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Study Summary
No. 2012-0054:.......Leukemia......Jorge Cortes......Leukemia
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Study Summary Title
Study Summary
Number:
2012-0054
Study Title:A Phase 2, Randomized, Open-Label Study of the Safety and Efficacy of Two Doses of Quizartinib (AC220; ASP2689) in Subjects with FLT3-ITD Positive Relapsed or Refractory Acute Myeloid Leukemia (AML)
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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:Astellas Pharma Global Development, Inc
Phase of Study:Phase IIReturn
Visit:
Treatment
Agents:
AC220Home Care:
Treatment Loc:Both at MDACC & and Other Sites
Estimated
Length of Stay
in Houston:
Description/
Intervention:
The goal of this clinical research study is to compare the safety and
effectiveness of 2 doses of quizartinib in people with relapsed or refractory
AML.

The study drug is designed to block the FLT3 receptor on leukemia cells to stop
the chemical reaction that happens when FLT3 is activated. This may slow or
stop cancer cell division, and may lead to cancer cell death. Quizartinib may
also work against other receptors that are involved in leukemia cell division
and growth.
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Study Objectives / Outcomes
The primary objectives are to:
  • Evaluate the composite complete remission rate (CRc) and to evaluate the rate of Grade 2 or higher QTcF prolongation at different doses of AC220

The secondary objectives are to:

  • Characterize the pharmacokinetics (PK) of AC220 and the active metabolite AC886
  • Evaluate the electrocardiogram (ECG) effects of AC220 in relation to plasma drug concentration
  • Evaluate the CR rate, overall survival (OS), event free survival (EFS), leukemia free survival (LFS), duration of remission, time to treatment response (TTR) and transplantation rate, i.e. percentage of patients undergoing a hematopoietic stem cell transplant (HSCT)
  • Assess overall safety

The exploratory objectives are to:

  • Evaluate the pharmacodynamic (PD) effect of AC220
  • Evaluate the mechanism of acquired resistance to AC220
  • Evaluate the PK-PD relationships
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Study Status Information
Study Activation / Registration Date:05/17/2012
IRB Review and Approval Date:05/17/2012
Study Type:Phase Ii Or Phase I/Ii
Recruitment Status:Closed
Projected Accrual:64
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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) Subject has provided an Institutional Review Board (IRB)/Independent Ethics Committee (IEC)-approved signed Informed Consent and privacy disclosure - as per national regulations (e.g., HIPAA Authorization for U.S. sites) prior to any study-related procedures (including withdrawal of prohibited medication, if applicable).

2) Subject is male or female >/=18 years of age.

3) Subject has morphologically documented primary AML or AML secondary to myelodysplastic syndrome (MDS) as defined by the World Health Organization (WHO) criteria, as determined by pathology review at the treating institution and has relapsed or is refractory after 1 second line (salvage) regimen or after HSCT.

4) Subject is positive for FLT3-ITD activating mutation in bone marrow or peripheral blood. The subject may be randomized based off local lab results. In addition, a sample will be sent to the central lab for confirmation (>10% allelic ratio). However, if the central lab result differs from local lab, the subject will be allowed to remain in the study.

5) ECOG performance status of 0 to 2.

6) In the absence of rapidly progressing disease clearly documented by the Investigator, the interval from prior treatment to time of AC220 administration will be at least 2 weeks (14 days) for prior cytotoxic agents or at least 5 half-lives for prior noncytotoxic agents, including immunosuppressive therapy post HSCT.

7) Persistent chronic clinically significant nonhematological toxicities from prior treatment (including chemotherapy, kinase inhibitors, immunotherapy, experimental agents, radiation, HSCT, or surgery) must be Grade </= 1.

8) Patients - both males and females - with reproductive potential are eligible if the following criteria is met: Female subject is: Post-menopausal (defined as at least 2 years without menses) prior to Screening visit; or surgically sterile (at least 1 month prior to Screening visit; or of childbearing potential with contraception. Female subject of childbearing potential has a negative pregnancy test at the Screening visit and agrees to use contraception consisting of two forms of birth control (one of which must be a barrier method) throughout the study period. Male subject with partner(s) of childbearing potential must agree to use contraception consisting of two forms of birth control (one of which must be a barrier method) and agrees to no sperm donation throughout the study period.

9) Subject must have adequate renal, hepatic, and coagulation parameters as indicated by the following laboratory values: Aspartate aminotransferase (AST), and alanine aminotransferase (ALT) </= 2.5 x institutional upper limit of normal (ULN); Total bilirubin </= 1.5 x institutional ULN; Serum creatinine </= 1.5 x institutional ULN and glomerular filtration rate (GFR) > 30 mL/min (calculated by Cockcroft and Gault formula).

10) Subject is able to comply with study procedures and follow-up examinations.

Exclusion Criteria:1) Subject received previous treatment with AC220.

2) Subject has diagnosis of acute promyelocytic leukemia.

3) Subject has diagnosis of chronic myelogenous leukemia (CML) in blast crisis.

4) Subject has AML or antecedent MDS secondary to prior chemotherapy.

5) Subject has had HSCT and has either of the following: Is within 100 days of transplant; Is still taking immunosuppressive drugs; Has clinically significant graft-versus-host disease requiring treatment; Has Grade > 1 persistent nonhematological toxicity related to the transplant. Donor lymphocyte infusion (DLI) is not permitted during the study or </= 30 days prior to study entry.

6) Subject has clinically active CNS leukemia. A subject is considered eligible if CNS leukemia is controlled and subject is receiving intrathecal ( IT) therapy at study entry. Subjects should continue to receive IT therapy (or cranial radiation) as clinically indicated.

7) Subject has received concurrent chemotherapy, immunotherapy, or radiotherapy within 14 days prior to the first dose of AC220, or any ancillary therapy that is considered to be investigational (i.e., used for non-approved indications(s) and in the context of a research investigation) within 30 days or 5 half-lives (whichever is longer) prior to the first dose of study drug.

8) Subject requires treatment with concomitant drugs that prolong QT/QTc interval or with strong inhibitors or inducers of cytochrome P450-isozyme3A4 (CYP3A4) with the exception of antibiotics, antifungals, and antivirals that are used as standard of care post-transplant or to prevent or treat infections and other such drugs that are considered absolutely essential for the care of the subject.

9) Subject requires treatment with anticoagulant therapy.

10) Subject has a known positive test for human immunodeficiency virus, hepatitis C, or hepatitis B surface antigen.

11) Subject had major surgery within 4 weeks prior to first dose of AC220.

12) Subject has uncontrolled or significant cardiovascular disease, including: A myocardial infarction within 12 months prior to the start of study drug; Uncontrolled angina within 6 months prior to the start of study drug; History of congestive heart failure (CHF) New York Heart Association (NYHA) class 3 or 4. If a screening echocardiogram (ECHO) or Multiple Gate Acquisition Scan (MUGA) is performed within 1 month prior to, or during study screening and the result is a left ventricular ejection fraction (LVEF) that is >/= 45% (or MDACC lower limit of normal value which is 50%) then this is not exclusionary; Heart rate < 50 beats per minute at Screening ECG; Diagnosed or suspected congenital long QT syndrome; Known family history of congenital long QT syndrome; QTc interval calculated by Fridericia's correction factor (QTcF) at Screening >/= 450 ms. The QTcF will be derived from the average QTcF in triplicate;

13) **continued from above: Any history of second or third degree heart block; Uncontrolled hypertension defined as systolic blood pressure >/= 180 mmHg or diastolic blood pressure >/= 110 mmHg; Obligate need for a cardiac pacemaker or defibrillator; Complete left bundle branch block; Atrial fibrillation documented within 2 weeks prior to first dose of study drug; or History of arrhythmia (multifocal premature ventricular contractions, bigeminy, trigeminy, ventricular tachycardia) that was symptomatic or required treatment (CTCAE Grade 3).

14) Subject has a pre-existing disorder predisposing the subject to a serious or life-threatening infection (e.g. cystic fibrosis, congenital or acquired immunodeficiency, bleeding disorder, or cytopenias).

15) Subject has an active uncontrolled acute or chronic systemic fungal, bacterial, viral, or other infection.

16) Subject has any of the following laboratory values: Serum potassium < 4.0 mmol/L despite supplementation, or > 5.5 mmol/L; Serum magnesium below the institutional normal limit despite supplementation, or > 3 mg/dL (1.23 mmol/L); Serum calcium > 11.5 mg/dL (2.9 mmol/L) or ionized calcium > 1.5 mmol/L.

17) Subject is a woman of childbearing potential (WOCBP) or a male subject with female partner of childbearing potential who is unwilling or unable to use an acceptable contraceptive method to avoid pregnancy for the entire study period and for at least 3 months after the last dose of study drug.

18) Subject is a female with a positive pregnancy test, pregnant, or breastfeeding.

19) Subject has any medical, psychiatric, addictive or other kind of disorder which compromises the ability of the subject to give written informed consent and/or to comply with procedures.

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Links
Registration Number: NCT01565668
Study Information on Clinical Trials Registry (clinicaltrials.gov)

Other Links:
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Results


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