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.

NCT02292446 | Completed Phase 3 Results

• 1 other identifier: CINC424B2001X
• Study Type: interventional
• Target: 161
• Locations: 12 countries
• Sites: 65

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

Conditions

Polycythemia Vera

Keywords

Polycythemia Vera; Hematologic Diseases; Myeloproliferative Disorders; INC424; Ruxolitinib; hydroxyurea resistant; adult

Outcome Measures

Primary Outcomes (1)

• Number of Participants With Adverse Events - All Grades

  Summary of adverse events (all grades).

  Baseline up to approximately 26 months

Secondary Outcomes (3)

• Change From Baseline in Hematocrit Levels at All Visits

  Up to approximately 26 months

• Change From Baseline in Spleen Length

  Up to approximately 26 months

• Change From Baseline in Myeloproliferative Neoplasm Symptom Assessment Form Total Symptom Score (MPN-SAF TSS)

  Up to approximately 26 months

Study Arms (1)

All patients

EXPERIMENTAL

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

Interventions

Ruxolitinib DRUG

supplied as 5 mg, 10 mg and 20 mg tablets to be taken orally

All patients

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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.

Sponsors & Collaborators

• Novartis Pharmaceuticals: lead

Study Sites (65)

Novartis Investigative Site

Linz, A-4010, Austria

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Novartis Investigative Site

Salzburg, 5020, Austria

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Novartis Investigative Site

Wels, A 4600, Austria

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Novartis Investigative Site

Antwerp, 2060, Belgium

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Novartis Investigative Site

Bruges, 8000, Belgium

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Novartis Investigative Site

Brussels, 1070, Belgium

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Novartis Investigative Site

Leuven, 3000, Belgium

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Novartis Investigative Site

Liège, 4000, Belgium

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Novartis Investigative Site

Yvoir, 5530, Belgium

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Novartis Investigative Site

Pleven, 5800, Bulgaria

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Novartis Investigative Site

Plovdiv, 4002, Bulgaria

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Novartis Investigative Site

Sofia, 1413, Bulgaria

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Novartis Investigative Site

Sofia, 1431, Bulgaria

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Novartis Investigative Site

Vancouver, British Columbia, V5Z 1M9, Canada

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Novartis Investigative Site

Hamilton, Ontario, L8V 5C2, Canada

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Novartis Investigative Site

Viña del Mar, Región de Valparaíso, 2540364, Chile

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Novartis Investigative Site

Santiago, 8420383, Chile

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Novartis Investigative Site

Santiago, Chile

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Novartis Investigative Site

Bayonne, Bayonne Cedex, 64109, France

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Novartis Investigative Site

Le Mans, Cedex 09, 72037, France

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Novartis Investigative Site

Angers, 49033, France

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Novartis Investigative Site

Avignon, 84902, France

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Novartis Investigative Site

Bordeaux, 33076, France

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Novartis Investigative Site

Chambéry, 73011, France

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Novartis Investigative Site

Marseille, 13273, France

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Novartis Investigative Site

Meaux, 77104, France

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Novartis Investigative Site

Metz, 57000, France

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Novartis Investigative Site

Mulhouse, 68070, France

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Novartis Investigative Site

Nice, 06202, France

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Novartis Investigative Site

Paris, 75010, France

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Novartis Investigative Site

Perpignan, 66046, France

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Novartis Investigative Site

Pringy, 74374, France

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Novartis Investigative Site

Toulouse, 31059, France

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Novartis Investigative Site

Vandœuvre-lès-Nancy, 54511, France

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Novartis Investigative Site

Villejuif, 94805, France

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Novartis Investigative Site

Mannheim, Baden-Wurttemberg, 68305, Germany

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Novartis Investigative Site

Aschaffenburg, 63739, Germany

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Novartis Investigative Site

Augsburg, 86150, Germany

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Novartis Investigative Site

Bad Soden, 65812, Germany

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Novartis Investigative Site

Berlin, 13357, Germany

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Novartis Investigative Site

Bottrop, 46236, Germany

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Novartis Investigative Site

Eisenach, 99817, Germany

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Novartis Investigative Site

Erlangen, 91054, Germany

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Novartis Investigative Site

Frankfurt, 60389, Germany

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Novartis Investigative Site

Frankfurt, 60596, Germany

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Novartis Investigative Site

Friedrichshafen, 88045, Germany

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Novartis Investigative Site

Hamburg, 22081, Germany

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Novartis Investigative Site

Hamm, 59063, Germany

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Novartis Investigative Site

Heidelberg, 69115, Germany

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Novartis Investigative Site

Heilbronn, 74072, Germany

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Novartis Investigative Site

Koblenz, 56068, Germany

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Novartis Investigative Site

Mutlangen, 73557, Germany

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Novartis Investigative Site

Stuttgart, 70376, Germany

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Novartis Investigative Site

Würzburg, 97080, Germany

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Novartis Investigative Site

Monterrey, Nuevo León, 64000, Mexico

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Novartis Investigative Site

Fredrikstad, NO-1603, Norway

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Novartis Investigative Site

Tromsø, 9038, Norway

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Novartis Investigative Site

Lisbon, 1099 023, Portugal

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Novartis Investigative Site

Lisbon, 1749-035, Portugal

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Novartis Investigative Site

Luleå, SE 971 80, Sweden

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Novartis Investigative Site

Uddevalla, 451 80, Sweden

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Novartis Investigative Site

Khon Kaen, THA, 40002, Thailand

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Novartis Investigative Site

Bangkok, 10400, Thailand

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Novartis Investigative Site

Bangkok, 10700, Thailand

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Novartis Investigative Site

Chiang Mai, 50200, Thailand

Location

MeSH Terms

Conditions

Polycythemia Vera; Hematologic Diseases; Myeloproliferative Disorders

Interventions

ruxolitinib

Condition Hierarchy (Ancestors)

Bone Marrow Neoplasms; Hematologic Neoplasms; Neoplasms by Site; Neoplasms; Bone Marrow Diseases; Hemic and Lymphatic Diseases

Results Point of Contact

• Title: Study Director
• Organization: Novaratis Pharmaceuticals

novartis.email@novartis.com

888-669-6682

Study Officials

• Novartis Pharmaceuticals

  Novartis Pharmaceuticals

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 5, 2014
• First Posted: November 17, 2014
• Study Start: November 21, 2014
• Primary Completion: December 29, 2017
• Study Completion: December 29, 2017
• Last Updated: July 18, 2019
• Results First Posted: April 2, 2019

Record last verified: 2019-07

Locations

SE (2); BE (6); CA (2); CL (3); ME (1); PT (2); TH (4); AU (3); FR (17); DE (19); NO (2); BU (4)