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.

• 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: Novartis Pharmaceuticals
• Information provided by (Responsible Party): Novartis ( Novartis Pharmaceuticals )

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

Condition or disease	Intervention/treatment	Phase
Polycythemia Vera	Drug: Ruxolitinib	Phase 3

Study Design

• 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

Arms and Interventions

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

Outcome Measures

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.

Eligibility Criteria

• 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.

Contacts and Locations

  Show 65 Study Locations

Sponsors and Collaborators

Novartis Pharmaceuticals

Investigators

• 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

More Information

• Responsible Party: Novartis Pharmaceuticals
• ClinicalTrials.gov Identifier: NCT02292446 History of Changes
• Other Study ID Numbers: CINC424B2001X
• First Posted: November 17, 2014
• Results First Posted: April 2, 2019
• Last Update Posted: July 18, 2019
• Last Verified: July 2019

• 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:

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