Trial Outcomes & Findings for To Evaluate Efficacy and Safety of Parsaclisib and Ruxolitinib in Participants With Myelofibrosis Who Have Suboptimal Response to Ruxolitinib (LIMBER-304) (NCT NCT04551053)

Last Updated: 2025-10-02

Results Overview

Participants had an MRI of the upper and lower abdomen and pelvis to determine the spleen volume. A CT scan was substituted for participants who were not candidates for MRI or when MRI was not readily available.

Recruitment status

TERMINATED

Study phase

PHASE3

Target enrollment

177 participants

Primary outcome timeframe

Baseline; Week 24

Results posted on

2025-10-02

Participant Flow

This study was conducted across sites in Austria, China, Finland, France, Germany, Hungary, Italy, Japan, South Korea, Norway, Poland, Spain, Turkey, the United Kingdom, and the United States.

Participant milestones

Participant milestones
Measure	Parsaclisib Plus Ruxolitinib Participants were randomized to receive parsaclisib plus ruxolitinib beginning on Day 1. Participants received parsaclisib at a dose of 5 milligrams (mg) once daily (QD). Participants also received the stable dose of ruxolitinib they were taking for the 8 weeks prior to Day 1. Treatment with parsaclisib plus ruxolitinib continued for as long as the participant tolerated the regimen and did not meet any discontinuation criteria.	Placebo Plus Ruxolitinib Participants were randomized to receive placebo plus ruxolitinib beginning on Day 1 and continuing until Week 24. Participants received matching placebo at a dose of 5 mg QD and received the stable dose of ruxolitinib they were taking for the 8 weeks prior to Day 1. After 24 weeks, participants randomized to receive placebo plus ruxolitinib had to switch to treatment with parsaclisib plus ruxolitinib or discontinue treatment. Treatment continued for as long as the regimen was tolerated and the participant did not meet any discontinuation criteria. Participants who demonstrated worsening symptomatic splenomegaly could have switched to treatment with parsaclisib plus ruxolitinib early.
Overall Study STARTED	90	87
Overall Study COMPLETED	0	0
Overall Study NOT COMPLETED	90	87

Reasons for withdrawal

Reasons for withdrawal
Measure	Parsaclisib Plus Ruxolitinib Participants were randomized to receive parsaclisib plus ruxolitinib beginning on Day 1. Participants received parsaclisib at a dose of 5 milligrams (mg) once daily (QD). Participants also received the stable dose of ruxolitinib they were taking for the 8 weeks prior to Day 1. Treatment with parsaclisib plus ruxolitinib continued for as long as the participant tolerated the regimen and did not meet any discontinuation criteria.	Placebo Plus Ruxolitinib Participants were randomized to receive placebo plus ruxolitinib beginning on Day 1 and continuing until Week 24. Participants received matching placebo at a dose of 5 mg QD and received the stable dose of ruxolitinib they were taking for the 8 weeks prior to Day 1. After 24 weeks, participants randomized to receive placebo plus ruxolitinib had to switch to treatment with parsaclisib plus ruxolitinib or discontinue treatment. Treatment continued for as long as the regimen was tolerated and the participant did not meet any discontinuation criteria. Participants who demonstrated worsening symptomatic splenomegaly could have switched to treatment with parsaclisib plus ruxolitinib early.
Overall Study Death	10	9
Overall Study Lost to Follow-up	0	2
Overall Study Study Terminated by Sponsor	57	65
Overall Study Withdrawal by Subject	12	5
Overall Study Adverse Event	2	0
Overall Study Lack of Efficacy	2	1
Overall Study Transitioned to Rollover per Protocol	5	4
Overall Study Splenapendectomi	1	0
Overall Study Medical Decision	1	1

Baseline Characteristics

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

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Parsaclisib Plus Ruxolitinib n=90 Participants Participants were randomized to receive parsaclisib plus ruxolitinib beginning on Day 1. Participants received parsaclisib at a dose of 5 milligrams (mg) once daily (QD). Participants also received the stable dose of ruxolitinib they were taking for the 8 weeks prior to Day 1. Treatment with parsaclisib plus ruxolitinib continued for as long as the participant tolerated the regimen and did not meet any discontinuation criteria.	Placebo Plus Ruxolitinib n=87 Participants Participants were randomized to receive placebo plus ruxolitinib beginning on Day 1 and continuing until Week 24. Participants received matching placebo at a dose of 5 mg QD and received the stable dose of ruxolitinib they were taking for the 8 weeks prior to Day 1. After 24 weeks, participants randomized to receive placebo plus ruxolitinib had to switch to treatment with parsaclisib plus ruxolitinib or discontinue treatment. Treatment continued for as long as the regimen was tolerated and the participant did not meet any discontinuation criteria. Participants who demonstrated worsening symptomatic splenomegaly could have switched to treatment with parsaclisib plus ruxolitinib early.	Total n=177 Participants Total of all reporting groups
Age, Continuous	64.0 years STANDARD_DEVIATION 9.88 • n=5 Participants	62.3 years STANDARD_DEVIATION 9.92 • n=7 Participants	63.19 years STANDARD_DEVIATION 9.88 • n=5 Participants
Sex: Female, Male Female	37 Participants n=5 Participants	38 Participants n=7 Participants	75 Participants n=5 Participants
Sex: Female, Male Male	53 Participants n=5 Participants	49 Participants n=7 Participants	102 Participants n=5 Participants
Race/Ethnicity, Customized White or Caucasian	45 Participants n=5 Participants	52 Participants n=7 Participants	97 Participants n=5 Participants
Race/Ethnicity, Customized Black or African American	1 Participants n=5 Participants	1 Participants n=7 Participants	2 Participants n=5 Participants
Race/Ethnicity, Customized Asian	37 Participants n=5 Participants	26 Participants n=7 Participants	63 Participants n=5 Participants
Race/Ethnicity, Customized Not Reported	7 Participants n=5 Participants	4 Participants n=7 Participants	11 Participants n=5 Participants
Race/Ethnicity, Customized Hispanic or Latino	2 Participants n=5 Participants	10 Participants n=7 Participants	12 Participants n=5 Participants
Race/Ethnicity, Customized Not Hispanic or Latino	70 Participants n=5 Participants	57 Participants n=7 Participants	127 Participants n=5 Participants
Race/Ethnicity, Customized Unknown	2 Participants n=5 Participants	4 Participants n=7 Participants	6 Participants n=5 Participants
Race/Ethnicity, Customized Captured as "Other" in Database	9 Participants n=5 Participants	12 Participants n=7 Participants	21 Participants n=5 Participants

PRIMARY outcome

Timeframe: Baseline; Week 24

Population: Intent-to-Treat (ITT) Population: all randomized participants. Participants were analyzed if they had both Baseline and Week 24 measurements, or discontinued treatment before 03MAR2023 or switched treatment before Week 24, or reached Week 24 before 03MAR2023 but were missing Week 24 assessments. For participants who switched to parsaclisib plus ruxolitinib treatment early, data was truncated at the time of switch.

Outcome measures

Outcome measures
Measure	Parsaclisib Plus Ruxolitinib n=72 Participants Participants were randomized to receive parsaclisib plus ruxolitinib beginning on Day 1. Participants received parsaclisib at a dose of 5 milligrams (mg) once daily (QD). Participants also received the stable dose of ruxolitinib they were taking for the 8 weeks prior to Day 1. Treatment with parsaclisib plus ruxolitinib continued for as long as the participant tolerated the regimen and did not meet any discontinuation criteria.	Placebo Plus Ruxolitinib n=72 Participants Participants were randomized to receive placebo plus ruxolitinib beginning on Day 1 and continuing until Week 24. Participants received matching placebo at a dose of 5 mg QD and received the stable dose of ruxolitinib they were taking for the 8 weeks prior to Day 1. After 24 weeks, participants randomized to receive placebo plus ruxolitinib had to switch to treatment with parsaclisib plus ruxolitinib or discontinue treatment. Treatment continued for as long as the regimen was tolerated and the participant did not meet any discontinuation criteria. Participants who demonstrated worsening symptomatic splenomegaly could have switched to treatment with parsaclisib plus ruxolitinib early.	Placebo Swith to Parsaclisib After 24 weeks, participants randomized to receive placebo plus ruxolitinib from Day 1 to Week 24 could have switched to treatment with parsaclisib plus ruxolitinib per the regimen received during the first 24 weeks of the study. Treatment continued for as long as the regimen was tolerated and the participant did not meet any discontinuation criteria. Participants who demonstrated worsening symptomatic splenomegaly could have switched to treatment with parsaclisb plus ruxolitinib early.
Percentage of Participants Achieving ≥25% Reduction in Spleen Volume From Baseline to Week 24 as Measured by Magnetic Resonance Imaging (MRI) (or Computed Tomography [CT] Scan in Applicable Participants)	16.7 percentage of participants	9.7 percentage of participants	—

SECONDARY outcome

Timeframe: Baseline; Week 24

Population: ITT Population. Participants were analyzed if they had both Baseline and Week 24 measurements, or discontinued treatment before 03MAR2023 or switched treatment before Week 24, or reached Week 24 before 03MAR2023 but were missing Week 24 assessments. For participants who switched to parsaclisib plus ruxolitinib treatment early, data was truncated at the time of switch.

Symptoms of myelofibrosis were assessed using the MFSAF v4.0 diary. The MFSAF v4.0 is composed of 7 individual symptom scores (fatigue, night sweats, itchiness, abdominal discomfort, pain under left ribs, early satiety, bone pain), each collected daily using a 0- (no symptoms) to 10-point (worst imaginable symptoms) scale. The daily TSS (0 to 70) is the sum of the 7 individual symptom scores collected in a day. A higher TSS corresponds to more severe symptoms. The TSS was marked as missing if there were any missing individual scores. Observations with missing dates were excluded from the analysis. The Baseline/Week 24 total score was defined as the average of the daily total scores from the last 7 days before the first dose of parsaclisib, placebo, or ruxolitinib/the Week 24 visit. The Baseline/Week 24 total score was marked as missing if there were ≥4 out of the 7 daily TSSs missing.

Outcome measures

Outcome measures
Measure	Parsaclisib Plus Ruxolitinib n=70 Participants Participants were randomized to receive parsaclisib plus ruxolitinib beginning on Day 1. Participants received parsaclisib at a dose of 5 milligrams (mg) once daily (QD). Participants also received the stable dose of ruxolitinib they were taking for the 8 weeks prior to Day 1. Treatment with parsaclisib plus ruxolitinib continued for as long as the participant tolerated the regimen and did not meet any discontinuation criteria.	Placebo Plus Ruxolitinib n=71 Participants Participants were randomized to receive placebo plus ruxolitinib beginning on Day 1 and continuing until Week 24. Participants received matching placebo at a dose of 5 mg QD and received the stable dose of ruxolitinib they were taking for the 8 weeks prior to Day 1. After 24 weeks, participants randomized to receive placebo plus ruxolitinib had to switch to treatment with parsaclisib plus ruxolitinib or discontinue treatment. Treatment continued for as long as the regimen was tolerated and the participant did not meet any discontinuation criteria. Participants who demonstrated worsening symptomatic splenomegaly could have switched to treatment with parsaclisib plus ruxolitinib early.	Placebo Swith to Parsaclisib After 24 weeks, participants randomized to receive placebo plus ruxolitinib from Day 1 to Week 24 could have switched to treatment with parsaclisib plus ruxolitinib per the regimen received during the first 24 weeks of the study. Treatment continued for as long as the regimen was tolerated and the participant did not meet any discontinuation criteria. Participants who demonstrated worsening symptomatic splenomegaly could have switched to treatment with parsaclisb plus ruxolitinib early.
Percentage of Participants Who Have a ≥50% Reduction in Total Symptom Score (TSS) From Baseline to Week 24 as Measured by the Myelofibrosis Symptom Assessment Form v.4.0 (MFSAF v4.0) Diary	17.1 percentage of participants	14.1 percentage of participants	—

SECONDARY outcome

Timeframe: Baseline; Week 24

Population: ITT Population. Only participants with data available were analyzed.

Outcome measures

Outcome measures
Measure	Parsaclisib Plus Ruxolitinib n=88 Participants Participants were randomized to receive parsaclisib plus ruxolitinib beginning on Day 1. Participants received parsaclisib at a dose of 5 milligrams (mg) once daily (QD). Participants also received the stable dose of ruxolitinib they were taking for the 8 weeks prior to Day 1. Treatment with parsaclisib plus ruxolitinib continued for as long as the participant tolerated the regimen and did not meet any discontinuation criteria.	Placebo Plus Ruxolitinib n=82 Participants Participants were randomized to receive placebo plus ruxolitinib beginning on Day 1 and continuing until Week 24. Participants received matching placebo at a dose of 5 mg QD and received the stable dose of ruxolitinib they were taking for the 8 weeks prior to Day 1. After 24 weeks, participants randomized to receive placebo plus ruxolitinib had to switch to treatment with parsaclisib plus ruxolitinib or discontinue treatment. Treatment continued for as long as the regimen was tolerated and the participant did not meet any discontinuation criteria. Participants who demonstrated worsening symptomatic splenomegaly could have switched to treatment with parsaclisib plus ruxolitinib early.	Placebo Swith to Parsaclisib After 24 weeks, participants randomized to receive placebo plus ruxolitinib from Day 1 to Week 24 could have switched to treatment with parsaclisib plus ruxolitinib per the regimen received during the first 24 weeks of the study. Treatment continued for as long as the regimen was tolerated and the participant did not meet any discontinuation criteria. Participants who demonstrated worsening symptomatic splenomegaly could have switched to treatment with parsaclisb plus ruxolitinib early.
Change in TSS From Baseline to Week 24 as Measured by the MFSAF v4.0 Diary Baseline	17.2 scores on a scale Standard Deviation 11.61	21.6 scores on a scale Standard Deviation 14.28	—
Change in TSS From Baseline to Week 24 as Measured by the MFSAF v4.0 Diary Change from Baseline at Week 24	-2.7 scores on a scale Standard Deviation 8.67	-2.7 scores on a scale Standard Deviation 9.98	—

SECONDARY outcome

Timeframe: Baseline; up to Week 24

Population: ITT Population. For participants who switched to parsaclisib plus ruxolitinib treatment early, data was truncated at the time of switch. Participants with valid, calculated Baseline TSS and at least 1 postbaseline TSS who did not have a ≥50% reduction in TSS at the time of analysis were censored at the time of the last valid calculated TSS. If the participants had no valid, calculated Baseline or postbaseline TSS, they were censored at the date of randomization.

Symptoms were assessed using the MFSAF v4.0 diary. The MFSAF v4.0 is composed of 7 individual symptom scores (fatigue, night sweats, itchiness, abdominal discomfort, pain under left ribs, early satiety, bone pain), each collected daily using a 0- (no symptoms) to 10-point (worst imaginable symptoms) scale. The daily TSS (0 to 70) is the sum of the 7 individual symptom scores collected in a day. A higher TSS corresponds to more severe symptoms. The TSS was marked as missing if there were any missing individual scores. Observations with missing dates were excluded from the analysis. The Baseline/Week 24 total score was defined as the average of the daily total scores from the last 7 days before the first dose of parsaclisib, placebo, or ruxolitinib/the Week 24 visit. The Baseline/Week 24 total scores was marked as missing if there were ≥4 out of the 7 daily TSSs missing. The 95% confidence interval was calculated using the Brookmeyer and Crowley's method with log-log transformation.

Outcome measures

Outcome measures
Measure	Parsaclisib Plus Ruxolitinib n=90 Participants Participants were randomized to receive parsaclisib plus ruxolitinib beginning on Day 1. Participants received parsaclisib at a dose of 5 milligrams (mg) once daily (QD). Participants also received the stable dose of ruxolitinib they were taking for the 8 weeks prior to Day 1. Treatment with parsaclisib plus ruxolitinib continued for as long as the participant tolerated the regimen and did not meet any discontinuation criteria.	Placebo Plus Ruxolitinib n=87 Participants Participants were randomized to receive placebo plus ruxolitinib beginning on Day 1 and continuing until Week 24. Participants received matching placebo at a dose of 5 mg QD and received the stable dose of ruxolitinib they were taking for the 8 weeks prior to Day 1. After 24 weeks, participants randomized to receive placebo plus ruxolitinib had to switch to treatment with parsaclisib plus ruxolitinib or discontinue treatment. Treatment continued for as long as the regimen was tolerated and the participant did not meet any discontinuation criteria. Participants who demonstrated worsening symptomatic splenomegaly could have switched to treatment with parsaclisib plus ruxolitinib early.	Placebo Swith to Parsaclisib After 24 weeks, participants randomized to receive placebo plus ruxolitinib from Day 1 to Week 24 could have switched to treatment with parsaclisib plus ruxolitinib per the regimen received during the first 24 weeks of the study. Treatment continued for as long as the regimen was tolerated and the participant did not meet any discontinuation criteria. Participants who demonstrated worsening symptomatic splenomegaly could have switched to treatment with parsaclisb plus ruxolitinib early.
Time to the First ≥50% Reduction in TSS as Measured by the MFSAF v4.0 Diary	NA days Interval 160.0 to The median and the upper limit of the confidence interval were not estimable because there were too few participants with a ≥50% reduction in TSS at the time of study termination.	NA days The median and the upper and lower limits of the confidence interval were not estimable because there were too few participants with a ≥50% reduction in TSS at the time of study termination.	—

SECONDARY outcome

Timeframe: up to 917 days

Population: ITT Population

Overall survival was defined as the interval between the randomization date and the date of death due to any cause.

Outcome measures

Outcome measures
Measure	Parsaclisib Plus Ruxolitinib n=90 Participants Participants were randomized to receive parsaclisib plus ruxolitinib beginning on Day 1. Participants received parsaclisib at a dose of 5 milligrams (mg) once daily (QD). Participants also received the stable dose of ruxolitinib they were taking for the 8 weeks prior to Day 1. Treatment with parsaclisib plus ruxolitinib continued for as long as the participant tolerated the regimen and did not meet any discontinuation criteria.	Placebo Plus Ruxolitinib n=87 Participants Participants were randomized to receive placebo plus ruxolitinib beginning on Day 1 and continuing until Week 24. Participants received matching placebo at a dose of 5 mg QD and received the stable dose of ruxolitinib they were taking for the 8 weeks prior to Day 1. After 24 weeks, participants randomized to receive placebo plus ruxolitinib had to switch to treatment with parsaclisib plus ruxolitinib or discontinue treatment. Treatment continued for as long as the regimen was tolerated and the participant did not meet any discontinuation criteria. Participants who demonstrated worsening symptomatic splenomegaly could have switched to treatment with parsaclisib plus ruxolitinib early.	Placebo Swith to Parsaclisib After 24 weeks, participants randomized to receive placebo plus ruxolitinib from Day 1 to Week 24 could have switched to treatment with parsaclisib plus ruxolitinib per the regimen received during the first 24 weeks of the study. Treatment continued for as long as the regimen was tolerated and the participant did not meet any discontinuation criteria. Participants who demonstrated worsening symptomatic splenomegaly could have switched to treatment with parsaclisb plus ruxolitinib early.
Overall Survival	NA days Due to study termination, the follow-up time was not long enough to estimate the median and the upper and lower limits of the confidence interval.	NA days Due to study termination, the follow-up time was not long enough to estimate the median and the upper and lower limits of the confidence interval.	—

SECONDARY outcome

Timeframe: up to 917 days

Population: Safety Population: all randomized participants who received at least 1 dose of parsaclisib, placebo, or ruxolitinib. Treatment groups for this population were determined according to the actual treatment the participant received regardless of assigned study drug treatment.

An adverse event (AE) is defined as any untoward medical occurrence associated with the use of a drug in humans, whether or not it is considered drug related. An AE could therefore be any unfavorable or unintended sign (including an abnormal laboratory finding), symptom, or disease (new or exacerbated) temporally associated with the use of study treatment. A TEAE is defined as an AE reported for the first time or the worsening of a pre-existing event after the first dose of study treatment.

Outcome measures

Outcome measures
Measure	Parsaclisib Plus Ruxolitinib n=90 Participants Participants were randomized to receive parsaclisib plus ruxolitinib beginning on Day 1. Participants received parsaclisib at a dose of 5 milligrams (mg) once daily (QD). Participants also received the stable dose of ruxolitinib they were taking for the 8 weeks prior to Day 1. Treatment with parsaclisib plus ruxolitinib continued for as long as the participant tolerated the regimen and did not meet any discontinuation criteria.	Placebo Plus Ruxolitinib n=87 Participants Participants were randomized to receive placebo plus ruxolitinib beginning on Day 1 and continuing until Week 24. Participants received matching placebo at a dose of 5 mg QD and received the stable dose of ruxolitinib they were taking for the 8 weeks prior to Day 1. After 24 weeks, participants randomized to receive placebo plus ruxolitinib had to switch to treatment with parsaclisib plus ruxolitinib or discontinue treatment. Treatment continued for as long as the regimen was tolerated and the participant did not meet any discontinuation criteria. Participants who demonstrated worsening symptomatic splenomegaly could have switched to treatment with parsaclisib plus ruxolitinib early.	Placebo Swith to Parsaclisib n=41 Participants After 24 weeks, participants randomized to receive placebo plus ruxolitinib from Day 1 to Week 24 could have switched to treatment with parsaclisib plus ruxolitinib per the regimen received during the first 24 weeks of the study. Treatment continued for as long as the regimen was tolerated and the participant did not meet any discontinuation criteria. Participants who demonstrated worsening symptomatic splenomegaly could have switched to treatment with parsaclisb plus ruxolitinib early.
Number of Participants With Any Treatment-emergent Adverse Event (TEAE)	81 Participants	76 Participants	33 Participants

SECONDARY outcome

Timeframe: up to 917 days

Population: Safety Population

An AE is defined as any untoward medical occurrence associated with the use of a drug in humans, whether or not it is considered drug related. A TEAE is defined as an AE reported for the first time or the worsening of a pre-existing event after the first dose of study treatment. The severity of AEs was assessed using Common Terminology Criteria for Adverse Events (CTCAE) v5.0 Grades 1 through 5. Grade 1: mild; asymptomatic or mild symptoms; clinical or diagnostic observations only; intervention not indicated. Grade 2: moderate; minimal, local or noninvasive intervention indicated; limiting instrumental activities of daily living (ADL). Grade 3: severe or medically significant but not immediately life-threatening; hospitalization or prolongation of hospitalization indicated; disabling; limiting self care ADL. Grade 4: life-threatening urgent intervention indicated. Grade 5: death related to AE.

Outcome measures

Outcome measures
Measure	Parsaclisib Plus Ruxolitinib n=90 Participants Participants were randomized to receive parsaclisib plus ruxolitinib beginning on Day 1. Participants received parsaclisib at a dose of 5 milligrams (mg) once daily (QD). Participants also received the stable dose of ruxolitinib they were taking for the 8 weeks prior to Day 1. Treatment with parsaclisib plus ruxolitinib continued for as long as the participant tolerated the regimen and did not meet any discontinuation criteria.	Placebo Plus Ruxolitinib n=87 Participants Participants were randomized to receive placebo plus ruxolitinib beginning on Day 1 and continuing until Week 24. Participants received matching placebo at a dose of 5 mg QD and received the stable dose of ruxolitinib they were taking for the 8 weeks prior to Day 1. After 24 weeks, participants randomized to receive placebo plus ruxolitinib had to switch to treatment with parsaclisib plus ruxolitinib or discontinue treatment. Treatment continued for as long as the regimen was tolerated and the participant did not meet any discontinuation criteria. Participants who demonstrated worsening symptomatic splenomegaly could have switched to treatment with parsaclisib plus ruxolitinib early.	Placebo Swith to Parsaclisib n=41 Participants After 24 weeks, participants randomized to receive placebo plus ruxolitinib from Day 1 to Week 24 could have switched to treatment with parsaclisib plus ruxolitinib per the regimen received during the first 24 weeks of the study. Treatment continued for as long as the regimen was tolerated and the participant did not meet any discontinuation criteria. Participants who demonstrated worsening symptomatic splenomegaly could have switched to treatment with parsaclisb plus ruxolitinib early.
Number of Participants With Any Grade 3 or Higher TEAE	54 Participants	37 Participants	18 Participants

SECONDARY outcome

Timeframe: up to 898 days

Population: ITT Population

The time to the first ≥25% reduction in spleen volume is defined as the time from randomization to the first time participants had ≥25% reduction in spleen volume. Participants with a Baseline and post-Baseline MRI or CT scan who did not have ≥25% reduction in spleen volume at the time of analysis were censored at the time of the last MRI or CT scan. If the participants had no Baseline or post-Baseline MRI or CT scan, they were censored at the date of randomization.

Outcome measures

Outcome measures
Measure	Parsaclisib Plus Ruxolitinib n=90 Participants Participants were randomized to receive parsaclisib plus ruxolitinib beginning on Day 1. Participants received parsaclisib at a dose of 5 milligrams (mg) once daily (QD). Participants also received the stable dose of ruxolitinib they were taking for the 8 weeks prior to Day 1. Treatment with parsaclisib plus ruxolitinib continued for as long as the participant tolerated the regimen and did not meet any discontinuation criteria.	Placebo Plus Ruxolitinib n=87 Participants Participants were randomized to receive placebo plus ruxolitinib beginning on Day 1 and continuing until Week 24. Participants received matching placebo at a dose of 5 mg QD and received the stable dose of ruxolitinib they were taking for the 8 weeks prior to Day 1. After 24 weeks, participants randomized to receive placebo plus ruxolitinib had to switch to treatment with parsaclisib plus ruxolitinib or discontinue treatment. Treatment continued for as long as the regimen was tolerated and the participant did not meet any discontinuation criteria. Participants who demonstrated worsening symptomatic splenomegaly could have switched to treatment with parsaclisib plus ruxolitinib early.	Placebo Swith to Parsaclisib After 24 weeks, participants randomized to receive placebo plus ruxolitinib from Day 1 to Week 24 could have switched to treatment with parsaclisib plus ruxolitinib per the regimen received during the first 24 weeks of the study. Treatment continued for as long as the regimen was tolerated and the participant did not meet any discontinuation criteria. Participants who demonstrated worsening symptomatic splenomegaly could have switched to treatment with parsaclisb plus ruxolitinib early.
Time to the First ≥25% Reduction in Spleen Volume	NA days The median and the upper and lower limits of the confidence interval were not estimable because too few participants had a ≥25% reduction in spleen volume.	NA days The median and the upper and lower limits of the confidence interval were not estimable because too few participants had a ≥25% reduction in spleen volume.	—

SECONDARY outcome

Timeframe: up to 898 days

Population: ITT Population. Only those participants who had at least 1 measurement of ≥25% reduction from Baseline were to be analyzed. At the time of study termination, only a limited number of ≥25% reduction in spleen volume responses were observed; therefore, as specified in the Statistical Analysis Plan, analysis of duration of a 25% reduction in spleen volume was not performed.

The duration of ≥25% reduction from Baseline in spleen volume was defined as the interval between the first spleen volume measurement that was a ≥25% reduction from Baseline and the date of the first measurement that was no longer a ≥25% reduction from Baseline. If the end date was not observed before the database cutoff, the duration was censored at the last assessment.

Outcome measures

Outcome data not reported

Adverse Events

Parsaclisib Plus Ruxolitinib

Serious events: 33 serious events

Other events: 71 other events

Deaths: 10 deaths

Placebo Plus Ruxolitinib

Serious events: 15 serious events

Other events: 58 other events

Deaths: 7 deaths

Placebo Swith to Parsaclisib

Serious events: 8 serious events

Other events: 25 other events

Deaths: 2 deaths

Serious adverse events

Other adverse events

Additional Information

Study Director

Incyte Corporation

Phone: 1-855-463-3463

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee Following the first publication, the Institution and/or Principal Investigator may publish data or results from the Study, provided, however, that the Institution and/or Principal Investigator submits the proposed publication to the Sponsor for review at least sixty (60) days prior to the date of the proposed publication. Sponsor may remove from the proposed publication any information that is considered confidential and/or proprietary other than Study data and results.
Publication restrictions are in place

Restriction type: OTHER