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
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
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.
- 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.
Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
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).
3-Month Response Evaluation
Srdan Verstovsek, MD
UT MD Anderson Cancer Center
United States: Food and Drug Administration
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