A Clinical Study of Ruxolitinib in Patients With Primary Myelofibrosis (PM), Post-polycythemia Vera (PV) Myelofibrosis, or Post-essential Thrombocythemia (ET) Myelofibrosis

NCT02087059 | Completed Phase 3 Results

• 1 other identifier: CINC424AJP01
• Study Type: interventional
• Target: 51
• Locations: 1 country
• Sites: 29

Brief Summary

This is an open-label, multicenter clinical study in order to collect and examine data concerning the safety and efficacy of ruxolitinib in patients with Primary Myelofibrosis (MF), Post-Polycythemia Vera (PV) MF, Post-Essential Thrombocythemia (ET) MF.

Conditions

Primary Myelofibrosis (MF)

Keywords

Post-Polycythemia Vera (PV) MF; Post-Essential Thrombocythemia (ET) MF

Outcome Measures

Primary Outcomes (1)

• Number of Participants With Adverse Events as a Measure of Safety and Tolerability

  24 weeks

Secondary Outcomes (4)

• Charge in Spleen Size From Baseline at Specified Week

  Baseline, 24 weeks

• Charge in Spleen Size From Baseline up to the Specified Week

  Baseline, 24 weeks

• Summary of Total Symptom Score as Measured by Seven-day Modified Myelofibrosis Symptom Assessment Form (MFSAF) v2.0 by Time

  24 weeks

• Summary of Summary of EORTC QLQ-C30 Responses by Time

  24 weeks

Study Arms (1)

Ruxolitinib

EXPERIMENTAL

Ruxolitinib was administered orally twice daily at the starting dose of 5 mg, 15 mg or 20 mg bid based on Baseline platelet counts. The dosage was subsequently adjusted for safety and efficacy so that each patient was titrated to their most appropriate dose.

Drug: Ruxolitinib

Interventions

Ruxolitinib DRUG

Ruxolitinib

Eligibility Criteria

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

You may qualify if:

• ≥18 years of age
• Diagnosis of PMF, PPV-MF, or PET-MF, regardless of JAK2 mutational status. The diagnostic of PMF will be according to the World Health Organization (WHO) criteria (Thiele et al., 2008) and PPV-MF and PET-MF according to the International Working Group for Myelofibrosis Research and Treatment (IWG-MRT) criteria (Barosi et al., 2008).
• At least one risk factors provided in the definition of IWG-MRT (Cervantes et al., 2009; classified as intermediate risk-1, intermediate risk-2, or high risk)
• Patients with intermediate risk-1 (patients who have only one of the IMG-MRT risk factors indicated above ) must have palpable splenomegaly with a length of ≥5 cm from the costal margin to the point of the greatest spleen protrusion.
• Proportion of blasts in peripheral blood \<10%
• ECOG performance status of 0 to 2
• The following values for bone marrow function prior to treatment:
• Absolute neutrophil count ≥1,000/μL, and
• Platelet count ≥50,000/μL without administration of a growth factor, thrombopoietin, or platelet transfusion
• Stem cell transplantation is not a treatment option at present because it is not indicated or because there are no suitable donors.
• All drugs used to treat MF were discontinued at least 28 days before treatment initiation.
• Informed consent form should be signed before any screening procedures is performed

You may not qualify if:

• Hepatic or renal impairment as indicated by the following:
• Direct bilirubin ≥2-fold than the upper limit of normal (ULN)
• Alanine aminotransferase (ALT) \>2.5-fold ULN
• Creatinine \>2.0 mg/dL
• Clinically significant infection by bacteria, fungus, mycobacteria, parasite, or virus (screening and enrollment postponed until completion of antibiotic treatment in patients with an acute bacterial infection that requires antibiotic use)
• Active hepatitis A, B, or C or HIV infection defined by a positive IgM-HA Ab test \[hepatitis A virus antibody (immunoglobulin M \[IgM\])\], HBs Ag test (hepatitis B surface antigen), HCV Ab test (hepatitis C virus antibody), or HIV Ab (human immunodeficiency virus antibody) at screening.
• History of malignancy within the previous 3 years, except for early-stage squamous cell carcinoma and basal cell carcinoma.
• History of serious congenital or acquired hemorrhagic disease
• Previous platelet count \<25,000/μL or absolute neutrophil count \<500/μL, except for patients currently undergoing treatment for a myeloproliferative neoplasm or cytotoxic therapy for any other reason.
• Splenic irradiation within 12 months before screening
• Administration of hematopoietic growth factor receptor agonists (erythropoietin, granulocyte colony stimulating factor, romiplostim, eltrombopag) within 14 days before screening or 28 days before treatment initiation.
• Currently receiving another investigational drug, or received another investigational drug within 30 days before the start of treatment.
• History of myocardial infarction or acute coronary syndrome within 6 months before screening
• Poorly controlled or unstable angina at present
• Rapid or paroxysmal atrial fibrillation at present

Sponsors & Collaborators

• Novartis Pharmaceuticals: lead

Study Sites (29)

Novartis Investigative Site

Nagoya, Aichi-ken, 467-8602, Japan

Location

Novartis Investigative Site

Akita, Akita, 010-8543, Japan

Location

Novartis Investigative Site

Matsuyama, Ehime, 790-8524, Japan

Location

Novartis Investigative Site

Tōon, Ehime, 791-0295, Japan

Location

Novartis Investigative Site

Fukuoka, Fukuoka, 812-8582, Japan

Location

Novartis Investigative Site

Kurume, Fukuoka, 830-0011, Japan

Location

Novartis Investigative Site

Gifu, Gifu, 501-1194, Japan

Location

Novartis Investigative Site

Maebashi, Gunma, 371-8511, Japan

Location

Novartis Investigative Site

Sapporo, Hokkaido, 060-8543, Japan

Location

Novartis Investigative Site

Sapporo, Hokkaido, 060-8648, Japan

Location

Novartis Investigative Site

Kobe, Hyōgo, 650-0017, Japan

Location

Novartis Investigative Site

Kobe, Hyōgo, 650-0047, Japan

Location

Novartis Investigative Site

Kumamoto, Kumamoto, 860-8556, Japan

Location

Novartis Investigative Site

Kyoto, Kyoto, 606-8507, Japan

Location

Novartis Investigative Site

Tsu, Mie-ken, 514-8507, Japan

Location

Novartis Investigative Site

Sendai, Miyagi, 980-8574, Japan

Location

Novartis Investigative Site

Miyazaki, Miyazaki, 889-1692, Japan

Location

Novartis Investigative Site

Okayama, Okayama-ken, 700-8558, Japan

Location

Novartis Investigative Site

Hirakata, Osaka, 573-1191, Japan

Location

Novartis Investigative Site

Sayama, Osaka, 589-8511, Japan

Location

Novartis Investigative Site

Suita, Osaka, 565-0871, Japan

Location

Novartis Investigative Site

Shimotsuke, Tochigi, 329-0498, Japan

Location

Novartis Investigative Site

Bunkyo-ku, Tokyo, 113-8431, Japan

Location

Novartis Investigative Site

Bunkyo-ku, Tokyo, 113-8519, Japan

Location

Novartis Investigative Site

Bunkyo-ku, Tokyo, 113-8603, Japan

Location

Novartis Investigative Site

Bunkyo-ku, Tokyo, 113-8677, Japan

Location

Novartis Investigative Site

Shinjuku-ku, Tokyo, 160-0023, Japan

Location

Novartis Investigative Site

Shinjuku-ku, Tokyo, 162-8666, Japan

Location

Novartis Investigative Site

Chūō, Yamanashi, 409-3898, Japan

Location

MeSH Terms

Conditions

Primary Myelofibrosis

Interventions

ruxolitinib

Condition Hierarchy (Ancestors)

Myeloproliferative Disorders; Bone Marrow Diseases; Hematologic Diseases; Hemic and Lymphatic Diseases

Results Point of Contact

• Title: Clinical Disclosure Office
• Organization: Novartis Pharmaceuticals

862-778-8300

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: March 12, 2014
• First Posted: March 14, 2014
• Study Start: April 1, 2014
• Primary Completion: March 1, 2015
• Study Completion: April 1, 2015
• Last Updated: July 11, 2016
• Results First Posted: July 11, 2016

Record last verified: 2016-05

Locations

JP (29)