Study to Determine the Safety and Efficacy of Ruxolitinib (INCB018424) in Patients With Polycythemia Vera or Essential Thrombocythemia

This study is ongoing, but not recruiting participants.

Sponsor:

Information provided by (Responsible Party):

Incyte Corporation

ClinicalTrials.gov Identifier:

NCT00726232

First received: July 29, 2008

Last updated: April 25, 2013

Last verified: April 2013

History of Changes

Results First Received: January 20, 2012

Study Type:	Interventional
Study Design:	Allocation: Randomized; Endpoint Classification: Safety/Efficacy Study; Intervention Model: Parallel Assignment; Masking: Open Label; Primary Purpose: Treatment
Conditions:	Polycythemia Vera Essential Thrombocythemia
Intervention:	Drug: Ruxolitinib

Participant Flow

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Recruitment Details

Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
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Pre-Assignment Details

Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
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Reporting Groups

	Description
PV: Ruxolitinib 10 mg BID	Participants with Polycythemia Vera received 10 mg Ruxolitinib orally twice a day (BID) for 56 days (two 28-day cycles) during the dose-ranging phase. After patients completed 2 cycles of treatment at the randomized dose, Investigators were permitted to adjust the dose/regimen on an individual basis to achieve an optimal balance of efficacy and safety. Treatment continued until a patient met a withdrawal criterion, had intolerable toxicity, progression of disease, or withdrew consent.
PV: Ruxolitinib 25 mg BID	Participants with Polycythemia Vera received 25 mg Ruxolitinib orally twice a day (BID) for 56 days (two 28-day cycles) during the dose-ranging phase. After patients completed 2 cycles of treatment at the randomized dose, Investigators were permitted to adjust the dose/regimen on an individual basis to achieve an optimal balance of efficacy and safety. Treatment continued until a patient met a withdrawal criterion, had intolerable toxicity, progression of disease, or withdrew consent.
PV: Ruxolitinib 50 mg QD	Participants with Polycythemia Vera received 50 mg Ruxolitinib orally once a day (QD) for 56 days (two 28-day cycles) during the dose-ranging phase. After patients completed 2 cycles of treatment at the randomized dose, Investigators were permitted to adjust the dose/regimen on an individual basis to achieve an optimal balance of efficacy and safety. Treatment continued until a patient met a withdrawal criterion, had intolerable toxicity, progression of disease, or withdrew consent.
ET: Ruxolitinib 10 mg BID	Participants with Essential Thrombocythemia received 10 mg Ruxolitinib orally twice a day (BID) for 56 days (two 28-day cycles) during the dose-ranging phase. After patients completed 2 cycles of treatment at the randomized dose, Investigators were permitted to adjust the dose/regimen on an individual basis to achieve an optimal balance of efficacy and safety. Treatment continued until a patient met a withdrawal criterion, had intolerable toxicity, progression of disease, or withdrew consent.
ET: Ruxolitinib 25 mg BID	Participants with Essential Thrombocythemia received 25 mg Ruxolitinib orally twice a day (BID) for 56 days (two 28-day cycles) during the dose-ranging phase. After patients completed 2 cycles of treatment at the randomized dose, Investigators were permitted to adjust the dose/regimen on an individual basis to achieve an optimal balance of efficacy and safety. Treatment continued until a patient met a withdrawal criterion, had intolerable toxicity, progression of disease, or withdrew consent.
ET: Ruxolitinib 50 mg QD	Participants with Essential Thrombocythemia received 50 mg Ruxolitinib orally once a day (QD) for 56 days (two 28-day cycles) during the dose-ranging phase. After patients completed 2 cycles of treatment at the randomized dose, Investigators were permitted to adjust the dose/regimen on an individual basis to achieve an optimal balance of efficacy and safety. Treatment continued until a patient met a withdrawal criterion, had intolerable toxicity, progression of disease, or withdrew consent.

Participant Flow: Overall Study

	PV: Ruxolitinib 10 mg BID	PV: Ruxolitinib 25 mg BID	PV: Ruxolitinib 50 mg QD	ET: Ruxolitinib 10 mg BID	ET: Ruxolitinib 25 mg BID	ET: Ruxolitinib 50 mg QD
STARTED	19 ^[1]	8	7	8	22 ^[2]	9
COMPLETED	14 ^[3]	7	7	7	17	5
NOT COMPLETED	5	1	0	1	5	4
Withdrawal by Subject	2	0	0	0	0	2
Adverse Event	1	1	0	1	2	1
Disease progression	1	0	0	0	0	0
No response	1	0	0	0	3	1

[1]	The starting dose selected for the PV expansion cohort was 10 mg bid hence the sample size is larger
[2]	The starting dose selected for the ET expansion cohort was 25 mg bid hence the sample size is larger
[3]	Represents patients ongoing in the study as of the data cut-off date of 18 June 2010.

Baseline Characteristics

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Outcome Measures

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1. Primary:

Percentage of Polycythemia Vera Participants With a Confirmed Clinical Partial Response (PR) or Complete Response (CR) [ Time Frame: Assessed after 2 cycles (56 days) of treatment on Day 1 of Cycle 3 ]

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2. Primary:

Percentage of Essential Thrombocythemia (ET) Participants With a Confirmed Clinical Partial Response (PR) or Complete Response (CR) [ Time Frame: Assessed after 2 cycles (56 days) of treatment on Day 1 of Cycle 3. ]

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3. Secondary:

Percentage of Polycythemia Vera Participants Who Achieved Individual Components of Clinical Response at 12 Weeks [ Time Frame: Baseline and Week 12 (Cycle 4, Day 1) ]

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4. Secondary:

Percentage of Polycythemia Vera Participants Who Achieved Individual Components of Clinical Response at 24 Weeks [ Time Frame: Baseline and Week 24 (Cycle 7, Day 1) ]

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5. Secondary:

Percentage of Polycythemia Vera Participants Who Achieved Individual Components of Clinical Response at 36 Weeks [ Time Frame: Baseline and Week 36 (Cycle 10, Day 1) ]

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6. Secondary:

Percentage of Essential Thrombocythemia Participants Who Achieved Individual Components of Clinical Response at 4 Weeks [ Time Frame: Baseline and 4 weeks (Cycle 2, Day 1) ]

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7. Secondary:

Percentage of Essential Thrombocythemia Participants Who Achieved Individual Components of Clinical Response at 24 Weeks [ Time Frame: Baseline and 24 weeks (Cycle 7, Day 1) ]

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8. Secondary:

Percentage of Essential Thrombocythemia Participants Who Achieved Individual Components of Clinical Response at 36 Weeks [ Time Frame: Baseline and 36 weeks (Cycle 10, Day 1) ]

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9. Secondary:

Change From Baseline to Week 4 in Polycythemia Vera Symptoms [ Time Frame: Baseline and Week 4 (Cycle 2, Day 1) ]

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10. Secondary:

Change From Baseline to Week 4 in Essential Thrombocythemia Symptoms [ Time Frame: Baseline and Week 4 (Cycle 2, Day 1) ]

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11. Secondary:

Change From Baseline to Week 4 in Health-related Quality of Life [ Time Frame: Baseline and Week 4 (Cycle 2, Day 1) ]

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Serious Adverse Events

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Other Adverse Events

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More Information

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Certain Agreements:

Principal Investigators are NOT employed by the organization sponsoring the study.

There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.

The agreement is:

	The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is less than or equal to 60 days. The sponsor cannot require changes to the communication and cannot extend the embargo.
	The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is more than 60 days but less than or equal to 180 days. The sponsor cannot require changes to the communication and cannot extend the embargo.
	Other disclosure agreement that restricts the right of the PI to discuss or publish trial results after the trial is completed. Restriction Description: Following the first publication, the Institution and/or Principal Investigator may publish data or results from the Study; provided, however, that the Institution and/or Principal Investigator submits the proposed publication to the Sponsor for review at least sixty (60) days prior to the date of the proposed publication. Sponsor may remove from the proposed publication any information that is considered confidential and/or proprietary other than Study data and results.

Limitations and Caveats

Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
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Results Point of Contact:

Name/Title: Study Director
Organization: Incyte Corporation
phone: 1-855-463-3463

No publications provided

Responsible Party:	Incyte Corporation
ClinicalTrials.gov Identifier:	NCT00726232 History of Changes
Other Study ID Numbers:	INCB 18424-256
Study First Received:	July 29, 2008
Results First Received:	January 20, 2012
Last Updated:	April 25, 2013
Health Authority:	United States: Food and Drug Administration Italy: Ministry of Health

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