Clinical trial NCT00360035: Safety and Efficacy of Obatoclax Mesylate (GX15-070MS) in the Treatment of Myelofibrosis With Myeloid Metaplasia
Official title: A Multi-Center, Open-Label, Phase II Study of Single-Agent GX15-070MS Administered as a 24-Hour Infusion Every 2 Weeks to Patients With Myelofibrosis With Myeloid Metaplasia (MF)
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Myelofibrosis
Lead sponsor: Teva Branded Pharmaceutical Products R&D, Inc.
Sponsor class: INDUSTRY
First posted: 2006-08-03
Enrollment: 22
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: Defects in the apoptotic process can lead to the onset of cancer by allowing cells to grow unchecked when an oncogenic 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.

Detailed description: This is a multi-center, open-label, Phase II study of single-agent obatoclax administered in 2-week cycles as a 24-hour infusion every 2 weeks at a fixed dose of 60 mg to patients with myelofibrosis. Infusions may be administered on an outpatient basis. No investigational or commercial agents or therapies other than those described in the protocol may be administered with the intent to treat the patient's malignancy. Supportive care measures including those directed at controlling symptoms resulting from myelofibrosis (blood products, growth factor, hydroxyurea, etc.) are allowed.

Eligibility criteria: Inclusion Criteria:

* Histologically confirmed myelofibrosis with myeloid metaplasia.
* No limitations on allowable type and amount of prior therapy.
* Patients must have normal organ function.
* Must be willing to submit to blood sampling for planned PK and PD analyzes.
* Must have ability to understand and willingness to sign a written informed consent form.

Exclusion Criteria:

* No other agents or therapies administered with the intent to treat malignancy.
* Patients with prior exposure to obatoclax.
* Uncontrolled, intercurrent illness.
* Pregnant women and women who are breast feeding.

Primary outcomes:
measure: Determine the steady-state pharmacokinetic parameters and pharmacodynamic response.
timeFrame: 1 Year
Secondary outcomes:
measure: Peripheral blood counts
timeFrame: 4 Weeks to 1 Year
measure: Bone marrow aspirates and biopsies
timeFrame: 8 weeks to 1 year
measure: Transfusion and growth factor requirements
timeFrame: 8 weeks to 1 year
Other outcomes:
Locations:
city: Washington
country: United States
facility: Georgetown University Medical Center
state: District of Columbia
city: Tampa
country: United States
facility: James A. Haley Veterans Hospital
state: Florida
city: Atlanta
country: United States
facility: Emory University
state: Georgia
city: Chicago
country: United States
facility: The University of Chicago
state: Illinois
city: Worcester
country: United States
facility: University of Massachusetts Medical Center
state: Massachusetts
city: Houston
country: United States
facility: MD Anderson Cancer Center
state: Texas
city: Toronto
country: Canada
facility: Princess Margaret Hospital
state: Ontario
Study officials:
affiliation: Gemin X, Inc.
name: Jean Viallet, MD
role: STUDY_DIRECTOR
References:
citation: Parikh SA, Kantarjian H, Schimmer A, Walsh W, Asatiani E, El-Shami K, Winton E, Verstovsek S. Phase II study of obatoclax mesylate (GX15-070), a small-molecule BCL-2 family antagonist, for patients with myelofibrosis. Clin Lymphoma Myeloma Leuk. 2010 Aug;10(4):285-9. doi: 10.3816/CLML.2010.n.059.