Chronic Myeloproliferative DisordersPhase 2NCT00381693
Azacitidine in Treating Patients With Myelofibrosis
A Phase 2 study testing azacitidine in people with Chronic Myeloproliferative Disorders and Secondary Myelofibrosis, run by Mayo Clinic at 2 sites in United States.
Trial summary
RATIONALE: Drugs used in chemotherapy, such as azacitidine, work in different ways to stop the growth of abnormal cells, either by killing the cells or by stopping them from dividing. PURPOSE: This phase II trial is studying how well azacitidine works in treating patients with myelofibrosis. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Chronic Myeloproliferative Disorders, Secondary Myelofibrosis
- What is being tested?
- azacitidine
- How many people
- 10 participants
- Where
- 2 sitesUnited States
- Study dates
- Aug 2006to Apr 2009
- 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
DISEASE CHARACTERISTICS:
- Histologically confirmed myelofibrosis with myeloid metaplasia (MMM), including any of the following subtypes:
- Agnogenic myeloid metaplasia
- Post-polycythemic myeloid metaplasia
- Post-thrombocythemic myeloid metaplasia
- Evaluable and symptomatic disease, defined as 1 of the following:
- Anemia (hemoglobin < 10 g/dL or erythrocyte transfusion-dependent, requiring 1 transfusion ≤ 8 weeks)
- Treatment required* for symptomatic palpable splenomegaly (palpable hepatomegaly is acceptable if previously splenectomized) NOTE: *Subjective but painful enough to mandate intervention
- Absence of t(9;22) by fluorescent in situ hybridization (FISH) or standard cytogenetics (by peripheral blood or marrow)
- Previous demonstration of a lack of this translocation (at any point) is sufficient
- No advanced malignant hepatic tumors
PATIENT CHARACTERISTICS:
- ECOG performance status 0-2
- Absolute neutrophil count ≥ 1,000/mm³
- Platelet count ≥ 50,000/mm³
- Creatinine ≤ 2.0 mg/dL
- Total bilirubin ≤ 2.0 mg/dL OR direct bilirubin ≤ 2.0 mg/dL if total bilirubin elevated (unless attributed to underlying disease)
- AST and ALT ≤ 2 times upper limit of normal (unless clinically attributed to hepatic extramedullary hematopoiesis)
- Not pregnant or nursing
- Negative pregnancy test
- Fertile patients must use effective contraception
- No baseline peripheral or autonomic neuropathy ≥ grade 2
- No condition, including the presence of laboratory abnormalities, that would preclude study compliance
- No hypersensitivity to mannitol or azacitidine
- Not incarcerated in a municipality (i.e., county, state, or federal prison)
PRIOR CONCURRENT THERAPY:
- At least 14 days since prior chemotherapy, including interferon alfa, anagrelide, or other myelosuppressive agents
- At least 14 days since prior systemic corticosteroids
- At least 14 days since prior investigational agents
Trial locations and contacts
2 sites in 1 country.
United States2 sites
Mayo Clinic
Rochester, Minnesota, 55905
Mayo Clinic in Arizona
Scottsdale, Arizona
About this study
Official title: Phase II Study of Azacitidine in Myelofibrosis
RATIONALE: Drugs used in chemotherapy, such as azacitidine, work in different ways to stop the growth of abnormal cells, either by killing the cells or by stopping them from dividing.
PURPOSE: This phase II trial is studying how well azacitidine works in treating patients with myelofibrosis.
Read the detailed description
OBJECTIVES:
Primary
- Determine the efficacy of azacitidine in patients with myelofibrosis (MF) with myeloid metaplasia.
- Evaluate the safety of azacitidine in these patients. Secondary
- Evaluate pertinent biologic characteristics of MF before and during therapy with azacitidine.
- Assess the effects of study treatment on constitutional symptoms in these patients.
- Estimate time to event distributions for overall survival and progression. OUTLINE: Patients receive azacitidine subcutaneously once daily on days 1-5. Treatment repeats every 4 weeks for up to 12 courses in the absence of disease progression or unacceptable toxicity.
After completion of study treatment, patients are followed periodically for up to 3 years.
PROJECTED ACCRUAL: A total of 35 patients will be accrued for this study.
- Being tested
- azacitidine
- Keywords
- primary myelofibrosis, polycythemia vera, essential thrombocythemia, secondary myelofibrosis
- Medical subject headings
- Myeloproliferative Disorders, Primary Myelofibrosis, Polycythemia Vera, Thrombocythemia, Essential
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
azacitidineDrug
What is being measured
The primary outcome is the main question the study is designed to answer.
Patients With Confirmed Response (Complete Remission or Partial Remission on 2 Consecutive Evaluation at Least 4 Weeks Apart) During the First 4 Months of Treatment
Response Definitions:
- Completion Remission (CR):complete resolution of disease-related symptoms, ultrasound-documented resolution of hepastosplenomegaly, normalization of the peripheral blood count, white cell differential, and smear, normalization of bone marrow histology including disappearance of fibrosis and osteosclerosis. Residual cytogenetic abnormalities are allowed.
- Partial Remission (PR): a major response in any baseline applicable criteria (except constitutional symptoms) without progression in any other category.
Measured: 4 months
Secondary outcomes (3)
Overall Survival (OS)
OS was defined as the time from registration to death due to any cause or time from registration to 3 years after registration if patient is still alive.
Measured: From date of registration until death or 3 years after registration if patient is still alive
Time to Progression
Time to progression was defined as the time from registration to progression of disease. Those who die without documentation of disease progression will be considered to have had disease progression at the time of their death unless documented evidence clearly indicates no progression has occured.
Measured: up to 3 years
Number of Participants With Treatment Related Adverse Events
Adverse events (AE) that are classified as either possibly, probably, or definitely related to study treatment according to the National Cancer Institute Common Toxicity Criteria for Adverse Events (NCI CTCAE version 3.0). The maximum grade for each type of AE will be recorded for each patient. Grade refers to the severity of the AE.
Grade 1: Mild AE, Grade 2: Moderate AE, Grade 3: Severe AE, Grade 4: Life-threatening or disabling AE, Grade 5: Death related AE
Measured: Every 4 weeks during treatment
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Sep 2006 (estimated)
- Study startAug 2006
- Primary completionMar 2008
- Study completionApr 2009
Why it stopped: Due to lack of accrual and trial has demonstrated too little clinical benefit
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 Chronic Myeloproliferative DisordersStudies of the same condition, open and under way.
- More trials run by Mayo ClinicThe 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
- Masking
- None (open label)
- Enrolment
- 10 participants (actual)
Sponsor and investigators
- Lead sponsor
- Mayo Clinic
- Sponsor type
- University, hospital or other
- Collaborators
- National Cancer Institute (NCI)
- Investigators
- Ruben A. Mesa, MD, Study chair, Mayo Clinic
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00381693
- Sponsor's protocol ID
- CDR0000503972
- Other IDs
- P30CA015083, MC058D, 05-004297
- Status last verified
- Apr 2011
- First submitted
- 26 Sep 2006
- Last updated
- 21 Apr 2011
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 21 Apr 2011. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase II Study of Azacitidine in Myelofibrosis
- ClinicalTrials.gov ID
- NCT00381693
- Acronym / sponsor ID
- CDR0000503972
- Keywords
- primary myelofibrosis, polycythemia vera, essential thrombocythemia, secondary myelofibrosis
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Due to lack of accrual and trial has demonstrated too little clinical benefit
- Enrolment
- 10 participants (actual)
- Lead sponsor
- Mayo ClinicUniversity, hospital or other
- Collaborators
- National Cancer Institute (NCI)
- Study start
- Aug 2006
- Primary completion
- Mar 2008 (actual)
- Study completion
- Apr 2009 (actual)
- First posted
- 28 Sep 2006 (estimated)
- Results
- Posted 24 Jan 2011
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 azacitidine, work in different ways to stop the growth of abnormal cells, either by killing the cells or by stopping them from dividing.
PURPOSE: This phase II trial is studying how well azacitidine works in treating patients with myelofibrosis.
Detailed description
OBJECTIVES:
Primary
- Determine the efficacy of azacitidine in patients with myelofibrosis (MF) with myeloid metaplasia.
- Evaluate the safety of azacitidine in these patients. Secondary
- Evaluate pertinent biologic characteristics of MF before and during therapy with azacitidine.
- Assess the effects of study treatment on constitutional symptoms in these patients.
- Estimate time to event distributions for overall survival and progression. OUTLINE: Patients receive azacitidine subcutaneously once daily on days 1-5. Treatment repeats every 4 weeks for up to 12 courses in the absence of disease progression or unacceptable toxicity.
After completion of study treatment, patients are followed periodically for up to 3 years.
PROJECTED ACCRUAL: A total of 35 patients will be accrued for this study.
Study design
- Primary purpose
- Treatment
- Masking
- None (open label)
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
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 |
|---|---|---|
| azacitidine | Drug | – |
Primary outcome
Patients With Confirmed Response (Complete Remission or Partial Remission on 2 Consecutive Evaluation at Least 4 Weeks Apart) During the First 4 Months of Treatment
Time frame: 4 months
Response Definitions: * Completion Remission (CR):complete resolution of disease-related symptoms, ultrasound-documented resolution of hepastosplenomegaly, normalization of the peripheral blood count, white cell differential, and smear, normalization of bone marrow histology including disappearance of fibrosis and osteosclerosis. Residual cytogenetic abnormalities are allowed. * Partial Remission (PR): a major response in any baseline applicable criteria (except constitutional symptoms) without progression in any other category.
3 secondary outcomes
Overall Survival (OS)
Time frame: From date of registration until death or 3 years after registration if patient is still alive
Time to Progression
Time frame: up to 3 years
Number of Participants With Treatment Related Adverse Events
Time frame: Every 4 weeks during treatment
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 21 Apr 2011.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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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.
Ruben A. Mesa, MD study chair
Mayo Clinic
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing azacitidine in people with Chronic Myeloproliferative Disorders and Secondary Myelofibrosis, run by Mayo Clinic at 2 sites 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?
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: stopped early, as of the sponsor's last update on 21 Apr 2011. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites 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 24 Jan 2011). 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?
Patients With Confirmed Response (Complete Remission or Partial Remission on 2 Consecutive Evaluation at Least 4 Weeks Apart) During the First 4 Months of Treatment (time frame: 4 months). 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 21 Apr 2011.