Azacitidine in Treating Patients With Myelofibrosis

NCT00381693 | Terminated Phase 2 Results DMC

• 4 other identifiers: CDR0000503972P30CA015083MC058D05-004297
• Study Type: interventional
• Target: 10
• Locations: 1 country
• Sites: 2

Brief 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.

Conditions

Chronic Myeloproliferative Disorders; Secondary Myelofibrosis

Keywords

primary myelofibrosis; polycythemia vera; essential thrombocythemia; secondary myelofibrosis

Outcome Measures

Primary Outcomes (1)

• 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.

  4 months

Secondary Outcomes (3)

• Overall Survival (OS)

  From date of registration until death or 3 years after registration if patient is still alive

• Time to Progression

  up to 3 years

• Number of Participants With Treatment Related Adverse Events

  Every 4 weeks during treatment

Interventions

azacitidine DRUG

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

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

Contact the study team to discuss eligibility requirements. They can help determine if this study is right for you.

Sponsors & Collaborators

• Mayo Clinic: lead
• National Cancer Institute (NCI): collaborator

Study Sites (2)

Mayo Clinic in Arizona

Scottsdale, Arizona, United States

Location

Mayo Clinic

Rochester, Minnesota, 55905, United States

Location

MeSH Terms

Conditions

Myeloproliferative Disorders; Primary Myelofibrosis; Polycythemia Vera; Thrombocythemia, Essential

Interventions

Azacitidine

Condition Hierarchy (Ancestors)

Bone Marrow Diseases; Hematologic Diseases; Hemic and Lymphatic Diseases; Bone Marrow Neoplasms; Hematologic Neoplasms; Neoplasms by Site; Neoplasms; Blood Coagulation Disorders; Thrombocytosis; Blood Platelet Disorders; Hemorrhagic Disorders

Intervention Hierarchy (Ancestors)

Aza Compounds; Organic Chemicals; Cytidine; Pyrimidine Nucleosides; Pyrimidines; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Nucleosides; Nucleic Acids, Nucleotides, and Nucleosides; Ribonucleosides

Results Point of Contact

• Title: Dr. Ruben A. Mesa
• Organization: Mayo Clinic

mesa.ruben@mayo.edu

507-284-3533

Study Officials

• Ruben A. Mesa, MD

  Mayo Clinic

  STUDY CHAIR

Publication Agreements

• PI is Sponsor Employee: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Masking: NONE
• Purpose: TREATMENT
• Sponsor Type: OTHER

Study Record Dates

• First Submitted: September 26, 2006
• First Posted: September 28, 2006
• Study Start: August 1, 2006
• Primary Completion: March 1, 2008
• Study Completion: April 1, 2009
• Last Updated: April 21, 2011
• Results First Posted: January 24, 2011

Record last verified: 2011-04

Locations

US (2)