Working…
ClinicalTrials.gov
ClinicalTrials.gov Menu

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.

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
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 )

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

Layout table for study information
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.




Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Layout table for eligibility information
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


  Hide Study Locations
Locations
Layout table for location information
Austria
Novartis Investigative Site
Linz, Austria, A-4010
Novartis Investigative Site
Salzburg, Austria, 5020
Novartis Investigative Site
Wels, Austria, A 4600
Belgium
Novartis Investigative Site
Antwerp, Belgium, 2060
Novartis Investigative Site
Brugge, Belgium, 8000
Novartis Investigative Site
Bruxelles, Belgium, 1070
Novartis Investigative Site
Leuven, Belgium, 3000
Novartis Investigative Site
Liege, Belgium, 4000
Novartis Investigative Site
Yvoir, Belgium, 5530
Bulgaria
Novartis Investigative Site
Pleven, Bulgaria, 5800
Novartis Investigative Site
Plovdiv, Bulgaria, 4002
Novartis Investigative Site
Sofia, Bulgaria, 1413
Novartis Investigative Site
Sofia, Bulgaria, 1431
Canada, British Columbia
Novartis Investigative Site
Vancouver, British Columbia, Canada, V5Z 1M9
Canada, Ontario
Novartis Investigative Site
Hamilton, Ontario, Canada, L8V 5C2
Chile
Novartis Investigative Site
Vina del Mar, Valparaiso, Chile, 2540364
Novartis Investigative Site
Santiago, Chile, 8420383
Novartis Investigative Site
Santiago, Chile
France
Novartis Investigative Site
Bayonne, Bayonne Cedex, France, 64109
Novartis Investigative Site
Le Mans, Cedex 09, France, 72037
Novartis Investigative Site
Angers Cedex 1, France, 49033
Novartis Investigative Site
Avignon cedex 9, France, 84902
Novartis Investigative Site
Bordeaux, France, 33076
Novartis Investigative Site
Chambéry Cedex, France, 73011
Novartis Investigative Site
Marseille, France, 13273
Novartis Investigative Site
Meaux cedex, France, 77104
Novartis Investigative Site
Metz, France, 57000
Novartis Investigative Site
Mulhouse cedex, France, 68070
Novartis Investigative Site
Nice Cedex, France, 06202
Novartis Investigative Site
Paris, France, 75010
Novartis Investigative Site
Perpignan, France, 66046
Novartis Investigative Site
Pringy cedex, France, 74374
Novartis Investigative Site
Toulouse Cedex 9, France, 31059
Novartis Investigative Site
Vandoeuvre Les Nancy, France, 54511
Novartis Investigative Site
Villejuif Cedex, France, 94805
Germany
Novartis Investigative Site
Mannheim, Baden-Wuerttemberg, Germany, 68305
Novartis Investigative Site
Aschaffenburg, Germany, 63739
Novartis Investigative Site
Augsburg, Germany, 86150
Novartis Investigative Site
Bad Soden, Germany, 65812
Novartis Investigative Site
Berlin, Germany, 13357
Novartis Investigative Site
Bottrop, Germany, 46236
Novartis Investigative Site
Eisenach, Germany, 99817
Novartis Investigative Site
Erlangen, Germany, 91054
Novartis Investigative Site
Frankfurt, Germany, 60389
Novartis Investigative Site
Frankfurt, Germany, 60596
Novartis Investigative Site
Friedrichshafen, Germany, 88045
Novartis Investigative Site
Hamburg, Germany, 22081
Novartis Investigative Site
Hamm, Germany, 59063
Novartis Investigative Site
Heidelberg, Germany, 69115
Novartis Investigative Site
Heilbronn, Germany, 74072
Novartis Investigative Site
Koblenz, Germany, 56068
Novartis Investigative Site
Mutlangen, Germany, 73557
Novartis Investigative Site
Stuttgart, Germany, 70376
Novartis Investigative Site
Wuerzburg, Germany, 97080
Mexico
Novartis Investigative Site
Monterrey, Nuevo Leon, Mexico, 64000
Norway
Novartis Investigative Site
Fredrikstad, Norway, NO-1603
Novartis Investigative Site
Tromso, Norway, 9038
Portugal
Novartis Investigative Site
Lisboa, Portugal, 1099 023
Novartis Investigative Site
Lisboa, Portugal, 1749-035
Sweden
Novartis Investigative Site
Lulea, Sweden, SE 971 80
Novartis Investigative Site
Uddevalla, Sweden, 451 80
Thailand
Novartis Investigative Site
Khon Kaen, THA, Thailand, 40002
Novartis Investigative Site
Bangkok, Thailand, 10400
Novartis Investigative Site
Bangkok, Thailand, 10700
Novartis Investigative Site
Chiang Mai, Thailand, 50200
Sponsors and Collaborators
Novartis Pharmaceuticals
Investigators
Layout table for investigator information
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


Layout table for additonal information
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: July 18, 2019
Last Verified: July 2019

Layout table for additional information
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:
Layout table for MeSH terms
Polycythemia Vera
Polycythemia
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