Trial · NCT04551053
To Evaluate Efficacy and Safety of Parsaclisib and Ruxolitinib in Participants With Myelofibrosis Who Have Suboptimal Response to Ruxolitinib (LIMBER-304)
NCT04551053, Phase 3, Terminated, Incyte Corporation; results first posted 2024-08-23
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. To Evaluate Efficacy and Safety of Parsaclisib and Ruxolitinib in Participants With Myelofibrosis Who Have Suboptimal Response to Ruxolitinib (LIMBER-304). Trials, record NCT04551053. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT04551053
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- To Evaluate Efficacy and Safety of Parsaclisib and Ruxolitinib in Participants With Myelofibrosis Who Have Suboptimal Response to Ruxolitinib (LIMBER-304)
- NCT number
- NCT04551053
- Status
- Terminated
- Phases
- Phase 3
- Enrollment
- 177 participants, actual
- Lead sponsor
- Incyte Corporation
- Conditions
- Myelofibrosis, Primary Myelofibrosis, Post Essential Thrombocythemia Myelofibrosis, Post Polycythemia Vera Myelofibrosis
- Interventions
- parsaclisib, ruxolitinib, placebo
- First posted
- 2020-09-16
- Last updated
- 2025-10-02
- Other registries' IDs
- eudract:2020-003415-98
- Results first posted
- 2024-08-23
- Limitations and caveats
- Following an interim analysis by an independent Data Monitoring Committee (DMC), the study was terminated early due to futility.
Source ClinicalTrials.gov
Registered outcomes9
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | 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) | Baseline; Week 24 | 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. |
| Secondary | 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 | Baseline; Week 24 | 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. |
| Secondary | Change in TSS From Baseline to Week 24 as Measured by the MFSAF v4.0 Diary | Baseline; Week 24 | 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. Change from Baseline was calculated as the Week 24 value minus the Baseline value. |
| Secondary | Time to the First ≥50% Reduction in TSS as Measured by the MFSAF v4.0 Diary | Baseline; up to Week 24 | 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. |
| Secondary | Overall Survival | up to 917 days | Overall survival was defined as the interval between the randomization date and the date of death due to any cause. |
| Secondary | Number of Participants With Any Treatment-emergent Adverse Event (TEAE) | up to 917 days | 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. |
| Secondary | Number of Participants With Any Grade 3 or Higher TEAE | up to 917 days | 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. |
| Secondary | Time to the First ≥25% Reduction in Spleen Volume | up to 898 days | 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. |
| Secondary | Duration of Maintenance of a ≥25% Reduction in Spleen Volume | up to 898 days | 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. |
Source ClinicalTrials.gov
Outcomes9
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | 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) | Baseline; Week 24 | 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. | percentage of participants | NUMBER |
| SECONDARY | 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 | Baseline; Week 24 | 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. | percentage of participants | NUMBER |
| SECONDARY | Change in TSS From Baseline to Week 24 as Measured by the MFSAF v4.0 Diary | Baseline; Week 24 | ITT Population. Only participants with data available were analyzed. | scores on a scale | MEAN |
| SECONDARY | Time to the First ≥50% Reduction in TSS as Measured by the MFSAF v4.0 Diary | Baseline; up to Week 24 | 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. | days | MEDIAN |
| SECONDARY | Overall Survival | up to 917 days | ITT Population | days | MEDIAN |
| SECONDARY | Number of Participants With Any Treatment-emergent Adverse Event (TEAE) | up to 917 days | 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. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Number of Participants With Any Grade 3 or Higher TEAE | up to 917 days | Safety Population | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Time to the First ≥25% Reduction in Spleen Volume | up to 898 days | ITT Population | days | MEDIAN |
| SECONDARY | Duration of Maintenance of a ≥25% Reduction in Spleen Volume | up to 898 days | 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. |
Source ClinicalTrials.gov
Results by group20
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
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) | Parsaclisib Plus Ruxolitinib | 16.7 | percentage of participants | ||
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) | Placebo Plus Ruxolitinib | 9.7 | percentage of participants | ||
| 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 | Parsaclisib Plus Ruxolitinib | 17.1 | percentage of participants | ||
| 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 | Placebo Plus Ruxolitinib | 14.1 | percentage of participants | ||
| Change in TSS From Baseline to Week 24 as Measured by the MFSAF v4.0 Diary | Parsaclisib Plus Ruxolitinib | Baseline | 17.2 | Standard Deviation 11.61 | scores on a scale |
| Change in TSS From Baseline to Week 24 as Measured by the MFSAF v4.0 Diary | Placebo Plus Ruxolitinib | Baseline | 21.6 | Standard Deviation 14.28 | scores on a scale |
| Change in TSS From Baseline to Week 24 as Measured by the MFSAF v4.0 Diary | Parsaclisib Plus Ruxolitinib | Change from Baseline at Week 24 | -2.7 | Standard Deviation 8.67 | scores on a scale |
| Change in TSS From Baseline to Week 24 as Measured by the MFSAF v4.0 Diary | Placebo Plus Ruxolitinib | Change from Baseline at Week 24 | -2.7 | Standard Deviation 9.98 | scores on a scale |
| Time to the First ≥50% Reduction in TSS as Measured by the MFSAF v4.0 Diary | Parsaclisib Plus Ruxolitinib | NA | 95% Confidence Interval 160.0 to NA | days | |
| Time to the First ≥50% Reduction in TSS as Measured by the MFSAF v4.0 Diary | Placebo Plus Ruxolitinib | NA | 95% Confidence Interval NA to NA | days |
1 to 10 of 20Source ClinicalTrials.gov
See all 20 results by groupParticipant flow6
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Parsaclisib Plus Ruxolitinib | 90 | |
| Overall Study | STARTED | Placebo Plus Ruxolitinib | 87 | |
| Overall Study | COMPLETED | Parsaclisib Plus Ruxolitinib | 0 | |
| Overall Study | COMPLETED | Placebo Plus Ruxolitinib | 0 | |
| Overall Study | NOT COMPLETED | Parsaclisib Plus Ruxolitinib | 90 | |
| Overall Study | NOT COMPLETED | Placebo Plus Ruxolitinib | 87 |
Source ClinicalTrials.gov
Adverse events255
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Abdominal pain upper | Gastrointestinal disorders | Parsaclisib Plus Ruxolitinib | 8 | 90 | 9 |
| other | Abdominal pain upper | Gastrointestinal disorders | Placebo Plus Ruxolitinib | 2 | 87 | 2 |
| other | Abdominal pain upper | Gastrointestinal disorders | Placebo Swith to Parsaclisib | 0 | 41 | 0 |
| other | Alanine aminotransferase increased | Investigations | Parsaclisib Plus Ruxolitinib | 8 | 90 | 9 |
| other | Alanine aminotransferase increased | Investigations | Placebo Plus Ruxolitinib | 3 | 87 | 4 |
| other | Alanine aminotransferase increased | Investigations | Placebo Swith to Parsaclisib | 4 | 41 | 4 |
| other | Anaemia | Blood and lymphatic system disorders | Parsaclisib Plus Ruxolitinib | 26 | 90 | 38 |
| other | Anaemia | Blood and lymphatic system disorders | Placebo Plus Ruxolitinib | 20 | 87 | 36 |
| other | Anaemia | Blood and lymphatic system disorders | Placebo Swith to Parsaclisib | 6 | 41 | 6 |
| other | Arthralgia | Musculoskeletal and connective tissue disorders | Parsaclisib Plus Ruxolitinib | 4 | 90 | 4 |
1 to 10 of 255Source ClinicalTrials.gov
See all 255 adverse eventsAdverse event totals9
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Parsaclisib Plus Ruxolitinib | 10 | 90 |
| Total, all-cause mortality | Placebo Plus Ruxolitinib | 7 | 87 |
| Total, all-cause mortality | Placebo Swith to Parsaclisib | 2 | 41 |
| Total, other adverse events | Parsaclisib Plus Ruxolitinib | 71 | 90 |
| Total, other adverse events | Placebo Plus Ruxolitinib | 58 | 87 |
| Total, other adverse events | Placebo Swith to Parsaclisib | 25 | 41 |
| Total, serious adverse events | Parsaclisib Plus Ruxolitinib | 33 | 90 |
| Total, serious adverse events | Placebo Plus Ruxolitinib | 15 | 87 |
| Total, serious adverse events | Placebo Swith to Parsaclisib | 8 | 41 |
Source ClinicalTrials.gov
Papers0
The papers the trial lists on ClinicalTrials.gov.
None in this release.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT04551053
GET https://conductscience.org/api/trials/v1/NCT04551053/events?page=2