A Phase II Study of Intravenous Azacitidine Alone in Patients With Myelodysplastic Syndromes

NCT00384956 | Completed Phase 2 Results DMC

• 1 other identifier: 06-0585
• Study Type: interventional
• Target: 25
• Locations: 1 country
• Sites: 1

Brief Summary

The primary endpoint of this study is to estimate morphologic complete remission rate. Estimation of response rate is also a secondary objection.

Conditions

Myelodysplastic Syndromes

Keywords

MDS either de novo or secondary, fitting any of the WHO classifications.

Outcome Measures

Primary Outcomes (1)

• Rate of Complete Remission (CR) and Partial Remission (PR)

  Defined according to the modified International Working Group (IWG) (2006) response criteria for myelodysplasia: CR=bone marrow with \<5% myeloblasts and 0% peripheral blasts, hemoglobin ≥11g/dL, platelets ≥ 100 x 10\^9/L, and neutrophils ≥1.0 x 10\^9/L. Residual dysplasia was allowed. PR= All of the CR criteria if abnormal before treatment except: bone marrow blasts decreased by ≥50% over pretreatment but still \>5%.

  After 4 cycles of therapy (up to 112 days after start of treatment)

Secondary Outcomes (5)

• Rate of Hematologic Improvement

  4 weeks following last azacitidine dose [median number of cycles 4.5 (1-20)]

• Rate of Transfusion Independence

  4 weeks following last azacitidine dose [median number of cycles 4.5 (1-20)]

• Rate of Cytogenetic Response

  2 years after first dose of study drug or until participant is lost to follow-up or dies

• Rate of Overall Survival

  2 years after first dose of study drug or until participant is lost to follow-up or dies

• Rate of Relapse After Hematopoietic Stem Cell Transplant in Individuals Treated With 5-azacitidine Prior to Transplant.

  2 years after first dose of study drug or until participant is lost to follow-up or dies

Study Arms (1)

Azacitidine

EXPERIMENTAL

Azacitidine 75 mg/m2 IV on days 1-5 of each 28 day cycle. Patients that do not respond after two cycles will have the dose increased to 100 mg/m2. Patients who achieve a CR will receive 3 additional 28 day cycles and then begin treatment on days 1-5 of a 56 day cycle. Individuals who demonstrate a loss of response will resume 28 day cycles.

Drug: Azacitidine

Interventions

Azacitidine DRUG

Also known as: 5-azacitidine, Vidaza

Azacitidine

Eligibility Criteria

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

You may qualify if:

• Pathological MDS either de novo or secondary, fitting any of the FAB classifications, confirmed by institutional pathologist within 2 weeks prior to start of treatment. Patients with 5% bone marrow blasts must also meet one of the following criteria:
• Symptomatic anemia with either hemoglobin less than 10.0 g/dL or requiring RBC transfusion
• Thrombocytopenia with a history of two or more platelet counts \< 50,000 / µL or a significant hemorrhage requiring platelet transfusions, or
• Neutropenia with two or more absolute neutrophil counts less than 1,000 /µL.
• ECOG performance status of 0-2.
• Must give written informed consent indicating their awareness of the investigational nature of this study and its potential hazards.
• Adequate renal and hepatic function (creatinine ≤ 150% of institutional upper limit of normal, total bilirubin ≤ 150% institutional upper limit of normal, AST ≤ 200% institutional upper limit of normal).
• Life expectancy of at least 12 weeks.
• Have not received any chemotherapy within 4 weeks of study enrollment and must have recovered from any treatment-related toxicities.
• Women of childbearing age must have a negative serum pregnancy test prior to initiating therapy.
• Sexually active women of childbearing potential must use effective birth control during the trial and for an appropriate period after the trial.
• Men must be willing to avoid fathering a new child while receiving therapy with azacitidine.
• ≥18 years, no upper age limit
• Individuals who are candidates for hematopoietic stem cell transplantation and who meet all other study criteria may participate in the study and receive intravenous azacitidine alone as a treatment prior to transplantation.

You may not qualify if:

• Known CNS leukemia.
• Previously received Azacitidine (Vidaza®, Pharmion Corp., Boulder CO) or decitabine (Dacogen®, MGI Pharma Inc. Bloomington, MN).
• Known or suspected hypersensitivity to azacitidine or mannitol.
• Receiving any other investigational agents within 30 days of first dose of study drug.
• Uncontrolled intercurrent illness including, but not limited to, ongoing or active infection, congestive heart failure of NYHA class 3 or 4, unstable angina pectoris, cardiac arrhythmia, or psychiatric illness/social situation that would limit compliance with study requirements.
• Known positive serology for HIV.
• Had radiotherapy within 14 days prior to study enrollment.
• Known presence of hepatic tumors.
• \<18 years of age
• Exclude women who are pregnant or breast feeding.

Sponsors & Collaborators

• Washington University School of Medicine: lead

Study Sites (1)

Washington University School of Medicine

St Louis, Missouri, 63110, United States

Location

Related Links

• Alvin J. Siteman Cancer Center at Barnes-Jewish Hospital and Washington University School of Medicine

MeSH Terms

Conditions

Myelodysplastic Syndromes; Neoplasm Metastasis

Interventions

Azacitidine

Condition Hierarchy (Ancestors)

Bone Marrow Diseases; Hematologic Diseases; Hemic and Lymphatic Diseases; Neoplastic Processes; Neoplasms; Pathologic Processes; Pathological Conditions, Signs and Symptoms

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: Ravi Vij, M.D.
• Organization: Washington University School of Medicine

rvij@dom.wustl.edu

314-454-8304

Study Officials

• Ravi Vij, M.D.

  Washington University School of Medicine

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 4, 2006
• First Posted: October 6, 2006
• Study Start: August 1, 2006
• Primary Completion: August 1, 2008
• Study Completion: March 1, 2010
• Last Updated: December 12, 2016
• Results First Posted: September 8, 2014

Record last verified: 2016-10

Locations

US (1)