Back to resultsExpanded Treatment Protocol (ETP) of Ruxolitinib in Patients With Polycythemia Vera Who Were Hydroxyurea Resistant or Intolerant and for Whom no Treatment Alternatives Was Available.
Primary Purpose
Polycythemia Vera
Status
Completed
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
Ruxolitinib
Sponsored by
About this trial
This is an interventional treatment trial for Polycythemia Vera focused on measuring Polycythemia Vera, Hematologic Diseases, Myeloproliferative Disorders, INC424, Ruxolitinib, hydroxyurea resistant, adult
Eligibility 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.
Sites / Locations
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
All patients
Arm Description
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
Outcomes
Primary Outcome Measures
Number of Participants With Adverse Events - All Grades
Summary of adverse events (all grades).
Secondary Outcome Measures
Change From Baseline in Hematocrit Levels at All Visits
Change in hematocrit levels from Baseline to each visit were measured
Change From Baseline in Spleen Length
Change in spleen length from Baseline to each visit
Change From Baseline in Myeloproliferative Neoplasm Symptom Assessment Form Total Symptom Score (MPN-SAF TSS)
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.
Full Information
NCT ID
NCT02292446
First Posted
November 5, 2014
Last Updated
July 16, 2019
Sponsor
Novartis Pharmaceuticals
1. Study Identification
Unique Protocol Identification Number
NCT02292446
Brief Title
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
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.
Study Type
Interventional
2. Study Status
Record Verification Date
July 2019
Overall Recruitment Status
Completed
Study Start Date
November 21, 2014 (Actual)
Primary Completion Date
December 29, 2017 (Actual)
Study Completion Date
December 29, 2017 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Novartis Pharmaceuticals
4. Oversight
Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No
5. Study Description
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
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Polycythemia Vera
Keywords
Polycythemia Vera, Hematologic Diseases, Myeloproliferative Disorders, INC424, Ruxolitinib, hydroxyurea resistant, adult
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
161 (Actual)
8. Arms, Groups, and Interventions
Arm Title
All patients
Arm Type
Experimental
Arm Description
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 Type
Drug
Intervention Name(s)
Ruxolitinib
Intervention Description
supplied as 5 mg, 10 mg and 20 mg tablets to be taken orally
Primary Outcome Measure Information:
Title
Number of Participants With Adverse Events - All Grades
Description
Summary of adverse events (all grades).
Time Frame
Baseline up to approximately 26 months
Secondary Outcome Measure Information:
Title
Change From Baseline in Hematocrit Levels at All Visits
Description
Change in hematocrit levels from Baseline to each visit were measured
Time Frame
Up to approximately 26 months
Title
Change From Baseline in Spleen Length
Description
Change in spleen length from Baseline to each visit
Time Frame
Up to approximately 26 months
Title
Change From Baseline in Myeloproliferative Neoplasm Symptom Assessment Form Total Symptom Score (MPN-SAF TSS)
Description
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.
Time Frame
Up to approximately 26 months
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility 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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Novartis Pharmaceuticals
Organizational Affiliation
Novartis Pharmaceuticals
Official's Role
Study Director
Facility Information:
Facility Name
Novartis Investigative Site
City
Linz
ZIP/Postal Code
A-4010
Country
Austria
Facility Name
Novartis Investigative Site
City
Salzburg
ZIP/Postal Code
5020
Country
Austria
Facility Name
Novartis Investigative Site
City
Wels
ZIP/Postal Code
A 4600
Country
Austria
Facility Name
Novartis Investigative Site
City
Antwerp
ZIP/Postal Code
2060
Country
Belgium
Facility Name
Novartis Investigative Site
City
Brugge
ZIP/Postal Code
8000
Country
Belgium
Facility Name
Novartis Investigative Site
City
Bruxelles
ZIP/Postal Code
1070
Country
Belgium
Facility Name
Novartis Investigative Site
City
Leuven
ZIP/Postal Code
3000
Country
Belgium
Facility Name
Novartis Investigative Site
City
Liege
ZIP/Postal Code
4000
Country
Belgium
Facility Name
Novartis Investigative Site
City
Yvoir
ZIP/Postal Code
5530
Country
Belgium
Facility Name
Novartis Investigative Site
City
Pleven
ZIP/Postal Code
5800
Country
Bulgaria
Facility Name
Novartis Investigative Site
City
Plovdiv
ZIP/Postal Code
4002
Country
Bulgaria
Facility Name
Novartis Investigative Site
City
Sofia
ZIP/Postal Code
1413
Country
Bulgaria
Facility Name
Novartis Investigative Site
City
Sofia
ZIP/Postal Code
1431
Country
Bulgaria
Facility Name
Novartis Investigative Site
City
Vancouver
State/Province
British Columbia
ZIP/Postal Code
V5Z 1M9
Country
Canada
Facility Name
Novartis Investigative Site
City
Hamilton
State/Province
Ontario
ZIP/Postal Code
L8V 5C2
Country
Canada
Facility Name
Novartis Investigative Site
City
Vina del Mar
State/Province
Valparaiso
ZIP/Postal Code
2540364
Country
Chile
Facility Name
Novartis Investigative Site
City
Santiago
ZIP/Postal Code
8420383
Country
Chile
Facility Name
Novartis Investigative Site
City
Santiago
Country
Chile
Facility Name
Novartis Investigative Site
City
Bayonne
State/Province
Bayonne Cedex
ZIP/Postal Code
64109
Country
France
Facility Name
Novartis Investigative Site
City
Le Mans
State/Province
Cedex 09
ZIP/Postal Code
72037
Country
France
Facility Name
Novartis Investigative Site
City
Angers Cedex 1
ZIP/Postal Code
49033
Country
France
Facility Name
Novartis Investigative Site
City
Avignon cedex 9
ZIP/Postal Code
84902
Country
France
Facility Name
Novartis Investigative Site
City
Bordeaux
ZIP/Postal Code
33076
Country
France
Facility Name
Novartis Investigative Site
City
Chambéry Cedex
ZIP/Postal Code
73011
Country
France
Facility Name
Novartis Investigative Site
City
Marseille
ZIP/Postal Code
13273
Country
France
Facility Name
Novartis Investigative Site
City
Meaux cedex
ZIP/Postal Code
77104
Country
France
Facility Name
Novartis Investigative Site
City
Metz
ZIP/Postal Code
57000
Country
France
Facility Name
Novartis Investigative Site
City
Mulhouse cedex
ZIP/Postal Code
68070
Country
France
Facility Name
Novartis Investigative Site
City
Nice Cedex
ZIP/Postal Code
06202
Country
France
Facility Name
Novartis Investigative Site
City
Paris
ZIP/Postal Code
75010
Country
France
Facility Name
Novartis Investigative Site
City
Perpignan
ZIP/Postal Code
66046
Country
France
Facility Name
Novartis Investigative Site
City
Pringy cedex
ZIP/Postal Code
74374
Country
France
Facility Name
Novartis Investigative Site
City
Toulouse Cedex 9
ZIP/Postal Code
31059
Country
France
Facility Name
Novartis Investigative Site
City
Vandoeuvre Les Nancy
ZIP/Postal Code
54511
Country
France
Facility Name
Novartis Investigative Site
City
Villejuif Cedex
ZIP/Postal Code
94805
Country
France
Facility Name
Novartis Investigative Site
City
Mannheim
State/Province
Baden-Wuerttemberg
ZIP/Postal Code
68305
Country
Germany
Facility Name
Novartis Investigative Site
City
Aschaffenburg
ZIP/Postal Code
63739
Country
Germany
Facility Name
Novartis Investigative Site
City
Augsburg
ZIP/Postal Code
86150
Country
Germany
Facility Name
Novartis Investigative Site
City
Bad Soden
ZIP/Postal Code
65812
Country
Germany
Facility Name
Novartis Investigative Site
City
Berlin
ZIP/Postal Code
13357
Country
Germany
Facility Name
Novartis Investigative Site
City
Bottrop
ZIP/Postal Code
46236
Country
Germany
Facility Name
Novartis Investigative Site
City
Eisenach
ZIP/Postal Code
99817
Country
Germany
Facility Name
Novartis Investigative Site
City
Erlangen
ZIP/Postal Code
91054
Country
Germany
Facility Name
Novartis Investigative Site
City
Frankfurt
ZIP/Postal Code
60389
Country
Germany
Facility Name
Novartis Investigative Site
City
Frankfurt
ZIP/Postal Code
60596
Country
Germany
Facility Name
Novartis Investigative Site
City
Friedrichshafen
ZIP/Postal Code
88045
Country
Germany
Facility Name
Novartis Investigative Site
City
Hamburg
ZIP/Postal Code
22081
Country
Germany
Facility Name
Novartis Investigative Site
City
Hamm
ZIP/Postal Code
59063
Country
Germany
Facility Name
Novartis Investigative Site
City
Heidelberg
ZIP/Postal Code
69115
Country
Germany
Facility Name
Novartis Investigative Site
City
Heilbronn
ZIP/Postal Code
74072
Country
Germany
Facility Name
Novartis Investigative Site
City
Koblenz
ZIP/Postal Code
56068
Country
Germany
Facility Name
Novartis Investigative Site
City
Mutlangen
ZIP/Postal Code
73557
Country
Germany
Facility Name
Novartis Investigative Site
City
Stuttgart
ZIP/Postal Code
70376
Country
Germany
Facility Name
Novartis Investigative Site
City
Wuerzburg
ZIP/Postal Code
97080
Country
Germany
Facility Name
Novartis Investigative Site
City
Monterrey
State/Province
Nuevo Leon
ZIP/Postal Code
64000
Country
Mexico
Facility Name
Novartis Investigative Site
City
Fredrikstad
ZIP/Postal Code
NO-1603
Country
Norway
Facility Name
Novartis Investigative Site
City
Tromso
ZIP/Postal Code
9038
Country
Norway
Facility Name
Novartis Investigative Site
City
Lisboa
ZIP/Postal Code
1099 023
Country
Portugal
Facility Name
Novartis Investigative Site
City
Lisboa
ZIP/Postal Code
1749-035
Country
Portugal
Facility Name
Novartis Investigative Site
City
Lulea
ZIP/Postal Code
SE 971 80
Country
Sweden
Facility Name
Novartis Investigative Site
City
Uddevalla
ZIP/Postal Code
451 80
Country
Sweden
Facility Name
Novartis Investigative Site
City
Khon Kaen
State/Province
THA
ZIP/Postal Code
40002
Country
Thailand
Facility Name
Novartis Investigative Site
City
Bangkok
ZIP/Postal Code
10400
Country
Thailand
Facility Name
Novartis Investigative Site
City
Bangkok
ZIP/Postal Code
10700
Country
Thailand
Facility Name
Novartis Investigative Site
City
Chiang Mai
ZIP/Postal Code
50200
Country
Thailand
12. IPD Sharing Statement
Learn more about this trial
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.
We'll reach out to this number within 24 hrs