INCB 50465-304
Research type
Research Study
Full title
A Randomized, Double-Blind, Placebo-Controlled Study of the PI3Kδ Inhibitor Parsaclisib Plus Ruxolitinib in Participants With Myelofibrosis Who Have Suboptimal Response to Ruxolitinib
IRAS ID
290797
Contact name
Ciro Rinaldi
Contact email
Sponsor organisation
Incyte Corporation
Eudract number
2020-003415-98
ISRCTN Number
ISRCTN00000000
Clinicaltrials.gov Identifier
Duration of Study in the UK
4 years, 2 months, 3 days
Research summary
Myelofibrosis (MF) is a type of blood cancer that affects the bone marrow. MF often causes the spleen to be enlarged which can cause significant problems in addition to MF symptoms. Drug therapies currently available for treating MF are not effective for all 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 who continue to have significant enlargement of the spleen and symptoms of MF while on treatment with ruxolitinib only.
Participants will be in the study for approximately 2 years. This will consist of a screening visit, then a baseline visit, before entering a 24-week treatment period. After the treatment period, participants can enter the extension period, during which those that had been taking placebo and ruxolitinib will be eligible to ‘crossover’ to instead receive parsaclisib and ruxolitinib. The extension period will continue as long as treatment is tolerated. An end-of-treatment (EOT)/early termination visit will take place once treatment is discontinued, and then participants will enter the follow-up period, with a visit 30-35 days after the EOT visit and then a survival follow-up phone call, email or visit every 12 weeks until the study finishes.
The study will recruit approximately 212 participants, with 50% receiving placebo and ruxolitinib, and 50% receiving parsaclisib and ruxolitinib, until the extension period where all will have the opportunity to take parsaclisib and ruxolitinib.
REC name
East Midlands - Derby Research Ethics Committee
REC reference
21/EM/0059
Date of REC Opinion
19 Apr 2021
REC opinion
Further Information Favourable Opinion