Asian Phase II Study of INC424 in Patients With Primary Myelofibrosis (MF), Post-PV MF or Post-ET MF

Novartis

Status and phase: Completed; Phase 2

Conditions: Primary Myelofibrosis (MF); Post-Polycythemia Vera (PV) MF; Post-Essential Thrombocythemia (ET) MF

Treatments: Drug: Ruxolitinib

Study type: Interventional

Funder types: Industry

Identifiers

NCT01392443

CINC424A2202

Details and patient eligibility

About

The objective of this study was 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 was also assessed.

Enrollment: 120 patients

Sex: All

Ages: 18 to 65 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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120 participants in 1 patient group

Ruxolitinib

Experimental group

Description:

Ruxolitinib was 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), increased or decreased per standardized dosing paradigm.

Treatment:

Drug: Ruxolitinib