NCT04551053

To Evaluate Efficacy and Safety of Parsaclisib and Ruxolitinib in Participants With Myelofibrosis Who Have Suboptimal Response to Ruxolitinib (LIMBER-304)

A Randomized, Double-Blind, Placebo-Controlled Study of the PI3Kδ Inhibitor Parsaclisib Plus Ruxolitinib in Participants With Myelofibrosis Who Have Suboptimal Response to Ruxolitinib

• Status: Terminated
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 177 (actual)
• Sponsor: Incyte Corporation · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

The purpose of the study is to compare the efficacy and safety of parsaclisib when combined with ruxolitinb versus placebo combined with ruxolitinib in participants with myelofibrosis who have suboptimal response while receiving ruxolitinib monotherapy.

Detailed description

Prospective participants must be on stable doses of ruxolitinib ranging from 5 mg BID to 25 mg BID and will have been on that dose for at least the last 8 weeks prior to Day 1. At least 3 months duration of prior ruxolitinib is required. Participants must meet Protocol-defined criteria for suboptimal response to ruxolitinib monotherapy. After participants have been determined to be eligible for the study and completed the baseline symptom diary assessment for 7 days, they will be randomized to 1 of 2 treatment groups, with stratification for platelet count (≥ 100 × 10\^9/L vs 50 to \< 100 × 10\^9/L inclusive) and DIPSS risk category (high vs intermediate-2 vs intermediate-1). Once a participant has completed the week 24 assessments, the participant's treatment assignment will then be unblinded and if found to be placebo, the participant will have the opportunity to crossover to begin receiving parsaclisib, together with continued ruxolitinib, as long as hematology parameters are adequate.

Conditions

• Myelofibrosis
• Primary Myelofibrosis
• Post Essential Thrombocythemia Myelofibrosis
• Post Polycythemia Vera Myelofibrosis

Interventions

Type	Name	Description
DRUG	parsaclisib	parsaclisib will be administered QD orally
DRUG	ruxolitinib	ruxolitinib will be administered BID orally
DRUG	placebo	placebo will be administered QD orally

Timeline

• Start date: 2021-05-26
• Primary completion: 2023-08-16
• Completion: 2024-08-21
• First posted: 2020-09-16
• Last updated: 2025-10-02
• Results posted: 2024-08-23

Locations

177 sites across 19 countries: United States, Austria, Belgium, China, Finland, France, Germany, Hungary, Israel, Italy, Japan, Norway, Poland, Romania, South Korea, Spain, Taiwan, Turkey (Türkiye), United Kingdom

Regulatory

• FDA-regulated drug study