LeukemiaPhase 2NCT00493129
Ontak (Denileukin Diftitox) in Patients With Systemic Mastocytosis (SM)
A Phase 2 study testing Ontak (Denileukin Diftitox) in people with Leukemia and Systemic Mastocytosis, run by M.D. Anderson Cancer Center at 1 site in United States.
Trial summary
Primary Objective: 1. To assess the response rate of ONTAK in Systemic Mastocytosis (SM) patients. Secondary Objectives: 1. To assess the safety of ONTAK in SM patients. 2. To evaluate the time to progression and duration of response following treatment with ONTAK. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Leukemia, Systemic Mastocytosis
- What is being tested?
- Ontak (Denileukin Diftitox)
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 8 participants
- Where
- 1 siteUnited States
- Study dates
- Jul 2004to Aug 2007
- Phase
- Phase 2
- Are there results?
- None posted in the registry
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 Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Patients with SM, including mast cell leukemia (MCL).
- ECOG Performance Status (PS) 0-3
- Adequate renal function (indicated by serum creatinine </= 2.5 mg/dL); adequate hepatic function (indicated by ALT </= 3 * upper limit of normal; total bilirubin </= 3 * upper limit of normal; and albumin >/= 2.8 g/dL).
- Provide written informed consent.
- Female patients of childbearing potential must have a negative pregnancy test within 14 days prior to first dose of study drug, and must agree to use an effective means of contraception following the pregnancy test, throughout the study and for at least three weeks after their last treatment on protocol.
Who cannot join
- History of hypersensitivity to diphtheria toxin.
- Active cardiovascular disease as defined by New York Heart Association (NYHA) Class III-IV categorization.
- Serious intercurrent medical illnesses or active infections requiring parenteral antibiotics that would interfere with the ability of the patient to carry out the treatment program.
- Concurrent malignancy (other than resected basal or squamous cell skin cancers or in-situ cervical cancer). Unless, patient has SM-associated clonal hematologic disease that does not require therapy, as judged by treating physician and approved by principal investigator.
- Female patients who are pregnant or breastfeeding.
- No chemotherapy, radiotherapy, immunotherapy, hormonal anticancer therapy, or experimental medications (including approved drugs tested in an investigational setting) may be administered while a patient is a participant in this protocol.
Trial locations and contacts
1 site in 1 country.
United States1 site
The University of Texas M.D. Anderson Cancer Center
Houston, Texas, 77030
About this study
Official title: ONTAK (Denileukin Diftitox) in Patients With Systemic Mastocytosis
Primary Objective:
- To assess the response rate of ONTAK in Systemic Mastocytosis (SM) patients.
Secondary Objectives:
- To assess the safety of ONTAK in SM patients.
- To evaluate the time to progression and duration of response following treatment with ONTAK.
Read the detailed description
Denileukin diftitox has been used for the treatment of a variety of disorders, in particular, malignant lymphoma, another blood-related disease. Denileukin diftitox is believed to be able to specifically attach to and kill malignant mast cells.
Before you can start treatment on this study, you will have what are called "screening tests". These tests will help the doctor decide if you are eligible to take part in the study. You will have blood (around 2 teaspoons) and bone marrow samples collected. To collect a bone marrow sample, an area of the hip bone is numbed with anesthetic and a small amount of bone marrow is withdrawn through a large needle. These samples will be used for tests to confirm the diagnosis of the disease. Women who are able to have children must have a negative blood pregnancy test.
If you are found to be eligible, you will receive denileukin diftitox as an injection by vein once a day for 5 days in a row. This will be repeated every 3 weeks (1 cycle). You will receive treatment on an outpatient basis. Treatment will continue as long as there is evidence that therapy is affecting the disease and is beneficial to you. If the disease gets worse or you experience any intolerable side effects, you will be taken off the study and your doctor will discuss other treatment options with you.
During treatment you will have blood (around 1 teaspoon) collected twice during every 3-week period. You will also have bone marrow samples collected every 3 months during the treatment.
After the end of treatment, blood and bone marrow samples will be collected every 3 months until the disease gets worse or you start a different therapy. The blood and bone marrow samples will be used to look at response to therapy.
This is an investigational study. Denileukin diftitox has been approved by the FDA for the treatment of skin T-cell lymphoma and is commercially available. The use of denileukin diftitox in this study is investigational. You will be provided denileukin diftitox free of charge. Up to 25 participants will take part in this study. All be enrolled at M. D. Anderson.
- Conditions
- Leukemia, Systemic Mastocytosis
- Being tested
- Ontak (Denileukin Diftitox)
- Keywords
- Systemic Mastocytosis, SM, Mast Cell Leukemia, Leukemia, Denileukin Diftitox, Ontak
- Medical subject headings
- Leukemia, Mastocytosis, Systemic, Leukemia, Mast-Cell
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Ontak (Denileukin Diftitox)Drug
Also called Denileukin Diftitox
9 µg/kg by vein Days 1-5 of a 21 day cycle.
| Study group | Role | What they receive |
|---|---|---|
| Ontak | Experimental | Ontak administered intravenously on Days 1-5 at the dose of 9 µg/kg/day, with a rest period from Days 6-21. Receives: Drug: Ontak (Denileukin Diftitox) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Objective Response Rate
Bone marrow samples used to look at response to therapy collected every 3 months until disease progression or different therapy started. Clinical efficacy assessed as objective response (major and partial response) for responding patients analyzed by the Kaplan-Meier method.
Measured: 3 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Jun 2007 (estimated)
- Study startJul 2004
- Primary completionAug 2007
- Study completionAug 2007
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- 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
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 8 participants (actual)
Sponsor and investigators
- Lead sponsor
- M.D. Anderson Cancer Center
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Srdan Verstovsek, MD, Principal investigator, M.D. Anderson Cancer Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00493129
- Sponsor's protocol ID
- 2004-0142
- Status last verified
- Feb 2012
- First submitted
- 26 Jun 2007
- Last updated
- 8 Feb 2012
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 8 Feb 2012. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- ONTAK (Denileukin Diftitox) in Patients With Systemic Mastocytosis
- ClinicalTrials.gov ID
- NCT00493129
- Acronym / sponsor ID
- 2004-0142
- Conditions
- Leukemia, Systemic Mastocytosis
- Keywords
- Systemic Mastocytosis, SM, Mast Cell Leukemia, Leukemia, Denileukin Diftitox, Ontak
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 8 participants (actual)
- Lead sponsor
- M.D. Anderson Cancer CenterUniversity, hospital or other
- Study start
- Jul 2004
- Primary completion
- Aug 2007 (actual)
- Study completion
- Aug 2007 (actual)
- First posted
- 28 Jun 2007 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Primary Objective:
- To assess the response rate of ONTAK in Systemic Mastocytosis (SM) patients.
Secondary Objectives:
- To assess the safety of ONTAK in SM patients.
- To evaluate the time to progression and duration of response following treatment with ONTAK.
Detailed description
Denileukin diftitox has been used for the treatment of a variety of disorders, in particular, malignant lymphoma, another blood-related disease. Denileukin diftitox is believed to be able to specifically attach to and kill malignant mast cells.
Before you can start treatment on this study, you will have what are called "screening tests". These tests will help the doctor decide if you are eligible to take part in the study. You will have blood (around 2 teaspoons) and bone marrow samples collected. To collect a bone marrow sample, an area of the hip bone is numbed with anesthetic and a small amount of bone marrow is withdrawn through a large needle. These samples will be used for tests to confirm the diagnosis of the disease. Women who are able to have children must have a negative blood pregnancy test.
If you are found to be eligible, you will receive denileukin diftitox as an injection by vein once a day for 5 days in a row. This will be repeated every 3 weeks (1 cycle). You will receive treatment on an outpatient basis. Treatment will continue as long as there is evidence that therapy is affecting the disease and is beneficial to you. If the disease gets worse or you experience any intolerable side effects, you will be taken off the study and your doctor will discuss other treatment options with you.
During treatment you will have blood (around 1 teaspoon) collected twice during every 3-week period. You will also have bone marrow samples collected every 3 months during the treatment.
After the end of treatment, blood and bone marrow samples will be collected every 3 months until the disease gets worse or you start a different therapy. The blood and bone marrow samples will be used to look at response to therapy.
This is an investigational study. Denileukin diftitox has been approved by the FDA for the treatment of skin T-cell lymphoma and is commercially available. The use of denileukin diftitox in this study is investigational. You will be provided denileukin diftitox free of charge. Up to 25 participants will take part in this study. All be enrolled at M. D. Anderson.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Ontak | Experimental | Drug: Ontak (Denileukin Diftitox)Ontak administered intravenously on Days 1-5 at the dose of 9 µg/kg/day, with a rest period from Days 6-21. |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (5 listed)
- Patients with SM, including mast cell leukemia (MCL).
- ECOG Performance Status (PS) 0-3
- Adequate renal function (indicated by serum creatinine </= 2.5 mg/dL); adequate hepatic function (indicated by ALT </= 3 * upper limit of normal; total bilirubin </= 3 * upper limit of normal; and albumin >/= 2.8 g/dL).
- Provide written informed consent.
- Female patients of childbearing potential must have a negative pregnancy test within 14 days prior to first dose of study drug, and must agree to use an effective means of contraception following the pregnancy test, throughout the study and for at least three weeks after their last treatment on protocol.
Key exclusion criteria (6 listed)
- History of hypersensitivity to diphtheria toxin.
- Active cardiovascular disease as defined by New York Heart Association (NYHA) Class III-IV categorization.
- Serious intercurrent medical illnesses or active infections requiring parenteral antibiotics that would interfere with the ability of the patient to carry out the treatment program.
- Concurrent malignancy (other than resected basal or squamous cell skin cancers or in-situ cervical cancer). Unless, patient has SM-associated clonal hematologic disease that does not require therapy, as judged by treating physician and approved by principal investigator.
- Female patients who are pregnant or breastfeeding.
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 |
|---|---|---|
| Ontak (Denileukin Diftitox)Also: Denileukin Diftitox9 µg/kg by vein Days 1-5 of a 21 day cycle. | Drug | Ontak |
Primary outcome
Objective Response Rate
Time frame: 3 months
Bone marrow samples used to look at response to therapy collected every 3 months until disease progression or different therapy started. Clinical efficacy assessed as objective response (major and partial response) for responding patients analyzed by the Kaplan-Meier method.
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 Feb 2012.
- M.D. Anderson's 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 principal investigator
M.D. 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 Ontak (Denileukin Diftitox) 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 5 inclusion and 6 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: finished, as of the sponsor's last update on 8 Feb 2012. 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?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Objective Response Rate (time frame: 3 months). 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 8 Feb 2012.