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Study Summary
No. 2004-0251:.......Leukemia......Hagop Kantarjian......Leukemia
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Study Summary Title
Study Summary
Number:
2004-0251
Study Title:A phase IA/II multicenter, dose-escalation study of oral AMN107 on a continuous daily dosing schedule in adult patients with GleevecŪ(imatinib)-resistant/intolerant CML in chronic or accelerated phase or blast crisis, relapsed/refractory Ph+ ALL, and other hematologic malignancies
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Physician New Patient Referral
Name:Hagop KantarjianPatients 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:713-792-8217
Contact us about clinical trials
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General Information
Disease Group:LeukemiaSupported By:N/A
Phase of Study:Phase I/Phase IIReturn
Visit:
Patients who have been on study for a minimum of 3 cycles, will be allowed to
have visits, which are not response or confirmation of response visits,
performed by a local physician. Patients will return to MDACC every 6 months.
Treatment
Agents:
AMN 107Home Care:Patients who have been on study for a minimum of 3 cycles, will be allowed to
have visits, which are not response or confirmation of response visits,
performed by a local physician. Patients will receive 6 months of study drug
Treatment Loc:Independent Multicenter Arrangements
Estimated
Length of Stay
in Houston:
N/A
Description/
Intervention:
The overall goal of this study is to look at the safety of AMN107 and to
further study the anti-cancer effects of AMN107. In addition, the study will
look at the internal workings of a cancer cell to find out how AMN107 effects
the abnormal way cancer cells grow.

The study is divided into two parts. The goal of the first part of the study
(Phase I) was to find the highest safe dose of AMN107 that can be given to
treat hematologic (blood related) diseases. Doses have been given once or twice
a day to find the highest dose of AMN107 that can be given without causing
unacceptable side effects.

The second part of the study (Phase II) will test how well AMN107 reduces the
growth of the tumor, and to further study the safety and anti-cancer effects of
AMN107. In addition, the study will look at the internal workings of a cancer
cell to find out how AMN107 effects the abnormal way cancer cells grow.
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Study Objectives / Outcomes
Primary Objectives:
Phase I
1. To determine the maximum tolerated dose (MTD) and dose-limiting toxicity (DLT) of AMN107 as a single agent when administered as an oral once-daily dose to adult patients with imatinib-resistant CML in accelerated or chronic phase or blast crisis, or relapsed/refractory Ph+ ALL
2. To determine the maximum-tolerated dose (MTD) and dose-limiting toxicity (DLT) of AMN107 as a single agent when administered orally on a twice-daily dosing schedule to adult patients with imatinib-resistant CML in accelerated or chronic phase or blast crisis, or relapsed/refractory Ph+ ALL
3. To characterize the pharmacokinetic profile of AMN107 in plasma serum and, where samples are available, in tumor cells and normal hematopoietic cells

Phase II

Phase II CML
To evaluate the efficacy and safety of AMN107 in patients with imatinib-resistant or intolerant CML-BC, imatinib-resistant or intolerant CML-AP and imatinib-resistant or intolerant CML-CP.

Phase II Ph + ALL

To evaluate safety and preliminary anticancer activity of AMN107 in relapsed/refractory patients with Ph+ ALL.

Phase II HES/CEL, and SM

To evaluate safety and preliminary anticancer activity of AMN107 in patients with HES/CEL and systemic mastocytosis.

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Study Status Information
Study Activation / Registration Date:06/08/2004
IRB Review and Approval Date:05/05/2004
Study Type:Therapeutic
Recruitment Status:Closed
Projected Accrual:859-907
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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) PHASEI: Patients with a cytopathologically confirmed diagnosis of Ph+ ALL who are either relapsed after or refractory to standard therapy, or patients with CML in blast crisis, or CML patients in accelerated or chronic phase who are resistant to imatinib. Patients with Ph+ ALL who have minimal residual disease (MRD) following stem cell transplantation may only be enrolled during the dose escalation portion of the study unless there is evidence of MRD indicating evolving relapse.

2) Age >/= 18 years old.

3) WHO Performance Status of </= 2.

4) Patients must have the following laboratory values: Cytoreductive therapy with vincristine or cyclophosphamide is permitted up to 7 days prior to first administration of AMN107. Corticosteroids can be administered up to 48 hours prior to first administration of AMN107. Hydroxyurea is permitted as clinically indicated during the dose escalation phase.

5) Potassium greater than or equal to LLN (lower limit of normal) or correctable with supplements.

6) Total calcium (corrected for serum albumin) greater than or equal to LLN or correctable with supplements.

7) Magnesium greater than or equal to LLN or correctable with supplements.

8) Phosphorus greater than or equal to LLN or correctable with supplements.

9) ALT and AST less than equal to 2.5 x ULN or less than or equal to 5.0 x ULN if considered due to tumor.

10) Alkaline phosphatase less than or equal to 2.5 x ULN.

11) Serum bilirubin less than or equal to 1.5 x ULN.

12) Serum creatinine less than or equal to 1.5 x ULN or 24-hour creatinine clearance greater than or equal to 50 ml/min.

13) Written informed consent.

14) PHASE II: Patients under consideration for participation in this study must meet one of the following disease inclusion criteria as defined in 1, 2, 3, 4, 5, and 6.

15) Imatinib resistant or intolerant Philadelphia chromosome-positive CML in blast crisis defined as at least 30% blasts in peripheral blood or bone marrow or extramedullary disease other than liver or spleen.

16) Imatinib resistant or intolerant Philadelphia chromosome-positive CML patients in accelerated phase defined as never in blast crisis before starting treatment, with one or more of the following criteria present within 4 weeks prior to beginning treatment: >/= 15% but <30% blasts in blood or bone marrow; >/=30% blasts plus promyelocytes in peripheral blood or bone marrow (providing that <30% blasts present in bone marrow); peripheral basophils >/=20%; thrombocytopenia <100 X 10^9/L unrelated to therapy.

17) Imatinib resistant or intolerant Philadelphia chromosome-positive CML in chronic phase defined as never in blast crisis or accelerated phase before starting treatment and the presence of the following criteria: < 15% blasts in peripheral blood and bone marrow; < 30% blasts plus promyelocytes in peripheral blood and bone marrow; < 20% basophils in the peripheral blood; >/= 100 x 10^9/L (>/= 100,000 /mm^3) platelets; No evidence of extramedullary leukemic involvement, with the exception of liver or spleen.

18) Relapsed or refractory Ph+ ALL: Patients with Ph+ ALL who have minimal residual disease (MRD) are eligible only if there is indication of evolving relapse defined as a >/= 2 log increase of Bcr-Abl transcript level (as reported by local laboratories), as compared to the minimum level achieved with prior therapy in peripheral blood; Patients with Ph+ALL whose disease exhibits features of biphenotypic acute leukemia are eligible.

19) Hypereosinophilic syndrome/chronic eosinophilic leukemia who have a clinical indication for treatment and meet the following criteria: eosinophilia greater than 1500/mm^3 for at least 6 months; exclusion of other causes of eosinophilia including clonal or abnormal T-cell populations, exclusion of reactive eosinophilia, and malignancies or T-cell disorders associated with eosinophilia.

20) Systemic mastocytosis who have a clinical indication for treatment and meet at least one major and one minor or three minor criteria: Major criterion: Multifocal dense infiltrates of mast cells (>15 mast cells in aggregates) in bone marrow biopsies and/or in sections of other extracutaneous organ(s). Minor criteria: >25% of all mast cells are atypical cells (type I or type II) on bone marrow smears or are spindle-shaped in mast cell infiltrates detected on sections of visceral organs; c-kit point mutation at codon 816 in the bone marrow or another extracutaneous organ,

21) #22 continued: Mast cells in bone marrow or blood or another extracutaneous organ express CD2 or/and CD25; Baseline serum tryptase concentration > 20 ng/ml (in the case of an unrelated myeloid neoplasm, is not valid as an SM criterion).

22) Prior imatinib therapy for patients with Ph+ ALL, HES/CEL and SM is permitted but is not required.

23) CML patients who have been treated with an investigational tyrosine kinase inhibitor who otherwise meet the definition of imatinib-resistance or intolerance are eligible.

24) The following inclusion criteria are mandatory for all patients:

25) Males or females >/= 18 years of age

26) WHO Performance Status of </= 2

27) Patients must have the following laboratory values:

28) Potassium >/= LLN (lower limit of normal) or corrected to within normal limits with supplements prior to the first dose of study medication

29) Total calcium (corrected for serum albumin) >/= LLN or correctable with supplements

30) Magnesium >/= LLN or corrected to within normal limits with supplements prior to the first dose of study medication

31) Phosphorus >/= LLN or correctable with supplements

32) ALT and AST </= 2.5 x ULN or </= 5.0 x ULN if considered due to tumor

33) Alkaline phosphatase </= 2.5 x ULN unless considered due to tumor

34) Serum bilirubin </= 1.5 x ULN

35) Serum creatinine </= 1.5 x ULN or 24-hour creatinine clearance >/= 50 ml/min

36) Serum amylase </= 1.5 x ULN and serum lipase </= 1.5 x ULN

37) Written informed consent prior to any study procedures being performed.

Exclusion Criteria:1) PHASE I: Cytopathologically confirmed CNS infiltration NB: in absence of suspicion of CNS involvement, lumbar puncture is not required.

2) Impaired cardiac function, including any one of the following

3) LVEF < 45% or below institutional lower limit of the normal range (which ever is higher) as determined by MUGA scan or echocardiogram.

4) Complete left bundle branch block.

5) Use of a cardiac pacemaker.

6) ST depression of > 1mm in 2 or more leads and/or T wave inversions in 2 or more contiguous leads.

7) Congenital long QT syndrome.

8) History of or presence of significant ventricular or atrial tachyarrhythmias.

9) Clinically significant resting bradycardia (< 50 beats per minute).

10) QTc > 450 msec on screening ECG (using the QTcF formula)

11) Right bundle branch block plus left anterior hemiblock, bifascicular block.

12) Myocardial infarction within 3 months prior to starting AMN107.

13) Unstable angina diagnosed or treated during the past 12 months.

14) Other clinically significant heart disease (e.g., congestive heart failure, uncontrolled hypertension, history of labile hypertension, or history of poor compliance with an antihypertensive regimen).

15) Impairment of gastrointestinal (GI) function or GI disease that may significantly alter the absorption of AMN107 (e.g., ulcerative diseases, uncontrolled nausea, vomiting, diarrhea, malabsorption syndrome, or small bowel resection).

16) Use of therapeutic warfarin.

17) Acute or chronic liver or renal disease considered unrelated to tumor.

18) Other concurrent severe and/or uncontrolled medical conditions (e.g., uncontrolled diabetes, active or uncontrolled infection) that could cause unacceptable safety risks or compromise compliance with the protocol.

19) Treatment with any hematopoietic colony-stimulating growth factors (e.g., G-CSF, GM-CSF)less than or equal to 1 week prior to starting study drug. Erythropoietin is allowed.

20) Patients who are currently receiving treatment with any of the medications listed in post-text supplement 4 Amendment 8 and the treatment cannot be either discontinued or switched to a different medication prior to starting study drug. The medications listed in post-text supplement 4 Amendment 8 have the potential to prolong the QT interval or are CYP3A4 inhibitors.

21) Patients who have received chemotherapy less than or equal to 1 week (6 weeks for nitrosurea or mitomycin-C) or who are within 5 half-lives of their last dose chemotherapy dose prior to starting study drug or who have not recovered from side effects of such therapy.

22) Patients who have received imatinib less than or equal to 1 week or who have not recovered from side effects of such therapy.

23) Patients who have received immunotherapy less than or equal to 1 week prior to starting study drug or who have not recovered from side effects of such therapy.

24) Patients who have received any investigational drug less than or equal to 4 weeks or investigational cytotoxic agent within 1 week (or who are within 5 half-lives of a previous investigational cytotoxic agent) prior to starting study drug or who have not recovered from side effects of such therapy.

25) Patients who have received wide field radiotherapy less than or equal to 4 weeks or limited field radiation for palliation < 2 weeks prior to starting study drug or who have not recovered from side effects of such therapy.

26) Patients who have undergone major surgery less than or equal to 2 weeks prior to starting study drug or who have not recovered from side effects of such therapy.

27) Patients who are pregnant or breast feeding, or adults of reproductive potential not employing an effective method of birth control. (Women of childbearing potential must have a negative serum pregnancy test within 48 hrs prior to administration of AMN107). Post menopausal women must be amenorrheic for at least 12 months to be considered of non-childbearing potential. Male and female patients must agree to employ an effective barrier method of birth control throughout the study and for up to 3 months following discontinuation of study drug.

28) Known diagnosis of human immunodeficiency virus (HIV) infection (HIV testing is not mandatory).

29) Patients with a history of another primary malignancy that is currently clinically significant or currently requires active intervention.

30) Patients unwilling or unable to comply with the protocol.

31) PHASE II: Cytopathologically confirmed CNS infiltration (in absence of suspicion of CNS involvement, lumbar puncture is not required).

32) Impaired cardiac function, including any one of the following: · LVEF < 45% as determined by MUGA scan or echocardiogram · Complete left bundle branch block · Use of a cardiac pacemaker · ST depression of > 1mm in 2 or more leads and/or T wave inversions in 2 or more contiguous leads · Congenital long QT syndrome · History of or presence of significant ventricular or atrial tachyarrhythmias · Clinically significant resting bradycardia (< 50 beats per minute) · QTc > 480 msec on screening ECG (using the QTcF formula) ·

33) #32 Continued Right bundle branch block plus left anterior hemiblock, bifascicular block · Myocardial infarction within 3 months prior to starting AMN107 · Uncontrolled angina pectoris · Other clinically significant heart disease (e.g., congestive heart failure, uncontrolled hypertension, history of labile hypertension, or history of poor compliance with an antihypertensive regimen).

34) Impairment of gastrointestinal (GI) function or GI disease that may significantly alter the absorption of AMN107 (e.g., ulcerative diseases, uncontrolled nausea, vomiting, diarrhea, malabsorption syndrome, or small bowel resection).

35) Use of therapeutic warfarin.

36) Acute or chronic liver or renal disease considered unrelated to tumor.

37) Other concurrent severe and/or uncontrolled medical conditions (e.g., uncontrolled diabetes, active or uncontrolled infection) that could cause unacceptable safety risks or compromise compliance with the protocol.

38) Treatment with any hematopoietic colony-stimulating growth factors (e.g., G-CSF, GM-CSF) </= 1 week prior to starting study drug. Erythropoietin is allowed.

39) Patients who are currently receiving treatment with any of the medications listed in Post-text supplement 4 and the treatment cannot be either discontinued or switched to a different medication prior to starting study drug. The medications listed in Post-text supplement 4 have the potential to prolong the QT interval.

40) Patients who have received chemotherapy </= 1 week or who are within 5 half-lives of their last dose of chemotherapy (6 weeks for nitrosurea or mitomycin-C) prior to starting study drug or who have not recovered from side effects of such therapy. Hydroxyurea is permitted at the investigators discretion prior to enrollment. Patients who have received imatinib </= 5 days prior or who have not recovered from side effects of such therapy.

41) Patients who have received immunotherapy </=1 week prior to starting study drug or who have not recovered from side effects of such therapy.

42) Patients who have received any investigational drug </= 4 weeks or investigational cytotoxic agent within 1 week (or who are within 5 half-lives of a previous investigational cytotoxic agent) prior to starting study drug or who have not recovered from side effects of such therapy.

43) Patients who have received wide field radiotherapy </= 4 weeks or limited field radiation for palliation < 2 weeks prior to starting study drug or who have not recovered from side effects of such therapy.

44) Patients who have undergone major surgery </= 2 weeks prior to starting study drug or who have not recovered from side effects of such therapy.

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Criteria truncated, please contact Prinicipal Investigator's office for full criteria

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Links
Registration Number: NCT00109707
Study Information on Clinical Trials Registry (clinicaltrials.gov)

Other Links:
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Results


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