New drug combo shows promise for rare bone marrow cancer

NCT ID NCT04279847

Disease control Sponsor: Incyte Corporation Source: ClinicalTrials.gov ↗

First seen Jun 26, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This early-stage trial tests an experimental drug called INCB057643, given alone or with the standard drug ruxolitinib, in about 140 adults with myelofibrosis or other advanced blood cancers. The main goal is to check safety and side effects, while also measuring if the drug can shrink an enlarged spleen. Because it is a Phase 1 study, it is too early to know if the drug will become a standard treatment.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance: INCB057643 (an experimental drug) and ruxolitinib (a standard therapy)
What this could lead to: If successful, this could point toward a new treatment option for myelofibrosis and similar blood cancers, possibly reducing spleen size and improving symptoms.
What could go wrong: This is an early Phase 1 trial focused on safety, not proof of effectiveness. The drug may cause side effects or fail to show benefit. Results may not apply to all patients.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 1

The first testing in people. Mainly checks safety and dose, usually in a small group.

Participants

140 people

The number who actually took part.

Started

Feb 2021

Expected to finish

Apr 2027

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

18 years and older

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: * Age 18 years and older at the time of signing the informed consent. * Part 1 Monotherapy: Participants with confirmed diagnosis of relapsed or refractory MF (primary, or post-PV and post-ET), MDS, MDS/MPN, or ET who have received at least 1 prior line of therapy; are either refractory, relapsed, or intolerant to the last therapy; and there is no available therapy that would provide clinical benefit in the opinion of the investigator. * a. MF with measurable disease (palpable spleen and symptoms) as defined in the protocol and risk category of intermediate 2 or high according to DIPSS. MF participants must have received a JAK inhibitor(s), such as ruxolitinib. * b. ET participants should have disease refractory to hydroxyurea as defined by the protocol. * Part 2 Combination with ruxolitinib. * a. Primary MF or secondary MFs (post-PV MF and post-ET MF), histologically or cytologically confirmed, with measurable disease (palpable spleen and symptoms) as defined in the protocol, either currently receiving ruxolitinib with suboptimal response or JAKi-naive. * b. Suboptimal response is defined as currently being treated with ruxolitinib monotherapy at a stable dose for ≥ 8 weeks immediately preceding the first dose of study treatment. One dose reduction due to toxicities within 8 weeks prior to Study Day 1 is permitted. * c. JAKi-naive is defined as those participants that have no prior use of any JAK inhibitor, including ruxolitinib, and; * d. Part 2 dose escalation: Risk category of intermediate-2 or high according to DIPSS. * e. Part 2 dose expansion: Risk category of intermediate-1, intermediate-2, or high according to DIPSS. * f. Part 2 dose expansion participants with chronic MF are defined as participants with bone marrow myeloblast percentage \< 5% (not applicable if dry tap or blast count deemed not reliable by the investigator) and blast count in peripheral blood \< 1% at screening and who are currently receiving ruxolitinib and having a suboptimal response. Note: Study treatment should be delayed if peripheral blood blast count at baseline is \> 3%; treatment should only be started with medical monitor approval. * g. Part 2 dose expansion participants with accelerated-phase MF are defined as having either a bone marrow myeloblast percentage ≥ 5% to \< 20% or a myeloblast percentage ≥ 10% in peripheral blood on 2 occasions at least 2 weeks apart, AND are currently receiving ruxolitinib and have a suboptimal response. * h. Part 2 dose expansion participants with JAKi-naive MF are eligible to receive ruxolitinib, with peripheral blood blast count of \< 10% at the screening hematology assessment. * Must not be a candidate for potentially curative therapy, including hematopoietic stem cell transplantation. * ECOG performance status 0 to 2. * Life expectancy ≥ 24 weeks. * Willingness to avoid pregnancy or fathering children based on criteria. * a. Men must agree to take appropriate precautions to avoid fathering children (with at least 99% certainty) from screening through 90 days after the last dose of study treatment and must refrain from donating sperm during this period. Permitted methods that are at least 99% effective in preventing pregnancy should be communicated to the participants and their understanding confirmed. * b. Women of childbearing potential must have a negative serum pregnancy test at screening and before the first dose on Day 1 and must agree to take appropriate precautions to avoid pregnancy (with at least 99% certainty) from screening through safety follow-up. Permitted methods that are at least 99% effective in preventing pregnancy should be communicated to the participants and their understanding confirmed. * c. Women of nonchildbearing potential (ie, surgically sterile with a hysterectomy and/or bilateral oophorectomy OR ≥ 12 months of amenorrhea without any other medical reasons such as treatment with anticancer agents) are eligible. Exclusion Criteria: * Prior receipt of a BET inhibitor. * Receipt of anticancer medications or investigational drugs within the protocol-defined interval before the first dose of study treatment. For Part 2 JAKi-naive, prior use of a JAK inhibitor (including ruxolitinib) and no use of experimental drug therapy for MF or any other standard drug (except hydroxyurea) used for MF or another indication within 3 months of starting study drug. For participants with suboptimal response to ruxolitinib, ruxolitinib will continue at the participants' current ongoing doses, no ruxolitinib washout is needed. * Participants with exclusionary laboratory values at screening defined as, including, but not limited to, * a. Platelets. Part 1 (monotherapy dose expansion, MF): \< 75 × 109/L. Part 1 (monotherapy dose expansion, ET): \< 450 × 109/L. Part 2 (combination dose escalation and expansion): \< 75 × 109/L. Part 2 (combination dose expansion, JAKi-naïve MF): \< 100 × 109/L. * b. Hemoglobin: Participants unwilling to receive red blood cell transfusion to treat low hemoglobin levels are excluded. * c. ANC \< 0.75 × 109/L. * inadequate renal, hepatic and coagulation functions as defined in the protocol. * Concurrent anticancer therapy other than the therapies being tested in this study. * Participants who have received allogeneic hematopoietic stem cell transplantation within 6 months of enrollment (unless approved by the medical monitor), or have active graft versus-host disease, or have received immunosuppressive therapy following allogeneic transplant within 2 weeks of the first dose of study treatment. * Unless approved by the medical monitor, may not have received autologous hematopoietic stem-cell transplant within 3 months before the first dose of study treatment. * Significant concurrent, uncontrolled medical condition, including but not limited to, significant GI disorder, history of or current clinically significant or uncontrolled cardiac disease, history or presence of an abnormal ECG that, in the investigator's opinion, is clinically meaningful, and history of bleeding disorder or at a high risk of bleeding. * Active bacterial, fungal, parasitic, or viral infection that requires therapy. * Current use of prohibited medication as described in the protocol, including the use of any potent CYP3A4 inhibitors or inducers within 14 days or 5 half lives (whichever is longer) before the first dose of study treatment. Other protocol-defined Inclusion/Exclusion Criteria may apply.

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Conditions

The condition(s) this trial relates to.

essential thrombocythemia myelodysplastic syndrome Myelodysplastic Syndromes myelodysplastic/myeloproliferative neoplasm myelofibrosis Myeloproliferative Disorders myeloproliferative neoplasm primary myelofibrosis Recurrence

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Et (essential thrombocythemia) Myelodysplastic syndrome Myelodysplastic/myeloproliferative neoplasm overlap syndrome Myelofibrosis Myeloproliferative neoplasm Relapsed or refractory primary myelofibrosis Secondary myelofibrosis (post-polycythemia vera myelofibrosis, post-essential thrombocythemia myelofibrosis)

Contacts and locations

Locations

Aou Policlinico S. Orsola-Malpighi

Bologna, 40138, Italy
Azienda Ospedaliero Universitaria San Luigi Gonzaga Di Orbassano

Orbassano, 10043, Italy
Azienda Ospedaliero-Universitaria Careggi (Aouc)

Florence, 50134, Italy
Centro Ricerche Cliniche Di Verona

Verona, 37134, Italy
Chiba University Hospital

Chiba, 260-8677, Japan
Emory University-Winship Cancer Institute

Atlanta, Georgia, 30322, United States
Fondazione Irccs Ca Granda Ospedale Maggiore

Milan, 20122, Italy
Fred Hutchinson Cancer Center

Seattle, Washington, 98109, United States
Fujita Health University Hospital

Aichi, 470-1192, Japan
Helsinki University Central Hospital

Helsinki, 00029, Finland
Henan Cancer Hostipal

Zhengzhou, 450003, China
Hospital Clinico Universitario de Salamanca

Salamanca, 37007, Spain
Hospital Universitari Germans Trias I Pujol

Badalona, 08916, Spain
Hospital Universitario 12 de Octubre

Madrid, 28041, Spain
Hospital Universitario Virgen de La Arrixaca

Murcia, 30120, Spain
Hospital Universitario de Gran Canaria Dr. Negrin

Las Palmas de Gran Canaria, 35010, Spain
Huntsman Cancer Institute At University of Utah

Salt Lake City, Utah, 84112, United States
Istituto Romagnolo Per Lo Studio Dei Tumori Dino Amadori

Meldola, 47014, Italy
Kumamoto Shinto General Hospital

Kumamoto, 862-8655, Japan
Kyushu University Hospital

Fukuoka, Japan
Lincoln County Hospital

Lincoln, LN2 5QY, United Kingdom
McGill University Jewish General Hospital

Montreal, Quebec, H3T 1E2, Canada
Md Anderson Cancer Center

Houston, Texas, 77030, United States
Nanfang Hospital_Southern Medical University

Guangzhou, 510515, China
National Cancer Center Hospital East

Chiba, 277-8577, Japan
Nyu Langone Health - Long Island Hospital

Mineola, New York, 11501, United States
Nyu Langone Laura and Isaac Perlmutter Cancer Center

New York, New York, 10016, United States
Ohio State University

Columbus, Ohio, 43210, United States
Oncology Consultants

Houston, Texas, 77030, United States
Oregon Health and Science University

Portland, Oregon, 97239, United States
Peking Union Medical College Hospital

Beijing, 100730, China
Princess Margaret Cancer Center

Toronto, Ontario, M5G 2M9, Canada
Rutgers Cancer Institute of Nj

New Brunswick, New Jersey, 08901, United States
St Paul'S Hospital

Vancouver, V6Z2A5, Canada
Texas Oncology-Baylor Sammons Cancer Center

Dallas, Texas, 75246, United States
The Christie Nhs Foundation Trust Uk

Manchester, M20 4BX, United Kingdom
The First Affiliated Hospital, Zhejiang University School of Medicine (Fahzu)

Hangzhou, 310003, China
United Lincolnshire Hospitals

Boston, PE21 9QS, United Kingdom
University of Alabama At Birmingham

Birmingham, Alabama, 35294, United States
University of Cincinnati Cancer Institute

Cincinnati, Ohio, 45267, United States
University of Colorado Cancer Center

Aurora, Colorado, 80045, United States
University of Iowa Hospital and Clinics

Iowa City, Iowa, 52242, United States
University of Miami Sylvester Comprehensive Cancer Center

Miami, Florida, 33136, United States
University of North Carolina At Chapel Hill

Chapel Hill, North Carolina, 27514, United States
University of Oxford

Oxford, OX3 7LE, United Kingdom
University of Yamanashi Hospital

Chūō, 409-3898, Japan
Washington University School of Medicine

St Louis, Missouri, 63110, United States
Weill Medical College of Cornell University

New York, New York, 10021, United States

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