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Study Summary
No. 2011-0359:.......Myeloproliferative Diseases......Srdan Verstovsek......Leukemia
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Study Summary Title
Study Summary
Number:
2011-0359
Study Title:AN OPEN LABEL ASSESSMENT OF SAFETY AND EFFICACY OF RUXOLITINIB IN SUBJECTS WITH PRIMARY MYELOFIBROSIS, POST ESSENTIAL THROMBOCYTHEMIAMYELOFIBROSIS AND POST POLYCYTHEMIA VERAMYELOFIBROSIS WHO HAVE PLATELET COUNTS OF 50 x10^9/L TO 100 x10^9/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:lncyte Corporation
Phase of Study:Phase IIReturn
Visit:
Treatment
Agents:
RuxolitinibHome Care:
Treatment Loc:Both at MDACC & and Other Sites
Estimated
Length of Stay
in Houston:
Description/
Intervention:
Unavailable
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Study Objectives / Outcomes

Primary Objectives:

·To determine the effects of ruxolitinib on spleen volume and symptomatic burden in patients with primary myelofibrosis (PMF), post polycythemia vera-myelofibrosis (PPV-MF) and post essential thrombocythemia-myelofibrosis (PET-MF) who have Baseline platelet count of 50 x10^9/L to 100 x10^9/L.
·To determine the safety and tolerability of ruxolitinib in patients with PMF, PPV-MF and PET-MF who have Baseline platelet count of 50 x109^/L to 100 x109^9/L.

Secondary Objective:

·To determine appropriate dosing for patients with low platelets.

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Study Status Information
Study Activation / Registration Date:
IRB Review and Approval Date:07/21/2011
Study Type:Phase Ii Or Phase I/Ii
Recruitment Status:Open
Projected Accrual:150
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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) Subjects who are able to understand and sign an informed consent document.

2) Subjects 18 years of age or older.

3) Subjects must be diagnosed with PMF, PPV-MF or PET-MF, according to the investigator's expert judgment, guided by the criteria outlined in the 2008 World Health Organization criteria for PMF, and the proposed criteria for PPV-MF and PET-MF outlined by the International Working Group for Myeloproliferative Neoplasms Research and Treatment (IWG-MRT) irrespective of JAK2 mutation status. A bone marrow biopsy will provide the relevant information; subjects without a prior biopsy report obtained in the 2 months prior to screening for review must have a biopsy at Screening or Baseline or will not be able to enroll in the study.

4) Subjects with MF requiring therapy must have at least 1 point using the DIPSS prognostic criteria developed by the IWG-MRT, using the information from laboratory assessments and ongoing medical history recorded at the Screening Visit.

5) Subjects in whom treatment of MF is indicated based on the Investigator's expert judgment.

6) Subjects with active symptoms of MF at the screening Visit as demonstrated by presence of one symptom score of at least 5 or two symptom scores of at least 3 using the Screening Symptom Assessment Form

7) Subjects who have platelet counts between 50 and 100 x10^9/L at the Screening and/or Baseline visits.

8) Subjects with hemoglobin value at the Screening visit equal to or more than 6.5 g/dL and who are willing to receive red blood cell transfusions to treat low hemoglobin levels.

9) Subjects must have discontinued all drugs used to treat underlying MF disease no later than Day -14.

10) Subjects must not currently have the option of stem cell transplantation, either because they are not a candidate, or because a suitable donor is not available.

11) Subjects with an ECOG performance status of 0, 1, 2 or 3 at the Screening Visit.

12) Subjects with peripheral blood blast count of < 5% at both Screening and Baseline visits

13) Subjects whose platelet count is between 50 x10^9/L to 100 x10^9/L.

Exclusion Criteria:1) Subjects with a life expectancy of less than 6 months.

2) Subjects in whom MF disease is well controlled with current therapy.

3) Females who are pregnant or are currently breastfeeding.

4) Subjects 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 greater than 55 years of age with history of amenorrhea for 1 year, OR (b) are surgically sterile for at least 3 months. • For females of childbearing potential, or for males, appropriate precautions are those that are at least 99% effective in preventing the occurrence of pregnancy. These methods should be communicated to the subjects and their understanding confirmed

5) Subjects with inadequate bone marrow reserve as demonstrated by: a. Absolute neutrophil count (ANC) that is < 1x 10^9/L at the Screening visit. b. Confirmed platelet count that is < 50 x 10^9/L without the assistance of growth factors, thrombopoietic factors or platelet transfusions at the Screening visit. Subjects must not have received growth factors for at least one month (30 days) prior to receiving the first dose of study drug. c. Subjects with known history of platelet counts < 25,000/µL, in the absence of cytoreductive therapy. d. Subjects who have received platelet transfusion(s) or ANC levels < 500/µL in the month (30 days) prior to Screening.

6) Subjects with platelet count >100 x10^9/L at BOTH the Screening and Baseline visits.

7) Subjects with recent (within 12 months of Screening) major bleed requiring transfusion(s) or resulting in a decrease in hemoglobin by 3 g/dL or more.

8) Subjects with inadequate coagulation parameters as follows: International Normalized Ratio (INR) > 1.5 or PTT > 1.5 x ULN.

9) Subjects with known history of esophageal or gastric varices, or any intracranial bleeding. Subjects with a history of an incidental finding of small varices (<5 mm) may be permitted in the study with sponsor approval.

10) Subjects with inadequate liver or renal function at Screening and Baseline visits as demonstrated by: a. Direct bilirubin equal to or more than 2 X upper limit of laboratory normal (ULN). (NOTE: direct bilirubin will only be determined if total bilirubin is equal to or more than 2.0 x ULN). b. Alanine aminotransferase (ALT) > 2.5x ULN. c. Creatinine > 2.0 mg/dL.

11) Subjects with clinically meaningful, active bacterial, fungal, parasitic or viral infection which require therapy, or who are HIV positive

12) Subjects with acute bacterial infections requiring antibiotic use should delay screening/enrollment until the course of antibiotic therapy has been completed and the event is considered resolved.

13) Subjects who are currently receiving therapy with a moderate or potent CYP 3A4 inhibitor. Subjects may enter Screening phase when therapy with the moderate or potent CYP 3A4 inhibitor is completed, and use of moderate inhibitors during the study will be permitted.

14) Subjects with an invasive malignancy over the previous 2 years except treated early stage carcinomas of the skin completely resected intraepithelial carcinoma of the cervix and completely resected papillary thyroid and follicular thyroid cancers.

15) Subjects with recent severe or unstable cardiac disease.

16) Subjects who have had splenic irradiation within 6 months prior to Day 1.

17) Subjects who have previously received treatment with a JAK inhibitor.

18) Subjects being treated concurrently with any prohibited medications

19) Subjects with active alcohol or drug addiction that would interfere with their ability to comply with the study requirements.

20) Subjects with any concurrent condition that, in the investigator's opinion, would jeopardize the safety of the subject or compliance with the protocol.

21) Subjects who are unable to complete the daily symptom diary, which is available in English and Spanish versions.

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Links
Registration Number: NCT01348490
Study Information on Clinical Trials Registry (clinicaltrials.gov)

Other Links:
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Results


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