Safety and Efficacy of Obatoclax Mesylate (GX15-070MS)for the Treatment of Myelodysplastic Syndromes (MDS)

A Phase II Study of Obatoclax Mesylate (GX15-070MS) in Patients With Previously-Untreated Myelodysplastic Syndromes (MDS) With Anemia and/or Thrombocytopenia

Defects in the apoptotic process can lead to the onset of cancer by allowing cells to grow unchecked when an oncogeneic signal is present. Obatoclax is designed to restore apoptosis through inhibition of the Bcl-2 family of proteins, thereby reinstating the natural process of cell death that is often inhibited in cancer cells.

This is a multi-center, open-label, Phase II study of obatoclax administered in 2-week cycles to patients with previously-untreated Myelodysplastic Syndromes with anemia and/or thrombocytopenia. Treatment may be administered on an outpatient basis. No investigational or commercial agents or therapies other than those described herein may be administered with the intent to treat the patient's malignancy. Supportive care measures including those directed at controlling symptoms resulting from Myelodysplastic Syndromes are allowed

Study Overview

• Status: Completed
• Conditions: Myelodysplastic Syndromes
• Intervention / Treatment: Drug: Obatoclax mesylate (GX15-070MS)

• Study Type: Interventional
• Enrollment (Actual): 24
• Phase: Phase 2

Contacts and Locations

Study Locations

• Canada
  • Alberta
    • Calgary, Alberta, Canada
      • Tom Baker Cancer Centre
  • Nova Scotia
    • Halifax, Nova Scotia, Canada, B3H 2Y9
      • QEII HSC
  • Ontario
    • Toronto, Ontario, Canada, M5G 2M9
      • Princess Margaret Hospital
  • Quebec
    • Montreal, Quebec, Canada, H2L4M1
      • Hospital Notre-Dame du Chum
    • Montreal, Quebec, Canada
      • Maisonneuve-Rosemont Hospital
• United States
  • California
    • Stanford, California, United States, 00000
      • Stanford University
  • District of Columbia
    • Washington, District of Columbia, United States, 20007
      • Georgetown University Medical Center
  • Florida
    • Tampa, Florida, United States, 33612
      • James A. Haley Veterans Hospital
  • Georgia
    • Atlanta, Georgia, United States, 30322
      • Emory University School of Medicine/ Winship Cancer Center
    • Marietta, Georgia, United States, 30060
      • Northwest Georgia Oncology Centers
  • Illinois
    • Chicago, Illinois, United States, 60637
      • University of Chicago
  • Massachusetts
    • Worcester, Massachusetts, United States, 01605
      • University of Massachusetts Medical Center
  • Michigan
    • Lansing, Michigan, United States, 48910
      • Michigan State University, Breslin Cancer Center CTO
  • Montana
    • Billings, Montana, United States, 59101
      • Hematology-Oncology Centers of the Northern Rockies
  • New York
    • New York, New York, United States, 01605
      • St. Vincent's Comprehensive Cancer Center
  • Oregon
    • Portland, Oregon, United States, 97210
      • Pacific Oncology
  • Tennessee
    • Memphis, Tennessee, United States, 38120
      • The West Clinic
    • Nashville, Tennessee, United States, 37203
      • Sarah Cannon Cancer Research Institute
  • Texas
    • Arlington, Texas, United States, 76012
      • Arlington Cancer Center
    • Dallas, Texas, United States, 75246
      • Mary Crowley Medical Research Center
    • Dallas, Texas, United States, 75231
      • Texax Oncology, P.A., Presbyterian/ Mary Crowley Clinical Research Centers
    • Houston, Texas, United States, 77030
      • MD Anderson Cancer Center (Protocol 2006-0688)

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

• Pathological confirmation of Myelodysplastic Syndromes (MDS)
• Patients must have had no prior systemic therapy
• Must have normal organ functions
• Must have the ability to understand and willingness to sign a written informed consent form

Exclusion Criteria:

• Must not be a result of prior chemotherapy and/or radiotherapy for another malignancy
• No other agents or therapies administered in the intent to treat
• Uncontrolled, intercurrent illness
• Pregnant women and women who are breast feeding

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Obatoclax Mesylate; Obatoclax Mesylate 30mg	Drug: Obatoclax mesylate (GX15-070MS)

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
International Working Group (IWG) Response Criteria for MDS	Determine the response rate according to bone marrow blast count less than or equal to 10%	52 weeks

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Peripheral blood counts; Bone marrow aspirates and biopsies; Transfusions and growth factor requirements	hemoglobin level less than 10 g/dL and or platelets less than 50 x 10 9/L. Eastern Cooperative Onocology Group (ECOG) performance status: 0 fully active-2 ambulatory 50% of the time; and total bilirubin less than or equal to 2 mg/dL; normal limits of SGOT/SGPT and creatinine according to laboratory standards	52 weeks

Collaborators and Investigators

• Sponsor: Gemin X

Publications and helpful links

General Publications

• Arellano ML, Borthakur G, Berger M, Luer J, Raza A. A phase II, multicenter, open-label study of obatoclax mesylate in patients with previously untreated myelodysplastic syndromes with anemia or thrombocytopenia. Clin Lymphoma Myeloma Leuk. 2014 Dec;14(6):534-9. doi: 10.1016/j.clml.2014.04.007. Epub 2014 Jun 12.

Study record dates

Study Major Dates

• Study Start: December 1, 2006
• Primary Completion (Actual): February 1, 2009
• Study Completion (Actual): November 1, 2009

Study Registration Dates

• First Submitted: December 18, 2006
• First Submitted That Met QC Criteria: December 18, 2006
• First Posted (Estimate): December 19, 2006

Study Record Updates

• Last Update Posted (Estimate): August 26, 2013
• Last Update Submitted That Met QC Criteria: August 16, 2013
• Last Verified: August 1, 2013

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Pathologic Processes; Neoplasms; Disease; Bone Marrow Diseases; Hematologic Diseases; Precancerous Conditions; Syndrome; Myelodysplastic Syndromes; Preleukemia; Molecular Mechanisms of Pharmacological Action; Enzyme Inhibitors; Obatoclax
• Other Study ID Numbers: GEM013