Alternative Dosing Strategy of Ruxolitinib in Patients With Myelofibrosis

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Incyte Corporation
ClinicalTrials.gov Identifier:
NCT01445769
First received: September 23, 2011
Last updated: September 16, 2014
Last verified: September 2014
Results First Received: March 31, 2014  
Study Type: Interventional
Study Design: Allocation: Non-Randomized;   Endpoint Classification: Safety/Efficacy Study;   Intervention Model: Single Group Assignment;   Masking: Open Label;   Primary Purpose: Treatment
Conditions: Primary Myelofibrosis
Post-Polycythemia Vera Myelofibrosis
Post-Essential Thrombocythemia Myelofibrosis
Intervention: Drug: Ruxolitinib

  Participant Flow
  Hide Participant Flow

Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
No text entered.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
No text entered.

Reporting Groups
  Description
Ruxolitinib Participants initially received ruxolitinib 10 mg twice a day (bid). Dose increases of 5 mg bid were permitted at Weeks 12 and 18 for subjects who did not meet criteria for or have a dose hold over the prior 6 weeks, had a platelet count ≥100 x 10^9/L at week 12 or ≥150 x 10^9/L at week 18, and had a self-reported Patients' Global Impression of Change (PGIC) score of 3 (minimally improved) to 7 (very much worse) OR the subject's palpable spleen length had been reduced by less than 40% at that visit relative to Baseline. The maximum dose was 15 mg BID at Week 12 and 20 mg bid at Week 18. There were also protocol-required dose decreases for protocol-defined anemia and thrombocytopenia.

Participant Flow:   Overall Study
    Ruxolitinib  
STARTED     45  
COMPLETED     37  
NOT COMPLETED     8  
Consent Withdrawn                 2  
Disease Progression                 1  
Lost to Follow-up                 5  



  Baseline Characteristics
  Hide Baseline Characteristics

Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
Intent-to-treat population: All enrolled participants. Note that this was a pilot study which was not sufficiently powered.

Reporting Groups
  Description
Ruxolitinib Participants initially received ruxolitinib 10 mg twice a day (bid). Dose increases of 5 mg bid were permitted at Weeks 12 and 18 for subjects who did not meet criteria for or have a dose hold over the prior 6 weeks, had a platelet count ≥ 100 x 10^9/L at week 12 or ≥ 150 x 10^9/L at week 18, and had a self-reported PGIC score of 3 (minimally improved) to 7 (very much worse) OR the subject's palpable spleen length had been reduced by less than 40% at that visit relative to Baseline. The maximum dose was 15 mg BID at Week 12 and 20 mg bid at Week 18. There were also protocol-required dose decreases for protocol-defined anemia and thrombocytopenia.

Baseline Measures
    Ruxolitinib  
Number of Participants  
[units: participants]
  45  
Age  
[units: years]
Mean ± Standard Deviation
  70.2  ± 9.10  
Gender  
[units: participants]
 
Female     21  
Male     24  



  Outcome Measures
  Show All Outcome Measures

1.  Primary:   Mean Percentage Change From Baseline in Spleen Volume at Week 24   [ Time Frame: Baseline to Week 24 ]

2.  Primary:   Median Percent Change From Baseline in Spleen Volume at Week 24   [ Time Frame: Baseline to Week 24 ]

3.  Secondary:   Mean Percentage Change From Baseline in the Total Symptom Score at Week 24   [ Time Frame: Baseline to Week 24 ]

4.  Secondary:   Median Percent Change From Baseline in the Total Symptom Score at Week 24   [ Time Frame: Baseline to Week 24 ]

5.  Secondary:   Percentage of Participants With a ≥ 35% Reduction From Baseline in Spleen Volume at Week 24   [ Time Frame: Baseline to Week 24 ]

6.  Secondary:   Percentage of Participants With a ≥ 10% Reduction From Baseline in Spleen Volume at Week 24   [ Time Frame: Baseline to Week 24 ]

7.  Secondary:   Percentage of Participants With a ≥ 50% Improvement From Baseline in Total Symptom Score at Week 24   [ Time Frame: Baseline to Week 24 ]

8.  Secondary:   Mean Percentage Change From Baseline in Palpable Spleen Length at Week 24   [ Time Frame: Baseline to Week 24 ]

9.  Secondary:   Median Percent Change From Baseline in Palpable Spleen Length at Week 24   [ Time Frame: Baseline to Week 24 ]

10.  Secondary:   Percentage of Participants With a ≥ 50% Improvement From Baseline in Their Transfusion Status or With New Transfusion Independence Status for Those Participants Who Were Transfusion Dependent at Baseline   [ Time Frame: Baseline to Week 24 ]

11.  Secondary:   Percentage of Participants With Clinically Notable Anemia   [ Time Frame: Baseline to Weeks 12, 18 and 24 ]

12.  Secondary:   Mean Percentage Change in Abdominal Symptom Scores at Week 24.   [ Time Frame: Week 24 ]

13.  Secondary:   Median Percentage Change in Abdominal Symptom Scores at Week 24.   [ Time Frame: Week 24 ]

14.  Secondary:   Number of Participants With Grade 3 or Grade 4 Adverse Events   [ Time Frame: Baseline to the end of the study ]

15.  Other Pre-specified:   Dose Distribution at Week 24   [ Time Frame: Week 24 ]


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats
  Hide 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
This was a single arm pilot study. The patient population differed from Phase III studies of ruxolitinib in myelofibrosis and therefore comparisons to standard dosing cannot be made from this study.


  More Information
  Hide More Information

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:
unchecked 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.
unchecked 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.


Results Point of Contact:  
Name/Title: Study Director
Organization: Incyte Corporation
phone: 855 463-3463


No publications provided


Responsible Party: Incyte Corporation
ClinicalTrials.gov Identifier: NCT01445769     History of Changes
Other Study ID Numbers: 18424-261
Study First Received: September 23, 2011
Results First Received: March 31, 2014
Last Updated: September 16, 2014
Health Authority: United States: Food and Drug Administration