Acute Myeloid Leukemia (AML)Phase 2NCT00666497
Phase 2 Study of Azacitidine (Vidaza) vs MGCD0103 vs Combination in Elderly Subjects With Newly Diagnosed Acute Myeloid Leukemia (AML) or Myelodysplastic Syndrome (MDS)
A Phase 2 study testing Azacitidine and MGCD0103 in people with Acute Myeloid Leukemia (AML) and Myelodysplastic Syndrome (MDS), run by Mirati Therapeutics Inc. at 8 sites in 3 countries.
Trial summary
The purpose of the study is to determine how effective azacitidine, MGCD0103, and the combination of azacitidine and MGCD0103 are in treating AML or MDS in people over 60 years of age. Read more about this study
- Who is it for?
- Ages 60 years and older
- Conditions the study is for
- Acute Myeloid Leukemia (AML), Myelodysplastic Syndrome (MDS)
- What is being tested?
- Azacitidine and MGCD0103
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 6 participants
- Where
- 8 sitesin 3 countries
- Study dates
- Jun 2008to Apr 2009
- 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 60 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Able to provide written informed consent, and be willing and able to comply with all the study procedures
- Must be 60 years of age or older
- Must have a pathologic confirmation of newly diagnosed (de novo or untreated secondary) AML or newly diagnosed Int-2 or high-risk MDS (IPSS classification) according to WHO criteria
- Must have a Eastern Cooperative Oncology Group (ECOG) performance status of 0, 1, or 2
- Must have adequate organ function, including total bilirubin ≤ 1.5 x upper limit of normal (ULN); AST \& ALT ≤ 2.5 x ULN; and serum creatinine ≤ 2.0 x ULN.
Who cannot join
- Considered fit for intensive chemotherapy and opt to be treated with intensive chemotherapy
- Prior transplantation or any prior anticancer therapy (standard or investigational, including chemotherapy, treatment with HDAC inhibitors, or combination HDAC and azacitidine) administered to treat AML or MDS.
- Clinical evidence of central nervous system (CNS) involvement by leukemia
- A diagnosis of promyelocytic leukemia
- Previous or concurrent malignancy except adequately treated basal cell or squamous cell skin cancer; in situ carcinoma of the cervix, or other solid tumor treated curatively, and without evidence of recurrence for at least 3 years prior to study entry
- Active and uncontrolled clinically significant infection
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- Known positive serology for hepatitis B surface antigen (HBsAg), hepatitis C antibody (HCV Ab) or human immunodeficiency virus (HIV)
- Less than 4 weeks elapsed since any major surgery
- Any prior or active disease that may interfere with the procedures or evaluations to be conducted in the study
Trial locations and contacts
8 sites in 3 countries.
8 sites in 3 countries
United Kingdom5 sites
University Hospital
Birmingham, B15 2TH
Royal Bournemouth Hospital
Bournemouth, BH7 7DW
St. Bartholomews Hospital
London, EC1A 7BE
Kings College Hospital
London, SE5 9RS
John Radcliffe Hospital
Oxford, OX3 9DU
Canada2 sites
Jewish General Hospital
Montreal, Quebec, H3T 1E2
Diamond Centre, Leukemia/BMT Program of BC
Vancouver, British Columbia, V5Z 4E3
About this study
Official title: A Phase 2, Randomized, Open-Label Study of Azacitidine (Vidaza) vs MGCD0103 vs Azacitidine in Combination With MGCD0103 for the Treatment of Elderly Subjects With Newly Diagnosed AML or Intermediate-2 or High-Risk MDS
The purpose of the study is to determine how effective azacitidine, MGCD0103, and the combination of azacitidine and MGCD0103 are in treating AML or MDS in people over 60 years of age.
Read the detailed description
This randomized, 3-arm Phase 2 study will compare the safety and efficacy of single-agent azacitidine (currently 1 of 3 approved treatments for myelodysplastic syndrome [MDS]) to that of single-agent MGCD0103 and to that of combination therapy with MGCD0103 and azacitidine in elderly patients with acute myelogenous leukemia (AML) or intermediate-2 (Int-2) or high-risk MDS, for whom no standard of care exists. The goal of the study is to determine which of the 3 treatment arms are worthy of further investigation in a subsequent Phase 3 study of elderly subjects with AML or Int-2 or high-risk MDS.
- Being tested
- Azacitidine, MGCD0103
- Keywords
- Acute Myeloid Leukemia, Myelodysplastic Syndrome, Azacitidine, Vidaza, MGCD0103, Histone deacetylase inhibitor, DNA methyltransferase inhibitor, Epigenetic Therapy
- Medical subject headings
- Leukemia, Myeloid, Acute, Myelodysplastic Syndromes
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AzacitidineDrug
Also called Vidaza
75 mg/m2/day for 5 days, subcutaneous (SC) injection, Days 1 - 5 of every 28-day cycle
MGCD0103Drug
90 mg, oral (PO) administration, 3 times per week for 12 doses, 28-day cycle
MGCD0103Drug
90 mg, oral (PO) administration, 3 times per week for 10 doses, 28-day cycle
| Study group | Role | What they receive |
|---|---|---|
| A | Experimental | Azacitidine Receives: Drug: Azacitidine |
| B | Experimental | MGCD0103 Receives: Drug: MGCD0103 |
| C | Experimental | Azacitidine + MGCD0103 Receives: Drug: Azacitidine; Drug: MGCD0103 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Overall response rate as assessed using IWG criteria for AML and MDS
Measured: After 45, 90, 135, and 180 subjects are enrolled and evaluated for response to treatment
Secondary outcomes (1)
Duration of response; Time to progression; Progression-free survival; RBC transfusion independence; Hematologic improvement; Quality of life; Safety profile; and Pharmacokinetics of azacitidine and MGCD0103
Measured: After 45, 90, 135, and 180 subjects are enrolled and evaluated for response to treatment
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Apr 2008 (estimated)
- Study startJun 2008
- Primary completionApr 2009
- Study completionApr 2009
Why it stopped: Celgene terminated its collaboration agreement with MethylGene for the development of MGCD0103. All Celgene-sponsored trials with MGCD0103 with be closed.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Acute Myeloid Leukemia (AML)Studies 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 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 6 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
- NCT00666497
- Sponsor's protocol ID
- 103 PH GL 2007 CL003
- Status last verified
- Apr 2015
- First submitted
- 23 Apr 2008
- Last updated
- 8 May 2015
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 8 May 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2, Randomized, Open-Label Study of Azacitidine (Vidaza) vs MGCD0103 vs Azacitidine in Combination With MGCD0103 for the Treatment of Elderly Subjects With Newly Diagnosed AML or Intermediate-2 or High-Risk MDS
- ClinicalTrials.gov ID
- NCT00666497
- Acronym / sponsor ID
- 103 PH GL 2007 CL003
- Keywords
- Acute Myeloid Leukemia, Myelodysplastic Syndrome, Azacitidine, Vidaza, MGCD0103, Histone deacetylase inhibitor, DNA methyltransferase inhibitor, Epigenetic Therapy
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Celgene terminated its collaboration agreement with MethylGene for the development of MGCD0103. All Celgene-sponsored trials with MGCD0103 with be closed.
- Enrolment
- 6 participants (actual)
- Lead sponsor
- Mirati Therapeutics Inc.Industry (pharma, biotech, devices)
- Study start
- Jun 2008
- Primary completion
- Apr 2009 (actual)
- Study completion
- Apr 2009 (actual)
- First posted
- 25 Apr 2008 (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.
The purpose of the study is to determine how effective azacitidine, MGCD0103, and the combination of azacitidine and MGCD0103 are in treating AML or MDS in people over 60 years of age.
Detailed description
This randomized, 3-arm Phase 2 study will compare the safety and efficacy of single-agent azacitidine (currently 1 of 3 approved treatments for myelodysplastic syndrome [MDS]) to that of single-agent MGCD0103 and to that of combination therapy with MGCD0103 and azacitidine in elderly patients with acute myelogenous leukemia (AML) or intermediate-2 (Int-2) or high-risk MDS, for whom no standard of care exists. The goal of the study is to determine which of the 3 treatment arms are worthy of further investigation in a subsequent Phase 3 study of elderly subjects with AML or Int-2 or high-risk MDS.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| A | Experimental | Drug: AzacitidineAzacitidine |
| B | Experimental | Drug: MGCD0103MGCD0103 |
| C | Experimental | Drug: Azacitidine, Drug: MGCD0103Azacitidine + MGCD0103 |
Who may qualify
- Ages
- 60 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- Able to provide written informed consent, and be willing and able to comply with all the study procedures
- Must be 60 years of age or older
- Must have a pathologic confirmation of newly diagnosed (de novo or untreated secondary) AML or newly diagnosed Int-2 or high-risk MDS (IPSS classification) according to WHO criteria
- Must have a Eastern Cooperative Oncology Group (ECOG) performance status of 0, 1, or 2
- Must have adequate organ function, including total bilirubin ≤ 1.5 x upper limit of normal (ULN); AST \& ALT ≤ 2.5 x ULN; and serum creatinine ≤ 2.0 x ULN.
Key exclusion criteria (9 listed)
- Considered fit for intensive chemotherapy and opt to be treated with intensive chemotherapy
- Prior transplantation or any prior anticancer therapy (standard or investigational, including chemotherapy, treatment with HDAC inhibitors, or combination HDAC and azacitidine) administered to treat AML or MDS.
- Clinical evidence of central nervous system (CNS) involvement by leukemia
- A diagnosis of promyelocytic leukemia
- Previous or concurrent malignancy except adequately treated basal cell or squamous cell skin cancer; in situ carcinoma of the cervix, or other solid tumor treated curatively, and without evidence of recurrence for at least 3 years prior to study entry
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 |
|---|---|---|
| AzacitidineAlso: Vidaza75 mg/m2/day for 5 days, subcutaneous (SC) injection, Days 1 - 5 of every 28-day cycle | Drug | A, C |
| MGCD010390 mg, oral (PO) administration, 3 times per week for 12 doses, 28-day cycle | Drug | B |
| MGCD010390 mg, oral (PO) administration, 3 times per week for 10 doses, 28-day cycle | Drug | C |
Primary outcome
Overall response rate as assessed using IWG criteria for AML and MDS
Time frame: After 45, 90, 135, and 180 subjects are enrolled and evaluated for response to treatment
1 secondary outcome
Duration of response; Time to progression; Progression-free survival; RBC transfusion independence; Hematologic improvement; Quality of life; Safety profile; and Pharmacokinetics of azacitidine and MGCD0103
Time frame: After 45, 90, 135, and 180 subjects are enrolled and evaluated for response to treatment
Results and safety
The study was stopped early (reason given: Celgene terminated its collaboration agreement with MethylGene for the development of MGCD0103. All Celgene-sponsored trials with MGCD0103 with be closed.). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 May 2015.
- The Myelodysplastic Syndromes FoundationLink provided by the sponsor.
- Leukemia Research FoundationLink 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.
Gregory Reid, MSc, MBA study director
MethylGene Inc.
8 sites in 3 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Azacitidine and MGCD0103 in people with Acute Myeloid Leukemia (AML) and Myelodysplastic Syndrome (MDS), run by Mirati Therapeutics Inc. at 8 sites in 3 countries.
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 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 8 May 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 Kingdom, Canada, 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 was stopped early (reason given: Celgene terminated its collaboration agreement with MethylGene for the development of MGCD0103. All Celgene-sponsored trials with MGCD0103 with be closed.). Results are not available in the source record.
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?
Overall response rate as assessed using IWG criteria for AML and MDS (time frame: After 45, 90, 135, and 180 subjects are enrolled and evaluated for response to treatment). 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 May 2015.