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Expanded Treatment Protocol (ETP) of Ruxolitinib in Patients With Polycythemia Vera Who Were Hydroxyurea Resistant or Intolerant and for Whom no Treatment Alternatives Was Available.

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ClinicalTrials.gov Identifier: NCT02292446
Recruitment Status : Completed
First Posted : November 17, 2014
Results First Posted : April 2, 2019
Last Update Posted : July 18, 2019
Sponsor:
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )

Tracking Information
First Submitted Date  ICMJE November 5, 2014
First Posted Date  ICMJE November 17, 2014
Results First Submitted Date  ICMJE December 19, 2018
Results First Posted Date  ICMJE April 2, 2019
Last Update Posted Date July 18, 2019
Actual Study Start Date  ICMJE November 21, 2014
Actual Primary Completion Date December 29, 2017   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: December 19, 2018)
Number of Participants With Adverse Events - All Grades [ Time Frame: Baseline up to approximately 26 months ]
Summary of adverse events (all grades).
Original Primary Outcome Measures  ICMJE
 (submitted: November 12, 2014)
Number of Participants with Adverse Events as a Measure of Safety and Tolerability [ Time Frame: 0 to 39 months ]
Change History Complete list of historical versions of study NCT02292446 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: December 19, 2018)
  • Change From Baseline in Hematocrit Levels at All Visits [ Time Frame: Up to approximately 26 months ]
    Change in hematocrit levels from Baseline to each visit were measured
  • Change From Baseline in Spleen Length [ Time Frame: Up to approximately 26 months ]
    Change in spleen length from Baseline to each visit
  • Change From Baseline in Myeloproliferative Neoplasm Symptom Assessment Form Total Symptom Score (MPN-SAF TSS) [ Time Frame: Up to approximately 26 months ]
    The MPN-SAF (Appendix 6) was a disease specific questionnaire comprised of 10 items that measures fatigue related to MPN disease and the severity of nine of the most prevalent associated symptoms including: early satiety, abdominal discomfort, inactivity, concentration, night sweats, itching, bone pain, fever and weight loss. There were three recall periods used in this questionnaire, which were 24 hours for fatigue, the past week for symptoms of early satiety, abdominal discomfort, inactivity, concentration, night sweats, itching, bone pain and fever, and the past 6 months for weight loss, Each item was scored on a scale ranging from 0 (no fatigue/absent) to 10 (As bad as you can imagine/worst imaginable). The MPN-SAF TSS was computed as the average of the observed items multiplied by 10 to achieve a 0-to-100 scale. The MPN-SAF TSS thus had a possible score range of 0 to 100.
Original Secondary Outcome Measures  ICMJE
 (submitted: November 12, 2014)
  • Change in hematocrit [ Time Frame: Up to 39 months ]
    Change in Hct levels from Baseline to each visit where measured
  • Change in spleen [ Time Frame: Up to 39 months ]
    Change in spleen length from Baseline to each visit where measured.
  • Change in MPN-SAF [ Time Frame: Up to 39 months ]
    Change in Myeloproliferative Neoplasm Symptom Assessment Form Total Symptom Score (MPN-SAF) from baseline to each visit where measured.
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Expanded Treatment Protocol (ETP) of Ruxolitinib in Patients With Polycythemia Vera Who Were Hydroxyurea Resistant or Intolerant and for Whom no Treatment Alternatives Was Available.
Official Title  ICMJE An Open-label, Multi-center, Expanded Treatment Protocol (ETP) of Ruxolitinib in Patients With Polycythemia Vera Who Are Hydroxyurea Resistant or Intolerant and for Whom no Treatment Alternatives Are Available.
Brief Summary The purpose of this open-label, single arm, multi-center Expanded Treatment Protocol (ETP) was to provide early access to ruxolitinib and evaluate safety information in patients with polycythemia vera (PV) who were hydroxyurea (HU) resistant or intolerant and who had no other standard treatment option, nor did they qualify for another clinical study for PV
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Polycythemia Vera
Intervention  ICMJE Drug: Ruxolitinib
supplied as 5 mg, 10 mg and 20 mg tablets to be taken orally
Study Arms  ICMJE Experimental: All patients
All patients will receive ruxolitinib at a starting dose of 10 mg twice daily which could be titrated to most appropriate dose. Dose was not to exceed 25 mg bid nor be less than 5 mg once a day
Intervention: Drug: Ruxolitinib
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: December 19, 2018)
161
Original Estimated Enrollment  ICMJE
 (submitted: November 12, 2014)
1500
Actual Study Completion Date  ICMJE December 29, 2017
Actual Primary Completion Date December 29, 2017   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

•Confirmed diagnosis of PV according to the 2008 World Health Organization criteria, palpable spleen, Resistant to or intolerant of hydroxyurea, ECOG performance status of 0, 1 or 2; did not have access to a comparable or satisfactory alternative treatment

Exclusion Criteria:

•Inadequate liver or renal function, Significant bacterial, fungal, parasitic, or viral infection requiring treatment, Active malignancy within the past 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., Women who were pregnant or nursing.

Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Austria,   Belgium,   Bulgaria,   Canada,   Chile,   France,   Germany,   Mexico,   Norway,   Portugal,   Sweden,   Thailand
Removed Location Countries Colombia,   Indonesia,   Jordan,   Lebanon,   Singapore,   Slovakia
 
Administrative Information
NCT Number  ICMJE NCT02292446
Other Study ID Numbers  ICMJE CINC424B2001X
Has Data Monitoring Committee No
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Novartis ( Novartis Pharmaceuticals )
Study Sponsor  ICMJE Novartis Pharmaceuticals
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: Novartis Pharmaceuticals Novartis Pharmaceuticals
PRS Account Novartis
Verification Date July 2019

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP