Phase 1
N=69
Study of the JAK Inhibitor Ruxolitinib Administered Orally to Patients With Primary Myelofibrosis (PMF), Post-Polycythemia Vera-Myelofibrosis (PPV-MF) or Post-Essential Thrombocythemia-Myelofibrosis (PET-MF)
Myelofibrosis
Bottom Line
View on ClinicalTrials.gov: NCT01317875 ↗Enrolled (actual)
69
Serious AEs
55.1%
Results posted
Mar 2022
Primary outcome: Primary: Number of Participants With Dose Limiting Toxicities — 0; 0; 0; 0 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 1
- Interventions
- Ruxolitinib (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- Incyte Corporation
- Primary completion
- Dec 2019
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Participants With Dose Limiting Toxicities |
0; 0; 0; 0; 0; 0 | — |
| SECONDARY Number of Treatment Emergent Adverse Events (TEAE's) |
8; 20; 16; 7; 18 | — |
| SECONDARY Number of Subjects Achieving ≥ 50% Reduction in Palpable Spleen Length |
3; 6; 4; 1; 3 | — |
| SECONDARY Change in Spleen Length as Measure by Palpation Over Time |
-2.8; -3.7; -3.3; -5.4; -5.2; -5.4 | — |
| SECONDARY PK- C Reactive Protein Levels by PK Quartile (AUC0-12) |
14; 12; 21.2; 2.6; 7.6; 5.51 | — |
| SECONDARY PK- Interleukin 1 Receptor Antagonist Levels by PK Quartile (AUC0-12) |
188; 105; 267; 588; 592; 379 | — |
| SECONDARY PK- Tissue Necrosis Factor Receptor 2 Levels by PK Quartile (AUC0-12) |
24; 6.2; 20.5; 25.7; 14; 22.6 | — |
| SECONDARY AUC 0-Inf |
1027; 1930; 3756 | — |
Summary
This is a Phase IB, open-label, dose-finding study of the JAK 1 and 2 inhibitor ruxolitinib in patients with myelofibrosis (MF). The study consists of two periods: the core study period, comprising the dose escalation stage and the safety extension phase up to Week 24, then the extension study period beyond Week 24 and up to 3 years, to further characterize the safety and efficacy of ruxolitinib in this patient population. The dose escalation phase will enroll successive cohorts of patients who receive increasing doses of ruxolitinib until the maximum safe starting dose (MSSD) is determined. In the safety expansion phase, additional patients will be treated with ruxolitinib at the MSSD defined during dose escalation. The primary objective is to establish the MSSD of ruxolitinib in patients with MF and starting platelet counts < 100 x 10 ^9/L
Eligibility Criteria
Inclusion Criteria
- Require treatment for MF and classified at least as intermediate risk level 1 defined by the International Working Group.
- Platelet count < 100x10 ^9/L at screening or at Study Day 1.
Exclusion Criteria
- Received platelet transfusion within 14 days prior to Screening evaluations.
Data sourced from ClinicalTrials.gov (NCT01317875). 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.