AzTrials
AzTrials

MyelofibrosisPhase 3NCT04551053

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

A Phase 3 study testing parsaclisib and ruxolitinib in people with Myelofibrosis and Primary Myelofibrosis, run by Incyte Corporation at 177 sites in 19 countries.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Official record
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Trial 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. Read more about this study

Who is it for?
Ages 18 years and older
What is being tested?
parsaclisib and ruxolitinib
Type of treatment
Pill or oral medicine
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
177 participants
Where
177 sitesin 19 countries
Study dates
26 May 2021to 21 Aug 2024
Phase
Phase 3
Are there results?
Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Diagnosis of PMF, PPV-MF, or PET-MF.
  • DIPSS risk category of intermediate-1, intermediate-2, or high.
  • Treated with ruxolitinib for ≥ 3 months with a stable dose for at least the last 8 weeks prior to Day 1
  • Palpable spleen of ≥ 5 cm below the left costal margin on physical examination at the screening visit.
  • Active symptoms of MF at the screening visit, as demonstrated by the presence of a TSS of ≥ 10 using the Screening Symptom Form.
  • Participants with an ECOG performance status score of 0, 1, or 2.
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  • Screening bone marrow biopsy specimen and pathology report(s) available that was obtained within the prior 2 months or willingness to undergo a bone marrow biopsy at screening/baseline; willingness to undergo bone marrow biopsy at Week 24 and every 24 weeks there after. Screening/baseline biopsy specimen must show diagnosis of MF.
  • Life expectancy of at least 24 weeks.
  • Willingness to avoid pregnancy or fathering children.

Who cannot join

  • Prior therapy with any drug that inhibits PI3K (examples of drugs targeting this pathway include but are not limited to INCB040093, idelalisib, duvelisib, buparlisib, copanlisib, and umbralisib).
  • Use of experimental drug therapy for MF or any other standard drug used for MF (whether for treatment of MF or another indication) with the exception of ruxolitinib, within 3 months of starting study drug, and/or lack of recovery from all toxicities from previous therapy (except ruxolitinib) to Grade 1 or better.
  • Inability to swallow food or any condition of the upper gastrointestinal tract that precludes administration of oral medications.
  • Recent history of inadequate bone marrow reserve.
  • Inadequate liver and renal function at screening.
  • Active bacterial, fungal, parasitic, or viral infection that requires therapy.
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  • Active HBV or HCV infection that requires treatment or at risk for HBV reactivation.
  • Known HIV infection.
  • Uncontrolled, severe, or unstable cardiac disease that in the investigator's opinion may jeopardize the safety of the participant or compliance with the Protocol.
  • Active invasive malignancy over the previous 2 years.
  • Splenic irradiation within 6 months before receiving the first dose of study drug.
  • Concurrent use of any prohibited medications.
  • Active alcohol or drug addiction that would interfere with the ability to comply with the study requirements.
  • Use of any potent CYP3A4 inhibitors or inducers within 14 days or 5 half lives(whichever is longer) before the first dose of study drug or anticipated during the study.
  • Inadequate recovery from toxicity and/or complications from a major surgery before starting therapy.
  • Currently breastfeeding or pregnant.
  • Any condition that would, in the investigator's judgment, interfere with full participation in the study, including administration of study drug and attending required study visits; pose a significant risk to the participant; or interfere with interpretation of study data.
  • History of Grade 3 or 4 irAEs from prior immunotherapy.
  • Receipt of any live vaccine within 30 days of the first dose of study drug
  • Unwillingness to receive RBC transfusions to treat low hemoglobin levels.
  • Known hypersensitivity or severe reaction to parsaclisib or ruxolitinib or excipients of parsaclisib/matching placebo or ruxolitinib formulations.

Trial locations and contacts

177 sites in 19 countries.

177 sites in 19 countries

United States35 sites

  • Emory University

    Atlanta, Georgia, 30322

  • Augusta University - Medical College of Georgia

    Augusta, Georgia, 30912

  • Sutter Health Alta Bates Summit Medical Center Absmc Alta Bates Summit Comprehensive Cancer Center

    Berkeley, California, 94704

  • Massachusetts General Hospital

    Boston, Massachusetts, 02114

  • Beth Israel Deaconess Medical Center

    Boston, Massachusetts, 02215

  • New Jersey Hematology Oncology Associates Llc

    Brick, New Jersey, 08724

  • Cleveland Clinic

    Cleveland, Ohio, 44195

  • Advent Health Hendersonville Park Ridge Hospital Hendersonville

    Clyde, North Carolina, 28721

  • Oregon Health & Science University

    Columbus, Ohio, 43210

  • Texas Oncology - Medical City Dallas

    Dallas, Texas, 75230

  • Texas Oncology - Baylor Sammons Cancer Center

    Dallas, Texas, 75246-2092

  • University of Texas Southwestern Medical Center

    Dallas, Texas, 75390-8565

  • Duke Cancer Center

    Durham, North Carolina, 27710

  • Providence Regional Medical Center Everett

    Everett, Washington, 98201

  • CCARE

    Fresno, California, 93720

  • Westchester Medical Center Advanced Oncology

    Hawthorne, New York, 10532

  • Renovatio Clinical

    Houston, Texas, 77005

  • Kelsey Seybold Clinic

    Houston, Texas, 77025

  • Indiana University Melvin and Bren Simon Cancer Center

    Indianapolis, Indiana, 46202

  • Midamerica Cancer Care

    Kansas City, Missouri, 64114

  • California Research Institute (Cri)

    Los Angeles, California, 90027-6005

  • Baptist Cancer Center

    Memphis, Tennessee, 38120

  • Morristown Medical Center - Atlantic Health System

    Morristown, New Jersey, 07960

  • Tulane University

    New Orleans, Louisiana, 70112

  • Mount Sinai School of Medicine

    New York, New York, 10029

  • Memorial Sloan Kettering Cancer Center

    New York, New York, 10065

  • Hillman Cancer Center

    Pittsburgh, Pennsylvania, 15232

  • Oregon Health & Science University

    Portland, Oregon, 97239

  • Emad Ibrahim Md Inc

    Redlands, California, 92373

  • Scripps Clinic

    San Diego, California, 92103

  • Coastal Integrated Cancer Care - Cicc

    San Luis Obispo, California, 93401

  • Stamford Hospital - Medical Oncology Hematology

    Stamford, Connecticut, 06904

  • Montefiore Medical Center

    The Bronx, New York, 10467

  • Georgetown University

    Washington D.C., District of Columbia, 20057

  • University of Kansas Hospital Authority

    Westwood, Kansas, 66205

Other trials in United States

China30 sites

  • Peking University People'S Hospital (Pkuph) - Institute of Hematology

    Beijing, 100044

  • Xuanwu Hospital Capital Medical University

    Beijing, 100053

  • Peking University Third Hospital

    Beijing, 100091

  • Peking University Third Hospital

    Beijing, 100191

  • The First Hospital of Jilin University

    Changchun, 130021

  • The First Peoples Hospital of Changzhou

    Changzhou, 213003

  • Fujian Medical University Union Hospital

    Fuzhou, 350001

  • Nanfang Hospital

    Guangzhou, 510515

  • The First Affiliated Hospital of Zhejiang University

    Hangzhou, 310003

  • Harbin Institute of Hematology and Oncology

    Harbin, 150010

  • University of Science and Technology of China-First Affiliated Hospital (Anhui Provincial Hospital)

    Hefei, 230022

  • The Affiliated Hospital of Inner Mongolia Medical University

    Hohhot, 10050

  • Jinan Central Hospital

    Jinan, 250013

  • The Second Affiliated Hospital of Kunming Medical University

    Kunming, 650101

  • The First Hospital of Lanzhou University

    Lanzhou, 730000

  • Lanzhou University Second Hospital

    Lanzhou, 730030

  • Jiangxi Provincial of People Hospital

    Nanchang, 330000

  • The First Affiliated Hospital of Nanchang University

    Nanchang, 330006

  • Jiangsu Province Hospital

    Nanjing, 210029

  • The Affiliated Hospital of Qingdao University

    Qingdao, 266003

  • The First Hospital of China Medical University

    Shenyang, 110001

  • Shenzhen University Hospital

    Shenzhen, 518055

  • Tianjin Medical University General Hospital

    Tianjin, 300052

  • Institute of Hematology and Hospital of Blood Diseases, Chinese Academy of Medical Sciences and Pek

    Tianjin, 510080

  • Union Hospital Affiliated to Tongji Medical College of Huazhong University of Science and Technology

    Wuhan, 430022

  • Tongji Hospital Huazhong University of Science and Technology

    Wuhan, 430030

  • The Second Affiliated Hospital of Xi an Jiaotong University

    Xi'an, 710004

  • Yantai Yuhuangding Hospital

    Yantai, 264000

  • Henan Provincial Peoples Hospital

    Zhengzhou, 450003

  • Henan Cancer Hostipal

    Zhengzhou, 450008

Other trials in China

Italy23 sites

  • Azienda Ospedaliera San Giuseppe Moscati

    Avellino, 83100

  • Aou Policlinico Consorziale Di Bari

    Bari, 70124

  • Lstituto Di Ematologia Lorenzo Ea.Seragnoli Universita Degli Studi Di Bologna - Policlinico S. Or

    Bologna, 40138

  • Azienda Ospedaliera Di Rilievo Nazionale E Di Alta Specializzazione Garibaldi

    Catania, 11111

  • University of Florence

    Florence, 50134

  • Irccs Azienda Ospedaliera Universitaria San Martino

    Genova, 16132

  • Fondazione Irccs Ca Granda Ospedale Maggiore

    Milan, 20122

  • Universita Di Napoli Federico Ii

    Naples, 80131

  • Azienda Ospedaliero Universitaria Maggiore Della Carita Di Novara

    Novara, 28100

  • Azienda Ospedaliera Ospedali Riuniti Villa Sofia - Cervello

    Palermo, 90146

  • Ospedale S.Maria Della Misericordia

    Perugia, 06156

  • Ospedale S.Maria Della Misericordia

    Perugia, 6156

  • Presidio Ospedaliero Pescara

    Pescara, 65124

  • Azienda Ospedaliero Universitaria Pisana

    Pisa, 56126

  • Grande Ospedale Metropolitano Bianchi-Melacrino-Morelli

    Reggio Calabria, 89133

  • Univ. Di Roma Facolta Di Armacia E Medicina

    Roma, 00161

  • Policlinico Universitario Agostino Gemelli Universita Cattolica Del Sacro Cuore

    Roma, 00168

  • Ospedale Sant. Eugenio

    Roma, 144

  • Policlinico Universitario Agostino Gemelli Universita Cattolica Del Sacro Cuore

    Rome, 00168

  • Azienda Ospedaliera Universitaria Senese Policlinico Santa Maria Alle Scotte

    Siena, 53100

  • Azienda Ospedaliera San Giuseppe Moscati

    Taranto, 74123

  • Azienda Sanitaria Universitaria Friuli Centrale Asu Fc

    Udine, 33100

  • A.O. Universitaria Ospedale Di Circolo E Fondazione Macchi

    Varese, 21100

Other trials in Italy

Japan18 sites

  • Kyushu University Hospital

    Fukuoka, 812-8582

  • Japanese Red Cross Society Himeji Hospital

    Himeji-shi, 670-8540

  • Kansai Medical University Hospital

    Hirakata, 573-1191

  • Tokai University Hospital

    Isehara, 2591193

  • Juntendo University Hospital

    Izunokuni, 410-2211

  • Kagoshima University Hospital

    Kagoshima, 890-8520

  • Hospital of the University of Occupation and Environmental Health

    Kitakyushu-shi, 807-8556

  • Kobe City Medical Center General Hospital

    Kobe, 650-0047

  • Kumamoto Shinto General Hospital

    Kumamoto, 862-8655

  • University of Miyazaki Hospital

    Miyazaki, 889-1692

  • Japanese Red Cross Nagoya Daini Hospital

    Nagoya, 453-8511

  • Ogaki Municipal Hospital

    Ōgaki, 5038502

  • Osaka Metropolitan University Hospital

    Osaka, 545-8585

  • Dokkyo Medical University Saitama Medical Center

    Saitama, 343-8555

  • Hokuyukai Sapporo Hokuyu Hospital

    Sapporo, 003-0006

  • Juntendo University Hospital

    Tokyo, 113-8431

  • Nippon Medical School Hospital

    Tokyo, 113-8603

  • University of Yamanashi Hospital

    Yamanashi, 409-3898

Other trials in Japan

Spain15 sites

  • Hospital General Unviersitario de Alicante

    Alicante, 03010

  • Ico Hospital Germans Trias I Pujol

    Badalona, 08916

  • Hospital Del Mar

    Barcelona, 08003

  • Hospital General Universitario Vall D Hebron

    Barcelona, 08035

  • Institut Catala Doncologia Ico - Hospital Duran I Reynals Location

    Barcelona, 08908

  • Hospital Universitario Insular de Gran Canaria

    Las Palmas de Gran Canaria, 35010

  • Hospital Universitario 12 de Octubre

    Madrid, 24041

  • Fundacion Jimenez Diaz University Hospital

    Madrid, 28040

  • Hospital General Universitario Morales Meseguer

    Murcia, 30008

  • Hospital Universitario Virgen de La Arrixaca

    Murcia, 30120

  • Hospital Clinico Universitario de Salamanca

    Salamanca, 37007

  • Hospital Universitario Virgen Del Rocio

    Seville, 41013

  • Hospital Universitari I Politecnic La Fe

    Valencia, 46000

  • Hospital Universitario Doctor Peset

    Valencia, 46017

  • Hospital Universitario Miguel Servet

    Zaragoza, 50009

Other trials in Spain

France10 sites

  • Centre Hospitalier Universitaire Grenoble Alpes (Chu Grenoble Alpes) - Hopital Albert Michallon

    La Tronche, 38700

  • Chu de Limoges

    Limoges, 87042

  • CHU Limoges - Hopital Duputren

    Limoges, 87042

  • Centre Hospitalier Universitaire de Nantes (Chu de Nantes) - Hotel-Dieu

    Nantes, 44093

  • Chu Nimes

    Nîmes, 30900

  • Ap-Hp Groupe Hospitalier Saint-Louis Lariboisiere Fernand-Widal Site Saint Louis (Paris)

    Paris, 75010

  • Hospital Saint Antoine

    Paris, 75012

  • Hospices Civils de Lyon Centre Hospitalier Lyon Sud

    Pierre-Bénite, 69495

  • Institut Universitaire Du Cancer de Toulouse Oncopole

    Toulouse, 31059

  • Chu Vandoeuvre-Les-Nancy Hopital Brabois

    Vandœuvre-lès-Nancy, 54500

Other trials in France

Israel6 sites

  • Samson Assuta Ashdod University Hospital

    Ashdod, 7747629

  • Rambam Health Care Campus

    Haifa, 31999

  • Hadassah Hebrew University Medical Center Ein Karem Hadassah

    Jerusalem, 91120

  • Kaplan Medical Center

    Rehovot, 76100

  • Tel Aviv Sourasky Medical Center

    Tel Aviv, 64239

  • Assuta Ramat Hahayal

    Tel Aviv, 69710

Other trials in Israel

Turkey (Türkiye)6 sites

  • Baskent University Adana Hospital

    Adana, 01250

  • Gazi University Hospital Gazi University Faculty of Medicine

    Ankara, 06500

  • Istanbul Medipol Universitesi Ibagcilar Medipol Mega Universitesi Hastanesi

    Istanbul, 34214

  • Baskent University Istanbul Hospital

    Istanbul, 34662

  • Ege University Hospital

    Izmir, 35040

  • Ondokuz Mayis University Medicine Faculty

    Samsun, 55200

Other trials in Turkey (Türkiye)

Poland5 sites

  • Pratia Hematologia Katowice

    Katowice, 40-040

  • Pratia Hematologia Katowice

    Katowice, 41-081

  • Samodzielny Publiczny Zoz Szpital Uniwersytecki W Krakowie

    Krakow, 31-501

  • Samodzielny Publiczny Szpital Kliniczny Nr 1

    Lublin, 20-081

  • Institute of Hematology and Transfusion Medicine

    Warsaw, 02-776

Other trials in Poland

United Kingdom5 sites

  • Grampian Health Board

    Aberdeen, AB15 6RE

  • United Lincolnshire Hospitals

    Boston, PE21 9QS

  • Gloucestershire Royal Hospital

    Gloucester, GL1 3NN

  • University College London Hospitals Nhs Foundation Trust

    London, NW1 2BU

  • James Cook University Hospital

    Middlesbrough, TS4 3BW

Other trials in United Kingdom

Belgium4 sites

  • A.Z. St.-Jan A.V.

    Bruges, 08000

  • Jessa Ziekenhuis

    Hasselt, 3500

  • AZ Delta

    Roeselare, 8800

  • Chu Ucl Namur University Hospital Mont-Godinne

    Yvoir, 05530

Other trials in Belgium

Germany4 sites

  • Universitatsklinikum Bonn Aoer

    Bonn, 53127

  • Universitaetsmedizin Greifswald

    Greifswald, 17475

  • Universitatsklinikum Halle (Saale)

    Halle, 06120

  • Universitaetsmedizin Rostock

    Rostock, 18057

Other trials in Germany

South Korea4 sites

  • Pusan National University Yangsan Hospital

    Busan, 49241

  • Seoul National University Hospital

    Seoul, 03080

  • Samsung Medical Center

    Seoul, 06351

  • The Catholic University of Korea Seoul St. Mary?S Hospital

    Seoul, 06591

Other trials in South Korea

Hungary3 sites

  • Semmelweis Egyetem

    Budapest, 01088

  • Markhot Ferenc Korhaz

    Eger, 03300

  • Petz Aladar County Teaching Hospital

    Győr, 09024

Other trials in Hungary

Taiwan3 sites

  • Kaohsiung Chang Gung Memorial Hospital

    Kaohsiung City, 83301

  • China Medical University Hospital

    Taichung, 00404

  • National Cheng Kung University (Ncku) Hospital

    Tainan, 00704

Other trials in Taiwan

Norway2 sites

  • Haukeland University Hospital

    Bergen, 05021

  • Akershus University Hospital

    Lorenskog, 01478

Other trials in Norway

Romania2 sites

  • Spitalul Clinic Coltea - Clinica Hematologie

    Bucharest, 30171

  • Spitalul Clinic Judetean de Urgenta Targu Mures

    Târgu Gânguleşti, 540139

Other trials in Romania

Austria1 site

  • Hanusch-Krankenhaus Wiener Gebietskrankenkasse

    Vienna, 01140

Other trials in Austria

Finland1 site

  • Helsinki University Central Hospital

    Helsinki, FI-00029

Other trials in Finland

About this study

Official 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

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.

Read the 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.

Being tested
parsaclisib, ruxolitinib, placebo
Keywords
INCB050465, ruxolitinib, parsaclisib, LIMBER, LIMBER-304, MF, MPN, Myeloproliferative Neoplasm, Myelofibrosis, Myoproliferative Neoplasms
Medical subject headings
Primary Myelofibrosis, Myeloproliferative Disorders

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • parsaclisibDrug

    Also called INCB050465

    parsaclisib will be administered QD orally

  • ruxolitinibDrug

    Also called Jakafi, Jakavi

    ruxolitinib will be administered BID orally

  • placeboDrug

    placebo will be administered QD orally

Study groups in NCT04551053
Study groupRoleWhat they receive
Group A : ruxolitinib +parsaclisibExperimentalParticipants will receive parsaclisib starting from Day 1 for the duration of study, while continuing to receive the stable dose of ruxolitinib they were taking for the 8 weeks prior to Day 1.
Receives: Drug: parsaclisib; Drug: ruxolitinib
Group B : ruxolitinib + placeboPlacebo comparatorParticipants will receive placebo starting from Day 1 for the duration of study, while continuing to receive the stable dose of ruxolitinib they were taking for the 8 weeks prior to Day 1.
Receives: Drug: ruxolitinib; Drug: placebo

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. 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)

    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.

    Measured: Baseline; Week 24

Secondary outcomes (8)

  1. 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

    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.

    Measured: Baseline; Week 24

  2. Change in TSS From Baseline to Week 24 as Measured by the MFSAF v4.0 Diary

    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.

    Measured: Baseline; Week 24

  3. Time to the First ≥50% Reduction in TSS as Measured by the MFSAF v4.0 Diary

    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.

    Measured: Baseline; up to Week 24

  4. Overall Survival

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

    Measured: up to 917 days

  5. Number of Participants With Any Treatment-emergent Adverse Event (TEAE)

    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.

    Measured: up to 917 days

  6. Number of Participants With Any Grade 3 or Higher TEAE

    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.

    Measured: up to 917 days

  7. Time to the First ≥25% Reduction in Spleen Volume

    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.

    Measured: up to 898 days

  8. Duration of Maintenance of a ≥25% Reduction in Spleen Volume

    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.

    Measured: up to 898 days

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted16 Sep 2020
  2. Study start26 May 2021
  3. Primary completion16 Aug 2023
  4. Study completion21 Aug 2024

Why it stopped: The study was terminated due to futility.

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 3
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Masking
Triple blind
Who is masked
Participant, Investigator, Outcomes assessor
Enrolment
177 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT04551053
Sponsor's protocol ID
INCB 50465-304/LIMBER-304
Other IDs
2020-003415-98
Status last verified
Sep 2025
First submitted
11 Sep 2020
Last updated
2 Oct 2025
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
Yes: Incyte shares data with qualified external researchers after a research proposal is submitted. These requests are reviewed and approved by a review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations. The trial data availability is according to the criteria and process described on https://www.incyte.com/our-company/compliance-and-transparency

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing parsaclisib and ruxolitinib in people with Myelofibrosis and Primary Myelofibrosis, run by Incyte Corporation at 177 sites in 19 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 9 inclusion and 21 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: stopped early, as of the sponsor's last update on 2 Oct 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 177 sites in United States, China, Italy, Japan, Spain and 14 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 23 Aug 2024). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

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) (time frame: Baseline; Week 24). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT04551053.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 2 Oct 2025.

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