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Study Summary
No. 2012-0062:.......Leukemia......Jorge Cortes......Leukemia
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Study Summary Title
Study Summary
Number:
2012-0062
Study Title:A Phase 1B Study to Evaluate the Safety and Preliminary Efficacy of Pf-04449913, an Oral Hedgehog Inhibitor, in Combination with Intensive Chemotherapy, Low Dose Ara-C or Decitabine in Patients with Acute Myeloid Leukemia or High-Risk Myelodysplastic Syndrome
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Physician New Patient Referral
Name:Jorge CortesPatients 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:713-794-5783
Contact us about clinical trials
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General Information
Disease Group:LeukemiaSupported By:Pfizer, Inc.
Phase of Study:Phase IReturn
Visit:
Treatment
Agents:
Cytarabine
Daunorubicin
PF-04449913
Home Care:
Treatment Loc:Both at MDACC & and Other Sites
Estimated
Length of Stay
in Houston:
Description/
Intervention:
The goal of the screening part of this clinical research study is find the
group that you will be assigned to in the main study.

The goal of the main part of the study is to learn the highest tolerable dose
of PF-04449913 that can be given with chemotherapy to patients with AML or
high-risk MDS.

PF-04449913 is designed to stop the growth of certain cancer stem cells that
may allow cancer to grow.
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Study Objectives / Outcomes
Primary Objective (Safety Cohorts)
  • To determine the maximum tolerated dose (MTD) and recommended Phase 2 dose (RP2D) of PF-04449913 in combination with low dose ARA-C (LDAC; Arm A), decitabine (Arm B) or cytarabine/daunorubicin (Arm C) when administered to adults with previously untreated Acute Myeloid Leukemia (AML) or high-risk Myelodysplastic Syndrome (MDS).

Secondary Objectives (Safety Cohorts)
  • To assess the safety and tolerability of PF-04449913 when administered in combination with LDAC (Arm A), decitabine (Arm B) or cytarabine/daunorubicin (Arm C) to adults with previously untreated AML or high-risk MDS.
  • To evaluate the pharmacodynamics of PF-04449913 when administered in combination with LDAC (Arm A), decitabine (Arm B) or cytarabine/daunorubicin (Arm C) to adults with previously untreated AML or high-risk MDS.
  • To evaluate the pharmacokinetics of PF-04449913 and the potential for a drug-drug interaction (DDI) between PF-04449913 and LDAC (Arm A), decitabine (Arm B) or cytarabine/daunorubicin (Arm C) when administered to adults with previously untreated AML or high-risk MDS.
  • To assess any preliminary evidence of clinical efficacy of PF-04449913 when administered in combination with LDAC (Arm A), decitabine (Arm B) or cytarabine/daunorubicin (Arm C) to adults with previously untreated AML or high-risk MDS.
  • To characterize the effects of PF-04449913 on QTc interval when administered in combination with LDAC (Arm A), decitabine (Arm B) or cytarabine/daunorubicin (Arm C) to adults with previously untreated AML or high-risk MDS.

Primary Objective (Efficacy Expansion Cohorts)
  • To determine the combined rate of complete remission (CR) and complete remission with incomplete blood count recovery (CRi) of PF-04449913 when administered in combination with LDAC (Arm A), decitabine (Arm B) or cytarabine/daunorubicin (Arm C) to adults with previously untreated AML or high-risk MDS.

Secondary Objectives (Efficacy Expansion Cohorts)
  • To assess clinical efficacy measures (including disease-specific measures) of PF-04449913 when administered in combination with LDAC (Arm A), decitabine (Arm B) or cytarabine/daunorubicin (Arm C) to adults with previously untreated AML or high-risk MDS.
  • To assess the safety and tolerability of PF-04449913 when administered in combination with LDAC (Arm A), decitabine (Arm B) or cytarabine/daunorubicin (Arm C) to adults with previously untreated AML or high-risk MDS.
  • To evaluate the pharmacodynamics of PF-04449913 when administered in combination with LDAC (Arm A), decitabine (Arm B) or cytarabine/daunorubicin (Arm C) to adults with previously untreated AML or high-risk MDS.
  • To evaluate the pharmacokinetics of PF-04449913 when administered in combination with LDAC (Arm A), decitabine (Arm B) or cytarabine/daunorubicin (Arm C) to adults with previously untreated AML or high-risk MDS.
  • To further characterize the effects of PF-04449913 on QTc interval when administered in combination with LDAC (Arm A) or decitabine (Arm B) to adults with previously untreated AML or high-risk MDS.
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Study Status Information
Study Activation / Registration Date:
IRB Review and Approval Date:10/04/2012
Study Type:Other
Recruitment Status:Open
Projected Accrual:175
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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) Patients with AML or RAEB-2 High-Risk MDS who are newly diagnosed according to the WHO 2008 Classification and previously untreated. Eligible patients with AML (arising from an antecedent hematologic disease [AHD]) or MDS may have had one approved prior targeted therapy (eg, azacytadine or decitabine) for the treatment of their prior hematologic disease. The patients may not have had a prior therapy for their AML.

2) AML patients include de-novo AML, AML evolving from MDS or other AHD and AML after previous cytotoxic therapy or radiation (secondary AML). For a diagnosis of AML, a bone marrow blast count of 20% or more is required; For AML defined by cytogenetic aberrations t(8;21), inv(16) or t(16;16) and some cases of erythroleukemia the proportion of bone marrow blasts may be <20%; In AML FAB M6a (erythroid leukemia) >/=30% of non-erythroid cells in the bone marrow must be leukemic blasts; In AML with monocytic or myelomonocytic differentiation, monoblasts and promonocytes, but not abnormal monocytes, are counted as blast equivalents.

3) For a diagnosis of high-risk Myelodysplastic Syndrome RAEB-2 the patient must have 10-19% bone marrow blasts.

4) Men and women >/= 18 years old.

5) ECOG Performance Status 0, 1, or 2.

6) Patients with AML or High-Risk MDS who are age 18 years or older and have one or more of the criteria below are considered "unfit" for intensive chemotherapy and are eligible for Arms A and B only: Age >/= 75 years; ECOG of 2; Serum creatinine >1.3 mg/dL; Severe cardiac disease (eg, LVEF < 45% by multigated acquisition [MUGA] or echocardiography [ECHO] at screening).

7) Patients with AML or high risk MDS who are age 18 to 74 years and have none of the following criteria are considered "fit" for more intensive chemotherapy and are eligible for Arm C only: ECOG of 2; Serum creatinine >1.3 mg/dL; Severe cardiac disease (eg, LVEF < 45% by MUGA or ECHO at screening).

8) Adequate Organ Function as defined by the following: Serum aspartate transaminase (AST) and serum alanine transaminase (ALT) </=3 x upper limit of normal (ULN), or AST and ALT </=5 x ULN if liver function abnormalities are due to underlying malignancy; Total serum bilirubin </=2 x ULN (except patients with documented Gilbert's syndrome); Serum creatinine </=1.5 x ULN or estimated creatinine clearance >/=60 mL/min as calculated using the method standard for the institution.

9) All anti-cancer treatments (unless specified) should be discontinued >/=2 weeks from study entry, for example: targeted chemotherapy, radiotherapy, investigational agents, hormones, anagrelide or cytokines; For control of rapidly progressing leukemia, hydroxyurea or leukapheresis may be used before and for up to 1 week after first dose of PF-04449913 for all three arms of the study; Patients with controlled CNS leukemia (documented by two consecutive assessments of zero blast count in cerebrospinal fluid), and who are still receiving intrathecal (IT) therapy at study entry are considered eligible, and will continue to receive IT therapy.

10) Resolved acute effects of any prior therapy to baseline severity or Grade </=1 CTC AE except for AEs not constituting a safety risk by investigator judgment.

11) Serum/urine pregnancy test (for females of childbearing potential) that is negative within 72 hours prior to initiation of treatment (first dose). Male and female patients of childbearing potential must agree to use a highly effective method of contraception throughout the study and for at least 90 days after the last dose of assigned treatment. A patient is of childbearing potential if, in the opinion of the investigator, he/she is biologically capable of having children and is sexually active.

12) Evidence of a personally signed and dated informed consent document indicating that the patient (or a legal representative) has been informed of all pertinent aspects of the study.

Exclusion Criteria:1) AML M3 Acute Promyelocytic Leukemia (APL) or patients with a t(9:22) cytogenetic translocation.

2) Hyperleukocytosis (leukocytes >/= 30 x 10[9]/L) at study entry. These patients may be treated with hydroxyurea or receive leukocytapheresis treatment according to routine practice, and enrolled in the study when the leukocyte count falls below 30 x 10[9]/L.

3) Patients in whom, at the time of study entry, a stem cell transplant is planned within the next 6 months.

4) Patient is known to be refractory to platelet or packed red cell transfusions per Institutional Guidelines, or a patient who refuses blood product support.

5) Patients with active malignancy with the exception of basal cell carcinoma, non-melanoma skin cancer, cervical carcinoma-in-situ. Patients with history of other cancers should be free of disease for at least 2 years. Other concurrent malignancies will be considered on a case-by-case basis.

6) For Arm C Only: LVEF <45% by ECHO or MUGA scan.

7) Any one of the following currently or in the previous 6 months: myocardial infarction, congenital long QT syndrome, torsades de points or clinically significant ventricular arrhythmias.

8) QTc interval >470 msec using the Fridericia (QTcF) or Bazett (QTcB) correcion methods, whichever is longer.

9) Patients with an active, life threatening or clinically significant uncontrolled systemic infection.

10) Patients with known active uncontrolled central nervous system (CNS) leukemia.

11) Known human immunodeficiency virus (HIV) or acquired immunodeficiency syndrome (AIDS)-related illness or active Hepatitis B or C infection.

12) Known malabsorption syndrome or other condition that may impair absorption of study medication (eg, gastrectomy or lap band).

13) Major surgery or radiation within 4 weeks of starting study treatment.

14) Prior treatment with a Hedgehog inhibitor.

15) Prior treatment with decitabine or azacitidine (Arm B only), cytarabine (Arm A and C only) or daunorubicin/other anthracycline if received maximum allowable dose (Arm C only).

16) The presence of any one of the following: For Arm A and C only: Hypersensitivity to cytarabine (not including drug fever or exanthema); For Arm B: hypersensitivity to decitabine; For Arm C: hypersensitivity to daunorubicin.

17) Concurrent treatment with any investigational or approved oncology agents (unless specified in the protocol).

18) Concurrent administration of herbal preparations.

19) Current use or anticipated need for food or drugs that are known strong/moderate CYP3A4 inhibitors, including their administration within 7-days prior to study entry.

20) Current use or anticipated need for drugs that are known strong CYP3A4 inducers, including their administration within 7-days prior to study entry.

21) Current use or anticipated need for drugs that are CYP3A4 substrates and have a narrow therapeutic index or P-glycoprotein inhibitors/inducers, including their administration within 7-days prior to study entry.

22) Current drug or alcohol abuse.

23) Other severe acute or chronic medical or psychiatric condition or laboratory abnormality that may increase the risk associated with study participation or investigational product administration or may interfere with the interpretation of study results and, in the judgment of the investigator, would make the patient inappropriate for entry into this study.

24) Patients who are investigational site staff members or relatives of those site staff members or patients who are Pfizer employees directly involved in the conduct of the trial.

25) Pregnant females; breastfeeding females; males and females of childbearing potential not using highly effective contraception or not agreeing to continue highly effective contraception for at least 90 days after last dose of investigational product; males and females of childbearing potential not using two (2) methods of highly effective contraception or not agreeing to continue two (2) methods of highly effective contraception for at least 90 days after last dose of investigational product.

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Links
Registration Number: NCT01546038
Study Information on Clinical Trials Registry (clinicaltrials.gov)

Other Links:
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Results


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