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Study Summary
No. 2010-0808:.......Myeloproliferative Diseases......Srdan Verstovsek......Leukemia
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Study Summary Title
Study Summary
Number:
2010-0808
Study Title:Randomized, open label, multicenter phase III study of Efficacy and Safety in POlycythemia vera subjects who are resistant to or intolerant of hydroxyurea: JAK iNhibitor ruxolitinib tablets verSus bEst available care (The RESPONSE Trial)
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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/Novartis Oncology
Phase of Study:Phase IIIReturn
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 compare INC424 (referred to from
here on as ruxolitinib) to the best available therapy when given to patients
with PV.
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Study Objectives / Outcomes
Primary Objective
To compare the efficacy of ruxolitinib to Best Available Therapy as assessed by both the absence of phlebotomy eligibility and reduction in spleen volume

Key Secondary Objectives
• To compare the proportion of subjects randomized to ruxolitinib vs Best Available Therapy achieving both durable absence of phlebotomy eligibility and durable spleen volume reduction
• To compare the proportion of subjects randomized to ruxolitinib vs Best Available Therapy achieving complete hematologic remission

Other Secondary Objectives
• To determine the proportion of subjects achieving a durable spleen volume reduction
• To estimate the proportion of subjects achieving a durable complete hematologic remission
• To estimate the proportion of subjects achieving a durable phlebotomy independence
To estimate the duration of both the absence of phlebotomy eligibility and reduction in spleen volume
• To determine the overall clinicohematologic response rate
• To estimate the proportion of subjects achieving a durable complete or partial clinicohematologic response
• To estimate the duration of the overall clinicohematologic response
• To evaluate the safety of ruxolitinib and Best Available Therapy.

Exploratory Objectives
• To compare the proportion of subjects achieving white blood cells count control to a defined level
• To compare the proportion of subjects achieving platelets count control to a defined level • To evaluate changes in Patient-Reported Outcomes
• To evaluate changes in JAK2V617F allele burden, and to evaluate cytokines and other plasma proteins as potential PD biomarkers.
• To assess pharmacokinetics of ruxolitinib in PV subjects
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Study Status Information
Study Activation / Registration Date:
IRB Review and Approval Date:04/18/2011
Study Type:Phase Iii
Recruitment Status:Closed
Projected Accrual:200
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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 18 years of age or older.

2) Subjects must be diagnosed with PV for at least 24 weeks prior to Screening according to the 2008 World Health Organization criteria

3) Subjects must have a treatment history for PV that meets the definition of resistance or intolerance to hydroxyurea (HU) by exhibiting at least one of the following five criteria: • HU Resistance, defined at least 12 weeks into a course of HU therapy at a dose of at least 2 grams/day OR at the subject's maximally tolerated dose if that dose is less than 2 grams/day: a. Need for phlebotomy to keep hematocrit < 45%, OR b. Platelet count > 400 x 109/L AND white blood cell count > 10 x 109/L, OR

4) Cont. of #3: c. Failure to reduce splenomegaly extending greater than 10 cm below the costal margin by more than 50%, as measured by palpation. • HU Intolerance: a. Absolute neutrophil count < 1.0 x 109/L OR platelet count < 100 x 109/L OR hemoglobin < 100 g/L (i.e. 10 g/dL) at the lowest dose of HU required to achieve a response: hematocrit < 45% without phlebotomy AND/OR all of the following three items: platelet count equal to or less than 400 x 109/L, white blood cell count equal to or less than 10 x 109/L, and non-palpable spleen), OR b. Presence of leg ulcers or other unacceptable HU-related non- hematological toxicities, such as mucocutaneous manifestations, gastrointestinal symptoms, pneumonitis or fever at any dose of HU defined as: a) CTCAE version 3.0 Grade 3-4 OR b) more than 1 week of CTCAE version 3.0 Grade 2, OR c) permanent discontinuation of HU, OR d) interruption of HU until toxicity resolved, OR e) hospitalization du to HU toxicity.

5) Subjects must have required at least 2 phlebotomies in the 24 weeks prior to Screening AND at least 1 phlebotomy in the 16 weeks prior to Screening, AND the most distant and the most recent phlebotomy within the 24 weeks prior to screening must be at least 4 weeks apart OR subjects will be considered to have met this criterion if they have required a phlebotomy within the 16 weeks prior to Screening AND they exhibit a hematocrit > 45% at Screening

6) Subjects with splenomegaly, defined as: a) Spleen palpable below the costal margin, provided the MRI (or CT in applicable subjects) spleen assessment during Screening confirms that the spleen is enlarged, defined with a volume of > 450 cm^3 OR b) Spleen non palpable below the costal margin due to body habitus (eg. In obese subjects), provided the MRI (or CT in spplicable subjects) spleen assessment during Screening confirms that the spleen is enlarged, defined with a volume of > 450 cm^3 .

7) Subjects with ANC equal to or more than 1.5 x 109/L at Screening.

8) Subjects with peripheral blood blast count of 0% at Screening.

9) Subjects with an ECOG performance status of 0, 1 or 2 at Screening and Baseline.

10) Subjects on a therapeutic regimen for PV must have been on a stable dose and schedule for at least 2 weeks prior to Screening and no less than 4 weeks prior to randomization (Study Day 1).

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) Women of child bearing potential, defined as all women physiologically capable of becoming pregnant, including women whose career, lifestyle or sexual orientation precludes intercourse with a male partner and women whose partners have been sterilized by vasectomy or other means, UNLESS they are using two birth control methods. The two methods can be a double barrier method or a barrier method plus a hormonal method. A) adequate barrier methods of contraception include: diaphragm, condom (by the partner), intrauterine device (copper or hormonal), sponge or spermicide. B) Hormonal contraceptives include any marketed contraceptive agaent that includes an estrogen and/or progestational agent. C) Reliable contraception should be maintained throughout the study and for 5 half lives of study drug after study tertment discontinuation. Cont. on #3

3) Cont. from #2: D) Women considered port- menopausal and not of child bearing potential if they have had 12 months of natural (spontaneous) amenorrhea with an appropriate clinical profile (eg. Age appropriate, history of vasomotor symptoms) or six months of spontaneous amenorrhea with serum FSH levels >40 mIU/mL and estradiol < 20 pg/mL or have had surgical bilateral oopherectomy (with or without hysterectomy) at least six weeks ago. In the case of oopherectomy alone, only when the reproductive status of the women has been confirmed by follow up hormone level assessment is she considered not of child bearing potential. E) Sexually active males must use a condom during intercourse while taking the drug for five half lives after stopping treatment and should not father a child in this period. A condom is require d to be used also be vastectomized men in order to prevent delivery of the drug via seminal fluid.

4) Subjects with inadequate liver or renal function at Screening as demonstrated by: a. Encephalopathy Grade 2 or more as per Child-Pugh System. b. Known hepatocellular disease (for example, hepatitis B or C, cirrhosis or other hepatocellular disease) c. Direct bilirubin equal to or more than 2 X upper limit of laboratory normal (ULN). d. Alanine aminotransferase (ALT) > 2.5x ULN. e. MDRD-eGFR < 30 mL/min/1.73m2 or on dialysis.

5) Subjects with impairment of gastrointestinal (GI) function or GI disease that may significantly alter the absorption of oral INC424 (e.g., ulcerative diseases, uncontrolled nausea, vomiting, diarrhea, malabsorption syndrome, small bowel resection).

6) Subjects with clinically significant bacterial, fungal, parasitic or viral infection which requires therapy: • Subjects with acute bacterial infections requiring antibiotic use should delay screening/enrollment until the course of antibiotic therapy has been completed. • Subjects with known active hepatitis A, B or C at Screening or with known HIV positivity.

7) Subjects with diagnosed primary immunodeficiency syndromes such as X-Linked Agammaglobulinemia and Common Variable Immune Deficiency

8) Subjects 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.

9) Subjects with clinically significant cardiac disease (NYHA Class III or IV).

10) Subjects receiving PEG-IFN-alpha-2a within 5 weeks of Screening or having a prior history of 32P therapy.

11) Subjects being treated concurrently with a potent systemic inhibitor of CYP3A4 at the time of Screening (ketoconazole, clarithromycin, itraconazole, nefazodone or telithromycin).

12) Subjects being treated concurrently with any prohibited medications

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

14) Subjects being treated concurrently with any investigational agent or prior participation in an investigational study within 30 days prior to enrollment or within 5-half lives of the investigational product, whichever is longer.

15) Subjects who are unable to comprehend or are unwilling to sign an informed consent form (ICF)

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

17) Subjects with an uncontrolled intercurrent illness or any concurrent condition that, in the investigator's opinion, would jeopardize the safety of the subject or compliance with the protocol.

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Links
Registration Number: NCT01243944
Study Information on Clinical Trials Registry (clinicaltrials.gov)

Other Links:
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Results


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