INCB 50465-313
Research type
Research Study
Full title
A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study of the Combination of PI3Kδ Inhibitor Parsaclisib and Ruxolitinib in Participants With Myelofibrosis
IRAS ID
291598
Contact name
Sebastian Francis
Contact email
Sponsor organisation
Incyte Corporation
Eudract number
2020-003130-21
ISRCTN Number
ISRCTN00000000
Duration of Study in the UK
4 years, 8 months, 1 days
Research summary
Myelofibrosis (MF) is a type of blood cancer affecting the bone marrow. MF often causes the spleen to be enlarged leading to significant problems in addition to MF symptoms. Drug therapies currently available for treating MF are ineffective for some patients. In this study, the investigational drug, parsaclisib, will be tested in combination with an already approved drug, ruxolitinib. Parsaclisib is a molecule that binds to an enzyme called PI3K and decreases its activity. PI3K enzyme levels are often increased in individuals with MF. Ruxolitinib targets genes that help abnormal blood cells in MF to grow, and can also reduce an enlarged spleen.
The main purpose of this study is to compare the safety and therapeutic effects of adding parsaclisib or placebo to ruxolitinib therapy in participants with MF.
Participants will be in the study for approximately 3 years. This will consist of 3 main parts. In part 1, 50% of participants will receive placebo and ruxolitinib, and 50% will receive parsaclisib and ruxolitinib. After 24 weeks, participants will move to part 2 of the study – the extension period – where they will continue receiving the same drug combination as part 1 for as long as they tolerate treatment. Once the last participant has completed their Week-24 assessments, the study will be unblinded, and participants taking placebo and ruxolitinib will have the opportunity to ‘crossover’ to receive parsaclisib and ruxolitinib. Crossover for some participants may occur early if they have spleen growth and worsening symptoms. Treatment will continue for as long as is tolerated. An end-of-treatment (EOT)/early termination visit will occur once treatment is discontinued, then participants will enter the follow-up period, with a visit 30-35 days after the EOT visit and then survival follow-up phone calls every 12 weeks until the study finishes.
The study will recruit approximately 440 participants.
REC name
Yorkshire & The Humber - Sheffield Research Ethics Committee
REC reference
21/YH/0080
Date of REC Opinion
5 May 2021
REC opinion
Further Information Favourable Opinion