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Study Summary
No. 2011-0382:.......Leukemia; Lymphoma......Patrick Zweidler-McKay......Leukemia
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Study Summary Title
Study Summary
Number:
2011-0382
Study Title:Phase 1 Ascending Multiple-Dose Study to Evaluate the Safety, Pharmacokinetics and Pharmacodynamics of BMS-906024 in Subjects with Relapsed/Refractory T-cell Acute Lymphoblastic Leukemia or T-cell Lymphoblastic Lymphoma
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Physician New Patient Referral
Name:Patrick Zweidler-McKayPatients 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:713-745-4616
Contact us about clinical trials
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General Information
Disease Group:Leukemia
Lymphoma
Supported By:Bristol-Myers Squibb
Phase of Study:Phase IReturn
Visit:
Treatment
Agents:
BMS-906024Home 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 find the highest tolerable dose
of BMS-906024 that can be given to patients with relapsed or refractory T cell
leukemia or lymphoma. The safety of this drug will also be studied.

BMS-906024 is designed to block an enzyme called gamma secretase. This enzyme
is important in most T cell leukemias and lymphomas. Blocking this enzyme may
help to control the disease. This is the first study using BMS-906024 in
humans.
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Study Objectives / Outcomes
Primary Objective: To assess the safety and tolerability of multiple weekly IV doses of BMS-906024 and to establish the RP2D in subjects with T-ALL or T-LL refractory to or relapsed from standard therapies.

Secondary Objectives:
  • To assess the PK of BMS-906024 and its metabolite BMS-911557 following the first IV dose and after repeated weekly doses
  • To assess pharmacodynamic changes in the expression of Notch pathway-related genes Hes1 and Deltex1 in peripheral blood following treatment
  • To describe preliminary anti-cancer activity of BMS-906024 in T-ALL and T-LL

Exploratory Objectives:
  • To assess pre-treatment Notch pathway-related genes in blood samples, bone marrow and tumor biopsies (DNA, mRNA, and protein) as potential predictive biomarkers in association with clinical responses
  • To assess pharmacodynamic changes in Notch pathway-related genes (mRNA and/or protein) in bone marrow following treatment
  • To assess the effects of BMS-906024 and its metabolite BMS-911557 on ECG parameters
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Study Status Information
Study Activation / Registration Date:01/27/2012
IRB Review and Approval Date:01/27/2012
Study Type:Phase I
Recruitment Status:Open
Projected Accrual:30
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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 written consent

2) Subjects with T-ALL or T-LL refractory to or relapsed from standard therapies

3) Pre-treatment tumor biopsies for subjects with T-LL: (1) Biopsies (other than bone marrow) are not mandatory for subjects with tumor involvement of peripheral blood or bone marrow sufficient for biomarker assessment

4) Pre-treatment tumor biopsies for subjects with T-ALL (continued): (2) Subjects without sufficient tumor involvement in blood or bone marrow for biomarker assessment: (a) During dose escalation: (i) Pre-treatment biopsies are required from subjects with tumor that can be biopsied at acceptable clinical risk as judged by the Investigator (ii) Subjects without accessible tumor for biopsy at acceptable clinical risk as judged by the Investigator are eligible (b) During dose expansion: (i) Pre-treatment biopsies are required from subjects with tumor that can be biopsied at acceptable clinical risk (ii) Subjects without accessible tumor for biopsy at acceptable clinical risk as judged by the Investigator may be enrolled after discussion between the Sponsor/Medical Monitor and Investigator provided either: 1. Archived tissue is available for biomarker assessment, or 2. Biomarker criteria for the expansion cohort can be fulfilled without excluding the subject

5) Pre-treatment tumor biopsies for subjects with T-LL (continued): (c) If a required pre-treatment biopsy is unsuccessful, it need not be repeated and the subject remains eligible

6) Life expectancy of at least 2 months

7) ECOG performance status score 0-1; subjects with ECOG performance status 2 due to disease-related symptoms are also eligible.

8) Previous Treatment: a) Prior anti-cancer treatments are permitted (ie, chemotherapy, radiotherapy, hormonal, or immunotherapy) b) Toxicity (except for alopecia) related to prior anti-cancer therapy and/or surgery must be stable or improving

9) Previous Treatment (continued): c) At least 42 weeks or 5 half-lives (whichever is longer) must have elapsed from the last dose of prior anti-cancer therapy and the initiation of study therapy, provided that subjects have recovered from treatment-related myelosuppression and gastrointestinal toxicities prior to the first dose of BMS-906024, with the following exceptions: i) Medications typically dosed on a daily schedule as part of a maintenance therapy for ALL, such as glucocorticoids or mercaptopurine, may be administered up to 3 days prior to the first dose of BMS-906024 ii) Vinca alkaloids or intrathecal chemotherapy may be dosed up to 7 days prior to first dose of BMS-906024

10) Previous Treatment (continued): c) At least 4 weeks or 5 half-lives (whichever is longer) must have elapsed from the last dose of prior anti-cancer therapy and the initiation of study therapy with the exception of the following: iii) At least 6 weeks must have elapsed between prior therapy with nitrosoureas, mitomycin C and liposomal doxorubicin. For biologics (eg, monoclonal antibodies) and extended-release formulations (other than intrathecal agents), the washout period must extend 1 month beyond the recommended dosing interval iv) At least 4 weeks or 5 half-lives (whichever is longer) must have elapsed since the last dose of any investigational drug. i) At least 6 months must have elapsed after prior therapy with any investigational nucleoside analogue.

11) Age, Sex, and Reproductive Status a) Men and women, 18 years of age or greater b) Women of childbearing potential (WOCBP) and men must be using an acceptable method of contraception to avoid pregnancy throughout the study and for up to 12 weeks after the last dose of investigational product in such a manner that the risk of pregnancy is minimized c) Women must have a negative serum or urine pregnancy test (minimum sensitivity 25 IU/L or equivalent units of human chorionic gonadotrophin [HCG]) within 72 hours prior to the start of investigational product d) Women must not be breastfeeding e) Sexually active fertile men must use effective birth control for the entire study period and for up to 12 weeks after the last dose of investigational product if their partners are WOCBP

Exclusion Criteria:1) Target Disease Exceptions a) Subjects with known or suspected central nervous system (CNS) leukemia or lymphoma. Evaluation of CSF is not required if there are no suggestive signs or symptoms

2) Medical History and Concurrent Diseases a) Evidence of uncontrolled, active infection</= 7 days prior to administration of study medication b) Current or recent (within 3 months of study drug administration) gastrointestinal disease such as chronic or intermittent diarrhea, or disorders that increase the risk of diarrhea, such as inflammatory bowel disease. Non-chronic conditions (e.g. infectious diarrhea) that are completely resolved for at least 2 weeks prior to starting study treatment are not exclusionary c) Conditions requiring chronic systemic (not inhaled) glucocorticoid use, such as autoimmune disease or severe asthma d) Active (symptomatic or requiring current medical treatment) graft versus host disease (GVHD)

3) Medical History and Concurrent Diseases (continued): e) Uncontrolled or significant cardiovascular disease including: i) Uncontrolled hypertension despite optimal medical management ii) Congestive heart failure NYHA (New York Heart Association) class 3 or greater within 3 months iii) Left ventricular ejection fraction (LVEF) < 45% as determined by MUGA or quantitative echocardiogram iv) Active coronary artery disease, unstable or newly diagnosed angina or myocardial infarction in the past 6 months v) History of congenital long QT syndrome or clinically significant ventricular arrhythmias (such as ventricular tachycardia, ventricular fibrillation or Torsade de Pointes). Controlled atrial fibrillation is not an exclusion criterion

4) Medical History and Concurrent Diseases (continued): f) History of medically significant thromboembolic events (excluding events associated with venous access devices) or bleeding diathesis (other than bleeding associated with thrombocytopenia) within the past 6 months, such as cerebrovascular accident (including transient ischemic attacks), pulmonary embolism, pulmonary hemorrhage > 2 teaspoonfuls/24hrs or repeated pulmonary hemorrhage or gastrointestinal hemorrhage requiring transfusion or procedural intervention. Subjects may receive prophylactic platelet transfusions in accordance with institutional policy

5) Medical History and Concurrent Diseases (continued): g) Required anticoagulation therapy with an agent such as warfarin or heparin i) Prophylactic anticoagulation for venous access devices with low-dose heparin or similar (e.g. heparin catheter flush) will be permitted. If warfarin or other vitamin K antagonists are used, the prothrombin time must be </= 1.2 times the institutional upper limit of normal (ULN) or INR < 2 ii) For antiplatelet agents, prophylactic doses are permitted (e.g. aspirin </= 100 mg daily) but must be discussed in advance with the Sponsor/Medical Monitor. Anti-platelet agents should be interrupted during periods of thrombocytopenia < 50,000 cells/mm^3

6) Medical History and Concurrent Diseases (continued): h) Subjects with concomitant second malignancies (except adequately treated non-melanomatous skin cancers or in situ bladder, breast or cervical cancers) are excluded unless a complete remission was achieved and no additional therapy is required or anticipated to be required during the study period i) A serious uncontrolled medical disorder which would impair the ability of the subject to receive protocol therapy j) Inability to be venipunctured and/or tolerate venous access k) Any other sound medical, psychiatric and/or social reason as determined by the Investigator

7) Physical and Laboratory Test Findings: a) Inadequate hepatic function defined as: i) Total bilirubin > 1.5 times the institutional ULN (except known Gilbert's Syndrome); ii) Alanine transaminase (ALT) or aspartate transaminase (AST) > 3 times the institutional ULN b) Uncontrolled (>/= Grade 2) hypertriglyceridemia defined as fasting triglycerides > 300 mg/dL (3.42 mmol/L) c) Inadequate renal function defined as: i) Blood creatinine > 1.5 times the institutional ULN d) Any of the following on 12-lead electrocardiogram (ECG) prior to study drug administration, confirmed by repeat: i) QRS > 120 msec, except right bundle branch block ii) QTcF > 450 msec e) Positive blood screen for hepatitis C antibody, hepatitis B surface antigen, or HIV-1, -2 antibody 4) Allergies and Adverse Drug Reaction a) History of allergy to BMS-906024-related compounds b) History of Grade 3 or 4 infusion-related reaction to CremophorŽ-containing compounds despite premedication with H1- and H2-blockers

8) Physical and Laboratory Test Findings (continued): 5) Sex and Reproductive Status a) WOCBP who are unwilling or unable to use an acceptable method to avoid pregnancy for the entire study period and for up to 12 weeks after the last dose of investigational product b) WOCBP using a prohibited contraceptive method (e.g. hormonal methods). Hormonal contraceptives may be used for other indications (e.g. menstrual bleeding) provided it is not the primary method of contraception c) Women who are pregnant or breastfeeding d) Women with a positive pregnancy test on enrollment or prior to investigational product administration

9) Physical and Laboratory Test Findings (continued): 6) Prohibited Treatments and/or Therapies a) Prior exposure to BMS-906024 or other Notch inhibitors b) Use of any herbal supplements within 1 week prior to study drug administration c) Use of medications causing Torsades de Pointes within 1 week or 5 half-lives (whichever is longer) d) Use of strong inhibitors of CYP3A4 within 1 week or 5 half-lives (whichever is longer) or strong inducers of CYP3A4 within 2 weeks or 5 half-lives (whichever is longer)

10) Physical and Laboratory Test Findings (continued): 7) Concomitant Therapies a) Concurrent chemotherapy, hormonal therapy, immunotherapy regimens, or radiation therapy, standard or investigational, with the following exceptions: i) Glucocorticoids may be administered for treatment of infusion reaction and as premedication to prevent further infusion reaction, and for treatment of severe diarrhea ii) Palliative radiation therapy to a limited field (e.g. painful bone metastasis, painful lumps), if it is not the sole site of measurable and/or assessable disease, is allowed any time during study participation with prior approval of the Sponsor/Medical Monitor iii) Other permitted use of concomitant anticancer therapy 8) Other Exclusion Criteria a) Prisoners or subjects who are involuntarily incarcerated b) Subjects who are compulsorily detained for treatment of either a psychiatric or physical (eg, infectious disease) illness

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Links
Registration Number: NCT01363817
Study Information on Clinical Trials Registry (clinicaltrials.gov)

Other Links:
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Results


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