Myelodysplastic SyndromePhase 1/2NCT00324220
A Phase I/II Study of MGCD0103 With Azacitidine in Patients With High-Risk Myelodysplastic Syndrome (MDS) or Acute Myelogenous Leukemia
A Phase 1/2 study testing MGCD0103 in people with Myelodysplastic Syndrome and Acute Myelogenous Leukemia, run by Mirati Therapeutics Inc. at 8 sites in United States.
Trial summary
In this study, MGCD0103, a new anticancer drug under investigation, is given three times weekly in combination with azacitidine to patients with high-risk myelodysplastic syndromes or acute myelogenous leukemia. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Myelodysplastic Syndrome, Acute Myelogenous Leukemia
- What is being tested?
- MGCD0103
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 66 participants
- Where
- 8 sitesUnited States
- Study dates
- Jan 2006to Nov 2008
- Phase
- Phase 1/2
- 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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Patients must have high-risk MDS (≥ 10% BM blasts) or AML
- RAEB (RA with excess blasts) with ≥10% BM blasts: 10%-20% blasts in BM, <5% blasts in peripheral blood
- RAEB-T (RAEB in transformation): 21%-30% blasts in BM, <5% blasts in peripheral blood, absolute monocytosis (>109/L)
- AML
- Disease may be relapsed/refractory or de novo. Once the MTD has been determined, all subsequent patients in the phase II portion of the study should have no prior azacitidine
- ECOG performance status of 0, 1, or 2
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- Age ≥18 years
- Laboratory requirements
- Patients or their legal representative must be able to read, understand, and sign a written informed consent (approved by the institutional review board/Ethics Committee (IRB/EC)) within 14 days prior to start of treatment
Who cannot join
- Patients with another active cancer (excluding basal cell carcinoma or cervical intraepithelial neoplasia (CIN / cervical in situ)). Prior history of cancer is allowed, as long as there is no active disease
- Pregnant or lactating women. Women of child-bearing potential (WOCBP) must have a negative serum pregnancy test documented within 7 days prior start of study drug
- WOCBP and men whose partners are WOCBP must use an acceptable method of contraception while enrolled on this study, and for a period of 3 months following study drug treatment. Patients unwilling or unable to follow this guideline will be excluded. Examples of acceptable forms of contraception include an oral contraceptive or a double barrier method, such as condom with diaphragm
- Patients with uncontrolled intercurrent illness, active or uncontrolled infections, or a fever >38.5˚C on the day of scheduled dosing
- Patients with serious illnesses, medical conditions, or other medical history, including laboratory results, which, in the investigator's opinion, would be likely to interfere with a patient's participation in the study, or with the interpretation of the results
- Patients who have been treated with any investigational drug within 30 days prior to study initiation (an investigational drug is one for which there is no approved indication), or who are receiving concurrent treatment with other experimental drugs or anti-cancer therapy
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- Known hypersensitivity to HDAC inhibitors, to any of the components of MG-0103 or Vidaza, including mannitol
- Prior treatment with azacitidine during the expanded phase II portion only
- Known HIV or active Hepatitis B or C
- Any condition (e.g., known or suspected poor compliance, psychological instability, geographical location, etc) that, in the judgment of the investigator, may affect the patient's ability to sign the informed consent and undergo study procedures
- Any condition that will put the patient at undue risk or discomfort as a result of adherence to study procedures. For example, consider requirement to take MG-0103 with an acidic drink and recommendation to avoid agents that increase gastric-pH.
Trial locations and contacts
8 sites in 1 country.
8 sites in 1 country
United States8 sites
St. Francis Hospital & Health Center
Beech Grove, Indiana, 46107
University of Texas, MD Anderson Cancer Center
Houston, Texas, 77030
University of Southern California
Los Angeles, California, 90033
Memorial Sloan-Kettering Cancer Center
New York, New York, 10021
University of Pennsylvania
Philadelphia, Pennsylvania, 19104
Thomas Jefferson University Hospital
Philadelphia, Pennsylvania, 19107
Thomas Jefferson University
Philadelphia, Pennsylvania, 19107
The Western Pennsylvania Hospital
Pittsburgh, Pennsylvania, 15224
About this study
Official title: A Phase I/II Study of MGCD0103 (MG-0103) in Combination With Azacitidine in Patients With High-Risk Myelodysplastic Syndrome or Acute Myelogenous Leukemia
In this study, MGCD0103, a new anticancer drug under investigation, is given three times weekly in combination with azacitidine to patients with high-risk myelodysplastic syndromes or acute myelogenous leukemia.
Read the detailed description
Phase I: The purpose of the first part (Phase 1) of this study is to find out what side effects the experimental drug MG-0103 in combination with azacitidine has on your body. The first part, or Phase, of the study will find out how much MG-0103 can be given safely along with azacitidine to people with cancer without causing side effects that are too severe. Patients are given MG-0103 and azacitidine and are watched closely to see what side effects may develop and to make sure that if side effects are seen, they can be taken care of rapidly. If the side effects are not severe, then more patients are asked to join the study and are given the same or a slightly higher dose of MG-0103. If there are no severe side effects, patients joining the study later on will get higher doses of MG-0103 than patients who join earlier. All patients will get the same dose of azacitidine. This will continue until a dose of MG-0103 is found that causes severe side effects in a high enough portion of patients. This will be the maximum dose of MG-0103 that can be given to patients in this study. Doses higher than that will not be given. Additional patients may be asked to join the study and receive MG-0103 and possibly azacitidine at lower doses that did not cause severe side effects.
Phase II: MG-0103 in combination with azacitidine may also have some effect on your disease. The purpose of the second part (Phase 2) of this study is to find out what, if any, effect there is. This Phase of the study will also find out more information about side effects of this combination of drugs. In this Phase, patients will receive a slightly lower dose than the maximum dose found in the first part of the study (which causes tolerable side effects). If a certain effect on the disease of patients is seen, then more patients are asked to join the study. Additional patients may also be asked to join the study and receive a lower dose if information collected during the study suggests that this should be done.
- Being tested
- MGCD0103
- Keywords
- Phase I/II
- Medical subject headings
- Myelodysplastic Syndromes, Leukemia, Myeloid, Acute
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
MGCD0103Drug
MGCD0103 Oral administration 3 times per week.
| Study group | Role | What they receive |
|---|---|---|
| 1 | Experimental | MGCD0103 oral administration 3 times per week. Receives: Drug: MGCD0103 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Maximum tolerated dose in combination with azacitidine
Measured: 1 year (anticipated)
Clinical response
Measured: 1 year (anticipated)
Secondary outcomes (3)
Dose limiting toxicities
Measured: 1 year (anticipated)
Pharmacokinetics
Measured: 1 year (anticipated)
Objective response
Measured: 1 year (anticipated)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 May 2006 (estimated)
- Study startJan 2006
- Primary completionNov 2008
- Study completionNov 2008
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Myelodysplastic SyndromeStudies of the same condition, open and under way.
- 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 1/2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 66 participants (actual)
Sponsor and investigators
- Lead sponsor
- Mirati Therapeutics Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Gregory Reid, MSc, MBA, Study director, MethylGene Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00324220
- Sponsor's protocol ID
- 0103-005
- Status last verified
- Jun 2015
- First submitted
- 8 May 2006
- Last updated
- 1 Jul 2015
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 1 Jul 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase I/II Study of MGCD0103 (MG-0103) in Combination With Azacitidine in Patients With High-Risk Myelodysplastic Syndrome or Acute Myelogenous Leukemia
- ClinicalTrials.gov ID
- NCT00324220
- Acronym / sponsor ID
- 0103-005
- Keywords
- Phase I/II
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 66 participants (actual)
- Lead sponsor
- Mirati Therapeutics Inc.Industry (pharma, biotech, devices)
- Study start
- Jan 2006
- Primary completion
- Nov 2008 (actual)
- Study completion
- Nov 2008 (actual)
- First posted
- 10 May 2006 (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.
In this study, MGCD0103, a new anticancer drug under investigation, is given three times weekly in combination with azacitidine to patients with high-risk myelodysplastic syndromes or acute myelogenous leukemia.
Detailed description
Phase I: The purpose of the first part (Phase 1) of this study is to find out what side effects the experimental drug MG-0103 in combination with azacitidine has on your body. The first part, or Phase, of the study will find out how much MG-0103 can be given safely along with azacitidine to people with cancer without causing side effects that are too severe. Patients are given MG-0103 and azacitidine and are watched closely to see what side effects may develop and to make sure that if side effects are seen, they can be taken care of rapidly. If the side effects are not severe, then more patients are asked to join the study and are given the same or a slightly higher dose of MG-0103. If there are no severe side effects, patients joining the study later on will get higher doses of MG-0103 than patients who join earlier. All patients will get the same dose of azacitidine. This will continue until a dose of MG-0103 is found that causes severe side effects in a high enough portion of patients. This will be the maximum dose of MG-0103 that can be given to patients in this study. Doses higher than that will not be given. Additional patients may be asked to join the study and receive MG-0103 and possibly azacitidine at lower doses that did not cause severe side effects.
Phase II: MG-0103 in combination with azacitidine may also have some effect on your disease. The purpose of the second part (Phase 2) of this study is to find out what, if any, effect there is. This Phase of the study will also find out more information about side effects of this combination of drugs. In this Phase, patients will receive a slightly lower dose than the maximum dose found in the first part of the study (which causes tolerable side effects). If a certain effect on the disease of patients is seen, then more patients are asked to join the study. Additional patients may also be asked to join the study and receive a lower dose if information collected during the study suggests that this should be done.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| 1 | Experimental | Drug: MGCD0103MGCD0103 oral administration 3 times per week. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (9 listed)
- Patients must have high-risk MDS (≥ 10% BM blasts) or AML
- Disease may be relapsed/refractory or de novo. Once the MTD has been determined, all subsequent patients in the phase II portion of the study should have no prior azacitidine
- ECOG performance status of 0, 1, or 2
- Age ≥18 years
- Laboratory requirements
Key exclusion criteria (11 listed)
- Patients with another active cancer (excluding basal cell carcinoma or cervical intraepithelial neoplasia (CIN / cervical in situ)). Prior history of cancer is allowed, as long as there is no active disease
- Pregnant or lactating women. Women of child-bearing potential (WOCBP) must have a negative serum pregnancy test documented within 7 days prior start of study drug
- WOCBP and men whose partners are WOCBP must use an acceptable method of contraception while enrolled on this study, and for a period of 3 months following study drug treatment. Patients unwilling or unable to follow this guideline will be excluded. Examples of acceptable forms of contraception include an oral contraceptive or a double barrier method, such as condom with diaphragm
- Patients with uncontrolled intercurrent illness, active or uncontrolled infections, or a fever >38.5˚C on the day of scheduled dosing
- Patients with serious illnesses, medical conditions, or other medical history, including laboratory results, which, in the investigator's opinion, would be likely to interfere with a patient's participation in the study, or with the interpretation of the results
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 |
|---|---|---|
| MGCD0103MGCD0103 Oral administration 3 times per week. | Drug | 1 |
Primary outcome
Maximum tolerated dose in combination with azacitidine
Time frame: 1 year (anticipated)
Clinical response
Time frame: 1 year (anticipated)
3 secondary outcomes
Dose limiting toxicities
Time frame: 1 year (anticipated)
Pharmacokinetics
Time frame: 1 year (anticipated)
Objective response
Time frame: 1 year (anticipated)
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 1 Jul 2015.
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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.
Gregory Reid, MSc, MBA study director
MethylGene Inc.
8 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing MGCD0103 in people with Myelodysplastic Syndrome and Acute Myelogenous Leukemia, run by Mirati Therapeutics Inc. at 8 sites 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 9 inclusion and 11 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 1 Jul 2015. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 8 sites 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 combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
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?
Maximum tolerated dose in combination with azacitidine (time frame: 1 year (anticipated)). The study lists 2 primary outcomes in all. 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 1 Jul 2015.