidence interval.
Measured: up to 5 years
Overall survival (OS)
Date of first dose of study drug to the date of death from any cause. Described using Kaplan-Meier models to plot these endpoints and estimate median times with a 95% confidence interval.
Measured: up to 5 years
Biomarkers of response to vosaroxin and azacitidine therapy
Serial estimate of biomarker expression will be plotted and summarized over time using means and standard deviations, possibly on a log scale
Measured: Up to 5 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Aug 2013 (estimated)
- Study start22 Nov 2013
- Primary completion5 Dec 2016
- Study completion29 Apr 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Myelodysplastic SyndromesStudies of the same condition, open and under way.
- More trials run by Washington University School of MedicineThe 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
- 35 participants (actual)
Sponsor and investigators
- Lead sponsor
- Washington University School of Medicine
- Sponsor type
- University, hospital or other
- Collaborators
- Sunesis Pharmaceuticals
- Responsible party
- Sponsor
- Investigators
- Meagan Jacoby, M.D., Ph.D., Principal investigator, Washington University School of Medicine
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01913951
- Sponsor's protocol ID
- 201309091
- Status last verified
- Apr 2024
- First submitted
- 30 Jul 2013
- Last updated
- 2 May 2024
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Lancet JE, Ravandi F, Ricklis RM, Cripe LD, Kantarjian HM, Giles FJ, List AF, Chen T, Allen RS, Fox JA, Michelson GC, Karp JE. A phase Ib study of vosaroxin, an anticancer quinolone derivative, in patients with relapsed or refractory acute leukemia. Leukemia. 2011 Dec;25(12):1808-14. doi: 10.1038/leu.2011.157. Epub 2011 Jul 15.
PubMed 21760592
Study at a glance
From the registry record, last updated by the sponsor on 2 May 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase I Study of Vosaroxin Plus Azacitidine for Patients With Myelodysplastic Syndrome
- ClinicalTrials.gov ID
- NCT01913951
- Acronym / sponsor ID
- 201309091
- Conditions
- Myelodysplastic Syndromes
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 35 participants (actual)
- Lead sponsor
- Washington University School of MedicineUniversity, hospital or other
- Collaborators
- Sunesis Pharmaceuticals
- Study start
- 22 Nov 2013 (actual)
- Primary completion
- 5 Dec 2016 (actual)
- Study completion
- 29 Apr 2024 (actual)
- First posted
- 1 Aug 2013 (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 phase I trial studies the side effects and the best dose of vosaroxin when given together with azacitidine in treating patients with myelodysplastic syndromes. Drugs used in chemotherapy, such as vosaroxin and azacitidine, work in different ways to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Treatment (vosaroxin, azacitidine) | Experimental | Drug: vosaroxin, Drug: AzacitidinePatients receive vosaroxin IV over 10 minutes on days 1 and 4 and azacitidine SC or IV over 15 minutes on days 1-7. Treatment repeats every 28 days for up to 6 cycles in the absence of disease progression or unacceptable toxicity. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (15 listed)
- Diagnosis of myelodysplastic syndrome and one of the following:
- Age ≥18 years old
- Adequate renal and hepatic function defined as all of the following:
- ECOG performance status ≤2
- Ability to understand and willingness to sign an IRB approved written informed consent document (or that of legally authorized representative, if applicable).
Key exclusion criteria (5 listed)
- Prior treatment with four or more cycles of hypomethylator therapy.
- Receiving concomitant chemotherapy, radiation therapy, or immunotherapy during the duration of treatment on protocol.
- Known seropositivity for or active viral infection with human immunodeficiency virus (HIV), hepatitis B virus (HBV), or hepatitis C virus (HCV). Patients who are seropositive because of hepatitis B virus vaccine are eligible. Patients who are seropositive for HCV, but have a negative viral load are also eligible. Documentation that the patients have completed a course of therapy for HCV is required and will be obtained.
- Uncontrolled intercurrent illness including, but not limited to, ongoing or active infection, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, or psychiatric illness/social situations that would limit compliance with study requirements
- Pregnant and/or breastfeeding.
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 |
|---|---|---|
| vosaroxinAlso: voreloxinGiven IV over 10 minutes on Day 1 and 4 | Drug | Treatment (vosaroxin, azacitidine) |
| AzacitidineAlso: Vidaza, LadakamycinGiven SC or IV over 15 minutes on days 1-7 | Drug | Treatment (vosaroxin, azacitidine) |
Primary outcome
MTD of vosaroxin in combination with azacitidine
Time frame: 28 days
Defined as the highest dose of vosaroxin that results in a DLT in =< 1 of 6 patients graded according to the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) 4.0
9 secondary outcomes
Best response (including hematologic improvement)
Time frame: At 3 cycles
Best overall response
Time frame: Up to 7 months
Incidence of adverse events
Time frame: Up to 7 months
Time to response
Time frame: Up to 7 months
Event-free survival
Time frame: up to 5 years
Progression-free survival (PFS)
Time frame: up to 5 years
Disease-free survival (DFS)
Time frame: up to 5 years
Overall survival (OS)
Time frame: up to 5 years
Biomarkers of response to vosaroxin and azacitidine therapy
Time frame: Up to 5 years
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 2 May 2024.
- Background: PubMed 21760592Lancet JE, Ravandi F, Ricklis RM, Cripe LD, Kantarjian HM, Giles FJ, List AF, Chen T, Allen RS, Fox JA, Michelson GC, Karp JE. A phase Ib study of vosaroxin, an anticancer quinolone derivative, in patients with relapsed or refractory acute leukemia. Leukemia. 2011 Dec;25(12):1808-14. doi: 10.1038/leu.2011.157. Epub 2011 Jul 15.
- Alvin J. Siteman Cancer Center at Barnes-Jewish Hospital and Washington University School of MedicineLink provided by the sponsor.
- Individual participant dataSharing planned: No
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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.
Meagan Jacoby, M.D., Ph.D. principal investigator
Washington University School of Medicine
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 vosaroxin and Azacitidine in people with Myelodysplastic Syndromes, run by Washington University School of Medicine 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 15 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: finished, as of the sponsor's last update on 2 May 2024. 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?
MTD of vosaroxin in combination with azacitidine (time frame: 28 days). 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 NCT01913951, 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 2 May 2024.