LeukemiaPhase 2NCT00918931
Obatoclax for Systemic Mastocytosis
A Phase 2 study testing Obatoclax Mesylate in people with Leukemia and Systemic Mastocytosis, run by M.D. Anderson Cancer Center at 1 site in United States.
Trial summary
The goal of this clinical research study is to learn if obatoclax mesylate can help to control systemic mastocytosis. The safety of this drug will also be tested. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Leukemia, Systemic Mastocytosis
- What is being tested?
- Obatoclax Mesylate
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 3 participants
- Where
- 1 siteUnited States
- Study dates
- Jun 2009to Oct 2010
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Patients with Systemic Mastocytosis (SM); including mast cell leukemia.
- Age equal to or greater than 18 years
- Minimum of two weeks since any major surgery or completion of radiation
- Eastern Cooperative Oncology Group (ECOG) performance status equal to or less than 2
- 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.
- Signed informed consent
Who cannot join
- 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.
- Treatment with any conventional (specifically, interferon or cladribine) or investigational medicine for SM within the preceding 4 weeks
- Chronic treatment with systemic steroids (unless limited to 10 mg prednisone equivalent per day or less) or another immunosuppressive agent
- 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).
- 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)
- A known history of human immunodeficiency virus (HIV) seropositivity
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- 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.
- Patients with a known hypersensitivity to obatoclax mesylate or to its excipients (PEG 300 and Tween 20)
- Patients unwilling to or unable to comply with the protocol
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Evaluation of Obatoclax Mesylate as Therapy for Patients With Systemic Mastocytosis
The goal of this clinical research study is to learn if obatoclax mesylate can help to control systemic mastocytosis. The safety of this drug will also be tested.
Read the detailed description
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.
- Conditions
- Leukemia, Systemic Mastocytosis
- Being tested
- Obatoclax Mesylate
- Keywords
- Leukemia, Systemic Mastocytosis, SM, Obatoclax mesylate, GX15-070MS
- Medical subject headings
- Leukemia, Mastocytosis, Systemic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Obatoclax MesylateDrug
30 mg given by vein over 3 hours on Days 1-3 of each 14-day study cycle.
| Study group | Role | What they receive |
|---|---|---|
| Obatoclax Mesylate | Experimental | 30 mg by vein over 3 hours Days 1-3, 14-day cycle Receives: Drug: Obatoclax Mesylate |
What is being measured
The primary outcome is the main question the study is designed to answer.
Response Rate
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).
Measured: 3-Month Response Evaluation
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 Jun 2009 (estimated)
- Study startJun 2009
- Primary completionOct 2010
- Study completionOct 2010
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for LeukemiaStudies of the same condition, open and under way.
- More trials run by M.D. Anderson Cancer CenterThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 3 participants (actual)
Sponsor and investigators
- Lead sponsor
- M.D. Anderson Cancer Center
- Sponsor type
- University, hospital or other
- Collaborators
- Gemin X
- Responsible party
- Sponsor
- Investigators
- Srdan Verstovsek, MD, Study chair, UT MD Anderson Cancer Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00918931
- Sponsor's protocol ID
- 2008-0792
- Status last verified
- Oct 2011
- First submitted
- 9 Jun 2009
- Last updated
- 4 Nov 2011
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 4 Nov 2011. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Evaluation of Obatoclax Mesylate as Therapy for Patients With Systemic Mastocytosis
- ClinicalTrials.gov ID
- NCT00918931
- Acronym / sponsor ID
- 2008-0792
- Conditions
- Leukemia, Systemic Mastocytosis
- Keywords
- Leukemia, Systemic Mastocytosis, SM, Obatoclax mesylate, GX15-070MS
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Terminated
- Enrolment
- 3 participants (actual)
- Lead sponsor
- M.D. Anderson Cancer CenterUniversity, hospital or other
- Collaborators
- Gemin X
- Study start
- Jun 2009
- Primary completion
- Oct 2010 (actual)
- Study completion
- Oct 2010 (actual)
- First posted
- 11 Jun 2009 (estimated)
- Results
- Posted 25 Apr 2011
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The goal of this clinical research study is to learn if obatoclax mesylate can help to control systemic mastocytosis. The safety of this drug will also be tested.
Detailed description
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Obatoclax Mesylate | Experimental | Drug: Obatoclax Mesylate30 mg by vein over 3 hours Days 1-3, 14-day cycle |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- Patients with Systemic Mastocytosis (SM); including mast cell leukemia.
- Age equal to or greater than 18 years
- Minimum of two weeks since any major surgery or completion of radiation
- Eastern Cooperative Oncology Group (ECOG) performance status equal to or less than 2
- 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.
Key exclusion criteria (9 listed)
- 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.
- Treatment with any conventional (specifically, interferon or cladribine) or investigational medicine for SM within the preceding 4 weeks
- Chronic treatment with systemic steroids (unless limited to 10 mg prednisone equivalent per day or less) or another immunosuppressive agent
- 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).
- 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)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Obatoclax Mesylate30 mg given by vein over 3 hours on Days 1-3 of each 14-day study cycle. | Drug | Obatoclax Mesylate |
Primary outcome
Response Rate
Time frame: 3-Month Response Evaluation
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).
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 4 Nov 2011.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- UT MD Anderson Cancer Center websiteLink provided by the sponsor.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Srdan Verstovsek, MD study chair
UT MD Anderson Cancer Center
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Obatoclax Mesylate in people with Leukemia and Systemic Mastocytosis, run by M.D. Anderson Cancer Center at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 4 Nov 2011. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 25 Apr 2011). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Response Rate (time frame: 3-Month Response Evaluation). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 4 Nov 2011.