This trial is active, not recruiting.

Conditions primary myelofibrosis (mf), post-polycythemia vera (pv) mf, post-essential thrombocythemia (et) mf
Treatment ruxolitinib
Phase phase 2
Target JAK
Sponsor Novartis Pharmaceuticals
Start date August 2011
End date November 2017
Trial size 120 participants
Trial identifier NCT01392443, CINC424A2202


The objective of this study is to determine the efficacy of INC424 as assessed by reduction in spleen volume in patients with primary myelofibrosis (MF), post-polycythemia vera (PV) MF, or post-essential thrombocythemia (ET) MF. The safety and tolerability of INC424 and the effects of INC424 on patient reported outcomes and the duration of response as assessed by reduction in spleen volume will also be assessed.

United States No locations recruiting
Other Countries No locations recruiting

Study Design

Intervention model single group assignment
Masking open label
Primary purpose treatment
Ruxolitinib is taken twice daily, unless instructed. Starting dose 15 mg BID for patients with baseline platelet count of 100,000/μL to 200,000/μL (inclusive) or 20 mg BID for those with baseline platelet count >200,000/μL (approximately 12 hours apart: morning and night), to be increased or decreased per standardized dosing paradigm.
ruxolitinib INC424
INC424 Tablet for oral use, provided in 5 mg bottles. The dosage strength is 5 mg/tablet INC424 phosphate (free base equivalent). Medication labels will be in the local language and comply with the legal requirements of each country. equivalent).

Primary Outcomes

Efficacy by reduction in spleen volume
time frame: 24 weeks

Secondary Outcomes

Safety/tolerability of INC424
time frame: Frequency, duration and severity of adverse events
patient reported outcomes
time frame: 24 weeks
Duration of response
time frame: about 3 years

Eligibility Criteria

Male or female participants from 18 years up to 65 years old.

Inclusion Criteria: 1. 18 years or older 2. Diagnosis of primary myelofibrosis (MF), post-polycythemia vera (PV) MF, or post-essential thrombocythemia (ET) MF 3. Enlarged spleen, measuring 5 cm or greater from the costal margin 4. Must have two or more of the following risk factors: 1. Over 65 years old 2. Have the following symptoms often associated with MF: loss of weight, fever, night sweats 3. Have a low red blood cell count (anemia - hemoglobin < 10 g/dL) 4. Have a high white blood cell count (history of white blood cell count > 25,000/uL) 5. Have high circulating blasts (> or = 1%) as measured by blood tests 5. Should have circulating blasts <10% (as measured by blood tests) 6. Should be capable of self-care 7. Should have adequate bone marrow reserve 8. Should not have the option of stem cell transplantation 9. Should discontinue any prior or ongoing treatment for myelofibrosis prior to entering the study 10. Had no prior treatment with another JAK inhibitor Exclusion Criteria: 1. Does not have adequate liver or kidney function (as measured by blood tests) 2. Has an active infection (bacterial, viral, etc.) 3. Has active hepatitis A, B, or C or positive for HIV 4. Has another cancer that needs active intervention 5. Had a history of bleeding disorder 6. Had a history of very low platelet counts (as measured by blood tests) not related to treatment of MF 7. Had radiation of the spleen within 1 year of joining the study 8. Does not have adequate heart function 9. Sufficient time has elapsed between stopping previous treatment for MF and joining the study 10. Females who are pregnant or breast-feeding 11. Not able to sign informed consent 12. Has any other active medical conditions that the doctor deems may compromise your safety or ability to join in the study Other protocol-defined inclusion/exclusion criteria may apply

Additional Information

Official title A Multi-national Open-label Phase II Study of the JAK Inhibitor INC424 in Patients With Primary Myelofibrosis, Post-polycythemia Vera Myelofibrosis or Post-essential Thrombocythemia Myelofibrosis
Trial information was received from ClinicalTrials.gov and was last updated in September 2016.
Information provided to ClinicalTrials.gov by Novartis.