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.

July 16, 2019 updated by: Novartis Pharmaceuticals

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.

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

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

161

Phase

  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Austria
      • Linz, Austria, A-4010
        • Novartis Investigative Site
      • Salzburg, Austria, 5020
        • Novartis Investigative Site
      • Wels, Austria, A 4600
        • Novartis Investigative Site
  • Belgium
      • 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
        • Novartis Investigative Site
  • Bulgaria
      • Pleven, Bulgaria, 5800
        • Novartis Investigative Site
      • Plovdiv, Bulgaria, 4002
        • Novartis Investigative Site
      • Sofia, Bulgaria, 1413
        • Novartis Investigative Site
      • Sofia, Bulgaria, 1431
        • Novartis Investigative Site
  • Canada
    • British Columbia
      • Vancouver, British Columbia, Canada, V5Z 1M9
        • Novartis Investigative Site
    • Ontario
      • Hamilton, Ontario, Canada, L8V 5C2
        • Novartis Investigative Site
  • Chile
      • Santiago, Chile, 8420383
        • Novartis Investigative Site
      • Santiago, Chile
        • Novartis Investigative Site
    • Valparaiso
      • Vina del Mar, Valparaiso, Chile, 2540364
        • Novartis Investigative Site
  • France
      • 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
        • Novartis Investigative Site
    • Bayonne Cedex
      • Bayonne, Bayonne Cedex, France, 64109
        • Novartis Investigative Site
    • Cedex 09
      • Le Mans, Cedex 09, France, 72037
        • Novartis Investigative Site
  • Germany
      • 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, 60596
        • Novartis Investigative Site
      • Frankfurt, Germany, 60389
        • 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
        • Novartis Investigative Site
    • Baden-Wuerttemberg
      • Mannheim, Baden-Wuerttemberg, Germany, 68305
        • Novartis Investigative Site
  • Mexico
    • Nuevo Leon
      • Monterrey, Nuevo Leon, Mexico, 64000
        • Novartis Investigative Site
  • Norway
      • Fredrikstad, Norway, NO-1603
        • Novartis Investigative Site
      • Tromso, Norway, 9038
        • Novartis Investigative Site
  • Portugal
      • Lisboa, Portugal, 1749-035
        • Novartis Investigative Site
      • Lisboa, Portugal, 1099 023
        • Novartis Investigative Site
  • Sweden
      • Lulea, Sweden, SE 971 80
        • Novartis Investigative Site
      • Uddevalla, Sweden, 451 80
        • Novartis Investigative Site
  • Thailand
      • Bangkok, Thailand, 10700
        • Novartis Investigative Site
      • Bangkok, Thailand, 10400
        • Novartis Investigative Site
      • Chiang Mai, Thailand, 50200
        • Novartis Investigative Site
    • THA
      • Khon Kaen, THA, Thailand, 40002
        • Novartis Investigative Site

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years and older (ADULT, OLDER_ADULT)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: TREATMENT
  • Allocation: NA
  • Interventional Model: SINGLE_GROUP
  • Masking: NONE

Arms and Interventions

Participant Group / 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

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Number of Participants With Adverse Events - All Grades
Time Frame: Baseline up to approximately 26 months
Summary of adverse events (all grades).
Baseline up to approximately 26 months

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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
Up to approximately 26 months
Change From Baseline in Spleen Length
Time Frame: Up to approximately 26 months
Change in spleen length from Baseline to each visit
Up to approximately 26 months
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.
Up to approximately 26 months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Novartis Pharmaceuticals

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (ACTUAL)

November 21, 2014

Primary Completion (ACTUAL)

December 29, 2017

Study Completion (ACTUAL)

December 29, 2017

Study Registration Dates

First Submitted

November 5, 2014

First Submitted That Met QC Criteria

November 12, 2014

First Posted (ESTIMATE)

November 17, 2014

Study Record Updates

Last Update Posted (ACTUAL)

July 18, 2019

Last Update Submitted That Met QC Criteria

July 16, 2019

Last Verified

July 1, 2019

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • CINC424B2001X

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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