Evaluation of Obatoclax Mesylate as Therapy for Patients With Systemic Mastocytosis
18 Years
N/A
Both
Leukemia, Systemic Mastocytosis
Trial Information
Evaluation of Obatoclax Mesylate as Therapy for Patients With Systemic Mastocytosis
The Study Drug:
Obatoclax mesylate is designed to block the growth of malignant mast cells. This may cause
cancer cells to die.
Study Drug Administration:
If you are found to be eligible to take part in this study, you will receive obatoclax
mesylate through a peripheral intravenous catheter (a needle in the vein of your arm). The
drug will be given over 3 hours each day on Days 1-3 of each 14-day study cycle.
If the study doctor thinks it is needed, you might receive the study drug though a
silicone-based central venous catheter (CVC). A central venous catheter is a sterile
flexible tube that will be placed into a large vein while you are under local anesthesia.
Some catheters cannot be used with the study drug. If you already have one of these
catheters in your body, it may need to be replaced before you can begin receiving the study
drug. You will sign a separate consent that will describe the procedure and its risks in
detail.
Study Visits:
One (1) time during each study cycle, the following tests and procedures will be performed:
- You will be asked about any side effects you may be having and about any other drugs
you may be taking.
- Blood (about 2 tablespoons) will be drawn for routine tests.
Every 3 months while you are taking the study drug, you will have a bone marrow
biopsy/aspirate to check the status of the disease.
Length of Study:
You will be able to continue receiving the study drug for as long as you are benefitting.
You will be taken off study if you have intolerable side effects or if the disease gets
worse.
Follow-Up:
Three months after your last dose of study drug, your doctor will call you to ask you how
you are feeling. This call should take about 10 minutes.
Additional Information:
- You must tell your doctor about any drug allergies you may have.
- You must tell your doctor about how much alcohol you consume and about any other drugs
you may be taking, including vitamins, herbal remedies, and over-the-counter,
prescription, and/or illegal drugs. Some of these will interact with the study drug.
- You must arrange to have someone drive you home after each treatment. You must not
drive or operate machinery (including kitchen appliances) after you have received the
study drug until any possible side effects have gone away.
- If you feel you any side effects or symptoms while you are on study, you must contact
your doctor right away.
This is an investigational study. Obatoclax mesylate is not FDA approved or commercially
available. At this time, obatoclax mesylate is only being used in research.
Up to 25 patients will take part in this study. All will be enrolled at M. D. Anderson.
Inclusion Criteria:
1. Patients with Systemic Mastocytosis (SM); including mast cell leukemia.
2. Age equal to or greater than 18 years
3. Minimum of two weeks since any major surgery or completion of radiation
4. Eastern Cooperative Oncology Group (ECOG) performance status equal to or less than 2
5. Adequate liver function as shown by serum bilirubin equal to or less than 1.5 * upper
limit of normal (ULN), and serum alanine aminotransferase (ALT) equal to or less than
3 * ULN.
6. Signed informed consent
Exclusion Criteria:
1. Patients with low blood cell counts (Grade 3 or 4), unless it is known that the low
blood cell count is due to systemic mastocytosis.
2. Treatment with any conventional (specifically, interferon or cladribine) or
investigational medicine for SM within the preceding 4 weeks
3. Chronic treatment with systemic steroids (unless limited to 10 mg prednisone
equivalent per day or less) or another immunosuppressive agent
4. Other malignancies within the past 3 years except for adequately treated carcinoma of
the cervix or basal or squamous cell carcinomas of the skin. Patient may have
SM-associated clonal hematologic disease that does not require therapy, as judged by
the treating physician and approved by the principal investigator).
5. Other concurrent severe and/or uncontrolled medical disease which could compromise
participation in the study as judged by the Principal Investigator (i.e., severely
impaired lung function, uncontrolled diabetes, uncontrolled hypertension, severe
infection, severe malnutrition, unstable angina, or congestive heart failure - New
York Heart Association Class III or IV, ventricular arrhythmias, active ischemic
heart disease, myocardial infarction within six months, chronic liver or renal
disease, active upper GI tract ulceration)
6. A known history of human immunodeficiency virus (HIV) seropositivity
7. Women who are pregnant or breast feeding,or women/men able to conceive and unwilling
to practice an effective method of birth control. Women of childbearing potential
must have a negative urine or serum pregnancy test within 48 hours prior to
administration of obatoclax. Women of childbearing potential is defined as women who
have not undergone a hysterectomy or bilateral oophorectomy, has not been naturally
postmenopausal for at least 24 consecutive months.
8. Patients with a known hypersensitivity to obatoclax mesylate or to its excipients
(PEG 300 and Tween 20)
9. Patients unwilling to or unable to comply with the protocol
Type of Study:
Interventional
Study Design:
Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Outcome Measure:
Response Rate
Outcome Description:
Response rate is defined as number of participants with objective response divided by number of participants evaluated. Objective response is complete response and partial response where 'complete response' constitutes total elimination of a symptom or sign of the disease; 'partial response' constitutes at least 50% improvement in a symptom or sign of the disease. Objective response definitions of response evaluated following guidelines proposed by Valent et al. (International working group consensus criteria).
Outcome Time Frame:
3-Month Response Evaluation
Safety Issue:
Yes
Principal Investigator
Srdan Verstovsek, MD
Investigator Role:
Study Chair
Investigator Affiliation:
UT MD Anderson Cancer Center
Authority:
United States: Food and Drug Administration
Study ID:
2008-0792
NCT ID:
NCT00918931
Start Date:
June 2009
Completion Date:
October 2010
Related Keywords:
- Leukemia
- Systemic Mastocytosis
- Leukemia
- Systemic Mastocytosis
- SM
- Obatoclax mesylate
- GX15-070MS
- Leukemia
- Mastocytosis
- Urticaria Pigmentosa
- Mastocytoma
- Mastocytosis, Systemic
Name | Location |
|---|---|
| UT MD Anderson Cancer Center | Houston, Texas 77030 |
