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Study Summary
No. 2010-0964:.......Myeloproliferative Diseases......Srdan Verstovsek......Leukemia
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Study Summary Title
Study Summary
Number:
2010-0964
Study Title:A Phase Ib, open-label, dose-finding study of the JAK
inhibitor ruxolitinib tablets administered orally to patients with
Primary Myelofibrosis (PMF), Post-Polycythemia Vera-
Myelofibrosis (PPV-MF) or Post-Essential
Thrombocythemia-Myelofibrosis (PET-MF) and baseline
platelet counts >/= 50x10^9/L and <100x10^9/L.
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Physician New Patient Referral
Name:Srdan VerstovsekPatients 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:713-792-7305
Contact us about clinical trials
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General Information
Disease Group:Myeloproliferative DiseasesSupported By:Incyte Corporation
Novartis
Phase of Study:Phase IReturn
Visit:
Treatment
Agents:
RuxolitinibHome 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
starting dose of ruxolitinib that can be given to patients with myelofibrosis
who have thrombocytopenia. The safety of this drug will also be studied.
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Study Objectives / Outcomes
Primary

· To establish the Maximum Starting Safety Dose (MSSD) of ruxolitinib in patients with MF and baseline PLT count < 100 x 109/L and equal to or more than 75 x 109/L (first stratum) and PLT count < 75 x 109L and equal to or more than 50 x 109/L (second stratum)

Secondary

· Safety: To characterize the safety of ruxolitinib
· Pharmacokinetics: To characterize the pharmacokinetics of ruxolitinib in this patient population
· Pharmacokinetic-pharmacodynamic relationship: To characterize the pharmacokinetic-pharmacodynamic relationship of this population
· Efficacy: To obtain estimates of efficacy

Exploratory

· Symptoms: To evaluate changes in symptoms of myelofibrosis (Patient-Reported Outcomes)
· Transfusion dependence: To evaluate changes in Packed Red Blood Cells (PRBC) transfusion dependence
· JAK2 mutational status: To evaluate the correlation between JAK2 mutational status and thrombocytopenia as well as between JAK2 mutational status and efficacy of ruxolitinib in these low-platelet MF patients

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Study Status Information
Study Activation / Registration Date:
IRB Review and Approval Date:04/15/2011
Study Type:Phase I
Recruitment Status:Open
Projected Accrual:62
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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 18 years of age or older

2) Patients must be diagnosed with PMF, PPV-MF or PET-MF irrespective of JAK2 mutation status, guided by the criteria outlined in the 2008 World Health Organization (WHO) criteria for PMF, and the proposed criteria for PPV-MF and PET-MF outlined by the International Working Group for Myelofibrosis Research and Treatment (IWG-MRT).

3) Patients with myelofibrosis requiring therapy must be classified at least as intermediate risk level 2 (2 or more prognostic factors) OR intermediate risk level 1 (1 or more prognostic factors). The prognostic factors, defined by the International Working Group are: • age > 65 yrs • presence of constitutional symptoms (weight loss > 10% of the baseline value in the year preceding the Screening Visit, unexplained fever, or excessive night sweats persisting for more than 1 month) • marked anemia (Hgb < 10g/dL)* • leukocytosis (history of WBC > 25 x10^9/L) • circulating blasts equal to or greater than 1% * A hemoglobin value < 10 g/dL must be demonstrated during the Screening for patients who are not transfusion dependent. Patients receiving regular transfusions of packed red blood cells will be considered to have hemoglobin < 10 g/dL for the purpose of evaluation of risk factors

4) Patients must have palpable spleen of at least 5 cm from the costal margin to the point of greatest splenic protrusion

5) Patients must have active symptoms of MF as demonstrated by one symptom score of at least 5 (0-10 point scale) or two symptom scores of at least 3 (0 to 10 point scale) on the Screening Symptom Form, SSF

6) Patients must fulfill all the following criteria both at Screening and at Study Day 1: • PLT counts < 100 x 10^9/L and equal to or greater than 75 x 10^9/L for the first stratum of the trial or PLT counts < 75 x 10^9/L and >/= 50 x 10^9/L for the second stratum • coagulation parameters as follows: INR and PTT < 1.2 x ULN, fibrinogen > 0.8 x ULN and D-dimer or fibrinogen degradation products (FDP) within normal limits NOTE: If at the planned day of study treatment initiation the PLT counts are outside the permitted range, for the respective stratum, then an additional PLT count assessment will be conducted in equal to or less than 7 days. The treatment will be started only if the PLT value of the additional assessment is within the permitted range for the respective stratum. The day at which the treatment will eventually start will be defined as the Study Day 1

7) Patients with ANC equal to or more than 1.5 x 10^9/L at Screening

8) Patients with peripheral blood blast count of < 5% at Screening

9) Patients with an ECOG performance status of 0, 1, or 2 at Screening

10) Patients must have discontinued all drugs used to treat underlying MF disease no later than 7 days prior to Screening evaluations visit

11) Patients who have not previously received treatment with a JAK inhibitor

Exclusion Criteria:1) Pregnant or nursing (lactating) women, where pregnancy is defined as the state of a female after conception (confirmed by a positive hCG laboratory test > 5 mIU/mL) and until the termination of gestation

2) Patients of childbearing potential who are unwilling to take appropriate precautions (from Screening through Follow-up) to avoid becoming pregnant or fathering a child • Females of non-childbearing potential are defined as women who (a) are equal to or more than 55 years of age with history of amenorrhea for 1 year with serum FSH levels > 40 mIU/mL, OR (b) are surgically sterile for at least 12 weeks • For females of childbearing potential, or for males, appropriate precautions (at least two highly effective contraception methods) are those that are at least 99% effective in preventing the occurrence of pregnancy. These methods should be communicated to the patients and their understanding confirmed

3) Patients undergoing treatment with hematopoietic growth factor receptor agonists (i.e. erythropoietin (EPO), granulocyte colony stimulating factor (GCSF), romiplostim, eltrombopag) for at least 30 days prior to receiving the first dose of study drug

4) Patients with any history of PLT counts < 45 x 10^9/L within 30 days prior to Screening, except during treatment for a myeloproliferative neoplasm or treatment with cytotoxic therapy for any other reason. Patients who have received PLT transfusion within 30 days prior to Screening evaluations.

5) Any history or predisposition to clinically significant bleeding

6) Any history of platelet dysfunction and/or bleeding diathesis

7) Any regular use of drugs that interferes with coagulation or inhibits PLT function. NOTE: low doses of aspirin equal to or less than 125 mg/day are allowed.

8) Patients with inadequate liver or renal function as demonstrated by: • encephalopathy grade 1 or more, as per West Haven Criteria • known hepatocellular disease (e.g. active hepatitis or cirrhosis) • total bilirubin equal to or more than 2 x ULN and subsequent determination of direct bilirubin equal to or more than 2 x ULN • alanine aminotransferase (ALT) > 2.5 x ULN • MDRD-eGFR < 30 mL/min/1.73m2 or on dialysis

9) Patients with clinically significant bacterial, fungal, parasitic or viral infection which require therapy. Patients with acute bacterial infections requiring antibiotic use should delay screening/enrollment until the course of antibiotic therapy has been completed

10) Patients with known active hepatitis A, B or C at Screening or with known HIV positivity

11) Patients being treated concurrently with a potent systemic inhibitor or a potent systemic inducer of CYP3A4 at the time of Screening

12) Patients with impairment of gastrointestinal (GI) function or GI disease that may significantly alter the absorption of oral ruxolitinib(e.g., ulcerative diseases, uncontrolled nausea, vomiting, diarrhea, malabsorption syndrome, small bowel resection)

13) Patients who currently are candidates for a stem cell transplantation at the time of the screening assessments, either because they are not a candidate for the procedure, in the investigator's expert judgment, or because a suitable donor is not available

14) Patients with an active malignancy over the previous 5 years, except treated cervical intraepithelial neoplasia, basal cell carcinoma of the skin, or squamous cell carcinoma of the skin, with no evidence for recurrence in the past 3 years

15) Patients with currently rapid or paroxysmal atrial fibrillation, currently uncontrolled or unstable angina, recent (approximately 6 months) myocardial infarction or acute coronary syndrome or any clinically significant cardiac disease

16) Patients under ongoing treatment with another investigational medication or having been treated with an investigational medication within 30 days or 5 half-lives (whichever is longer) prior to enrollment

17) Patients who have had splenic irradiation within 12 months prior to Screening

18) Patients who are unable to comprehend or are unwilling to sign an informed consent form

19) Patients with active alcohol or drug addiction that, in the investigator's expert judgment, would interfere with their ability to comply with the study requirements

20) Patients with any concurrent condition that, in the Investigator's opinion, would jeopardize the safety of the patient or compliance with the protocol

21) Patients who cannot operate electronic diary equipment, or who cannot read the screen or who cannot speak or read and understand the languages provided with the diary (as per e-diary vendor manual).

22) Subjects with a life expectancy of less than 6 months.

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Links
Registration Number: NCT01317875
Study Information on Clinical Trials Registry (clinicaltrials.gov)

Other Links:
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Results


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