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Study Summary
No. 2005-0813:.......Leukemia......Jorge Cortes......Leukemia
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Study Summary Title
Study Summary
Number:
2005-0813
Study Title:A Phase 1/2 Study of SKI-606 in Philadelphia Chromosome Positive Leukemias
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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:N/A
Phase of Study:Phase I/Phase IIReturn
Visit:
Every week for 1 month, then every 4 weeks for 12 weeks, then every 12 weeks
Treatment
Agents:
SKI-606Home Care:SKI-606 is self administered (oral)
Treatment Loc:Independent Multicenter Arrangements
Estimated
Length of Stay
in Houston:
None anticipated
Description/
Intervention:
The goal of this clinical research study is to find the highest tolerable dose
of the drug bosutinib (SKI-606) that can be given in the treatment of CML and
ALL. The safety and effectiveness of this drug will be studied. Researchers
will also perform blood tests to evaluate the tolerability to bosutinib to
monitor the status the leukemia, to learn how the body processes bosutinib, and
to develop better ways to measure the effects of bosutinib on blood or tumor
cells.
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Study Objectives / Outcomes
Part 1 - Dose Escalation Component:

Primary
  • Define the maximum tolerated dose, less than or equal to 1000 mg/day, in subjects with CML in chronic phase resistant or refractory to imatinib
  • Evaluate the overall PK parameters in this population

Secondary
  • Determine the rate of major cytogenetic response in chronic phase subjects at various dose levels of SKI-606
  • Obtain data on the ability of SKI-606 to inhibit phosphorylation of CrkL and BCR-Abl at various dose levels

Exploratory
  • Explore the pharmacogenomic effects of SKI-606 in subjects with chronic phase CML


Part 2 - Efficacy Component:

Primary
  • Determine the rate of attaining major cytogenetic response in subjects entering with imatinib-resistant chronic phase CML, who have no prior Src, Abl, or Src-Abl kinase inhibitor exposure other than imatinib
  • Determine the population PK parameters of this population

Secondary
  • Estimate the time to and duration of major cytogenetic response in subjects entering with imatinib-resistant chronic phase CML, who have no prior Src, Abl, or Src/Abl kinase inhibitor exposure other than imatinib
  • Estimate MCyR rate in CP CML subjects intolerant of imatinib, who have no prior Src, Abl, or Src/Abl kinase inhibitor exposure other than imatinib
  • Estimate the time to and duration of major cytogenetic response in CP CML subjects intolerant of imatinib, who have no prior Src, Abl, or Src/Abl kinase inhibitor exposure other than imatinib
  • Estimate the time to and duration of CHR in the imatinib-resistant and imatinib-intolerant groups
  • Estimate MCyR rate in CP CML subjects who have failed imatinib and are resistant to other tyrosine kinase inhibitors (dasatinib or nilotinib)
  • Estimate MCyR rate in CP CML subjects who have failed imatinib and are resistant to dasatinib
  • Estimate the overall survival (OS) and progression free survival (PFS) rates at 1 and 2 years
  • Estimate CHR rate in advanced leukemia (AP CML, BP CML, Ph+ ALL) subjects
  • Estimate OHR rate in imatinib-resistant accelerated phase and blast phase CML subjects
  • Assess the safety of SKI-606 during prolonged oral exposure in a leukemic population

Exploratory
  • Estimate the rate of molecular responses in those whose best prior response was CCyR and cytogenetic responses in those previously attaining only CHR.
  • Define PD effects of SKI-606 on activation of Src-family kinases and downstream substrates
  • Evaluate health outcomes endpoints by administering quality of life questionnaires
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Study Status Information
Study Activation / Registration Date:01/11/2006
IRB Review and Approval Date:11/16/2005
Study Type:Therapeutic
Recruitment Status:Open
Projected Accrual:530
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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) Signed and dated institutional review board (IRB) or independent ethics committee (IEC)-approved informed consent form before any protocol-specific screening procedures.

2) Cytogenetic or PCR based diagnosis of any phase of Ph+ CML or Ph+ ALL whose disease is resistant to full-dose imatinib (>/=600 mg), or are intolerant of any dose of imatinib.

3) Adequate duration of prior imatinib therapy.

4) ECOG Performance Status of 0 or 1 for Chronic Phase subjects, and 0, 1 or 2 for Advanced Stage Subjects.

5) No anti-proliferative treatment or anti-leukemia treatment within 7 days of the first dose of SKI-606 (except Hydroxyurea & Anagrelide).

6) Recovered to Grade 0-1, or to baseline, from any toxicities of prior treatment, other than alopecia.

7) At least 3 months post allogeneic stem cell transplantation.

8) Able to take daily oral capsules or tablets reliably.

9) Adequate bone marrow function (Chronic Phase patients with a history of imatinib resistance only): a) Absolute neutrophil count > 1000/mm(3) [>1 x 10(9)/L]; b) Platelets >/= 100,000/mm(3) [>100 x 10(9)/L] absent any platelet transfusions during the preceding 14 days.

10) Adequate hepatic, and renal function: a) AST/ALT </= 2.5 x upper limit of normal (ULN) or </= 5 x ULN if attributable to liver involvement of leukemia; b) Total bilirubin </= 1.5 x ULN; c) Creatinine </= 1.5 x ULN.

11) Age >/= 18 years.

12) Willingness of male and female subjects, who are not surgically sterile or postmenopausal, to use reliable methods of birth control (oral contraceptives, intrauterine devices, or barrier methods used with a spermicide) for the duration of the study and for 30 days after the last dose of SKI-606.

13) Documented normal INR if not on oral anticoagulant therapy (OAT), or, if on OAT consistent target INR </= 3.

Exclusion Criteria:1) Subjects with Philadelphia chromosome, and BCR-abl negative CML.

2) Subjects previously intolerant of imatinib - Part 1 (dose escalation only).

3) Overt leptomeningeal leukemia. Subjects must be free of CNS involvement for a minimum of 2 months. Subjects with symptoms of CNS involvement must have a diagnositc lumbar puncture prior to study enrollment.

4) Subjects with extramedullary disease only.

5) In part 1, no prior exposure to Src, Abl, or Src/Abl kinase inhibitors is allowed.

6) Ongoing requirement for warfarin or other OAT (Part 1 only).

7) Ongoing requirement for hydroxyurea or anagrelide (Part 1 only).

8) Graft versus Host Disease (GVHD): a) Part 1 - no previous GVHD allowed; b) Part 2 - no treated or untreated GVHD within 60 days of study start.

9) Major sugery within 14 days or radiotherapy within 7 days before the first dose of SKI-606 (recovery from any previous surgery should be complete before day 1).

10) Ongoing clinical requirement for administration of a strong inhibitor CYP-3A4 (part 1 only)

11) History of clinically significant or uncontrolled cardiac disease including: history of or active congestive heart failure; uncontrolled angina or hypertension within 3 months; myocardial infarction (within 12 months); clinically significant ventricular arrhythmia (such as ventricular tachycardia, ventricular fibrillation, or Torsades de pointes); diagnosed or suspected congenital or acquired prolonged QT syndrome; unexplained syncope; history of prolonged QTc.

12) Prolonged QTc (>0.45 sec, average of triplicate readings at screening).

13) Concomitant use of or need for medications known to prolong the QT interval.

14) Uncorrected hypomagnesemia or hypokalemia due to potential effects on the QT interval.

15) Recent (within 30 days of study entry) or ongoing clinically significant gastrointestinal disorder (e.g., malabsorption, short bowel syndrome, bleeding, or Grade > 1 diarrhea, nausea or emesis lasting more than 2 days, despite adequate medical therapy).

16) Pregnant or breastfeeding women.

17) Evidence of serious active infection, significant medical or psychiatric illness.

18) Known seropositivity to HIV, or current acute or chronic Hepatitis B or Hepatitis C (antigen positive), cirrhosis, or clinically significant abnormal laboratory finding that would, in the investigator's judgment, make the subject inappropriate for this study.

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Links
Registration Number: NCT00261846
Study Information on Clinical Trials Registry (clinicaltrials.gov)

Other Links:
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Results


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