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Trial record 1 of 1 for:    NCT02292446
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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 : April 2, 2019
Sponsor:
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )

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

Condition or disease Intervention/treatment Phase
Polycythemia Vera Drug: Ruxolitinib Phase 3

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 161 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: 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.
Actual Study Start Date : November 21, 2014
Actual Primary Completion Date : December 29, 2017
Actual Study Completion Date : December 29, 2017


Arm Intervention/treatment
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
Drug: Ruxolitinib
supplied as 5 mg, 10 mg and 20 mg tablets to be taken orally




Primary Outcome Measures :
  1. Number of Participants With Adverse Events - All Grades [ Time Frame: Baseline up to approximately 26 months ]
    Summary of adverse events (all grades).


Secondary Outcome Measures :
  1. 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

  2. Change From Baseline in Spleen Length [ Time Frame: Up to approximately 26 months ]
    Change in spleen length from Baseline to each visit

  3. 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.




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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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.


Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02292446


  Show 68 Study Locations
Sponsors and Collaborators
Novartis Pharmaceuticals
Investigators
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Study Director: Novartis Pharmaceuticals Novartis Pharmaceuticals
  Study Documents (Full-Text)

Documents provided by Novartis ( Novartis Pharmaceuticals ):
Study Protocol  [PDF] March 11, 2016
Statistical Analysis Plan  [PDF] March 15, 2018


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Responsible Party: Novartis Pharmaceuticals
ClinicalTrials.gov Identifier: NCT02292446     History of Changes
Other Study ID Numbers: CINC424B2001X
First Posted: November 17, 2014    Key Record Dates
Results First Posted: April 2, 2019
Last Update Posted: April 2, 2019
Last Verified: December 2018

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Novartis ( Novartis Pharmaceuticals ):
Polycythemia Vera
Hematologic Diseases
Myeloproliferative Disorders
INC424
Ruxolitinib
hydroxyurea resistant
adult

Additional relevant MeSH terms:
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Polycythemia
Polycythemia Vera
Hematologic Diseases
Bone Marrow Neoplasms
Hematologic Neoplasms
Neoplasms by Site
Neoplasms
Bone Marrow Diseases
Myeloproliferative Disorders
Hydroxyurea
Antineoplastic Agents
Antisickling Agents
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action
Nucleic Acid Synthesis Inhibitors