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Study Summary
No. 2011-0547:.......Leukemia......Farhad Ravandi-Kashani......Leukemia
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Study Summary Title
Study Summary
Number:
2011-0547
Study Title:A Phase 1 Study to Evaluate the Safety and Tolerability and Pharmacokinetic/Pharmacodynamics of MK-8242 Administered Alone and in Combination with Chemotherapy in Subjects with Refractory, or Recurrent Acute Myelogenous Leukemia
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Physician New Patient Referral
Name:Farhad Ravandi-KashaniPatients Call:800-392-1611 (in U.S.A.) 713-792-6161 (outside U.S.A.)
Dept:LeukemiaReferring MD
Call:
800-392-1611 (in U.S.A.) 713-792-6161 (outside U.S.A.)
Phone:713-792-5630
Contact us about clinical trials
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General Information
Disease Group:LeukemiaSupported By:Merck Sharp & Dohme Corp., a Subsidiary of Merck & Co., Inc.
Phase of Study:Phase IReturn
Visit:
Treatment
Agents:
MK-8242Home Care:
Treatment Loc:Both at MDACC & and Other Sites
Estimated
Length of Stay
in Houston:
Description/
Intervention:
The goal of this genetic sub-study is to look at how people's genes may
influence if and how well the study drug may affect the disease. This is
called pharmacogenetic (PGt) testing.

The genetic research in this sub-study involves looking at samples of DNA (the
genetic material of cells) and bone marrow samples.
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Study Objectives / Outcomes
Primary Trial Objectives:
Arm A MK-8242 Monotherapy, Part 1: dose escalation:

Objective: To establish the preliminary recommended Phase 2 dose (RP2D) for MK-8242 monotherapy based on safety, tolerability, and pharmacokinetics (PK) in subjects with, refractory, or recurrent acute myelogenous leukemia (AML).

Hypothesis: There is a dose of MK-8242 that can be administered orally as a single agent on a twice daily schedule for 7-days on and 7-days off repeated every 28 days for up to four cycles to subjects with refractory, or recurrent AML with acceptable tolerability and PK.

Arm A MK-8242 Monotherapy: Part 2: dose confirmation/expansion:

Objective: To evaluate the complete remission (CR) and complete remission with incomplete marrow recovery (CRi) rate of MK-8242 as a single agent administered orally at the RP2D in subjects with refractory, or recurrent wild type AML and p53 WT status.

Hypothesis: At the RP2D MK-8242 administered orally as a single agent will demonstrate efficacy as indicated by CR and CRi rates in refractory, or recurrent AML subjects with p53 WT tumor status.

Arm B MK-8242 Combination with Chemotherapy, Part 1 dose escalation:

Objective: To establish the preliminary RP2D for MK-8242 in combination with cytarabine based on safety, tolerability, and PK in subjects with recurrent AML.

Hypothesis: There is a dose of MK-8242 that can safely be administered on a twice daily oral dosing schedule, Days 1-7 and Days 15-21 in combination with intravenous (IV) cytarabine chemotherapy concurrent on Days 1-4 during a 28-day cycle for up to four cycles to subjects with recurrent AML with acceptable tolerability and PK.

Arm B MK-8242 Combination with Chemotherapy, Part 2 dose confirmation/expansion:

Objective: To evaluate the CR and CRi rate of MK-8242 in combination with cytarabine chemotherapy administered at the RP2D in subjects with recurrent and p53 WT status.

Hypothesis: At the RP2D MK-8242 administered orally in combination with cytarabine will demonstrate efficacy as indicated by CR and CRi rates in recurrent AML subjects with p53 WT tumor status.

Secondary Trial Objectives:
Parts 1 and 2: To evaluate MK-8242 alone and in combination with cytarabine for the following:
1) PK profile.
2) Pharmacodynamic (PD) endpoints such as p21 fold induction in bone marrow blasts and blood.
3) Responses defined as complete response (CR) and complete response with incomplete platelet
recovery (Cri) in Cheson et al(3) at dose levels other than RP2D.
4) Response duration.
5) Responses that do not meet the specified criteria of CR or CRi.

Exploratory Trial Objectives:
Parts 1 and 2:
1) Explore the relationship between MK-8242 PK and PD in whole blood and bone marrow.
2) Correlate predictive biomarkers with response; evaluate performance of additional PD biomarkers.
3) Correlate pharmacogenetic (PGt) relationships with PK/PD, response, and disease.
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Study Status Information
Study Activation / Registration Date:04/30/2012
IRB Review and Approval Date:04/30/2012
Study Type:Phase I
Recruitment Status:Open
Projected Accrual:120-180
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Enrollment Eligibility
If you do not meet the enrollment eligibility, there may be other treatment options for you. Please Contact the Referral Office for more information.

Inclusion Criteria:1) Each subject (or their legal representative) must be willing and able to provide written informed consent for the trial.

2) A subject must comply with either: a. For Arm A (monotherapy), *Part 1 (dose escalation): A subject must have refractory, or recurrent acute AML be >/= 18 years of age and, in the opinion of the Investigator, is not an appropriate candidate for standard therapy. b. For Arm A (monotherapy), Part 2 (dose confirmation/cohort expansion): A subject must have refractory, or recurrent AML, be >/= 18 years of age, not be, in the opinion of the Investigator, an appropriate candidate for standard therapy and have p53 WT tumor status. c. For Arm B (combination therapy), *Part 1 (dose escalation): A subject must have recurrent AML having achieved a CR or CRi of 6-12 months duration following initial therapy and be >/= 18 years old and less than 70 years old.

3) Continued from 2: d. For Arm B (combination therapy), Part 2 (cohort expansion): A subject must have recurrent AML having achieved a CR or CRi of 6-12 months duration from initial therapy, and >/= 18 years old and less than 70 years old, and have AML and p53 WT tumor status. * p53 WT status will be a required enrichment criterion beginning with the cohorts in the 3+3 escalation of Part 1 but may be performed sooner pending assay availability.

4) Subjects must have: a) Eastern Cooperative Oncology Group (ECOG) performance status of 0, 1, or 2 for those subjects selected for Arm A of the study, b) ECOG performance status of 0 or 1 for those subjects selected for Arm B of the study.

5) Subjects are of either sex and any race/ethnicity

6) Each subject must be able to adhere to dose and visit schedules.

7) Women of childbearing potential must have a negative pregnancy test within 72 hours of the first dose of study medication.

8) Female subjects of childbearing potential and male subjects and their partners who are of childbearing potential must agree to abstain from sexual intercourse or to use an acceptable method of contraception during the study and for 90 days following the last dose of study therapy.

9) Each subject must have adequate organ function within 72 hours of Cycle 1 Day 1 of treatment defined by: a. Creatinine <1.5 upper limit of normal (ULN) or calculated creatinine clearance >/= 60mL/min b. Total bilirubin <1.5 x ULN or direct bilirubin < ULN for subjects with total bilirubin levels > 1.5 ULN c. Aspartate aminotransferase (AST)/serum glutamic-oxalecetic transaminases (SGOT) and alanine aminotransferase (ALT)/serum glutamic-pyruvic transaminases (SGPT) <3 x upper limit of normal range (ULN) d. If alkaline phosphatase > 2.5 X ULN, then liver fraction should be </= 2.5 X ULN

10) Each subject must have recovered from the effects of any prior surgery, radiotherapy or anti-neoplastic treatment, with the exception of alopecia.

11) For participation in the pharmacogenetic analysis, the subject must be willing to give written informed consent for the pharmacogenetic testing and able to adhere to dose and visit schedules. Note: A subject unwilling to sign the informed consent for pharmacogenetic testing may be included in the trial; however, pharmacogenetic samples must not be obtained.

12) Each subject must be able to swallow, retain, and absorb oral medications and oral nutrition

13) Subjects must follow the appropriate washout period for prohibited treatments (eg.: 48-hour wash-out following the last hydroxyurea dose).

Exclusion Criteria:1) A subject with an active malignancy other than AML.

2) A subject who has known leptomeningeal leukemia requiring intrathecal therapy.

3) For Arm A and B, Part 1 only: a subject with a history of myelodysplastic syndrome (MDS). a. For Arm A and B, Part 2, subjects with AML in the background of MDS can be included (note this is after the 3+3 dose escalation and preliminary MTD determination).

4) A subject with isolated extramedullary leukemia without also meeting bone marrow criteria for acute leukemia.

5) A subject with AML blast crisis of Chronic Myelogenous Leukemia (CML).

6) A subject who has had a bond marrow transplant with active graft-versushost disease (GVHD) or who receives immunosuppressive therapy.

7) A subject with an uncontrolled active infection that requires systemic treatment,

8) A subject with clinically significant (CTCAE v. 4.0 based criteria) hepatitis at Screening, or a subject that is hepatitis C antibody positive, hepatitis B surface antigen positive, or human immunodeficiency virus (HIV) seropositive (subjects with risk factors, or clinically suspected infection should be screened for Hepatitis B/C and/or HIV).

9) A subject with persistent, unresolved, drug-related toxicity CTCAE (common terminology criteria for adverse events) > Grade 2, that is associated with previous treatment, except for alopecia. The inclusion of subjects with persistent neuropathy or hearing loss >/= Grade 2 due to previous treatment requires discussion with the sponsor.

10) A subject who has any clinically significant condition or situation, other than the condition being studied that, in the opinion of the investigator, would interfere with the study evaluations or optimal participation in the study.

11) A female subject who is breast-feeding, pregnant, intends to become pregnant or intends to breast feed during the study, or has a positive pregnancy test at Screening.

12) A subject participating in any other clinical study with a potentially therapeutic agent or who has received another investigational product within 5 half-lives (if the half-life is known) or 28 days (if the half-life is unknown) prior to Day 1 of Cycle 1. If there is a subject whose last dose was <28 days or 5 half-lives as above, but they have recovered from all toxicity, then discuss eligibility with sponsor.

13) A subject with any other medical or psychiatric condition which in the opinion of the investigator might interfere with participation in the trial or interfere with interpretation of the results.

14) A subject who, within the past 6 months, has had any of the following: myocardial infarction, coronary/peripheral artery bypass graft, cerebrovascular accident, transient ischemic attack, or uncontrolled seizure disorder (i.e., seizures within the past 6 months).

15) A subject who, at the time of Screening, presents with: unstabel or uncontrolled angina, New York Heart Association (NYHA) Class III or IV congestive heart failure, uncontrolled hypertension, clinically significant cardiac dysrhythmia or clinically significant electrocardiogram (ECG) abnormality.

16) A subject with a known bleeding disorder, e.g. hemophilia or disseminated intravascular coagulopathy or be on anti-coagulation therapy.

17) A subject unlikely to complete the study and appropriate follow-up visits.

18) The subject has received any treatment listed as Prohibited Medications During Screening and While on Treatment more recently than the indicated washout period prior to start of treatment or who must continue to receive treatment with the prohibited medications.

19) Subjects Screening for Arm B only: A subject with a known hypersensitivity to cytarabine.

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Links
Registration Number: NCT01451437
Study Information on Clinical Trials Registry (clinicaltrials.gov)

Other Links:
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Results


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