Phase 2
N=120
Asian Phase II Study of INC424 in Patients With Primary Myelofibrosis (MF), Post-PV MF or Post-ET MF
Primary Myelofibrosis (MF) · Post-Polycythemia Vera (PV) MF · Post-Essential Thrombocythemia (ET) MF
Bottom Line
View on ClinicalTrials.gov: NCT01392443 ↗Enrolled (actual)
120
Serious AEs
50.0%
Results posted
Sep 2019
Primary outcome: Primary: Percentage of Participants With at Least 35% Reduction in Spleen Volume From Baseline at Week 24 — 38 Participants — p=0.0007
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Ruxolitinib (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- Novartis Pharmaceuticals
- Primary completion
- Oct 2017
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Percentage of Participants With at Least 35% Reduction in Spleen Volume From Baseline at Week 24 |
38 | 0.0007 sig |
| SECONDARY Percentage of Participants With at Least 35% Reduction in Spleen Volume From Baseline at Each Scheduled Time Point - Best Response |
35.8; 40.0; 44.2; 44.2; 45.8; 45.8 | — |
| SECONDARY Kaplan Meier Estimates of Duration of Response of at Least ≥ 35% Reduction From Baseline in Spleen Volume Per Kaplan Meier Estimates |
1.00; 0.96; 0.96; 0.93; 0.93; 0.91 | — |
| SECONDARY Change in EORTC QLQ-C30 Scores From Baseline in at Week 24 |
5.2; 0.6; -0.2; 1.9; -4.0; 0.2 | — |
| SECONDARY Change in Total Symptom Score From Baseline at Week 24 as Measured by Seven-day Modified MFSAF v2.0 |
-5.0 | — |
Summary
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.
Eligibility Criteria
Inclusion Criteria
- 18 years or older
- Diagnosis of primary myelofibrosis (MF), post-polycythemia vera (PV) MF, or post-essential thrombocythemia (ET) MF
- Enlarged spleen, measuring 5 cm or greater from the costal margin
- Must have two or more of the following risk factors:
- Over 65 years old
- Have the following symptoms often associated with MF: loss of weight, fever, night sweats
- Have a low red blood cell count (anemia - hemoglobin 25,000/uL)
- Have high circulating blasts (> or = 1%) as measured by blood tests
- Should have circulating blasts <10% (as measured by blood tests)
- Should be capable of self-care
- Should have adequate bone marrow reserve
- Should not have the option of stem cell transplantation
- Should discontinue any prior or ongoing treatment for myelofibrosis prior to entering the study
- Had no prior treatment with another JAK inhibitor
Exclusion Criteria
- Does not have adequate liver or kidney function (as measured by blood tests)
- Has an active infection (bacterial, viral, etc.)
- Has active hepatitis A, B, or C or positive for HIV
- Has another cancer that needs active intervention
- Had a history of bleeding disorder
- Had a history of very low platelet counts (as measured by blood tests) not related to treatment of MF
- Had radiation of the spleen within 1 year of joining the study
- Does not have adequate heart function
- Sufficient time has elapsed between stopping previous treatment for MF and joining the study
- Females who are pregnant or breast-feeding
- Not able to sign informed consent
- Has any other active medical conditions that the doctor deems may compromise your safety or ability to join in the study
Data sourced from ClinicalTrials.gov (NCT01392443). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.