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.

Polycythemia Vera Clinical Trial

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.

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02292446
• Other study ID #: CINC424B2001X
• Status: Completed
• Phase: Phase 3
• Start date: November 21, 2014
• Est. completion date: December 29, 2017

Study information

• Verified date: July 2019
• Source: Novartis
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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

Recruitment information / eligibility

• Status: Completed
• Enrollment: 161
• Est. completion date: December 29, 2017
• Est. primary completion date: December 29, 2017
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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.

Related Conditions & MeSH terms

• Polycythemia
• Polycythemia Vera

Intervention

Drug:
• Ruxolitinib
supplied as 5 mg, 10 mg and 20 mg tablets to be taken orally

Locations

Country	Name	City	State
Austria	Novartis Investigative Site	Linz
Austria	Novartis Investigative Site	Salzburg
Austria	Novartis Investigative Site	Wels
Belgium	Novartis Investigative Site	Antwerp
Belgium	Novartis Investigative Site	Brugge
Belgium	Novartis Investigative Site	Bruxelles
Belgium	Novartis Investigative Site	Leuven
Belgium	Novartis Investigative Site	Liege
Belgium	Novartis Investigative Site	Yvoir
Bulgaria	Novartis Investigative Site	Pleven
Bulgaria	Novartis Investigative Site	Plovdiv
Bulgaria	Novartis Investigative Site	Sofia
Bulgaria	Novartis Investigative Site	Sofia
Canada	Novartis Investigative Site	Hamilton	Ontario
Canada	Novartis Investigative Site	Vancouver	British Columbia
Chile	Novartis Investigative Site	Santiago
Chile	Novartis Investigative Site	Santiago
Chile	Novartis Investigative Site	Vina del Mar	Valparaiso
France	Novartis Investigative Site	Angers Cedex 1
France	Novartis Investigative Site	Avignon cedex 9
France	Novartis Investigative Site	Bayonne	Bayonne Cedex
France	Novartis Investigative Site	Bordeaux
France	Novartis Investigative Site	Chambéry Cedex
France	Novartis Investigative Site	Le Mans	Cedex 09
France	Novartis Investigative Site	Marseille
France	Novartis Investigative Site	Meaux cedex
France	Novartis Investigative Site	Metz
France	Novartis Investigative Site	Mulhouse cedex
France	Novartis Investigative Site	Nice Cedex
France	Novartis Investigative Site	Paris
France	Novartis Investigative Site	Perpignan
France	Novartis Investigative Site	Pringy cedex
France	Novartis Investigative Site	Toulouse Cedex 9
France	Novartis Investigative Site	Vandoeuvre Les Nancy
France	Novartis Investigative Site	Villejuif Cedex
Germany	Novartis Investigative Site	Aschaffenburg
Germany	Novartis Investigative Site	Augsburg
Germany	Novartis Investigative Site	Bad Soden
Germany	Novartis Investigative Site	Berlin
Germany	Novartis Investigative Site	Bottrop
Germany	Novartis Investigative Site	Eisenach
Germany	Novartis Investigative Site	Erlangen
Germany	Novartis Investigative Site	Frankfurt
Germany	Novartis Investigative Site	Frankfurt
Germany	Novartis Investigative Site	Friedrichshafen
Germany	Novartis Investigative Site	Hamburg
Germany	Novartis Investigative Site	Hamm
Germany	Novartis Investigative Site	Heidelberg
Germany	Novartis Investigative Site	Heilbronn
Germany	Novartis Investigative Site	Koblenz
Germany	Novartis Investigative Site	Mannheim	Baden-Wuerttemberg
Germany	Novartis Investigative Site	Mutlangen
Germany	Novartis Investigative Site	Stuttgart
Germany	Novartis Investigative Site	Wuerzburg
Mexico	Novartis Investigative Site	Monterrey	Nuevo Leon
Norway	Novartis Investigative Site	Fredrikstad
Norway	Novartis Investigative Site	Tromso
Portugal	Novartis Investigative Site	Lisboa
Portugal	Novartis Investigative Site	Lisboa
Sweden	Novartis Investigative Site	Lulea
Sweden	Novartis Investigative Site	Uddevalla
Thailand	Novartis Investigative Site	Bangkok
Thailand	Novartis Investigative Site	Bangkok
Thailand	Novartis Investigative Site	Chiang Mai
Thailand	Novartis Investigative Site	Khon Kaen	THA

Sponsors (1)

Lead Sponsor	Collaborator
Novartis Pharmaceuticals

Countries where clinical trial is conducted

Austria,  Belgium,  Bulgaria,  Canada,  Chile,  France,  Germany,  Mexico,  Norway,  Portugal,  Sweden,  Thailand, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Number of Participants With Adverse Events - All Grades	Summary of adverse events (all grades).	Baseline up to approximately 26 months
Secondary	Change From Baseline in Hematocrit Levels at All Visits	Change in hematocrit levels from Baseline to each visit were measured	Up to approximately 26 months
Secondary	Change From Baseline in Spleen Length	Change in spleen length from Baseline to each visit	Up to approximately 26 months
Secondary	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.	Up to approximately 26 months